DRY POWDER INHALER FOR PARKINSON'S DISEASE

18th July 2015 - New research

A dry powder inhaler has been found to be a viable means of administering L-dopa. Because of its rapid onset of action, pulmonary administration of L-dopa is a possible alternative to the oral administration of L-dopa in Parkinson's Disease patients in an off period. Its means of administration could enable a very quick therapeutic effect.

Researchers studied the ability of people with Parkinson's Disease to operate a dry powder inhaler (DPI) correctly during an off period. They used an instrumented test inhaler with three different resistances to air flow. The volumes inhaled varied from 1.2 litres to 3.5 litres. Total inhalation time and the time to peak inspiratory flow rate both decreased with decreasing inhaler resistance. Nearly all of the patients could hold their breath for at least five seconds after inhalation and most of them could extend this time to ten seconds.

The data from this study indicate that patients with Parkinson's disease will be able to use a dry powder inhaler during an off period and they provide an adequate starting point for the development of an L-dopa powder inhaler.

An L-dopa inhaler using L-dopa in liquid form instead of a dry form, that takes only 10 minutes to start having effect, is already undergoing clinical trials in Parkinson's Disease. 

For more information go to : 

http://www.viartis.net/parkinsons.disease/news/150625.htm

Reference : PLoS One [2015] 10 (7) : e0132714 (M.Luinstra, A.W.Rutgers, H.Dijkstra, F. Grasmeijer, P.Hagedoorn, J.M.Vogelzang, H.W.Frijlink, A.H.de Boer)

Complete abstract : http://www.ncbi.nlm.nih.gov/pubmed/26173114

http://www.viartis.net/parkinsons.disease/news/150718.pdf mail@viartis.net

©2015 Viartis 

Study: Virtual research studies feasible

A new pilot study in Parkinson's disease suggests a new era of clinical research which removes the barrier of distance for both scientists and volunteers. The research, which appears in the journal Digital Health, could also enable researchers to leverage the rapid growth in personal genetic testing to better diagnose, and potentially treat, a wide range of diseases.

"These findings demonstrate that remote recruitment and conduct of research visits is feasible and well-received by participants," said Ray Dorsey, M.D., M.B.A., a neurologist at the University of Rochester and lead author of the study. "Direct-to-consumer genetic testing, when paired with telemedicine, has the potential to involve more people in clinical research and accelerate the process of identifying the genetic causes and variations in chronic diseases such as Parkinson's"

Published: Friday 17 July 2015 at 3am PST

"Giving clinicians the ability to recruit and assess patients remotely for research and clinical trials is a game changer," said Emily Drabant Conley, Ph.D., a research scientist and director of business development with 23andMe, and co-author of the study. "Leveraging genetically-defined groups of patients through direct-to-consumer genetic testing combined with self-reported data and remote assessment opens up exciting frontiers in research and may allow us to do things at a scale and speed that was previously not possible."

Parkinson's is a complex multi-system disease with many known genetic "clues" and a wide range of patient experiences, both in terms of the severity and progression of symptoms and an individual patient's responsiveness to the several available forms of treatment. While researchers have been able to identify many of the different phenotypes of the disease, this variation makes the process of diagnosis and treatment a challenge.

The ideal solution would be to identify the genetic signature of the various phenotypes and understand more precisely how these different forms of the disease are manifested in terms of symptoms and what treatments, or combination of treatments, provide the most effective relief.

However, this has proven to be a highly difficult undertaking given the previous high cost of genetic testing and the logistical obstacle of having to recruit from a geographically diverse pool of volunteers in order to create a sample large enough to arrive at scientifically meaningful conclusions.

Two new technologies now make this task possible: direct-to-consumer genetic testing - and the recent rapid decline in the cost of genetic sequencing - and telemedicine.

Researchers at the University of Rochester and Johns Hopkins University partnered with 23andMe, a personal genomics and biotechnology company based in California, to conduct a pilot study to determine if individuals with known genetic risk factors for Parkinson's disease could be diagnosed for the condition via telemedicine. The researchers also wanted to test the feasibility of conducting clinical research remotely.

