Clinical trials: Neuromuscular Disease
Solid Tumors
Dose Escalation of RMC-5552 Monotherapy in Relapsed/Refractory Solid Tumors
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) profiles of escalating doses of RMC-5552 monotherapy in adult participants with relapsed/refractory solid tumors…
- Trial name
- NCT04774952
- Testing
- RMC-5552
- Where
- 8 sites in United States
- Volunteers
- 58 people
- Sponsor
- Revolution Medicines, Inc.
Diabetic Polyneuropathy
Safety and Efficacy of MCC-257 in the Treatment of Diabetic Polyneuropathy
The primary objectives of the study are to evaluate the efficacy and safety of three doses of MCC-257 in patients with mild to moderate diabetic polyneuropathy
- Trial name
- NCT00307749
- Testing
- MCC-257
- Volunteers
- 420 people
- Sponsor
- Tanabe Pharma Corporation
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.
Muscular Atrophy, Spinal
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)
This was a phase IV Open-label, single-arm, single-dose, multicenter study, to evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with SMA with bi-allelic mutations in…
- Trial name
- OFELIA (NCT05073133)
- Testing
- OAV101
- Where
- 5 sites in Argentina, Brazil
- Volunteers
- 16 people
- Sponsor
- Novartis Pharmaceuticals
Poliomyelitis
Booster Dose of sIPV Co-administered With MMR and HepA-I.
This is an Open-labeled, Randomized, Controlled Phase IV Clinical Trial to Evaluate the Immunogenicity and Safety of Booster Dose of Sabin Strain Inactivated Poliovirus Vaccine (Vero cell) (sIPV) Co-administered with Measles…
- Trial name
- NCT06442449
- Testing
- sIPV, MMR, HepA-I
- Where
- China
- Volunteers
- 889 people, healthy volunteers welcome
- Sponsor
- Sinovac Biotech Co., Ltd
Cardiac Diseases, Muscular Weakness
Biomarker for Pompe Disease (BioPompe)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Pompe disease from blood (plasma)
- Conditions
- Cardiac Diseases, Muscular Weakness, Hepato-splenomegaly, Lung Disease
- Trial name
- BioPompe (NCT01457443)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Generalized Myasthenia Gravis
Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab for the treatment of participants with generalized myasthenia gravis (gMG).
- Trial name
- NCT03920293
- Testing
- Ravulizumab
- Where
- 106 sites in United States, Austria and 13 more
- Volunteers
- 175 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Multiple Sclerosis (MS)
Safety and Effectiveness of Nabiximols Oromucosal Spray as Add-on Therapy in Participants With Spasticity Due to Multiple Sclerosis
This trial is being conducted to demonstrate the efficacy of nabiximols, compared with placebo, when added to standard of care, in the treatment of muscle spasms associated with multiple sclerosis (MS).
- Trial name
- RELEASE MSS3 (NCT04203498)
- Testing
- Nabiximols
- Where
- 37 sites in United States, Czechia and 3 more
- Volunteers
- 139 people
- Sponsor
- Jazz Pharmaceuticals
Diabetic Neuropathy, Painful
Study Of Pregabalin (Lyrica) In Patients With Painful Diabetic Peripheral Neuropathy
Patients will be switched from their current medication for painful diabetic peripheral neuropathy to evaluate the safety and efficacy of pregabalin as compared to placebo.
- Trial name
- NCT01057693
- Testing
- pregabalin (Lyrica)
- Where
- 172 sites in United States, Canada and 2 more
- Volunteers
- 633 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Chronic Inflammatory Demyelinating Polyneuropathy
To Assess Efficacy and Safety of Batoclimab in Adult Participants With Active CIDP
This is a multi-center, randomized, quadruple-blind, placebo-controlled study to evaluate the efficacy and safety of batoclimab in adult participants with active CIDP.
