Innovations In Study Design
Beyond the traditional parallel group design, alternative designs can be more efficient in terms of cost and duration. One of these designs for instance, is the 2 Ã 2 factorial design, in which two interventions can be simultaneously tested. However, in the case of drug development trials, in which response to each intervention and adverse events need to be strictly monitored, factorial design appears to be inappropriate. Contrarily, for RCT testing approved drugs for repurposing or repositioning for disease-modification or other types of therapies, such as diet modification or physical exercise, when the interaction between the interventions is unlikely to result in severe adverse events, this type of design can be very useful .
Another alternative is the master protocol design, also called platform trials. These studies have a single overarching protocol developed to evaluate multiple hypotheses, with the general goal of improving efficiency . The platform basket trials, which evaluate a single targeted therapy on multiple diseases or umbrella trials, which test multiple interventions in a single disease can be feasible option to test a single drug in multiple neurodegenerative processes, for instance AD and PD, or to test one drug in multiple disease, for instance, an anti-tau therapy and AD and other tauopathies.
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Your participation will allow you to have firsthand experience with developing new medical treatments that may be beneficial to others. Current treatment methods for neurologic disorders are only available because of volunteer participants in clinical trials. Clinical trial information is excerpted from www.clinicaltrials.gov.
A Clinical Study Of Nly01 In Patients With Early Parkinson’s Disease
Objective: Phase 2 study designed to assess the safety, tolerability and efficacy of NLY01 in subjects with early untreated Parkinson’s disease Eligibility: Individuals 30 to 80 years old, with early-stage Parkinsons Disease, not on any current treatmentsP.I.: Emile Moukheiber, M.D.Contact: Kori Ribb
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Feasibility And Preliminary Effects Of Using A Music
Objective: The purpose of this clinical study is to evaluate the effects of music, tailored to the participant’s cadence, on adherence, quality of life, gait speed, functional mobility, and walking activity in individuals with Parkinson disease when used in the home and community environment.P.I.: Alex Pantelyat, M.D.Contact: Colin McGregor
Multimodal Mri In Psp
Eligibility: Individuals diagnosed w/ PSP or PPA willingness/ability to complete MRI and lumbar punctureP.I.: Alex Pantelyat, M.D.
Objective: Create an international repository to learn more about dystonia, treatment methods and patient responseEligibility: Individuals over the age of 18 who have primary dystoniaP.I.: Alex Pantelyat, M.D.Contact: Sydney Baybayan
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Parkinson Progression Marker Initiative Online
open to eligible people ages 18 years and up
Parkinson Progression Marker Initiative Online is an observational study collecting participant reported information from people with and without Parkinson’s disease , for the goal of better understanding risk and predictive factors for PD. PPMI Online is part of the broader Parkinson Progression Marker Initiative aimed at identifying markers of disease progression for use in clinical trials of therapies to reduce progression of PD disability.
San Francisco, California
Identification Of The Study Population
The study population is the subset of the population with the condition of interest defined by the eligibility criteria. In AD and PD trials, the current trend is to recruit patients at the pre-clinical or prodromal stages of these diseases. This approach was encouraged mainly by the recognition that AD and PD pathological processes start decades before the onset of clinical symptoms and the multiple negative trials for DMT in participants with an established diagnosis. However, once individuals in the pre-clinical stages do not have clinical symptoms, the identification of the study population needs to rely on the use of biomarkers and rigorous eligibility criteria. However, biomarkers, either clinical, radiological or laboratorial are not perfect, and one cannot predict with certainty if an individual with positive biomarkers will convert into a clinical phenotype, particularly when there is no gold standard for confirmation of the diagnosis âin vivoâ .
In AD research, biomarkers have been increasingly incorporated over the past decade to confirm that cognitive decline in a given individual is actually associated with AD instead of other differential diagnoses . Also, biomarkers have gained crucial importance in identifying individuals in the pre-clinical and prodromal phases of the disease and as surrogate endpoints in the early stages of drug development trials.