Working with 23andMe and the Michael J. Fox Foundation for Parkinson's Research, the researchers were able to ultimately recruit 50 individuals in 23 states who agreed to undergo a remote assessment consisting of cognitive and motor tests via a secure video conferencing developed by Vidyo. The participants also completed a survey.

http://www.medicalnewstoday.com/releases/296946.php?tw

Parkinson's nurses in short supply

By Josh Jennings

July 18, 2015

Parkinson's disease nurse Mary Jones says she fosters long-term relationships with many of her patients, given the number that live with Parkinson's for many decades. The duration of these relationships significantly enhances the satisfaction she derives from Parkinson's nursing too, she adds.

"To see people come in, have their appendix out and go home is terribly rewarding but it's not me," says Jones.

"I love the long-term relationships and being part of people's lives.

"It's a really chronic condition you share a life with."

Jones, a Parkinson's nurse specialist, works in private practice alongside a neurologist and plays a significant role in reducing the physical and psychological burden encountered by people who live with Parkinson's.

She says diagnosis is one of the most critical stages of the disease, citing research indicating that the way the diagnosis is presented to patients significantly affects the progression of the disease.

"It's not a nice diagnosis to get," says Jones. "People can be fairly shocked ...

"A lot of support goes on in the early stages and then right through the condition."

Jones began in private practice in 2009. Some of the main roles she's had during her career include nine years in an orthopaedic nursing role at the Alfred Hospital and a decade co-ordinating the movement disorders program at Eastern Health.

Presently, according to Parkinson's Victoria, there are very few Victorian Parkinson's nurses available for the 27,000 Victorians living with Parkinson's.

"We don't have an education system," Jones says of Parkinson's nursing. "The role isn't suited to that of a nurse practitioner because we don't want to prescribe medication but we do want to stand alone as highly specialised nurses looking after a very complex condition.

"Largely it's going to be self-taught and taught by your peers."

Next month, Parkinson's Victoria is presenting A Walk in the Park, an Australia-wide event staged to improve the quality of life for those living with Parkinson's. Jones, a regular participant in the event and former board member of Parkinson's Victoria, says she has become an active speaker on Parkinson's education as her career has developed. She delivers lectures for support groups and organisations such as Parkinson's Victoria and St Vincent's Hospital.

"A good outcome for me in the next year would be recognition of Parkinson's as a specialty," she says.

"At the moment, any patient I see has to be seen by the doctor as well, because we're not funded. Patients will ring up and say, 'I only need to see Mary'. But there's no way we can do it currently."

A Walk in the Park takes place on August 30. www.parkinsonswalk.com.au

The story Parkinson's nurses in short supply first appeared on The Sydney Morning Herald.

http://health.einnews.com/article_detail/276461120/tEFZbIUTvl7urp-c?n=2&code=ga_qGBxHZ2aVYO4P

Anti-malaria drugs could treat Parkinson’s disease

June 17, 2015 

Associate Professor at the Nanyang Technological University (NTU) School of Biological Sciences Yoon Ho Sup with a protein sample, which will then be inserted into a Nuclear Magnetic Resonance (NMR) spectroscopy machine to identify the compounds which could bind and activate Nurr1 in the brain. Photo: Jaslin Goh 

         

         Anti-malaria drugs could potentially treat Parkinson’s disease, scientists from Nanyang Technological University (NTU), and McLean Hospital and Harvard Medical School in the United States, have found.The drugs, chloroquine and amodiaquine, share a basic chemical structure that allows them to bind and activate a class of proteins called Nurr1, which protects the brain’s ability to generate dopamine neurons.

Parkinson’s disease disrupts the production of dopamine neurons, which progressively causes the loss of motor control as dopamine is an important neurotransmitter that affects motor control and movement of muscles in the body.

The research was a four-year collaboration between Professor Kwang-Soo Kim from McLean Hospital and Harvard Medical School and Associate Professor Yoon Ho Sup from NTU’s School of Biological Sciences.

They screened 1,000 US Food and Drug Administration (FDA)-approved drugs before discovering the two anti-malaria drugs that worked.

In laboratory tests, rats injected with the drugs improved in their behaviour and later showed no signs of suffering from Parkinson’s disease.

Prof Kim, a leading expert in Parkinson’s, said the current gold standard of treatment is to replenish the patient’s dopamine levels through medication or by deep-brain stimulation using electric currents.

“However, these pharmacological and surgical treatments address the patient’s symptoms, such as to improve mobility functions in the early stages of the disease, but the treatments cannot slow down or stop the disease process,” he said.

The team is modifying the two drugs to be more potent while having fewer side effects. They are also finding new chemical compounds which could activate Nurr1. If all goes well, Assoc Prof Yoon estimates clinical trials could start in three to five years’ time.

“Our research shows that existing drugs can be repurposed to treat other diseases and once several potential drugs are found, we can redesign them to be more effective in combating their targeted diseases while reducing side effects,” said the expert in drug discovery and design.

Chloroquine was used to treat malaria in the late 1940s to early 1950s until the malaria parasite grew resistant, while amodiaquine is still used in Africa today. Side effects include hallucinations, nausea and diarrhoea.

Parkinson’s disease affects three in 1,000 persons aged 50 and above, and is one of the most common neurodegenerative conditions in Singapore.

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There is currently no cure or treatment to slow down or halt the disease, whose symptoms include tremors, muscle stiffness and changes in speech.

http://health.einnews.com/article/276206159/yAP8kBtjdBcWuFRj

NTU scientists discover potential treatment for Parkinson's disease

July 16,2015

NANYANG TECHNOLOGICAL UNIVERSITY

CAPTION

This image shows NTU Assoc Prof Yoon Ho Sup (centre) with Dr Shin Joon (right) and Mr Nguyen Quoc Toan (left) in front of the NMR spectroscopy machine used in their experiments.

SCIENTISTS FROM SINGAPORE'S NANYANG TECHNOLOGICAL UNIVERSITY (NTU SINGAPORE) AND MCLEAN HOSPITAL AND HARVARD MEDICAL SCHOOL IN THE UNITED STATES HAVE FOUND THAT EXISTING ANTI-MALARIA DRUGS COULD BE A POTENTIAL TREATMENT FOR PARKINSON'S DISEASE.PARKINSON'S DISEASE IS A DEGENERATIVE DISORDER OF THE CENTRAL NERVOUS SYSTEM THAT CAUSES A PERSON TO LOSE CONTROL OF MOTOR MOVEMENTS, SUCH AS THE ABILITY TO MOVE HIS OR HER HANDS, ARMS, AND LEGS. PARKINSON'S DISEASE IS ONE OF THE MOST COMMON NEURODEGENERATIVE CONDITIONS IN SINGAPORE. IT AFFECTS THREE OUT OF EVERY 1,000 PERSONS AGED 50 YEARS AND ABOVE. WITH AN AGEING POPULATION IN SINGAPORE, CASES OF NEURODEGENERATIVE DISEASES ARE SET TO RISE. 

Currently, there is no cure or treatment which can slow down or stop Parkinson's disease, which affects an estimated 10 million people worldwide.

This groundbreaking research was published recently in Proceedings of the National Academy of Sciences of the United States of America (PNAS) online, a prestigious peer-reviewed scientific journal.

The multi-year research project was a partnership between Professor Kwang-Soo Kim from McLean Hospital and Harvard Medical School in the United States and Associate Professor Yoon Ho Sup from NTU's School of Biological Sciences.

The team of international scientists had discovered that by activating Nurr1, a class of proteins found in the brain, it protects the brain's ability to generate dopamine neurons. 

Dopamine, commonly known as the chemical in the brain that generates pleasurable feelings, is an important neurotransmitter that affects motor control and movement of muscles in the body.

Parkinson's disease disrupts the production of dopamine neurons and progressively causes the loss of motor control.

In laboratory tests, the scientists found that by activating Nurr1, the rats which had Parkinson's disease appeared to improve in their behaviour and showed no signs of suffering from the disease. 

Assoc Prof Yoon said the team had screened about 1000 FDA-approved drugs before they found two anti-malaria drugs which worked: Chloroquine and Amodiaquine.

