Clinical trials: Spinal Muscular Atrophy Type I
Spinal Muscular Atrophy Type I
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1.
- Trial name
- NCT07070999
- Testing
- GB221
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- Gemma Biotherapeutics
Spinal Muscular Atrophy Type I
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT05614531
- Testing
- EXG001-307 injection
- Where
- 2 sites in China
- Volunteers
- 12 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Spinal Muscular Atrophy
A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA
An Open-label, Long-term Follow-Up Study to Evaluate the Safety and Tolerability of Gene Therapy with EXG001-307 in SMA1 patients ,who joined the parent study (EXG001-307-102)
- Trial name
- NCT06588803
- Testing
- No intervention, only for observational studies
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
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.
- Conditions
- Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
- Trial name
- NCT04042025
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 31 sites in United States, Australia and 7 more
- Volunteers
- 85 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).
- Trial name
- NCT06632730
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Novartis
Muscular Atrophy, Spinal
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA).
- Trial name
- FIREFISH (NCT02913482)
- Testing
- Risdiplam
- Where
- 32 sites in United States, Belgium and 14 more
- Volunteers
- 62 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy Type I
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
This is a Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with spinal muscular atrophy (SMA) Type 1 and who are genetically defined by a biallelic…
- Trial name
- NCT03837184
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 4 sites in Japan, South Korea and 1 more
- Volunteers
- 2 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy (SMA)
A Study of the Safety and Preliminary Efficacy of NKG001 in Pediatric Patients With Type 1 or Type 2 Spinal Muscular Atrophy
This study is a single-center, open-label, single-arm, non-randomized, single-dose, dose-escalation investigator-initiated trial (IIT) designed to evaluate the safety, tolerability, and preliminary efficacy of NKG001 Injection…
- Trial name
- NCT07617779
- Testing
- NKG001
- Where
- China
- Volunteers
- 21 people
- Sponsor
- Nikegen Pharmaceutical (Hangzhou) Company Limited
SMA
Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in patients with spinal muscular atrophy (SMA) Type 1 who meet enrollment criteria and are genetically defined by a…
- Trial name
- STRIVE-EU (NCT03461289)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 10 sites in Belgium, France and 2 more
- Volunteers
- 33 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy 1
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi.
- Trial name
- START (NCT03421977)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy 1
A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I
This is a phase I/II clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atrophy Type I.
- Trial name
- NCT07554924
- Testing
- SKG0201 Injection
- Where
- 3 sites in China
- Volunteers
- 11 people
- Sponsor
- Lanyue Biotech (Hangzhou) Co., Ltd.
Spinal Muscular Atrophy 1
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS-101 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT02122952
- Testing
- AVXS-101
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Novartis Gene Therapies
Muscular Atrophy, Spinal
An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
This expanded access program (EAP) will provide access to risdiplam for eligible participants with Type 1 or Type 2 spinal muscular atrophy (SMA) before it is commercially available in the United States for the indication of SMA.
- Trial name
- NCT04256265
- Testing
- Risdiplam
- Where
- 29 sites in United States
- Sponsor
- Genentech, Inc.
Spinal Muscular Atrophy
An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)
An open-label, multi-part, first-in-human study of oral branaplam in infants with Type 1 spinal muscular atrophy. The purpose of this study was to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD)…
- Trial name
- NCT02268552
- Testing
- branaplam
- Where
- 14 sites in Belgium, Bulgaria and 5 more
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Infantile-onset Spinal Muscular Atrophy
Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
To provide access to nusinersen to eligible patients with Infantile-onset Spinal Muscular Atrophy (SMA) (consistent with Type 1) to address a high-unmet medical need.
- Trial name
- NCT02865109
- Testing
- Nusinersen
- Where
- 7 sites in Colombia, New Zealand and 1 more
- Sponsor
- Biogen
SMA - Spinal Muscular Atrophy, Gene Therapy
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
- Trial name
- STR1VE (NCT03306277)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 16 sites in United States
- Volunteers
- 22 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscle Atrophy
A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
This multicenter, non-interventional, prospective study with a retrospective component aims to evaluate the real-world clinical outcomes, safety profile, and motor function improvements of Risdiplam treatment in participants with…
- Trial name
- NCT07531719
- Where
- 2 sites in Egypt
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.
- Trial name
- NCT05824169
- Testing
- GC101
- Where
- 4 sites in China
- Volunteers
- 18 people
- Sponsor
- GeneCradle Inc
Type I Spinal Muscular Atrophy
Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I
The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy.
- Trial name
- NCT06576388
- Testing
- EXG001-307 injection
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd