Clinical trials: Muscular Diseases
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
Fibromyalgia
Pregabalin In Adolescent Patients With Fibromyalgia
The main purpose of this study is to evaluate the long-term safety of pregabalin in adolescent patients who participated in the previous fibromyalgia study (A0081180) and who wish to recieve open-label pregabalin
- Trial name
- NCT01020526
- Testing
- pregabalin
- Where
- 26 sites in United States, Czechia and 1 more
- Volunteers
- 63 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Dupuytren's Contracture
AA4500 (XIAFLEX™, Proposed Name) in the Treatment of Dupuytren's Contracture
This was a Phase 3, double-blind, randomized, placebo-controlled study conducted in the United States. Subjects with a diagnosis of Dupuytren's contracture in a metacarpophalangeal (MP) or proximal interphalangeal (PIP) joint…
- Trial name
- CORD-I (NCT00528606)
- Testing
- collagenase clostridium histolyticum
- Where
- 15 sites in United States
- Volunteers
- 308 people
- Sponsor
- Endo USA Inc., a Keenova Therapeutics Company
Temporomandibular Disorders
Treatment Outcomes for Temporomandibular Disorders (TMD) Via the Clayton Intra-aural Device (CID) Clinical Trial
The purpose of this study is to characterize the safety profile and assess the effectiveness of the CID in treating subjects with temporomandibular disorders (TMDs).
- Trial name
- NCT00815776
- Testing
- Clayton Intra-aural Device (CID), Mouth Splint, Exercise Group
- Where
- Mexico
- Volunteers
- 152 people
- Sponsor
- Ascentia Health, Inc.
Muscular Atrophy, Spinal
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA).
- Trial name
- FIREFISH (NCT02913482)
- Testing
- Risdiplam
- Where
- 32 sites in United States, Belgium and 14 more
- Volunteers
- 62 people
- Sponsor
- Hoffmann-La Roche
Fibromyalgia
A Study Comparing Duloxetine and Placebo in the Treatment of Fibromyalgia
The purpose of this study is to confirm the efficacy and safety of duloxetine 60-120 mg once daily in comparison to placebo on symptom improvement in patients meeting criteria for fibromyalgia aged 18 and older.
- Trial name
- NCT00673452
- Testing
- duloxetine hydrochloride
- Where
- 46 sites in United States, Puerto Rico
- Volunteers
- 530 people
- Sponsor
- Eli Lilly and Company
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
- Trial name
- NCT04886518
- Testing
- Pitolisant Oral Tablet
- Where
- 13 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- Harmony Biosciences Management, Inc.
ICU Acquired Weakness
Evaluation of FDY-5301 in Major Trauma Patients in ICU
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics (PK) of FDY-5301 compared to placebo in major trauma ICU patients at risk of intensive care unit acquired weakness (ICUAW)
- Trial name
- NCT04430283
- Testing
- FDY-5301
- Where
- 5 sites in United States
- Volunteers
- 8 people
- Sponsor
- Faraday Pharmaceuticals, Inc.
RYR-1 Myopathy
S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM)
This study proposes to test S 48168 (ARM210) in a Phase 1 trial in RYR1-RM patients, specifically. The objectives of this study are to explore the safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD)/target…
- Trial name
- NCT04141670
- Testing
- S48168
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- RyCarma Therapeutics, Inc.
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
Uterine Fibroids, Heavy Menstrual Bleeding
An Extension Study to Evaluate the Efficacy and Safety of Elagolix in Premenopausal Women With Heavy Menstrual Bleeding Associated With Uterine Fibroids
This is an extension study for women who have already received six months of treatment in the phase III clinical trial M12-815 (NCT02654054) or M12-817 (NCT02691494), and will evaluate the long-term efficacy and safety of…
- Trial name
- NCT02925494
- Testing
- Estradiol/Norethindrone Acetate, Elagolix
- Where
- 124 sites in United States, Canada and 1 more
- Volunteers
- 433 people
- Sponsor
- AbbVie
Duchenne Muscular Dystrophy
Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in participants with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).