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More On Symptom Treatment And Disease
In 2022, 32 trials will be completed, including three stem cell trials. The decisions about which will move on to the next steps will be captured in the 2023 update. The current research landscape holds much promise. We are eagerly awaiting the results of the 32 trials being completed this year, what trials graduate to the next phases, and what new therapeutic categories will join the pipeline over the course of this year.
McFarthing, K., Rafaloff, G., Baptista, M., Mursaleen, L., Fuest, R., Wyse, R. K., & Stott, S. . Parkinsons Disease Drug Therapies in the Clinical Trial Pipeline: 2022 Update. Journal of Parkinsons disease, 12, 10731082.
Parkinson Canada’s mission is to transform the lives of people living with Parkinson’s across Canada. Articles like this represent our commitment to building awareness and providing resources and support for people living with Parkinson’s and their care network.
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What Causes Parkinsons Disease
The precise cause of Parkinsons disease is not yet known. Much research is being completed to try an uncover this, as it will aid in prevention and treatment. Although it is not fatal by itself, the symptoms can seriously impact quality of life. Likewise, some complications leave a patient at greater risk of injury or other health issues.
In addition to dopamine-producing nerve cells, people with Parkinsons lose nerve endings responsible for producing norepinephrine, which regulates the autonomic nervous system. This leads to disruption in automatic functions like heart rate and blood pressure.
Current Parkinsons research focuses on the presence of Lewy bodies in patients brain cells. These unusual clumps of protein may be related to certain genetic mutations.
Parkinsons clinical trials attract more participants than many other chronic diseases. The Michael J. Fox foundation is a leading organization dedicated to finding more advanced treatments and a cure for Parkinsons disease. Millions of dollars from other outside resources have also been donated to study it. A variety of new clinical resources for Parkinsons are in advanced stages of development.
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Sample Size Statistical Analysis And Duration Of Follow Up
Even the most well-designed research question will be left unanswered if the study is underpowered. Undersized trials may end with negative results that are not interpretable, while oversized studies may find statistically significant differences that are not clinically relevant. To adequately estimate the sample size, several characteristics of the study need to be taken into account, including the experimental design, the study hypothesis, the nature of the outcome, the statistical test, the MCID, the significance threshold and power, and the expected attrition .
In AD, most trials have a parallel group design comparing intervention to placebo or two different doses of the intervention and placebo . The trials aimed to demonstrate the superiority of the intervention against placebo, typically using one of the rating scales previously discussed as continuous outcome measures. Because of the small changes expected in the outcome scales in participants at pre-clinical and prodromal stages, relatively large sample sizes have been required in AD trials, ranging from 1500 to 2000 .
A Study To Evaluate The Efficacy And Safety Of Intravenous Prasinezumab In Participants With Early Parkinson’s Disease
open to eligible people ages 50-85
This is a multicenter, randomized, double-blind, placebo-controlled study that will evaluate the efficacy and safety of intravenous prasinezumab versus placebo in participants with Early Parkinson’s Disease who are on stable symptomatic PD medication.
San Francisco, California
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Are There Any Promising Treatments For Parkinson’s Disease
“Neural transplants can be transplanted from a variety of sources, including twins and aborted fetuses.
Scientists are researching different ways to use neural transplants to treat Parkinson’s disease. One way is to transplant new brain cells into areas where the cells have died. Another way is to transplant cells into areas that are not affected by the disease. This could help prevent the disease from getting worse.” – Anonymous Online Contributor
Perspectives For Clinical Trials For Early Ad And Pd
Designing an RCT is definitely not an easy task. This is particularly more challenging when dealing with pre-clinical and prodromal phases of neurodegenerative disorders. By looking at the recent research in the fields of AD and PD, clinical trials in AD appeared to have achieved more sophistication, largely due to the advances in biomarker research. For that reason, AD research is closer to move to studies in the pre-clinical stage of the disease. On the other hand, the PD field is only starting to look at the prodromal stages of the disease and more questions needs to be addressed before moving research to the pre-clinical stages. Independent of these differences in timing, similar issues are faced in both areas.
