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Muscular Atrophy, Spinal clinical trials

View clinical trials related to Muscular Atrophy, Spinal.

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NCT ID: NCT03779334 Active, not recruiting - Clinical trials for Muscular Atrophy, Spinal

A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Rainbowfish
Start date: August 7, 2019
Phase: Phase 2
Study type: Interventional

A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).

NCT ID: NCT03421977 Active, not recruiting - Clinical trials for Spinal Muscular Atrophy 1

Long-Term Follow-up Study for Patients From AVXS-101-CL-101

START
Start date: September 21, 2017
Phase:
Study type: Observational

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. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years.

NCT ID: NCT03067857 Active, not recruiting - Clinical trials for Amyotrophic Lateral Sclerosis

Autologous Bone Marrow-Derived Stem Cell Therapy for Motor Neuron Disease

Start date: September 2016
Phase: Phase 1/Phase 2
Study type: Interventional

Herein, the investigators study the safety and efficacy of transplanting purified autologous bone marrow-derived stem cells transplanted via the intrathecal route by interventional radiology and the intravenous route.

NCT ID: NCT03032172 Active, not recruiting - Clinical trials for Spinal Muscular Atrophy

A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

Jewelfish
Start date: March 3, 2017
Phase: Phase 2
Study type: Interventional

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.

NCT ID: NCT02386553 Active, not recruiting - Clinical trials for Spinal Muscular Atrophy

A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

NURTURE
Start date: May 18, 2015
Phase: Phase 2
Study type: Interventional

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.

NCT ID: NCT01233817 Active, not recruiting - Clinical trials for Muscular Atrophy, Spinal

Progressive Strength Training in Spinal Muscular Atrophy

Start date: June 2010
Phase: N/A
Study type: Interventional

The proposed feasibility study is necessary to test if children and young adults will participate in and adhere to a 12-week, home-based, supervised progressive strength training exercise program and to obtain preliminary data that will subsequently allow us to determine the safety and impact of strength training in spinal muscular atrophy. Our pilot study will address 3 aims: (1) Ascertain the feasibility of, and potential barriers to, participation in and adherence to a 12-week home-based, supervised, progressive strength training exercise program in children and young adults aged 5-21 years with SMA types II and III; (2) Determine the safety and tolerability of progressive strength training in a pilot study sample of children and young adults with SMA types II and III; and (3) Determine candidate outcome measures.