Clinical trials: Myopathies
DM1, Muscular Dystrophies
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
AOC 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001…
- Conditions
- DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1
- Trial name
- MARINA-OLE (NCT05479981)
- Testing
- AOC 1001
- Where
- 8 sites in United States
- Volunteers
- 37 people
- Sponsor
- Avidity Biosciences, Inc.
Duchenne Muscular Dystrophy
Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S.
- Trial name
- NCT02592941
- Testing
- Deflazacort
- Where
- 42 sites in United States
- Sponsor
- PTC Therapeutics
Pain Associated With Fibromyalgia
An Open-Label Extension Study of DS-5565 for 52 Weeks in Pain Associated With Fibromyalgia
This is an open-label study of DS-5565 in subjects who either completed participation in a preceding Phase 3 study of DS-5565 in fibromyalgia (FM); i.e.
- Trial name
- NCT02234583
- Testing
- DS-5565
- Where
- 276 sites in United States, Australia and 27 more
- Volunteers
- 2,485 people
- Sponsor
- Daiichi Sankyo
Spasticity, Post-Stroke
A Multicenter, Open-label, Single Dose Study of the Safety and Efficacy of GSK1358820 (Botulinum Toxin Type A) in Chinese Subjects With Post-stroke Focal Upper Limb Spasticity
This trial is a multicenter, open-label study to evaluate the safety and efficacy of GSK1358820 for treatment in post-stroke subjects with focal wrist, finger and in some cases, thumb spasticity.
- Trial name
- NCT01205451
- Testing
- Botulinum toxin type A
- Where
- 13 sites in China
- Volunteers
- 109 people
- Sponsor
- GlaxoSmithKline
Rhabdomyolysis, Renal Failure
Myoglobin Removal by High Cut-off CVVHD
A high cut-off dialyzer (septeX) is tested for the removal of myoglobin which is not efficiently removed by standard high flux dialysis membranes.
- Trial name
- HicoRhabdo (NCT01467180)
- Testing
- septeX, HF CVVH
- Where
- Germany
- Volunteers
- 16 people
- Sponsor
- Vantive Health LLC
Tendinopathy
A Study Evaluating the Safety, Tolerability, and Pharmacokinetics of Multiple Ascending Doses of SM04755 Following Topical Administration to Healthy Subjects
This study is a single-center, randomized, single-blind, placebo-controlled, multiple ascending dose study of SM04755 solution applied topically once daily for 14 days to intact skin overlying the inner thigh of healthy subjects…
- Trial name
- NCT03229291
- Testing
- SM04755, Vehicle
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Biosplice Therapeutics, 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
Myositis
A Study of Nipocalimab in Participants With Active Idiopathic Inflammatory Myopathies
The purpose of this study is to evaluate the efficacy and safety of Nipocalimab versus placebo in participants with active idiopathic inflammatory myopathies (IIM).
- Trial name
- SPIREA (NCT05379634)
- Testing
- Nipocalimab, Glucocorticoids
- Where
- 57 sites in United States, Canada and 11 more
- Volunteers
- 36 people
- Sponsor
- Janssen Research & Development, LLC
Duchenne Muscular Dystrophy
A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy
This is a Phase 2 randomized, 2-period, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, efficacy, PK and PD of PF-06252616 administered to ambulatory boys diagnosed with Duchenne Muscular…
- Trial name
- NCT02310763
- Testing
- PF-06252616
- Where
- 66 sites in United States, Australia and 6 more
- Volunteers
- 121 people
- Sponsor
- Pfizer
Immune Mediated Necrotizing Myopathy
Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy
The purpose of the study is to evaluate the safety and efficacy of zilucoplan in patients with Immune-Mediated Necrotizing Myopathy (IMNM).
- Trial name
- NCT04025632
- Testing
- zilucoplan
- Where
- 18 sites in United States, France and 2 more
- Volunteers
- 27 people
- Sponsor
- Ra Pharmaceuticals
Fibrodysplasia Ossificans Progressiva
A Study to Examine the Safety, Tolerability and Effects on Abnormal Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva
This is a three period study design consisting of a 6-month, randomized, double-blind placebo-controlled treatment (period 1) followed by a 6-month, open-label treatment (period 2) and a follow-up treatment period (period 3).