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Dipraglurant For The Treatment Of Patients With Parkinson’s Disease Receiving Levodopa
open to eligible people ages 30-85
This study is designed to evaluate the safety and efficacy of dipraglurant in PD patients with dyskinesia for 12 weeks . The primary efficacy assessment will be based on the Unified Dyskinesia Rating Scale . Patients who complete the 12-week blinded treatment period may have the option to roll into an open-label safety extension study for an additional 12-month treatment period.
Treatments In Phase Ii Trials
Another strategy in the therapeutic research space is drug repurposing. This is when an existing medication for one condition is repurposed to treat an entirely different condition. Working with repurposed medications comes with many advantages including understanding its general safety. Repurposing an existing medication, rather than starting from scratch, typically requires fewer tests for safety as the drug has already met these requirements. This can reduce costs and speed up the process through the clinical trial pipeline. It can also lead to faster approvals, getting much-needed treatments into the hands of people with Parkinsons as soon as possible. There are a total of 74 therapies in Phase II trials and 44% are repurposed medications.
One exciting takeaway from Phase II trials this year is the progress made with stem cell therapies. While there are nine stem cell therapies being explored in Phase I, two stem cell therapies graduated to Phase II trials this year! Moving into Phase II means these treatments are being administered to a larger group of people to monitor their effectiveness and further evaluate their safety.
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The Promising Treatments In The 2022 Parkinsons Clinical Trials Pipeline
As we know the Parkinsons research community is hard at work to find therapies and treatments to improve the quality of life of Canadians living with Parkinsons and ultimately find a cure. An article outlining the current clinical trials pipeline for Parkinsons was recently published in the Journal of Parkinsons Disease. Here are some of the key highlights. For a refresher on clinical trials and what happens in each phase, here is a helpful FAQ.
As of January 2022, there were nearly 150 Parkinsons therapies active in the clinical trial pipeline:
- 50 products in Phase I
- 65 products in Phase II
- 17 products in Phase III
Most of the drugs in trials are for managing the symptoms of Parkinsons . Developments in disease-modifying treatments the types of treatment that change the course of Parkinsons progression have been slow to emerge. However, in 2022, there are 54 disease-modifying treatments in trial phases, with three graduating to Phase III trials!
Below youll find a brief overview of whats happening in each phase of the trials.
Irlab Celon Target Parkinsons Disease Motor Symptoms
While levodopa can reduce PD symptoms by increasing dopamine levels in the brain, prolonged use can cause increased bouts of dyskinesia during OFF time when the drug has reduced effects. IRLAB Therapeutics and Celon Pharma have Phase II trials aiming to reduce levodopa-induced dyskinesia using two different approaches.
IRLABs mesdopetam, which targets the dopamine D3 receptor, has placebo-controlled Phase II trial results expected the second half of this year. Mesdopetam has estimated peak sales of $226 million in 2026, according to GlobalDatas consensus forecast. GlobalData is the parent company of Clinical Trials Arena.
As a primary endpoint, the Phase II mesdopetam trial assesses change in ON time, defined as hours in the day where patients experience the positive effects of levodopa without the negative effects of dyskinesia. Before and after 12 weeks of treatment, patients record their hours of ON time over the course of 24 hours.
Meanwhile, Celons CPL500036, which targets phosphodiesterase 10A , also has placebo-controlled Phase II results expected this year. The four-week study uses a primary endpoint of the Unified Dyskinesia Rating Scale , which measures the severity of dyskinesia side effects.
Of the two primary endpoints, change in ON time is a better measure than the UDyRS, Kordower says. My understanding is that patients would much rather have less time spent in dyskinesias than decreased magnitude of dyskinesia, he explains.
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What Are 5 Signs And Symptoms Of Parkinson’s Disease
“The tremors associated with Parkinson’s disease typically start in one hand, foot, or leg, and eventually affect both sides of the body. Rigidity, bradykinesia, vocal symptoms, and postural instability are also common in Parkinson’s patients. Walking or gait difficulties, cognitive changes, and depression and anxiety are also common in those with Parkinson’s disease.” – Anonymous Online Contributor
What Is A Clinical Trial
A clinical trial is a research study that looks into the safety and usefulness of a drug in improving symptoms or slowing, stopping, or reversing the progression of a disease. Most drugs tested in clinical trials are not yet available in drug stores while some studies involve medications that are already available .
