Clinical trials: Motor Neuron Disorder
Metastatic Breast Cancer
Efficacy Evaluation of Enobosarm Monotherapy in Treatment of AR+/ER+/HER2- Metastatic Breast Cancer
To demonstrate the efficacy of enobosarmin the treatment of androgen receptor positive (AR+) and estrogen receptor positive (ER+) metastatic breast cancer (MBC) as measured by radiographic progression free survival (rPFS).
- Trial name
- ARTEST (NCT04869943)
- Testing
- Enobosarm, Exemestane
- Where
- 54 sites in United States, Poland and 2 more
- Volunteers
- 52 people
- Sponsor
- Veru Inc.
Primary Lateral Sclerosis, Adult, 1
"Individual Patient Expanded Access IND to Treat Primary Lateral Sclerosis"
This Individual Patient Expanded Access IND has been created as requested by an 83-year-old man who suffers Primary Lateral Sclerosis and for which the drugs currently approved are not providing an improvement over the…
- Trial name
- NCT04825613
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Amyotrophic Lateral Sclerosis
A Study to Evaluate the Safety, Tolerability and Efficacy of ILB in Patients With Amyotrophic Lateral Sclerosis
This is a Phase 2a single-centre, open single-arm study in patients with Amyotrophic Lateral Sclerosis (ALS) of intermediate progression rate. Eligible subjects will be administered weekly doses of ILB.
- Trial name
- NCT03613571
- Testing
- ILB
- Where
- Sweden
- Volunteers
- 13 people
- Sponsor
- TikoMed AB
Amyotrophic Lateral Sclerosis (ALS)
Efficacy and Safety Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis (ALS) Who Met Severity Classification III
The primary objective of this study is to evaluate the efficacy of 60mg of MCI-186 via intravenous drip once a day in patients with ALS whose severity is classified as grade III, based on the changes in the revised ALS functional…
- Trial name
- NCT00415519
- Testing
- MCI-186
- Volunteers
- 25 people
- Sponsor
- Shionogi
ALS (Amyotrophic Lateral Sclerosis)
Ciprofloxacin/Celecoxib Combination in Patients With ALS
This is an open label, off label study, to provide interested ALS patients with Ciprofloxacin/Celecoxib fixed dose combination, while assessing safety and tolerability, routine disease progression measures (ALSFRS-R and Vital…
- Trial name
- NCT04090684
- Testing
- Fixed dose combination Ciprofloxacin/Celecoxib
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- NeuroSense Therapeutics Ltd.
Amyotrophic Lateral Sclerosis
Dose Titration Study to Test Safety and Effects of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
A Phase II, double-blind, randomized, placebo-controlled ascending dose titration study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamic effects of multiple ascending doses of CK-2017357 to an individual…
- Trial name
- NCT01486849
- Testing
- CK-2017357, Riluzole 50 MG
- Where
- 11 sites in United States
- Volunteers
- 27 people
- Sponsor
- Cytokinetics
Muscular Atrophy, Spinal
A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy
The aim of this study was to evaluate treatment patterns, SMA-related complications and services, and all-cause medical encounters of patients with SMA receiving multiple DMTs in the real world.
- Trial name
- NCT07403214
- Where
- United States
- Volunteers
- 4,805 people
- Sponsor
- Novartis Pharmaceuticals
Amyotrophic Lateral Sclerosis (ALS)
A Long-Term Study in Patients With Amyotrophic Lateral Sclerosis (ALS)
The purpose of this study is to investigate the safety and efficacy of long-term E0302 administration in patients with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT00445172
- Testing
- E0302 (mecobalamin)
- Where
- 42 sites in Japan
- Volunteers
- 135 people
- Sponsor
- Eisai Co., Ltd.
Spinal Muscular Atrophy
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3
- Trial name
- SYNAPSE-SMA (NCT05794139)
- Testing
- NMD670
- Where
- 25 sites in United States, Belgium and 6 more
- Volunteers
- 52 people
- Sponsor
- NMD Pharma A/S
ALS, Amyotrophic Lateral Sclerosis
Clinical Trial - Analyzing Participation Experiences Of Amyotrophic Lateral Sclerosis Patients
Clinical trial participation has always been substantially skewed toward certain demographic groups. However, there has been little study on whether trial qualities impact participation in either a positive or negative way.
- Trial name
- NCT05423678
- Volunteers
- 500 people
- Sponsor
- Power Life Sciences Inc.
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.
Type I Spinal Muscular Atrophy
Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I
The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy.
- Trial name
- NCT06576388
- Testing
- EXG001-307 injection
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
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
Familial Amyotrophic Lateral Sclerosis
Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Caused by SOD1 Gene Mutations
This study will test the safety, tolerability and pharmacokinetics of single doses of ISIS 333611 administered into the spinal canal as 12 hour infusions.
- Trial name
- SOD-1 (NCT01041222)
- Testing
- ISIS 333611
- Where
- 4 sites in United States
- Volunteers
- 33 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Spinal Muscular Atrophy, SMA
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones…
- Conditions
- Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- OPAL (NCT07047144)
- Testing
- Apitegromab, Nusinersen, Risdiplam
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 52 people
- Sponsor
- Scholar Rock, Inc.
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
Food Supplement for the Treatment of Patients With Amyotrophic Lateral Sclerosis
The purpose of the study is to evaluate the safety of combining phospholipids with medicinal plants for treatment of patients with amyotrophic lateral sclerosis (ALS)
- Trial name
- ALS-PHL (NCT02588807)
- Testing
- Spirit1
- Where
- Israel
- Sponsor
- Herb Spirit
Amyotrophic Lateral Sclerosis
Therapeutic Nanocatalysis to Slow Disease Progression of Amyotrophic Lateral Sclerosis (ALS)
The objective of this trial was to assess the efficacy, safety, and PK/PD effects of CNM-Au8 as a disease-modifying agent for the treatment of ALS by utilizing electrophysiological measures to detect preservation of motor neuron…
- Trial name
- RESCUE-ALS (NCT04098406)
- Testing
- CNM-Au8
- Where
- 2 sites in Australia
- Volunteers
- 45 people
- Sponsor
- Clene Nanomedicine
Amyotrophic Lateral Sclerosis
Efficacy and Safety of Plasma Exchange With Albutein® 5% in Participants With Amyotrophic Lateral Sclerosis
This is a pilot, phase 2, prospective, open-label, single-arm study to evaluate disease progression, forced vital capacity, and the safety and tolerability of plasma exchange (PE) using Albutein® 5% in participants with…
- Trial name
- NCT02872142
- Testing
- Albutein 5%, Plasma Exchange
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Grifols Therapeutics LLC
Amyotrophic Lateral Sclerosis
RT001 in Amyotrophic Lateral Sclerosis
RT001-014 is a Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects with Amyotrophic Lateral Sclerosis
- Trial name
- NCT04762589
- Testing
- RT001
- Where
- 4 sites in Estonia, Latvia and 2 more
- Volunteers
- 43 people
- Sponsor
- Biojiva LLC