"Our discovery brings hope for the millions of people suffering from Parkinson's disease, as the drugs that we have found to have worked in the laboratory tests have already been used to treat malaria in patients for decades," said Assoc Prof Yoon, an expert in drug discovery and design.

"Our research also shows that existing drugs can be repurposed to treat other diseases and once several potential drugs are found, we can redesign them to be more effective in combating their targeted diseases while reducing the side effects."

Professor Kwang-Soo Kim, a leading expert in Parkinson's disease, said the current golden standard of treatment is to replenish the patients' dopamine levels through medication or by using a surgical method to do deep brain stimulation using electric currents.

"However, these pharmacological and surgical treatments address the patient's symptoms, such as to improve mobility functions in the early stages of the disease, but the treatments cannot slow down or stop the disease process," Prof Kim explains.

"Backed by various lines of scientific evidence, Nurr1 is known to be a potential drug target to treat Parkinson's. Despite great efforts from pharmaceutical companies and academia, no one has managed to find a molecule which can directly bind to it and activate it, except for us."

Both Chloroquine and Amodiaquine are approved by the US Food and Drug Administration and are used treat malaria infections. Chloroquine was used in the late 1940s to early 1950s, until the malaria parasite grew resistant while Amodiaquine is still being used in Africa today.

The scientists are now looking into studying more drugs which can halt and reverse the onset of Parkinson's disease. 

They also aim to design better drugs for the disease by modifying Chloroquine and Amodiaquine. The team hopes to carry out clinical trials with these modified drugs.

About Nanyang Technological University

A research-intensive public university, Nanyang Technological University (NTU) has 33,500 undergraduate and postgraduate students in the colleges of Engineering, Business, Science, and Humanities, Arts, & Social Sciences. It has a new medical school, the Lee Kong Chian School of Medicine, set up jointly with Imperial College London.

NTU is also home to world-class autonomous institutes - the National Institute of Education, S Rajaratnam School of International Studies, Earth Observatory of Singapore, and Singapore Centre on Environmental Life Sciences Engineering - and various leading research centres such as the Nanyang Environment & Water Research Institute (NEWRI), Energy Research Institute @ NTU (ERI@N) and the Institute on Asian Consumer Insight (ACI). 

A fast-growing university with an international outlook, NTU is putting its global stamp on Five Peaks of Excellence: Sustainable Earth, Future Healthcare, New Media, New Silk Road, and Innovation Asia. 

The University's main Yunnan Garden campus has been named one of the Top 15 Most Beautiful in the World. NTU also has a campus in Novena, Singapore's medical district.

For more information, visit http://www.ntu.edu.sg

http://www.eurekalert.org/pub_releases/2015-07/ntu-nsd071615.php

Neuroscientists decipher brain's noisy code

JULY 17, 2015

Study explains how output of single neurons can predict behavior on perceptual tests

By analyzing the signals of individual neurons, neuroscientists have deciphered the code the brain uses to make the most of its inherently 'noisy' neuronal circuits. These green and purple hills represent the average activity for many neurons in two different brain regions. These neuronal activity patterns will differ from time to time, even in response to exactly the same sensory stimulus, and those differences set the limit for how well the brain can sense things.
Credit: Xaq Pitkow/Rice University

By analyzing the signals of individual neurons in animals undergoing behavioral tests, neuroscientists at Rice University, Baylor College of Medicine, the University of Geneva and the University of Rochester have deciphered the code the brain uses to make the most of its inherently "noisy" neuronal circuits.

The human brain contains about 100 billion neurons, and each of these sends signals to thousands of other neurons each second. Understanding how neurons work, both individually and collectively, is important to better understand how humans think, as well as to treat neurological and psychiatric disorders like Alzheimer's disease, Parkinson's disease, autism, epilepsy, schizophrenia, depression, traumatic brain injury and paralysis.

"If the brain could always count on receiving the same sensory response to the same stimulus, it would have an easier time," said neuroscientist Xaq Pitkow, lead author of a new study this week in Neuron. "But noise is always there in the brain: studies have repeatedly shown that neurons give a variety of responses to the same stimulus."

