Clinical trials: Motor Neuron Diseases
Spinal Muscular Atrophy Type 3
Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) are one of the common physical disabilities in childhood. For SMA, progressive muscle weakness and early fatigue hamper the mobility of the sufferers.
- Trial name
- NCT03056144
- Testing
- whole body vibration therapy
- Where
- Hong Kong
- Volunteers
- 1 people
- Sponsor
- The Hong Kong Polytechnic University
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
Breast Cancer
Phase II Trial of SAHA & Tamoxifen for Patients With Breast Cancer
Phase II trial to explore the efficacy of vorinostat and tamoxifen combined.
- Trial name
- NCT00365599
- Testing
- suberoylanilide hydroxamic acid (SAHA, Vorinostat), tamoxifen citrate (Tamoxifen)
- Where
- 8 sites in United States
- Volunteers
- 43 people
- Sponsor
- H. Lee Moffitt Cancer Center and Research Institute
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
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
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, Respiratory Muscle Weakness
ALS and Airway Clearance (ALSAC) Therapy
Patients will receive one of three respiratory therapy interventions for airway clearance assistance: 1) High frequency chest wall oscillation (HFCWO) and mechanical insufflation/exsufflation (MIE), 2) HFCWO or 3) MIE.
- Trial name
- ALSAC (NCT04947436)
- Testing
- High Frequency Chest Wall Oscillation, Mechanical insufflation/exsufflation
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- The University of Texas Health Science Center at San Antonio
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
C9orf72 Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia
Safety and Therapeutic Potential of the FDA-approved Drug Metformin for C9orf72 ALS/FTD
The primary objective is to assess the safety and tolerability of Metformin in subjects with C9orf72 amyotrophic lateral sclerosis administered for 24 weeks.
- Trial name
- NCT04220021
- Testing
- Metformin
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- University of Florida
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
Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 4
Spinal Cord Stimulation in Spinal Muscular Atrophy
Spinal cord stimulation (SCS) has shown remarkable efficacy in restoring motor function in people with spinal cord injury by recruiting afferent input to enhance the responsiveness of spared neural circuits to residual cortical…
- Trial name
- SCSinSMA (NCT05430113)
- Testing
- Spinal Cord Stimulator (octopolar Medtronic Vectris Leads)
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Marco Capogrosso
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III
The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy types II or III; and to determine if the drug has an effect on SMN mRNA and protein levels.
- Trial name
- NPTUNE01 (NCT00439569)
- Testing
- sodium phenylbutyrate
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Westat
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
Small Cell Neuroendocrine Carcinoma, Prostate Cancer
Apalutamide Plus Cetrelimab in Patients With Treatment-Emergent Small Cell Neuroendocrine Prostate Cancer
Despite the low androgen receptor (AR) transcriptional activity of treatment-emergent small cell neuroendocrine prostate cancer, there is persistent AR expression observed in the majority of treatment-emergent small-cell…
- Conditions
- Small Cell Neuroendocrine Carcinoma, Prostate Cancer, Small Cell Carcinoma
- Trial name
- NCT04926181
- Testing
- Apalutamide, Cetrelimab
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Rahul Aggarwal