Clinical trials: Neuromuscular Manifestations
Muscular Diseases, Neuromuscular Manifestations
Study of Anitocabtagene Autoleucel in Participants With Non-Oncology Plasma Cell-Related Diseases
The goal of this clinical study is to learn more about the study drug Anitocabtagene autoleucel (anito-cel), and its safety, tolerability, dosing, and preliminary effectiveness in participants with generalized myasthenia gravis…
- Conditions
- Muscular Diseases, Neuromuscular Manifestations, Autoimmune, Autoimmune Diseases
- Trial name
- NCT06626919
- Testing
- anito-cel, Cyclophosphamide, Fludarabine
- Where
- 14 sites in United States
- Volunteers
- 30 people
- Sponsor
- Kite, A Gilead Company
Spinal Muscular Atrophy, SMA
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones…
- Conditions
- Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- OPAL (NCT07047144)
- Testing
- Apitegromab, Nusinersen, Risdiplam
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 52 people
- Sponsor
- Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- ONYX (NCT05626855)
- Testing
- Apitegromab
- Where
- 49 sites in United States, Belgium and 7 more
- Volunteers
- 238 people
- Sponsor
- Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved…
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- SAPPHIRE (NCT05156320)
- Testing
- Apitegromab
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 188 people
- Sponsor
- Scholar Rock, Inc.
Myasthenia Gravis, Neuromuscular Manifestations
Expediting Myasthenia Gravis (MG) Diagnostic Evaluation: A Novel, Proof-of-Concept for Undiagnosed, Symptomatic Patients That Uses Social Media Targeting and Self-Assessment
This is a fully remote, site-less, prospective, observational study enrolling adults in the United States (excluding U.S. territories) with undiagnosed neuromuscular symptoms.
- Conditions
- Myasthenia Gravis, Neuromuscular Manifestations, Neuromuscular Diseases
- Trial name
- NCT06381284
- Testing
- Social media recruitment, Self-Assessment, Follow-up
- Where
- United States
- Volunteers
- 215 people, healthy volunteers welcome
- Sponsor
- ZS Associates
Myasthenia Gravis, Neuromuscular Disease
Validation and Scaling of Screening Program for Undiagnosed Myasthenia Gravis-Social Media Campaign Paired With a Self-moderated Assessment
This study expands and validates the pilot study NCT06381284. It is a fully remote, site-less, prospective, observational study enrolling adults in the United States (excluding U.S.
- Conditions
- Myasthenia Gravis, Neuromuscular Disease, Neuromuscular Manifestations
- Trial name
- NCT06866483
- Testing
- Social media recruitment, Self-Assessment, Follow-up
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- ZS Associates
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- TOPAZ (NCT03921528)
- Testing
- SRK-015
- Where
- 16 sites in United States, Italy and 2 more
- Volunteers
- 58 people
- Sponsor
- Scholar Rock, Inc.