Clinical trials: Neuron Disease, Motor
- Condition Neuron Disease, Motor
- Funder type Industry (pharma, biotech, devices)
- Has posted results yes
Muscular Atrophy, Spinal
A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function
This is a multi-center, open-label, non-randomized, parallel-group, 2-part study to evaluate the effect of hepatic impairment on the PK and safety and tolerability of a single oral dose of risdiplam compared to matched healthy…
- Trial name
- NCT03920865
- Testing
- Risdiplam
- Where
- 3 sites in United States
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Amyotrophic Lateral Sclerosis, ALS
An Efficacy and Safety Study of Ravulizumab in ALS Participants
The purpose of the study is to assess the efficacy and safety of ravulizumab for the treatment of adult participants with ALS.
- Trial name
- NCT04248465
- Testing
- Ravulizumab
- Where
- 95 sites in United States, Australia and 15 more
- Volunteers
- 382 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Amyotrophic Lateral Sclerosis
Effects of Oral Levosimendan on Respiratory Function in Patients With Amyotrophic Lateral Sclerosis (ALS): Open-Label Extension
This study provides an opportunity for subjects in the REFALS (3119002; NCT03505021) study to continue treatment with oral levosimendan.
- Trial name
- REFALS-ES (NCT03948178)
- Testing
- Levosimendan
- Where
- 79 sites in United States, Australia and 12 more
- Volunteers
- 227 people
- Sponsor
- Orion Corporation, Orion Pharma
Spinal Muscular Atrophy
Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
The purpose of this trial is to evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with Spinal Muscular Atrophy with 3 copies of SMN2 and deletion of SMN1.
- Trial name
- STRONG (NCT03381729)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 11 sites in United States
- Volunteers
- 32 people
- Sponsor
- Novartis Gene Therapies
Amyotrophic Lateral Sclerosis
Study to Investigate the Efficacy and Safety of FAB122 (Daily Oral Edaravone) in Patients With Amyotrophic Lateral Sclerosis
Multicenter, multinational, double-blind, randomized (2:1), placebo-controlled Phase III study to investigate the efficacy and safety of 100 mg FAB122 once daily as oral formulation in ALS patients.
- Trial name
- ADORE (NCT05178810)
- Testing
- FAB122
- Where
- 38 sites in Belgium, France and 9 more
- Volunteers
- 313 people
- Sponsor
- Ferrer Internacional S.A.
Amyotrophic Lateral Sclerosis
Study of Ozanezumab (GSK1223249) Versus Placebo in the Treatment of Amyotrophic Lateral Sclerosis
This is a 48-week, randomised, multi-centre, double-blind, placebo-controlled, parallel group investigation of the efficacy and safety of intravenous (IV) ozanezumab (GSK1223249) compared to placebo in subjects with Amyotrophic…
- Trial name
- NCT01753076
- Testing
- Ozanezumab
- Where
- 35 sites in United States, Australia and 9 more
- Volunteers
- 304 people
- Sponsor
- GlaxoSmithKline
Amyotrophic Lateral Sclerosis
A 6-Month Extension Study to Assess the Long-Term Safety of Engensis in Amyotrophic Lateral Sclerosis
The purpose of this study is to evaluate the long-term safety of intramuscular administration of Engensis in Participants with Amyotrophic Lateral Sclerosis who were previously randomized, received treatment, and completed the…
- Trial name
- REViVALS-1B (NCT05176093)
- Testing
- Engensis
- Where
- 2 sites in United States, South Korea
- Volunteers
- 8 people
- Sponsor
- Helixmith Co., Ltd.
Pseudobulbar Affect (PBA)
Safety and Efficacy of AVP-923 in PBA Patients With ALS or MS
Objectives of the study are to evaluate the safety, tolerability, and efficacy of two different doses of AVP-923 (capsules containing either 30 mg of dextromethorphan hydrobromide and 10 mg of quinidine sulfate [AVP-923-30] or 20…
- Trial name
- STAR (NCT00573443)
- Testing
- dextromethorphan hydrobromide 20 mg and quinidine sulfate 10 mg, dextromethorphan hydrobromide 30 mg and quinidine sulfate 10 mg
- Where
- 62 sites in United States, Argentina and 1 more
- Volunteers
- 326 people
- Sponsor
- Avanir Pharmaceuticals
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
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.
