Clinical trials: Neuron Disease, Motor
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)
A Phase Ⅰ/Ⅱa Study of SNUG01 in Adult Subjects With ALS
The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy of SNUG01 in in adult subjects with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT07169175
- Testing
- SNUG01
- Where
- 4 sites in United States, China
- Volunteers
- 21 people
- Sponsor
- SineuGene Therapeutics Co., Ltd.
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
Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment
The Phoenix Trial is a randomized double blind placebo controlled Phase III trial to evaluate the safety and efficacy of AMX0035 for treatment of ALS
- Trial name
- Phoenix (NCT05021536)
- Testing
- AMX0035
- Where
- 69 sites in United States, Belgium and 10 more
- Volunteers
- 664 people
- Sponsor
- Amylyx Pharmaceuticals Inc.
ALS (Amyotrophic Lateral Sclerosis)
Intermediate-size Patient Population Expanded Access Protocol
The purpose of this Expanded Access Program is to provide tazbentetol to ALS patients who are not eligible to enroll in an ALS clinical trial.
- Trial name
- NCT07088159
- Testing
- Tazbentetol
- Where
- 16 sites in United States
- Sponsor
- Spinogenix
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, Spinal Muscular Atrophy Type 3
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved…
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- SAPPHIRE (NCT05156320)
- Testing
- Apitegromab
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 188 people
- Sponsor
- Scholar Rock, Inc.
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
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
Amyotrophic Lateral Sclerosis
Evaluation the Efficacy and Safety of Mutiple Lenzumestrocel (Neuronata-R® Inj.) Treatment in Patients With ALS
ALSUMMIT is a double-blind, randomized, placebo-controlled, multi-center, parallel, phase III clinical trial to evaluate and confirm the efficacy and long-term safety of repeated Lenzumestrocel (Neuronata-R® inj.) treatment.
- Trial name
- ALSummit (NCT04745299)
- Testing
- Lenzumestrocel, Riluzole
- Where
- 5 sites in South Korea
- Volunteers
- 123 people
- Sponsor
- Corestemchemon, Inc.
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
Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS
The goal of this clinical trial is to learn if the drug pridopidine works to treat amyotrophic lateral sclerosis in adults. It will also help to learn about the safety of pridopidine.
- Trial name
- PREVAiLS (NCT07322003)
- Testing
- Pridopidine
- Where
- 56 sites in United States, Belgium and 11 more
- Volunteers
- 500 people
- Sponsor
- Prilenia
Amyotrophic Lateral Sclerosis (ALS)
A Follow up Study to Protocol 101/2 - Continued Treatment by IPL344 IV
This is a prospective, open-label, follow up study to protocol 101/2 - continued treatment by IPL344 IV administered once a day in up to 15 participants with ALS.
- Trial name
- NCT03755167
- Testing
- IPL344
- Where
- 2 sites in Israel
- Volunteers
- 15 people
- Sponsor
- Immunity Pharma Ltd.
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.
Amyotrophic Lateral Sclerosis
Efficacy and Safety of Tazbentetol in ALS Participants
The objectives of this study are to examine the effects of tazbentetol on clinical measures of ALS, patient reported outcomes (PROs), long-term safety and tolerability.
- Trial name
- NCT07325591
- Testing
- Tazbentetol
- Volunteers
- 430 people
- Sponsor
- Spinogenix
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
Amyotrophic Lateral Sclerosis
An Open-Label Extension for the Phase 2 Study in Early Symptomatic Amyotrophic Lateral Sclerosis Patients on Stable Background Therapy to Assess Bioenergetic Catalysis With CNM-Au8 to Slow Disease Progression in ALS
This is an optional open-label extension to participants that have completed the clinical trial CNMAu8.205.
- Trial name
- NCT05299658
- Testing
- CNMAu8
- Where
- 2 sites in Australia
- Volunteers
- 40 people
- Sponsor
- Clene Nanomedicine
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