- Trial name
- NCT05581199
- Testing
- Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly, Batoclimab 340 mg SC weekly
- Where
- 98 sites in United States, Argentina and 19 more
- Volunteers
- 94 people
- Sponsor
- Immunovant Sciences GmbH
Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP)
International, multicenter, observational, longitudinal study to identify biomarker/s for the development of a new MS-based biomarker for the early and sensitive diagnosis of Transthyretin-Related Familial Amyloidotic…
- Conditions
- Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin Amyloid Cardiopathy
- Trial name
- BioTRAP (NCT02713880)
- Where
- 2 sites in Germany, India
- Sponsor
- CENTOGENE GmbH Rostock
Pain, Pain Syndrome
Long Term Efficacy and Tolerability of AP707 in Patients With Chronic Pain Due to Traumatic or Post-operative Peripheral Neuropathy
Over the last years a rising medical need for treatment of chronic pain was identified. Based on previous findings indicating the pain modulating effects of cannabinoids in chronic pain disorders, this clinical trial investigates…
- Conditions
- Pain, Pain Syndrome, Pain, Chronic, Chronic Pain
- Trial name
- NCT06071988
- Testing
- Adezunap (AP707)
- Where
- Germany
- Volunteers
- 558 people
- Sponsor
- Apurano Pharmaceuticals GmbH
Amyotrophic Lateral Sclerosis
Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS
The goal of this clinical trial is to learn if the drug pridopidine works to treat amyotrophic lateral sclerosis in adults. It will also help to learn about the safety of pridopidine.
- Trial name
- PREVAiLS (NCT07322003)
- Testing
- Pridopidine
- Where
- 56 sites in United States, Belgium and 11 more
- Volunteers
- 500 people
- Sponsor
- Prilenia
Amyotrophic Lateral Sclerosis, Fasciculation
Study to Evaluate Safety & Efficacy of FLX-787-ODT to Treat Fasciculations in Tongue and Appendicular Muscle in Adult Subjects With ALS
The FLX-787-107 study will determine how well FLX-787-ODT works to reduce fasciculations in patients with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT03334786
- Testing
- FLX-787-ODT
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Flex Pharma, Inc.
Generalized Myasthenia Gravis
Phase 3 Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants With Generalized Myasthenia Gravis
The purpose of this 4-period study is to confirm the efficacy and safety of batoclimab in participants with gMG. In Period 1, participants will be randomized 1:1:1 to receive batoclimab 680 milligrams (mg) subcutaneously (SC)…
- Trial name
- NCT05403541
- Testing
- Batoclimab 680 mg SC weekly, Batoclimab 340 mg SC weekly, Batoclimab 340 mg SC bi-weekly
- Where
- 93 sites in United States, Argentina and 14 more
- Volunteers
- 233 people
- Sponsor
- Immunovant Sciences GmbH
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)
This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NTLA-2001 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and…
- Conditions
- Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis
- Trial name
- NCT04601051
- Testing
- NTLA-2001
- Where
- 4 sites in France, New Zealand and 2 more
- Volunteers
- 72 people
- Sponsor
- Intellia Therapeutics
Generalized Myasthenia Gravis
Clinical Study of Cizutamig in Generalized Myasthenia Gravis (gMG)
The purpose of this study is to assess the safety, tolerability, PK, PD, immunogenicity, and preliminary clinical activity of Cizutamig in patients with Generalized Myasthenia Gravis.
- Trial name
- NCT07215650
- Testing
- Cizutamig
- Where
- 10 sites in China
- Volunteers
- 44 people
- Sponsor
- Candid Therapeutics
Mitochondrial Myopathies, Mitochondrial Pathology
Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)
SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to assess efficacy and safety of single daily subcutaneous (SC) administration of elamipretide as a treatment for subjects with primary…
- Conditions
- Mitochondrial Myopathies, Mitochondrial Pathology, Mitochondrial DNA Mutation, Mitochondrial Diseases
- Trial name
- NuPower (NCT05162768)
- Testing
- Elamipretide
- Where
- 32 sites in United States, Australia and 8 more
- Volunteers
- 102 people
- Sponsor
- Stealth BioTherapeutics Inc.
Poliomyelitis
IPV-102 Safety, Tolerability and Immunogenicity of TAK-195 in Healthy Infants, Toddlers and Adults
The purpose of this study is to select the optimal antigen dosage of the three Sabin poliovirus strains (types 1, 2, and 3) entering the composition of the stand-alone trivalent Sabin-based inactivated poliomyelitis vaccine…
- Trial name
- NCT03092791
- Testing
- Sabin-Based Inactivated Poliomyelitis Vaccine (sIPV), Reference IPV
- Where
- 4 sites in Panama
- Volunteers
- 340 people, healthy volunteers welcome
- Sponsor
- Takeda
Becker Muscular Dystrophy
Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)
A study of sevasemten (EDG-5506) in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON study was expanded to include an additional 175 adult participants in a cohort…
- Trial name
- NCT05291091
- Testing
- Sevasemten 10 mg, Sevasemten 5 mg, Sevasemten 12.5 mg
- Where
- 51 sites in United States, Australia and 10 more
- Volunteers
- 244 people
- Sponsor
- Edgewise Therapeutics, Inc.
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