Pitkow, assistant professor of neuroscience at Baylor and assistant professor of electrical and computer engineering at Rice, said "noise" can be described as anything that changes neural activity in a way that doesn't depend on the task the brain wants to accomplish.

Not only are neural responses noisy, but each neuron's noise is correlated with the noise in thousands of other neurons. That means that something that affects the output of one neuron may be amplified to affect many more. Because of these correlations, it is extraordinarily difficult for scientists to accurately model how small groups of neurons will affect the way a person or animal reacts to a given stimulus.

Given both these correlated responses and the inherently noisy nature of neuronal signals, scientists have struggled to explain a seeming paradox that was first observed in experiments more than 25 years ago.

"When neuroscientists first analyzed the output of individual neurons, they were surprised to find that the activity of just a single neuron sometimes predicted behavior in certain tasks," Pitkow said.

This perplexing find has turned up in numerous experiments, but neuroscientists have yet to explain it.

"A lot of people have studied this and offered up different kinds of models that make all sorts of assumptions," Pitkow said. "By integrating all of those ideas and applying some analytical techniques, we found there were two different ways this could happen."

He said one possibility is that many neurons are sharing the same information, processing it independently and arriving at the same answer. The other possibility is that each neuron is using different information and casting its vote for a slightly different answer but the brain is doing a poor job of coming to a consensus with the different votes.

"The first model is a bit like trying to find a needle in haystack, and the second is like trying to find a needle on a clean floor while looking backward through a pair of binoculars," Pitkow said. "Each piece of straw looks like a needle, which makes the haystack test very difficult. On the other hand, a needle should really stand out on a clean floor, but it will be hard to find with a bad searching method."

In each case, the neurons are correlated with one other, "but in the first instance the noise correlations can never be removed, and in the second they could and should be removed but they're not," Pitkow said. "And each of these scenarios has very different consequences for the brain's code, how it represents information. In terms of information theory, if the brain has a lot of information and it is not doing a good job of using it, there are very different implications than if all the neurons are correlated and they're all informative in the same way."

To determine which of these scenarios is at play in the brain, Pitkow and colleagues developed two mathematical models, one for each scenario. The models described how information and noise would flow through the network in the two opposing cases.

The team tested each model against the activity of single neurons in monkeys that were undergoing perceptual tests to measure how accurately they could perceive slight movements to the left or right. The experimenters found that some neurons predicted the animals' guesses about whether they were moving left or right.

"When we examined the output, we found that the monkeys' brains were not throwing away information," Pitkow said. "They were using each neuron's information very effectively. And we also saw that even though there were many neurons involved, the guess of any individual neuron was only slightly worse than the animal's actual guess during the test. These two pieces of evidence together indicate the neurons mostly share the same information."

But if every neuron is doing the same processing, why have so many? It's an obvious question, Pitkow said, but it's beyond the scope of what he and his colleagues could address in the current study.

"We didn't explore the value of redundancy in this study, but we are very interested in that question," Pitkow said. He pointed out that the vestibular sensors, the part of the inner ear dedicated to the sense of balance, contain only about 6,000 of the brain's 100 billion neurons. Even those few thousand might be redundant, which would mean that the rest of the neurons they contact also are redundant.

"One intriguing possibility that we are looking into is that redundancy allows the brain to reformat information and approach complex problems from many different angles," he said.

http://www.medicalnewstoday.com/releases/296942.php

Clinical Trials with the Apple Research Kit

von Andreas Griesser am 17.07.2015

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Too many clinical trials fail – where are the errors and how to remove them?

When looking at the overall process of a clinical trial, we can separate the sources of errors into the following classes:

• hypothesis: poor quality of the underlying hypothesis
• knowledge about the patient
• inherent statistical errors
• data errors during a trial

Looking at those types of trials where a paper-version of a CRF (case report form) is used, then one obvious source of error lies in the transcription phase: while the Form Filler at the study site manually enters data (i.e. answers to the predefined questions) into the CRF, the Data Enterer transcripts these data into a validated database. Since this manual step is erroneous, using an electronic CRF (eCRF) with direct or indirect link to the database can definitely improve data consistency.

