Clinical trials: Muscular Atrophy, Spinal
Spinal Muscular Atrophy
A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants
This will be a Phase I, 2-part, open-label, non-randomized study to investigate the safety, tolerability, and pharmacokinetics (PK) of a multiple-dosing regimen of risdiplam (Part 1) and the effect of risdiplam on the PK of…
- Trial name
- NCT03988907
- Testing
- Risdiplam, Midazolam
- Where
- United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
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
A Study of CK-2127107 in Patients With Spinal Muscular Atrophy
This study will evaluate the pharmacodynamic (PD) effect of CK-2127107 (hereafter referred to as reldesemtiv) versus placebo on measures of skeletal muscle function or fatigability in patients with Type II, III, or IV spinal…
- Trial name
- NCT02644668
- Testing
- Reldesemtiv 150 mg, Reldesemtiv 450 mg
- Where
- 19 sites in United States, Canada
- Volunteers
- 70 people
- Sponsor
- Cytokinetics
Spinal Muscular Atrophy
AveXis Managed Access Program Cohort for Access to AVXS-101
The purpose of this Cohort Treatment Protocol will allow access to AVXS-101 for eligible patients diagnosed with SMA.
- Trial name
- NCT03955679
- Testing
- AVXS-101
- Where
- United States
- Sponsor
- United BioSource, LLC
Muscular Atrophy, Spinal
A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)
The purpose of this open-label, single arm study is to further evaluate long-term tolerability, safety and efficacy outcomes of olesoxime in participants with Spinal Muscular Atrophy (SMA) who previously participated in one of…
- Trial name
- NCT02628743
- Testing
- Olesoxime
- Where
- 24 sites in Belgium, France and 5 more
- Volunteers
- 131 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy
A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers
The objective of this study is to assess the safety and tolerability of Risdiplam (RO7034067) in healthy people. The study will assess what the body does to Risdiplam (RO7034067) and what Risdiplam (RO7034067) does to the body.
- Trial name
- NCT02633709
- Testing
- Itraconazole, Risdiplam
- Where
- Netherlands
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Motor Neuron Disease
A Study to Assess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps.
The COMMEND Study will assess the safety and effectiveness of FLX-787 in men and women with Motor Neuron Disease [including Amyotrophic Lateral Sclerosis (ALS), Primary Lateral Sclerosis (PLS) or Progressive Muscular Atrophy…
- Trial name
- NCT03196375
- Testing
- FLX-787-ODT (orally disintegrating tablet)
- Where
- 35 sites in United States
- Volunteers
- 54 people
- Sponsor
- Flex Pharma, Inc.
Muscular Atrophy, Spinal
A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)
This multicenter, randomized, double-blind, 12-week, placebo-controlled multiple dose study will investigate the safety and tolerability of RO6885247 in adult and pediatric patients with spinal muscular atrophy (SMA).
- Trial name
- NCT02240355
- Testing
- RO6885247
- Where
- 26 sites in United States, Canada and 7 more
- Volunteers
- 9 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III Non Ambulant
Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.
Assess the efficacy and the safety of olesoxime in SMA type 2 or type 3 non ambulant patients aged 3-25 years
- Trial name
- NCT01302600
- Testing
- Olesoxime
- Where
- 22 sites in Belgium, France and 5 more
- Volunteers
- 165 people
- Sponsor
- Hoffmann-La Roche
Muscular Atrophy, Spinal
A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis
In this single center study blood samples for biomarker analysis will be collected from patients with spinal muscular atrophy. Up to 21 mL blood will be drawn from eligible patients at a single visit.
- Trial name
- NCT01910168
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- Hoffmann-La Roche
Healthy Volunteer, Muscular Atrophy, Spinal
Ability of Muscle Imaging and Motor Function Measure (MFM) to Detect Changes in Disease Progression in Ambulant Spinal Muscular Atrophy Patients Compared to Healthy Volunteers.
This non-drug, single center, 24-week, longitudinal study in ambulant spinal muscular atrophy (SMA) patients and in age- and gender-matched healthy volunteers will assess the detection of disease progression by magnetic resonance…
- Trial name
- NCT02044029
- Where
- Switzerland
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy (SMA), Carrier Screening
Multi-disease Carrier Screening Test Validation
The purpose of this study is to collect blood samples to enable validation of genetic testing for diseases within a multi-disease carrier screening panel.
- Conditions
- Spinal Muscular Atrophy (SMA), Carrier Screening, Genetic Testing
- Trial name
- NCT01663584
- Testing
- Blood draw
- Where
- United States
- Sponsor
- Natera, Inc.