- Trial name
- LUMINA-1 (NCT03188666)
- Testing
- REGN2477
- Where
- 11 sites in United States, Canada and 6 more
- Volunteers
- 44 people
- Sponsor
- Regeneron Pharmaceuticals
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
DMD/BMD is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Trial name
- NCT00592553
- Testing
- Ataluren
- Where
- 37 sites in United States, Australia and 9 more
- Volunteers
- 174 people
- Sponsor
- PTC Therapeutics
Myotonic Dystrophy 1
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared…
- Trial name
- FREEDOM2-DM1 (NCT06667453)
- Testing
- PGN-EDODM1
- Where
- 12 sites in Canada, New Zealand and 2 more
- Volunteers
- 24 people
- Sponsor
- PepGen Inc
Fibromyalgia
Study of Duloxetine Versus Placebo in the Treatment of Fibromyalgia Syndrome
To assess the efficacy of Duloxetine compared with placebo in the treatment of pain in patients with Fibromyalgia syndrome, with or without major depressive disorder.
- Trial name
- NCT00190866
- Testing
- Duloxetine
- Where
- 35 sites in United States, Puerto Rico
- Volunteers
- 210 people
- Sponsor
- Eli Lilly and Company
Fibromyalgia
Adolescent Fibromyalgia Study
This study will compare pregabalin with placebo over a 15 week period in adolescents with fibromyalgia aged 12-17 years to evaluate the safety and efficacy of pregabalin.
- Trial name
- NCT01020474
- Testing
- pregabalin (Lyrica)
- Where
- 36 sites in United States, Czechia and 2 more
- Volunteers
- 107 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Muscular Dystrophies
Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol
This is a single arm, open-label continued access protocol of drisapersen for the treatment of male subjects with Duchenne muscular dystrophy (DMD) having dystrophin mutations correctable by drisapersen-induced DMD Exon 51…
- Trial name
- NCT01890798
- Testing
- Drisapersen
- Sponsor
- GlaxoSmithKline
Fibromyalgia
Effect of PERMEAPROTECT on the Quality of Life of Patients With Fibromyalgia
Fibromyalgia is a medical disorder characterized by chronic widespread pain, and a heightened and painful response to pressure. Fibromyalgia symptoms are not restricted to pain, leading to the use of the alternative term…
- Trial name
- L2009-03 (NCT01469936)
- Testing
- PERMEAPROTECT
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Lescuyer Laboratory
Spasticity, Multiple Sclerosis
One Year Extension Study To Protocol C2/5/TZ:MS-05
Open label, one year extension study to evaluate the clinical efficacy and safety of 12 mg sublingual tizanidine administered once nightly in MS patients who successfully completed Phase I/II protocol C2/5/TZ:MS-05 at the Tel…
- Trial name
- NCT00464958
- Testing
- sublingual tizanidine 12 mg
- Where
- Israel
- Volunteers
- 10 people
- Sponsor
- Teva GTC
Duchenne Muscular Dystrophy (DMD)
A Trial to Investigate Safety and Pharmacokinetics of GRT6019, Including Food Effect, in Healthy Male Participants
The purpose of this trial is to evaluate the safety, tolerability, and Pharmacokinetics (PK) of a single oral dose of GRT6019 in healthy male participants, including food effect.
- Trial name
- NCT07317063
- Testing
- GRT6019
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Grünenthal GmbH
Tendinopathy, Insertional Achilles Tendinopathy
Evaluation of Collagen-based Medical Device Treatment Combined With Physiotherapy in Subjects With Achilles Tendinopathy.
Achilles tendinopathy is a condition characterized by inflammation of the Achilles tendon. Achilles tendinopathies are classified into insertional tendinitis and noninsertional tendinitis.
- Conditions
- Tendinopathy, Insertional Achilles Tendinopathy, Achilles Tendinopathy, Peritendinitis
- Trial name
- AKITENMED (NCT05464498)
- Testing
- MD-Tissue Collagen Medical Device, eccentric strengthening protocol
- Where
- Italy
- Volunteers
- 72 people
- Sponsor
- Guna S.p.a