An interesting source of error lies in the knowledge about the patient. Do we really know that much about the person and its physiology and its health status so that a correct diagnosis can be made? Is abdominal pain a side effect of the drug under test or does the patient have IBS (irritable bowel syndrome)? While not knowing everything about the patient (making a gene test with every patient before the study is often not doable), the diagnosis may be wrong. The more we know about the patient, i.e. the more data we have, the better a diagnosis can be. Studies then may be converted from a hypothesis based one to a data-driven one.

This Article demonstrates the usage of a new toolbox, the Apple ResearchKit, as a eCRF and how more and more reliable data from the patients can be collected during a clinical trial.

Overview of the ResearchKit

ResearchKit was announced in March 2015 and published to the community as open source software one month later, with magnificent feedback from the development community. It was developed by Apple with the help of the leading medical institutions and foundations around the world.

Such is the popularity of the iPhone, ResearchKit was an immediate success. Just days after being announced, the system helped Stanford University gather a year’s worth of applicants (11,000) for a cardiovascular study in just 24 hours. Praising “the power of the phone,” Alan Yeung, medical director of Stanford Cardiovascular Health, said to get over 10,000 applicants enrolled on a study without ResearchKit “would take a year and 50 medical centers around the country.”  ibtimes.co.uk

Apple published ResearchKit including a short video giving a rough overview. Now let us dive deeper into the insights: In general ResearchKit supports the creation of native iOS applications for research studies while focusing on three major aspects:

• Inform about the study and obtain patients consent
• Create surveys
• Create active tasks and collect sensor data

Strictly spoken, ResearchKit is a set of templates for fast creation of eCRFs with the additional aspect of having the patient filling in the data without the necessity of going to the study center.

Having an eye on data security, it is important to inform the patients in research studies about which sensitive information will be collected and shared as part of their enrollment and involvement. Appropriate customizable templates can be used to inform the patient about the details of the study as well obtaining his consent. Part of this is a dedicated template that asks the user to provide a signature directly on their device.

Example screenshots of the “Consent” module of the ResearchKit

The central “survey” module of the framework contains templates for the creation of dynamic surveys. This means that the authors of a study can select from a choice of currently more than 10 templates that best fit to the types of questions defined in the trial plan. Creating a question form is quite straightforward: just provide the text for the question, the type of answer expected and, if appropriate (e.g. multiple choice questions), the possible answers the patient can choose of.

Example screenshots of the “survey” module of the ResearchKit

All data entered (i.e. answers from multiple choices or free text) is being stored in a defined format and needs to be transferred to the backend of the research study. At this point it needs to be said that Apple or the contributors of ResearchKit do not have access to the patient’s data at any time. It is upon the choice of the developer to implement a secure data transmission to some database.

So far these templates cover the possibilities of an eCRF.

Now, the most interesting part of the framework is the ability of collecting sensor data from the iOS device while asking the patient to perform certain tasks. Hereby, a variety of templates as part of the “active tasks” module help gathering data for different categories like fitness, voice, gait and balance, spatial memory and tapping speed.

Typically, an active task is structured into several steps:

1. Provide information about the task itself and what the patient needs to do
2. Counter indicating when the task starts. Now the patient can prepare to perform the required task, e.g. put the iOS device onto a table
3. Record data during the duration of the task
4. Inform about the completion of the task

A tasks ends with a confirmation screen that informs about the successful completion and about the next steps in the research study. The recorded data are stored as raw data and will not be interpreted by ResearchKit. This remains at the owner of the study.

The following active tasks are currently supported:

Category	Task	Sensor	Data collected
Motor activity	Gait and Balance	AccelerometerGyroscope	Device motionPedometer
	Tapping speed	Multi-Touch displayAccelerometer (optional	Touch activity
Fitness	Fitness	Accelerometer	Device motionPedometerLocationHeart rate
Cognition	Spatial memory	Multi-Touch display	Touch activityCorrect and actual sequence
Voice	Sustained phonation	Microphone	Uncompressed audio

Example screenshots of active tasks currently integrated in the Research Kit

With the help of ResearchKit the creation speed of the eCRFs is dramatically increased. Instead of wasting time and money on the creation of forms, the developer can now concentrate on the data only.