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Biomarkers For Ataxia And Multiple System Atrophy
Objective: A major impediment to developing new treatments for neurological diseases is the absence of biological markers for early diagnosis and treatment response. Currently the diagnosis of ataxia and MSA is based on the presence of clinical signs and, in some cases, genetic testing. The research seeks to identify biomarkers for ataxia and MSA in an effort to improve diagnosis and therapeutics for these diseases.P.I.: Liana Rosenthal, M.D.Contact: Michelle Joyce
Getting Involved In Parkinson’s Clinical Trials
There is often a shortage of volunteers to take part in clinical trials. This may be due to a lack of easily available information on trials in a particular location or area, or to doctors being unaware of trials that are being held locally.
If you would like to become involved in a trial, it is essential to discuss this first with your doctor as it is likely that they will be required to refer you to the trial centre. You should find out as much information as possible about the trial so that you can fully discuss your participation. Your doctor will be able to advise you on the advantages and disadvantages for your own particular situation, and they may be able to refer you to other members of the multidisciplinary team who have more knowledge about a particular area of research.
Depending on what the trial is investigating, the research team will be looking for people who fit specific criteria called inclusion and exclusion criteria that ensure you are suitable for the trial. Screening usually involves answering questions about your Parkinsons, your current medication, treatment history and checking details such as your age and when you were diagnosed. This information is frequently available on the trial website. You may need to have an additional detailed examination which may include blood tests, scans or other clinical assessment tests. If you fit the trials eligibility criteria, you may then be invited to take part in the trial.
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Why Should I Participate In A Clinical Study
We can only reach breakthroughs in treatment and care if people participate in the studies.
Participating is safe and can help you
Every clinical study is reviewed thoroughly before your doctor asks you to participate. Clinical trials carry some risks, but your doctor is required by law to explain the risks to you clearly and make sure that you understand them. If your doctor tells you about the risks of participating in the study, ask yourself, What are the risks of not participating in the study? Most of the time, if you balance the possible benefits from participating against the risks, it is about the same as the risks of not being in the study.
On the other hand, the study may be of a new drug or treatment that could help you. If you dont participate, it may be years before you have a chance to try that drug.
Some people do not participate because there is no guarantee they will get the experimental therapy they might get the placebo. Again, think carefully about the risks and benefits of entering the study and getting the new treatment, entering the study and getting the placebo or not entering the study at all.
Your participation can help others
If you have PD or any other disease, the drugs, procedures and therapies you use now were scientifically tested, likely by thousands of volunteers. Participating in a clinical trial is your way to pay it forward for people diagnosed with Parkinsons in the future.
Amneal Tests A New Formulation
AmnealPharmaceuticals plans to report Phase III safety results for IPX-203, a reformulation of the common generic PD treatment combination of carbidopa and levodopa that could reduce symptom fluctuations. The company said the Phase III, open-label extension study will have results available by the end of the second quarter of 2022.
CD/LD can lead to troughs and spikes of plasma levels that generate side-effects like dyskinesia, Kordower explains. A new extended-release version of CD/LD could smooth out these drops, he notes.
If approved, IPX-203 will join several other marketed reformulations of CD/LD. Amneals own extended-release capsule Rytary, Schwarz Pharmas orally disintegrating tablet Parcopa, and AbbVie’s enteral suspension Duopa all have FDA approval in PD. A GlobalData consensus forecasts pegs peak IPX-203 sales at $127 million in 2028.
In a separate, placebo-controlled Phase III trial , IPX-203 resulted in 0.53 more hours of ON time than immediate-release CD/LD after seven weeks . Earlier, a six-week Phase II trial of IPX-203 reported no serious treatment-emergent adverse events among the 26 patients enrolled. Experts say the long-term safety data will be key in determining IPX-203s place among CD/LD formulations.
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