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10 Spinal Muscular Atrophy Trials near New York, NY

Power is an online platform that helps thousands of Spinal Muscular Atrophy patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

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This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:All
Sex:All
85 Participants Needed
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 4
Age:3 - 24
Sex:All
28 Participants Needed
A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Insert (USPI) in adult and pediatric participants with SMA. In this study, participants will be followed for the duration of the study or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study, if they agree to continue participating in the follow-up assessments. An optional sub study will assess the feasibility, acceptability, and adherence of remote assessment of motor and bulbar functions in participants with SMA using wearable and smartphone-based biosensors. Approximately 39 participants from the main study are planned to be enrolled in the sub study.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 4
Age:2+
Sex:All
402 Participants Needed
This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA) previously enrolled in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previously treated with nusinersen, olesoxime or AVXS-101.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Age:6 - 60
Sex:All
174 Participants Needed
The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA). Secondary objectives of this study are to examine the effects of Nusinersen in infants with genetically diagnosed and presymptomatic SMA.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Age:6 - 6
Sex:All
25 Participants Needed
This trial is testing a medication called Apitegromab to see if it is safe and effective for people with certain types of spinal muscular atrophy (SMA). It focuses on patients who have already participated in previous research. The goal is to see if the medication can help improve their muscle function over time.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:2+
Sex:All
238 Participants Needed
This trial tests the safety and effectiveness of combining two treatments, RO7204239 and risdiplam, for patients with spinal muscular atrophy (SMA). Risdiplam helps the body produce a protein needed for muscle movement, while RO7204239 allows muscles to grow bigger and stronger. The study includes SMA patients to see if this combination improves their muscle function and overall health.
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Age:2 - 25
Sex:All
259 Participants Needed
In this study, researchers will learn more about the use of a higher dose of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study will focus on teenagers and adults who are unable to walk on their own and who have previously taken another drug for SMA called risdiplam. The main goal of this study is to learn about the effect of high dose (HD) nusinersen on muscle and movement ability (motor function) in SMA. The main question that researchers want to answer is: - How do the scores of a movement test called the Revised Upper Limb Module change from the start of treatment? The Revised Upper Limb Module is a test used to measure a participant's ability to do specific tasks that involve their shoulders, arms, wrist, elbows, and hands. It measures the changes in their abilities over time. Researchers will also learn more about the safety of HD nusinersen. They will check participants for adverse events and changes in vital signs, heart tests, and laboratory tests including blood and urine tests. The study will be done as follows: * Participants will be screened to check if they can join the study. * After screening, participants will enter the Core Treatment period. * At the start of the Core Treatment period, they will receive 2 "loading" doses of nusinersen. These are 50 mg doses of nusinersen given 2 weeks apart. * Afterwards, they will continue to receive "maintenance" doses of nusinersen once every 4 months. These doses will be 28 mg. * The Core Treatment period will last about 2 years, with a follow-up visit 4 months after the last dose. * Participants who complete the Core Treatment period will have the option to continue receiving 28 mg of nusinersen in the Long-Term Extension (LTE) period for about 2 years. There will also be a follow-up visit 4 months after the last dose. * Nusinersen will be given through a lumbar puncture, which involves injecting the drug into the fluid around the spinal cord in the lower back. * In total, participants will have up to 18 study visits. They will also be called by researchers after each dose of nusinersen. * Participants will stay in the study for about 4.5 years if they complete both the Core Treatment and LTE periods.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:15 - 50
Sex:All
45 Participants Needed
This trial is testing a medicine called risdiplam, which is taken by mouth. It aims to help infants who have a genetic diagnosis of spinal muscular atrophy (SMA) but are not yet showing symptoms. The medicine works by helping the body make more of a protein that muscles need to stay healthy. The goal is to see if early treatment can prevent or lessen the severity of SMA.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:1 - 6
Sex:All
26 Participants Needed
This trial will test if taldefgrobep alfa can help people with SMA who are already on other treatments. The drug works by blocking a protein that stops muscle growth, potentially making muscles stronger and improving movement. Taldefgrobep alfa has shown promise in increasing muscle mass and function in SMA patients.
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Age:4 - 21
Sex:All
269 Participants Needed
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Frequently Asked Questions

How much do Spinal Muscular Atrophy clinical trials in New York, NY pay?

Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

How do Spinal Muscular Atrophy clinical trials in New York, NY work?

After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Spinal Muscular Atrophy trials in New York, NY 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length in New York, NY for Spinal Muscular Atrophy is 12 months.

How do I participate in a study as a "healthy volunteer"?

Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility in New York, NY several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

What does the "phase" of a clinical trial mean?

The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

Do I need to be insured to participate in a Spinal Muscular Atrophy medical study in New York, NY ?

Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

What are the newest Spinal Muscular Atrophy clinical trials in New York, NY ?

Most recently, we added Risdiplam for Spinal Muscular Atrophy, Long-Term Apitegromab for Spinal Muscular Atrophy and Taldefgrobep Alfa for Spinal Muscular Atrophy to the Power online platform.