Updated May 2026 · ClinicalTrials.gov
Phase 3 Spinal Muscular Atrophy Trials
6 Phase 3 trials for Spinal Muscular Atrophy, the final stage before a treatment can be submitted for FDA approval.
6 Phase 3 clinical trials for Spinal Muscular Atrophy are registered on ClinicalTrials.gov. Phase 3 is the final stage of testing before a treatment can be submitted for FDA approval, and the trials below come directly from the federal registry. Always talk to your doctor before contacting a study site.
What Phase 3 Means for Spinal Muscular Atrophy
Phase 3 trials are the largest and most expensive stage of clinical research before potential FDA approval. For Spinal Muscular Atrophy, a Phase 3 protocol typically enrolls several hundred to several thousand patients across many medical centers, randomizes participants between the investigational treatment and either a placebo or current standard of care (where ethically appropriate), and tracks them for months or years to confirm that the treatment is both effective and safe in a real-world patient population.
6 Phase 3 trials for Spinal Muscular Atrophy are listed on ClinicalTrials.gov. Smaller late-stage pipelines often correspond to rare conditions, niche subpopulations, or treatment areas where Phase 2 results are still being read out.
Biogen (2), Novartis Pharmaceuticals (1), GeneCradle Inc (1) lead the Phase 3 Spinal Muscular Atrophy sponsor list. The blend of industry, academic, and government sponsors on a condition's Phase 3 list is a useful signal of how broadly the research community is engaged with the disease.
Phase 3 Spinal Muscular Atrophy Trials on ClinicalTrials.gov
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial....
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With...
This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously...
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle...
The study objective is to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec for the treatment of SMA. The specific objectives are: * To determine...
A Study to Compare the Efficacy and Safety of BMS-986365 Versus the Investigator's Choice of Therapy in Participants...
The purpose of this study is to compare the efficacy and safety of BMS-986365 versus the investigator's choice of therapy in participants with Metastatic Castration-resistant...
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years...
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works...
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in...
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works...
What Participation Looks Like
Phase 3 trials for Spinal Muscular Atrophy typically enroll several hundred to several thousand participants across multiple sites. Participation involves a screening visit to confirm eligibility, randomization to either the investigational treatment or a comparator (often the current standard of care), regular study visits over months or years, and follow-up after the active treatment period. The protocols, time commitments, and visit schedules differ from trial to trial — read the per-trial page for the specifics before discussing participation with your doctor.
Each trial begins with informed consent and a screening visit, where the study team confirms eligibility against the inclusion and exclusion criteria. Randomization assigns participants to either the investigational treatment or a comparator. Standard-of-care portions of the protocol are typically billed to insurance; trial-specific procedures (extra imaging, biopsies, lab draws beyond standard care) are usually covered by the sponsor. Read each trial\'s detailed page for its specific time commitment and visit schedule.
Authoritative Resources for Spinal Muscular Atrophy Trials
Verify any individual trial directly on ClinicalTrials.gov. For the federal context on how Phase 3 results feed into approval decisions, see the FDA drug approval process. For oncology-specific trial resources, the National Cancer Institute publishes patient-oriented overviews. For trials registered outside the U.S., the WHO ICTRP aggregates registries from around the world.
Frequently Asked Questions
What is a Phase 3 Spinal Muscular Atrophy trial?
A Phase 3 trial is the final stage of clinical testing before a treatment can be submitted to the FDA for approval. For Spinal Muscular Atrophy, Phase 3 studies typically enroll hundreds to thousands of patients across multiple medical centers, comparing the new treatment to the current standard of care or a placebo (where ethically appropriate). The goal is to confirm efficacy, monitor side effects in a larger population, and generate the evidence the FDA needs to make an approval decision.
How many Phase 3 Spinal Muscular Atrophy trials are recruiting?
6 Phase 3 trials for Spinal Muscular Atrophy are currently registered on ClinicalTrials.gov. Recruitment status varies by trial — some are actively enrolling, some have closed enrollment but are still in the active treatment phase, and some are completing follow-up. Click any trial below to see its current status, eligibility criteria, and contact information.
Who can participate in a Phase 3 Spinal Muscular Atrophy trial?
Phase 3 eligibility depends entirely on the specific trial protocol. Each trial sets its own inclusion criteria (typically a confirmed diagnosis, certain disease stage or severity, age range) and exclusion criteria (often previous treatments, comorbidities, lab values that fall outside set ranges). The trial pages on this site translate the clinical eligibility criteria into plain English alongside the original text. Whether you fit any specific trial is a medical decision your doctor needs to confirm.
Is participating in a Phase 3 Spinal Muscular Atrophy trial safe?
Phase 3 trials use treatments that have already passed Phase 1 (safety in small groups) and Phase 2 (initial efficacy and side-effect monitoring), so the safety profile is better understood than in earlier-phase studies. That said, side effects can still emerge in larger populations, and the trial protocol may require additional procedures (lab draws, imaging, biopsies) beyond standard care. The informed consent document for any specific trial details the known risks. Discuss those risks with your physician before deciding whether to participate.
Where does this trial data come from?
All trial data is sourced from the ClinicalTrials.gov API v2, the federal registry maintained by the National Library of Medicine at NIH. Under FDAAA 801, most U.S. drug and device trials must register on ClinicalTrials.gov, making it the most comprehensive source of trial information. Sponsors are required to update trial status within 30 days of a change, but delays occur — always confirm the current status with the trial site before traveling for screening.
How This Page Is Built
The trial list is filtered to ClinicalTrials.gov registrations whose phase field includes Phase 3 and whose condition list includes Spinal Muscular Atrophy. Trial counts and the sponsor leaderboard are computed from the same record set. Plain-English eligibility translations on each linked trial page preserve the original clinical text alongside the accessible version. Read the full methodology for the data pipeline and known limitations.
Source: ClinicalTrials.gov API v2, maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData, Phase 3 Spinal Muscular Atrophy list, May 2026. Data: ClinicalTrials.gov."
Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.
Last updated 2026-05-08 · 6 Phase 3 trials tracked for Spinal Muscular Atrophy.
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