Clinical trials: Amyotrophic Lateral Sclerosis (ALS)
ALS
Riluzole Oral Soluble Film Safety and Tolerability in Amyotrophic Lateral Sclerosis
The primary objective of this study is to assess the safety and tolerability, with emphasis on the oral cavity, of ROSF (containing riluzole 50mg) in subjects with amyotrophic lateral sclerosis (ALS) administered twice daily for…
- Trial name
- NCT03457753
- Testing
- Riluzole Oral Soluble Film
- Where
- United States
- Sponsor
- Aquestive Therapeutics
Amyotrophic Lateral Sclerosis (ALS)
Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients
This study will evaluate the safety and efficacy of repeated administration of NurOwn® (MSC-NTF cells) therapy, which is based on transplantation of autologous bone marrow derived mesenchymal stromal cells (MSC), which are…
- Trial name
- NCT03280056
- Testing
- NurOwn® (MSC-NTF cells), Bone Marrow aspiration
- Where
- 6 sites in United States
- Volunteers
- 196 people
- Sponsor
- Brainstorm-Cell Therapeutics
Amyotrophic Lateral Sclerosis
Individual Patient Expanded Access IND of Autologous HBadMSCs for the Treatment of Amyotrophic Lateral Sclerosis
The drug for this submission is Hope Biosciences' autologous, adipose-derived culture-expanded mesenchymal stem cells (HB-adMSCs) for the treatment of a single patient with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT04514952
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Amyotrophic Lateral Sclerosis
Rho Kinase Inhibitor in Amyotrophic Lateral Sclerosis (REAL)
A Phase 2a Open-Label Preliminary Safety, Efficacy, and Biomarker Study of WP-0512 in Patients with Amyotrophic Lateral Sclerosis (ALS)
- Trial name
- REAL (NCT05218668)
- Testing
- Fasudil (WP-0512)
- Where
- 12 sites in United States, Australia
- Volunteers
- 40 people
- Sponsor
- Woolsey Pharmaceuticals
Amyotrophic Lateral Sclerosis
Clinical Trial of Ultra-high Dose Methylcobalamin for ALS
To examine the clinical efficacy and safety of ultra-high dose (50mg, im, twice a week) methylcobalamin in retarding the progression of symptoms in amyotrophic lateral sclerosis (ALS) patients, we enroll ALS patients diagnosed by…
- Trial name
- JETALS (NCT03548311)
- Testing
- methylcobalamin, saline solution
- Where
- 16 sites in Japan
- Volunteers
- 130 people
- Sponsor
- Eisai Co., Ltd.
Amyotrophic Lateral Sclerosis (Healthy Volunteers)
Single & Multiple Ascending Dose Study of SAR443820 in Healthy Adult Participants
This is a Phase 1, single-center study conducted in 2 parts: Part 1a, single ascending dose (SAD-TDU16519): Double-blind, randomized, placebo-controlled sequential ascending single oral doses including up to 6 cohorts.
- Trial name
- NCT05795907
- Testing
- SAR443820
- Where
- United States
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- Sanofi
ALS (Amyotrophic Lateral Sclerosis)
Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis
This clinical study is in participants with Amyotrophic Lateral Sclerosis and is designed to evaluate the safety and tolerability of the gene therapy CTx1000.
- Trial name
- KOANEWA (NCT07401121)
- Testing
- AAV9 Gene therapy
- Where
- Australia
- Volunteers
- 15 people
- Sponsor
- Celosia Therapeutics Pty Ltd
Neurologic Disorders, Nervous System Diseases
Neurologic Stem Cell Treatment Study
This is a human clinical study involving the isolation of autologous bone marrow derived stem cells (BMSC) and transfer to the vascular system and inferior 1/3 of the nasal passages in order to determine if such a treatment will…
- Conditions
- Neurologic Disorders, Nervous System Diseases, Neurodegenerative Diseases, Neurological Disorders
- Trial name
- NEST (NCT02795052)
- Testing
- Intravenous and Intranasal BMSC
- Where
- 3 sites in United States, United Arab Emirates
- Volunteers
- 500 people
- Sponsor
- MD Stem Cells
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
A Long-term Extension Study of TCH346 and Placebo Administered Once Daily in Patients With Amyotrophic Lateral Sclerosis(ALS)
This is a study to evaluate the safety and clinical effects of 4 oral doses of TCH346 compared to placebo in patients with mild or mild to moderate stages of ALS.
