Clinical trials: Motor Neuron Disorder
- Condition Motor Neuron Disorder
- Funder type Industry (pharma, biotech, devices)
- Has posted results yes
Spinal Muscular Atrophy
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in pre-symptomatic patients with SMA and 2 or 3 copies SMN2
- Trial name
- SPR1NT (NCT03505099)
- Testing
- onasemnogene abeparvovec-xioi
- Where
- 16 sites in United States, Australia and 4 more
- Volunteers
- 30 people
- Sponsor
- Novartis Gene Therapies
Amyotrophic Lateral Sclerosis
A Biomarker Study to Evaluate MN-166 in Subjects With Amyotrophic Literal Sclerosis (ALS)
This is a multi-center, open-label study of MN-166 (ibudilast) in subjects with ALS. To be eligible subjects must meet the El Escorial criteria of possible, laboratory-supported probable, probable, or definite criteria for a…
- Trial name
- NCT02714036
- Testing
- ibudilast
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- MediciNova
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
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
Safety and Efficacy of TRO19622 as add-on Therapy to Riluzole Versus Placebo in Treatment of Patients Suffering From ALS
The purpose of the assay is to assess the safety and the efficacy of TRO19622 330 mg QD as add-on therapy to riluzole 50 mg bid in the treatment of patients suffering from ALS, as compared to placebo, assessed by the 18-month…
- Trial name
- MITOTARGET (NCT00868166)
- Testing
- Olesoxime, Riluzole
- Where
- 15 sites in Belgium, France and 3 more
- Volunteers
- 512 people
- Sponsor
- Hoffmann-La Roche
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, 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 Study to Evaluate, Safety, Tolerability, and PK of AP-101 in Participants With ALS
The purpose of this study is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.
- Trial name
- NCT05039099
- Testing
- AP-101
- Where
- 13 sites in United States, Belgium and 4 more
- Volunteers
- 73 people
- Sponsor
- AL-S Pharma
Amyotrophic Lateral Sclerosis
Phase 3 Study of Dexpramipexole in ALS
The purpose of this study is to determine whether dexpramipexole (150 mg twice daily) is safe and effective in the treatment of Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- EMPOWER (NCT01281189)
- Testing
- Dexpramipexole
- Where
- 82 sites in United States, Australia and 9 more
- Volunteers
- 942 people
- Sponsor
- Knopp Biosciences
Spinal Muscular Atrophy
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA).
- Trial name
- NCT01839656
- Testing
- nusinersen
- Where
- 4 sites in United States, Canada
- Volunteers
- 21 people
- Sponsor
- Biogen
Type 2 Spinal Muscular Atrophy
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
This was a Phase III multi-center, single dose (1.2 x 10^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never…
- Trial name
- STEER (NCT05089656)
- Testing
- OAV101
- Where
- 42 sites in United States, Brazil and 11 more
- Volunteers
- 126 people
- Sponsor
- Novartis Pharmaceuticals
Amyotrophic Lateral Sclerosis
An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)
The primary objectives of Parts A and B of this study are to evaluate the safety, tolerability, and pharmacokinetics (PK) of ascending doses of tofersen in adults with ALS and a documented superoxide dismutase 1 (SOD1) mutation.
- Trial name
- VALOR (Part C) (NCT02623699)
- Testing
- Tofersen
- Where
- 40 sites in United States, Australia and 10 more
- Volunteers
- 176 people
- Sponsor
- Biogen
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
A Study of NIDO-361 in Patients With SBMA
The purpose of this study is to evaluate the safety, tolerability, and efficacy of NIDO-361 in adult patients with Spinal and Bulbar Muscular Atrophy (SBMA).
- Trial name
- PIONEER KD (NCT06411912)
- Testing
- NIDO-361
- Where
- 5 sites in Denmark, Italy and 2 more
- Volunteers
- 54 people
- Sponsor
- Nido Biosciences, 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 of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
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)…
- Trial name
- Jewelfish (NCT03032172)
- Testing
- Risdiplam
- Where
- 24 sites in United States, Belgium and 7 more
- Volunteers
- 174 people
- Sponsor
- Hoffmann-La Roche
Stroke, Muscle Spasticity
Safety Study of Botulinum Toxin Type A in Post-Upper Limb Stroke Patients With Reduced Lung Function
The purpose of this study is to evaluate the safety of injections of botulinum toxin Type A in patients with reduced lung function and focal upper limb poststroke spasticity
- Conditions
- Stroke, Muscle Spasticity, Motor Neuron Disease
- Trial name
- NCT00076687
- Testing
- botulinum toxin Type A, saline
- Where
- 4 sites in United States, Czechia and 2 more
- Volunteers
- 155 people
- Sponsor
- Allergan
Amyotrophic Lateral Sclerosis
Study to Investigate the Long-term Safety of FAB122 in Patients With Amyotrophic Lateral Sclerosis
A multicenter, open-label extension study to investigate the long-term safety of FAB122 in patients with Amyotrophic Lateral Sclerosis
- Trial name
- ADOREXT (NCT05866926)
- Testing
- FAB122
- Where
- Spain
- Volunteers
- 201 people
- Sponsor
- Ferrer Internacional S.A.
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
Amyotrophic Lateral Sclerosis
Efficacy and Safety of Plasma Exchange With Albutein® 5% in Participants With Amyotrophic Lateral Sclerosis
This is a pilot, phase 2, prospective, open-label, single-arm study to evaluate disease progression, forced vital capacity, and the safety and tolerability of plasma exchange (PE) using Albutein® 5% in participants with…
- Trial name
- NCT02872142
- Testing
- Albutein 5%, Plasma Exchange
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Grifols Therapeutics LLC
Muscular Atrophy, Spinal
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)
This was a phase IV Open-label, single-arm, single-dose, multicenter study, to evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with SMA with bi-allelic mutations in…
- Trial name
- OFELIA (NCT05073133)
- Testing
- OAV101
- Where
- 5 sites in Argentina, Brazil
- Volunteers
- 16 people
- Sponsor
- Novartis Pharmaceuticals