Clinical trials: Neuron Disease, Motor
Spinal Muscular Atrophy
A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).
The primary objective of Part 1 of this study is to assess the safety and tolerability of Nusinersen in participants with SMA who are not eligible to participate in the clinical studies ISIS 396443-CS3B (NCT02193074) or ISIS…
- Trial name
- EMBRACE (NCT02462759)
- Testing
- Nusinersen
- Where
- 7 sites in United States, Germany
- Volunteers
- 21 people
- Sponsor
- Biogen
Amyotrophic Lateral Sclerosis (ALS)
Humanitarian Device Exemption Post-Approval Study of NeuRx Diaphragm Pacing System for Amyotrophic Lateral Sclerosis
This post-approval study will follow 60 participants who have ALS, documented chronic hypoventilation, and bilateral phrenic nerve function, and who undergo the surgical implantation procedure to receive the NeuRx Diaphragm…
- Trial name
- NCT01605006
- Testing
- NeuRx Diaphragm Pacing System (DPS)
- Where
- 11 sites in United States
- Volunteers
- 97 people
- Sponsor
- Synapse Biomedical
Spinal Muscular Atrophy (SMA)
Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy
The primary objective of the study is to evaluate anxiety level during intrathecal administration (IT) under standard of care (SOC) and virtual reality (VR) conditions using a reliable self-rating scale.
- Trial name
- REALITY (NCT05354414)
- Testing
- Virtual reality, Standard of Care
- Where
- 12 sites in France
- Volunteers
- 63 people
- Sponsor
- Biogen
Amyotrophic Lateral Sclerosis
A Substudy of LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis
The main purpose of this study is to assess the long-term safety and tolerability of LY4256984 in participants with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT07571174
- Testing
- LY4256984
- Where
- 11 sites in Belgium, Canada and 3 more
- Volunteers
- 32 people
- Sponsor
- Eli Lilly and Company
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
Autologous Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF), in Patients With Amyotrophic Lateral Sclerosis (ALS)
The study will evaluate the safety, tolerability and therapeutic effects of transplantation of escalating doses of autologous cultured mesenchymal bone marrow stromal cells secreting neurotrophic factors (MSC-NTF), in patients…
- Trial name
- NCT01777646
- Testing
- MSC_NTF cells transplantation by multiple intramuscular injections at 24 separate sites, in addition to a single intrathechal injection into the CSF
- Where
- Israel
- Volunteers
- 14 people
- Sponsor
- Brainstorm-Cell Therapeutics
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
A Compassionate Use Protocol of AMX0035 for Treatment of Patients With Amyotrophic Lateral Sclerosis (ALS)
The protocol is intended to provide extended treatment with AMX0035 to patients who previously participated in an Amylyx sponsored study of AMX0035 for ALS.
- Trial name
- NCT04516096
- Testing
- AMX0035
- Where
- 5 sites in United States
- Volunteers
- 28 people
- Sponsor
- Amylyx Pharmaceuticals Inc.
Foot Drop, Thigh Muscles Weakness (Hamstrings or Quadriceps)
Evaluation of the Safety and Performance of the NESS L300 Plus System
The study is designed to evaluate the safety and efficacy of using the L300 Plus as a neuroprosthesis device for walking in patients following an UMN injury or disease.
- Conditions
- Foot Drop, Thigh Muscles Weakness (Hamstrings or Quadriceps), Upper Motor Neuron Injury or Disease
- Trial name
- L300Plus (NCT01237860)
- Testing
- NESS L300 Plus System
- Where
- Israel
- Volunteers
- 45 people
- Sponsor
- Bioness Inc
Spinal Muscular Atrophy
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)
The primary objective of this study is to examine the safety and tolerability of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA) who previously participated in ISIS…
- Trial name
- NCT01780246
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- Biogen
Amyotrophic Lateral Sclerosis
Study to Investigate the Safety of the Transplantation (by Injection) of Human Glial Restricted Progenitor Cells (hGRPs; Q-Cells®) Into Subjects With Amyotrophic Lateral Sclerosis (ALS)
This study is a non-randomized, open-label, partially blinded, sequential cohort, dose-escalation study designed to obtain preliminary data on the safety, tolerability, and early efficacy of Q-Cells® transplantation in subjects…
- Trial name
- NCT02478450
- Testing
- Q-Cells
- Volunteers
- 30 people
- Sponsor
- Q Therapeutics, Inc.
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
Amyotrophic Lateral Sclerosis (ALS)
A Phase II Multi-centre, Extension Study to Investigate the Long Term Safety of ONO-2506PO in Patients Diagnosed With Amyotrophic Lateral Sclerosis (ALS)
The objective of this study is to investigate the long term safety of ALS patients taking ONO-2506PO.
- Trial name
- NCT00694941
- Testing
- ONO-2506PO
- Where
- 25 sites in Belgium, France and 5 more
- Volunteers
- 15 people
- Sponsor
- Ono Pharmaceutical Co., Ltd.
Amyotrophic Lateral Sclerosis
Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis
This is a first-in-human trial of spinal derived stem cells transplanted into the spinal cord of patients with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- ALS (NCT01348451)
- Testing
- surgical implantation
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Neuralstem Inc.
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients
This is a phase 1/2a randomized, double-blind study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of study drug AJ201 in subjects with Spinal and Bulbar Muscular Atrophy (SBMA).
- Trial name
- NCT05517603
- Testing
- AJ201
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- AnnJi Pharmaceutical Co., Ltd.
Drop Foot, Muscle Weakness
Evaluation of Usability and Human Factors in the Novus System
The Novus system intended to provide ankle dorsiflexion and knee flexion or extension in individuals with foot drop and thigh muscle weakness, following an upper motor neuron injury or disease.
- Conditions
- Drop Foot, Muscle Weakness, Motor Neuron Disease, Upper
- Trial name
- NCT02738242
- Testing
- Novus system
- Where
- Israel
- Volunteers
- 16 people
- Sponsor
- Bioness Neuromodulation
Spinal Muscular Atrophy Type I
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT05614531
- Testing
- EXG001-307 injection
- Where
- 2 sites in China
- Volunteers
- 12 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Muscular Dystrophies, Spinal Muscular Atrophy
Schulze Muscular Dystrophy Ability Clinical Study
The primary objective of the Schulze study is to evaluate the function of the upper limbs of subjects diagnosed with neuromuscular disorders, with and without use of the Abilitech Assist device in the clinic and home…
- Conditions
- Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT05409079
- Testing
- Abilitech Assist
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- AbiliTech Medical Inc.
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2…
- Trial name
- HINALEA 1 (NCT05861986)
- Testing
- Risdiplam
- Where
- 16 sites in United States, Germany and 2 more
- Volunteers
- 28 people
- Sponsor
- Hoffmann-La Roche
Amyotrophic Lateral Sclerosis
Single-Ascending-Dose Safety/Tolerability of NP001 in Amyotrophic Lateral Sclerosis (ALS)
Primary objectives: To assess the safety and tolerability of ascending doses of NP001 compared to placebo in subjects with ALS. Secondary objective: To explore the effects of NP001 on biomarkers potentially relevant to ALS.
- Trial name
- NCT01091142
- Testing
- NP001
- Where
- 3 sites in United States
- Volunteers
- 56 people
- Sponsor
- Neuraltus Pharmaceuticals, Inc.