- Trial name
- NCT00230074
- Testing
- TCH346
- Where
- United States
- Volunteers
- 350 people
- Sponsor
- Novartis
Amyotrophic Lateral Sclerosis
Creation of a Bank of Fibroblast From Patients With Amyotrophic Lateral Sclerosis: Pilot Study
Patients with ALS will be included in the reference center for ALS in hospital La Pitié Salpêtrière, Paris. The study proposes to investigate the pathophysiology of ALS by setting up a fibroblast bank for studying molecular…
- Trial name
- ALSCELL (NCT01639391)
- Testing
- skin biopsy
- Where
- France
- Volunteers
- 19 people
- Sponsor
- Institut Pasteur
Amyotrophic Lateral Sclerosis, Lou Gehrig Disease
MRG-001 in Patients With Amyotrophic Lateral Sclerosis
The proposed study is an Open-Label, Single-Dose Study to Assess the Safety, and Pharmacodynamics (PD) signals of MRG-001 in Patients with Amyotrophic Lateral Sclerosis (ALS).
- Conditions
- Amyotrophic Lateral Sclerosis, Lou Gehrig Disease, Motor Neuron Disease, Motor Neuron Atrophy
- Trial name
- NCT06315608
- Testing
- MRG-001
- Volunteers
- 10 people
- Sponsor
- MedRegen LLC
Amyotrophic Lateral Sclerosis (ALS)
Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis
Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis
- Trial name
- UNITE-ALS (NCT07023835)
- Testing
- 50 mg Usnoflast, 75 mg Usnoflast
- Where
- 49 sites in United States, Australia and 10 more
- Volunteers
- 240 people
- Sponsor
- Zydus Therapeutics Inc.
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult participants with ALS, FTD, or mixed ALS/FTD phenotype with a documented…
- Trial name
- NCT05683860
- Testing
- WVE-004 10 mg Q12W
- Where
- 3 sites in Netherlands, United Kingdom
- Volunteers
- 8 people
- Sponsor
- Wave Life Sciences USA, Inc.
Amyotrophic Lateral Sclerosis Als
Evaluate the Safety and Efficacy of Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis
The goal of the study aims to evaluate the safety and tolerability of a single intrathecal injection of RJK002 in subjects with amyotrophic lateral sclerosis (ALS), and to determine the recommended Phase II dose (RP2D).
- Trial name
- NCT06493279
- Testing
- RJK002 Intrathecal injection
- Where
- China
- Volunteers
- 9 people
- Sponsor
- RJK Biopharma Ltd
Amyotrophic Lateral Sclerosis
A Study of VRG50635 in Participants With Amyotrophic Lateral Sclerosis (ALS)
The primary purpose of this study is to evaluate the safety and tolerability of VRG50635 in participants with ALS.
- Trial name
- NCT06215755
- Testing
- VRG50635
- Where
- 7 sites in Belgium, Canada and 2 more
- Volunteers
- 54 people
- Sponsor
- Verge Genomics
Amyotrophic Lateral Sclerosis (ALS)
A Clinical Trial to Evaluate NB-4746 in Participants With Amyotrophic Lateral Sclerosis.
The purpose of this trial is to learn about the effects of NB-4746 compared with placebo in people with amyotrophic lateral sclerosis.
- Trial name
- NCT07369076
- Testing
- NB-4746 High dose, NB-4746 Low dose, NB-4746 (Dose TBD)
- Where
- 12 sites in Australia, Canada and 2 more
- Volunteers
- 80 people
- Sponsor
- Nura Bio
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Amyotrophic Lateral Sclerosis
A Study Exploring the PK/PD Relationship of QRL-101 in Adults With ALS
This Phase 1 randomized, placebo-controlled, double-blind study will evaluate the PK/PD relationship of single doses of QRL-101 in 12 ALS participants.
- Trial name
- NCT06714396
- Testing
- QRL-101
- Where
- Netherlands
- Volunteers
- 12 people
- Sponsor
- QurAlis Corporation
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