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
Amyotrophic Lateral Sclerosis, ALS
Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis
The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow-derived stem cells("HYNR-CS inj"), through intrathecal delivery for the treatment in patients with ALS. This study consists of 2 steps.
- Trial name
- NCT01363401
- Testing
- HYNR-CS inj, Control group
- Where
- South Korea
- Volunteers
- 72 people
- Sponsor
- Corestemchemon, Inc.
Amyotrophic Lateral Sclerosis (ALS)
Safety Extension Study of Oral Edaravone Administered in Subjects With Amyotrophic Lateral Sclerosis (ALS)
This is a Phase 3, international, multicenter, open-label, long-term extension study. The primary objective of this study is to evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral…
- Trial name
- NCT04577404
- Testing
- MT-1186
- Where
- 44 sites in United States, Canada and 4 more
- Volunteers
- 124 people
- Sponsor
- Shionogi
Spinal Muscular Atrophy
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
The primary objective of the study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally (IT) to participants with infantile-onset with infantile-onset spinal muscular atrophy (SMA).
- Trial name
- ENDEAR (NCT02193074)
- Testing
- nusinersen
- Where
- 31 sites in United States, Australia and 11 more
- Volunteers
- 122 people
- Sponsor
- Biogen
Amyotrophic Lateral Sclerosis
Efficacy and Safety of Plasma Exchange With Albumin in Patients With Amyotrophic Lateral Sclerosis
Pilot, phase II, prospective, open-label, uncontrolled study of plasma exchange with 5% albumin in 10 subjects having a definite, possible, or probable diagnosis of Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT02479802
- Testing
- Albumin
- Where
- Spain
- Volunteers
- 13 people
- Sponsor
- Instituto Grifols, S.A.
Amyotrophic Lateral Sclerosis
Safety and Tolerability Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS)
This was a 2-part study of dexpramipexole in patients with ALS. Part 1 was a randomized, placebo-controlled, multi-center study to evaluate the safety, tolerability, and clinical effects of oral administration of 3 dosage levels…
- Trial name
- CL201 (NCT00647296)
- Testing
- Dexpramipexole 50 mg/day, Dexpramipexole 150 mg/day, Dexpramipexole 300 mg/day
- Where
- 21 sites in United States
- Volunteers
- 194 people
- Sponsor
- Knopp Biosciences
Amyotrophic Lateral Sclerosis, ALS
Study of Safety, Tolerability, and Biological Activity of LAM-002A in C9ORF72-Associated Amyotrophic Lateral Sclerosis
This is a clinical trial to evaluate the safety, tolerability, and biological effect of LAM-002A in adults with C9ORF72-associated ALS (C9ALS).
- Trial name
- NCT05163886
- Testing
- LAM-002A
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- IRulya Therapeutics Inc.
Amyotrophic Lateral Sclerosis, ALS
Open Label Extension Study of AMX0035 in Patients With ALS
This extension study, in which all participants received active treatment (AMX0035), was designed to assess the longer-term safety and therapeutic potential of AMX0035 for participants who have completed the Main Study (AMX3500…
- Trial name
- CENTAUR-OLE (NCT03488524)
- Testing
- AMX0035
- Where
- 24 sites in United States
- Volunteers
- 90 people
- Sponsor
- Amylyx Pharmaceuticals Inc.
Amyotrophic Lateral Sclerosis
A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)
The purpose of this study is to assess the long-term safety and tolerability of reldesemtiv in patients with ALS who have successfully completed dosing in the Phase 3 clinical trial, CY 5031 (also known as COURAGE-ALS)
- Trial name
- NCT05442775
- Testing
- Reldesemtiv
- Where
- 41 sites in United States, Australia and 7 more
- Volunteers
- 71 people
- Sponsor
- Cytokinetics
Spinal Muscular Atrophy
A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
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…
- Trial name
- NURTURE (NCT02386553)
- Testing
- Nusinersen
- Where
- 21 sites in United States, Australia and 5 more
- Volunteers
- 25 people
- Sponsor
- Biogen