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
Impact of Nuedexta on Bulbar Physiology and Function in ALS
Nuedexta is FDA approved for the treatment of pseudobulbar affect in ALS patients and anecdotal reports of improvements in speech, salivation or swallowing have been reported.
- Trial name
- NCT03883581
- Testing
- dextromethorphan HBr and quinidine sulfate
- Where
- 2 sites in United States
- Volunteers
- 28 people
- Sponsor
- University of Florida
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
Geometrical Facial Deformation According to Posture in Amyotrophic Lateral Sclerosis
The measurement of the facial deformation according to the body posture will be conducted by 3D scanning for each posture after marking the landmarks on the face.
- Trial name
- NCT04798183
- Testing
- 3-dimensional scan
- Where
- South Korea
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Pusan National University Yangsan Hospital
Amyotrophic Lateral Sclerosis
The Effect of RNS60 on ALS Biomarkers
Amyotrophic Lateral Sclerosis (ALS) is a rare lethal neurodegenerative disease involving inflammation. Riluzole, the only drug for ALS, improves median survival by 3 months. This prompts new treatments of ALS.
- Trial name
- RNS60 (NCT03456882)
- Testing
- RNS60
- Where
- 23 sites in United States, Italy
- Volunteers
- 147 people
- Sponsor
- Mario Negri Institute for Pharmacological Research
Amyotrophic Lateral Sclerosis
HEALEY ALS Platform Trial - Regimen A Zilucoplan
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.
- Trial name
- NCT04436497
- Testing
- Zilucoplan
- Where
- United States
- Volunteers
- 162 people
- Sponsor
- Merit E. Cudkowicz, MD
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 (ALS)
Trial of Safety and Efficacy of Rasagiline in Patients With Amyotrophic Lateral Sclerosis (ALS)
ALS is a disorder that weakens motor strength and lung function. Rapid loss of motor neurons in the brain and spinal cord of ALS patients causes the symptoms of increasing weakness and loss of muscle function.
- Trial name
- NCT01232738
- Testing
- rasagiline
- Where
- 10 sites in United States, Canada
- Volunteers
- 36 people
- Sponsor
- Yunxia Wang, MD
Amyotrophic Lateral Sclerosis
HEALEY ALS Platform Trial - Regimen F ABBV-CLS-7262
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.
- Trial name
- NCT05740813
- Testing
- ABBV-CLS-7262 Dose 1, ABBV-CLS-7262 Dose 2
- Where
- United States
- Volunteers
- 310 people
- Sponsor
- Merit E. Cudkowicz, MD
Familial Amyotrophic Lateral Sclerosis
SOD1 Inhibition by Pyrimethamine in Familial Amyotrophic Lateral Sclerosis (ALS)
The objective of this study will be to evaluate the safety, tolerability and effect on SOD1 levels by pyrimethamine in patients with familial amyotrophic lateral sclerosis.
- Trial name
- NCT01083667
- Testing
- Pyrimethamine
- Where
- 5 sites in United States, Germany and 2 more
- Volunteers
- 32 people
- Sponsor
- Weill Medical College of Cornell University
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
Muscular Atrophy, Spinal
A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Types II and III Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Types II and III SMA; and…
- Trial name
- NCT00568802
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Stanford University
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.
Muscular Atrophy, Spinal
A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Type I Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Type I SMA; and to detect the…
- Trial name
- NCT00568698
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Stanford University
Spinal Muscular Atrophy (SMA)
Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development
Spinal muscular atrophy (SMA) is the leading genetic cause of death of infants. Strong preclinical evidence suggests that effective therapy must be delivered as early as possible to prevent progression of the disease.
- Trial name
- NCT01736553
- Where
- 15 sites in United States
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- Ohio State University