- Trial name
- LELANTOS-1 (NCT04371666)
- Testing
- Pamrevlumab, Corticosteroids
- Where
- 53 sites in United States, Australia and 12 more
- Volunteers
- 98 people
- Sponsor
- Kyntra Bio
Giant Cell Tumors
Tenosynovial Giant Cell Tumors (TGCT) Observational Platform Project
TGCT is a rare disease that is difficult to manage, surgical resection is the primary treatment currently available. To date no disease registry exists and there is little data available detailing the management of patients with…
- Trial name
- TOP (NCT02948088)
- Where
- 15 sites in United States, Austria and 6 more
- Volunteers
- 183 people
- Sponsor
- Daiichi Sankyo
Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis
Study of Pimicotinib (ABSK021) for Tenosynovial Giant Cell Tumor (MANEUVER)
The goal of this clinical trial is to assess the efficacy and safety of Pimicotinib (ABSK021) in patients with Tenosynovial Giant Cell Tumor (TGCT).
- Conditions
- Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis, Giant Cell Tumor of Tendon Sheath
- Trial name
- NCT05804045
- Testing
- Pimicotinib(ABSK021)
- Where
- 40 sites in United States, Canada and 5 more
- Volunteers
- 94 people
- Sponsor
- Abbisko Therapeutics Co, Ltd
Myasthenia Gravis, MuSK, AChR Myasthenia Gravis
Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)
Evaluate the long-term safety of amifampridine phosphate in patients with MuSK antibody positive and AChR antibody positive myasthenia gravis.
- Trial name
- NCT03579966
- Testing
- Amifampridine Phosphate
- Where
- 3 sites in United States, Italy
- Volunteers
- 63 people
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Muscular Dystrophy, Duchenne
A Study of Tadalafil for Duchenne Muscular Dystrophy
The main purpose of this study is to determine if tadalafil can slow the decline in walking ability of boys who have Duchenne muscular dystrophy (DMD).
- Trial name
- NCT01865084
- Testing
- Tadalafil
- Where
- 62 sites in United States, Argentina and 14 more
- Volunteers
- 331 people
- Sponsor
- Eli Lilly and Company
Fibromyalgia
Trial Comparing the Effects of Xyrem (Sodium Oxybate) With Placebo for the Treatment of Fibromyalgia
The purpose of this study is to determine whether Xyrem (sodium oxybate) is effective when used alone to treat the pain and sleep disturbances of fibromyalgia.
- Trial name
- NCT00087555
- Testing
- Xyrem (sodium oxybate) oral solution
- Where
- 20 sites in United States
- Volunteers
- 195 people
- Sponsor
- Jazz Pharmaceuticals
Bladder Cancer
Safety Study of Recombinant Human Hyaluronidase (Chemophase) in Combination With Mitomycin in Participants With Superficial Bladder Cancer
The purpose of this study is to explore a treatment that potentially enhances the delivery of chemotherapy to tumors in participants with superficial bladder cancer.
- Trial name
- NCT00318643
- Testing
- Mitomycin C, Chemophase
- Where
- 5 sites in United States
- Volunteers
- 27 people
- Sponsor
- Halozyme Therapeutics
Spasticity With Multiple Sclerosis
Evaluation of the Effect of Nabiximols Oromucosal Spray on Clinical Measures of Spasticity in Participants With Multiple Sclerosis
This study will be conducted to evaluate the effect of multiple doses of nabiximols compared with placebo on a clinical measure of velocity-dependent muscle tone in the lower limbs (Lower Limb Muscle Tone-6 [LLMT-6]) in…
- Trial name
- RELEASE MSS5 (NCT04984278)
- Testing
- Nabiximols
- Where
- 30 sites in United States, Czechia and 3 more
- Volunteers
- 56 people
- Sponsor
- Jazz Pharmaceuticals
Spinal Muscular Atrophy
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies
The primary objective is to evaluate long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in…
- Trial name
- SHINE (NCT02594124)
- Testing
- nusinersen
- Where
- 47 sites in United States, Australia and 12 more
- Volunteers
- 292 people
- Sponsor
- Biogen