With the decision to publish ResearchKit as Open Source on githup, the community of iOS developers are able to push the framework forwards as they have already done with more than 600 updates to this day since the first release in April. By this approach, one can be sure that only the owner of the study sees the data.

The restriction to the Apple ecosystem ensures that the gathered data is not influenced by different hardware suppliers. The minimum hardware requirement is an iPod touch that can be handed over to a closed user group if they do not own an iOS device. ResearchKit supports external hardware like the Apple Watch to measure data like the heart rate and it can be adopted to support other external devices that collect medical data.

Currently not supported by ResearchKit is the collection of data in the background as it will be done within Apple’s health app, but as the framework and the health app is under control of Apple this can only be a matter of time. Another point that is yet not supported by ResearchKit and has to be realized by the developer is the ability to schedule surveys and active tasks. For example if the patient should perform an active task every morning, the developer has to setup a notification to remind the patient.

It will be exciting to see how ResearchKit develops in the next month and according to the news  the iPhone could become a new tool in genetic studies.

Active Task: Example Case

To give you an impression how an active task can look like we would like to give an example for voice recording. The goal of the task is to investigate the ability of sustained phonation that uses the microphone of the device and creates uncompressed audio. The described example demonstrates the usage of the various templates as well as how the forms can be filled with content. All the artwork is provided by ResearchKit and can easily be customized or replaced by other individual images or animations.

	Step 1: Inform about the task.Typically the patient will be informed about the task in general on a first screen.In this case the patient will be informed that the voice will be evaluated, using the microphone of the device.
	Step 2: Inform about the details.This screen informs about the concrete steps the patient will have to complete in the following.Here the patient will be informed that he needs to take a deep breath and say “Aaaaah” as long as possible. He will receive visual feedback in form of a power spectrum to involve his vocal volume.
	Step 3: Prepare for the activity.It is possible to display a countdown timer so that the patient can prepare for the evaluation.
	Step 4: Guide and give feedback.Within this step the patient will be guided to say “Aaaaah” and receives visual feedback from the microphone that helps him to keep his vocal volume constantly. No data analysis is done by ResearchKit; this can be defined according to the requirements.This step ends automatically as soon as the patient has stopped his voice.
	Step 5: Inform about the task result.This steps displays that the evaluation is successfully completed and what will happen next with the results.

Summary and Outlook

The ResearchKit provides templates for rapid development of iOS based eCRFs covering not only basic question types but also active tasks by utilizing available sensor data. Data can be entered everywhere and the patient is not required to go to the study center. This makes it very comfortable and in the end comes with less costs for a study.

Notice that the questionnaire is just part of a clinical trial; the clinical diagnostics has still be done (e.g. once in a month). Although this is the more expensive portion of a study, having the ability of collecting data on a higher frequency base (e.g. every day or on demand) helps the researchers finding out more about the person/drug under test and as such improve the quality of diagnosis.

The integration of sensor data opens the range of applicable trial types to areas, where sensor data was not available at all or where the study center required some additional measurement tools. Hereby, one can think of the measurement of the Range-of-Motion after surgical intervention, e.g. on the shoulder. Another idea would be the observation and tracking of activity and movement of elderly people.

From the trial plan to the integrated solution using the Apple Research Kit

Clinical Trials and Zühlke?

When moving from the ResearchKit to an integrated solution for clinical trials, the following tasks need to be done:

• create the customized App based on your trial plan and questionnaire and bring it onto your iOS devices (or put it in the AppStore)
• ensure a secure data transmission to a database and implement the whole data backend
• incorporate special tasks not integrated in the ResearchKit right now (e.g. Range-of-Motion tasks, optional)
• perform Data Analytics tasks, such as providing some sort of first analysis helping the researchers interpreting the raw sensor data (optional)
• develop and integrate dedicated sensor hardware/software, e.g. vitality sensors for iOS devices (optional)

Zühlke is supporting you at all these steps.

What is your experience with the ResearchKit? We are looking forward to your feedback to this blog.
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