Clinical trials: Myopathies
Multiple Sclerosis
IPX056 in Subjects With Established Spasticity Resulting From Multiple Sclerosis
The purpose of this study is to determine the effects, both good and bad, of IPX056 on subjects and their spasticity. This study will also determine the relationship between the amount of IPX056 in blood and the effects on…
- Trial name
- NCT00488839
- Testing
- IPX056 20 mg, IPX056 40 mg, Encapsulated Baclofen 20 mg
- Where
- 31 sites in United States, Canada and 3 more
- Volunteers
- 173 people
- Sponsor
- Impax Laboratories, LLC
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)
The goal of this clinical trial is to evaluate the long-term safety and tolerability of zagociguat in patients with MELAS who completed study medication treatment in the lead-in study TIS6463-203.
- Trial name
- NCT06961344
- Testing
- zagociguat 15mg
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 37 people
- Sponsor
- Tisento Therapeutics
Fibrodysplasia Ossificans Progressiva
A Study to Assess the Effectiveness and Safety of 2 Dosage Regimens of Oral Fidrisertib (IPN60130) for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP).
Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by the presence of bone in soft tissue where bone normally does not exist, known as Heterotopic Ossification (HO).
- Trial name
- FALKON (NCT05039515)
- Testing
- IPN60130
- Where
- 25 sites in United States, Argentina and 12 more
- Volunteers
- 113 people
- Sponsor
- Clementia Pharmaceuticals Inc.
Muscular Dystrophy, Duchenne
Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
The PolarisDMD study is a Phase 3, global study to evaluate the efficacy and safety of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD.
- Trial name
- PolarisDMD (NCT03703882)
- Testing
- Edasalonexent
- Where
- 40 sites in United States, Australia and 6 more
- Volunteers
- 131 people
- Sponsor
- Catabasis Pharmaceuticals
Fibrodysplasia Ossificans Progressiva
Study of Single-Ascending Doses of DS-6016a in Healthy Japanese Subjects
This study will assess the safety, tolerability, and pharmacokinetics of DS-6016a after subcutaneous injection in healthy Japanese participants.
- Trial name
- NCT04818398
- Testing
- DS-6016a
- Where
- Japan
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Daiichi Sankyo Co., Ltd.
Muscle Spasticity, Stroke
Long-Term Study of OnabotulinumtoxinA Treatment in Adult Patients With Upper Limb Spasticity
This is a long-term safety and efficacy study of onabotulinumtoxinA in poststroke patients with upper limb spasticity.
- Trial name
- NCT02145689
- Testing
- onabotulinumtoxinA
- Sponsor
- Allergan
Myofascial Pain Syndromes
Study to Assess the Efficacy and Safety of Dysport® in Upper Back Myofascial Pain Syndrome
The main purpose of this study is to determine which is the best dose of a drug known as Dysport to give when treating one's type of upper back pain.
- Trial name
- NCT00134810
- Testing
- Botulinum toxin type A
- Where
- 22 sites in Czechia, Germany and 4 more
- Volunteers
- 381 people
- Sponsor
- Ipsen
Facioscapulohumeral Muscular Dystrophy, Limb Girdle Muscular Dystrophy
Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)
ATYR1940-C-006 is a multi-national, multicenter study being conducted at centers in the United States (US) and Europe who participated in Study ATYR1940-C-003 (Stage 1 only) or Study ATYR1940-C-004 (that is, the parent studies).
- Trial name
- FSHD (NCT02836418)
- Testing
- ATYR1940
- Where
- 5 sites in United States, Denmark and 1 more
- Volunteers
- 8 people
- Sponsor
- aTyr Pharma, Inc.
Spasticity
Efficacy and Safety Study of MYOBLOC® in the Treatment of Adult Lower Limb Spasticity
Phase 2/3, randomized, double-blind, placebo-controlled, single-treatment, multicenter trial assessing the efficacy and safety of MYOBLOC for the treatment of lower limb spasticity, in adults followed by an open-label extension…
- Trial name
- NCT04099667
- Testing
- Phase 2; Low Dose MYOBLOC (15,000 Units), Phase 2; High Dose MYOBLOC (20,000 Units), Phase 3; MYOBLOC
- Where
- 9 sites in United States, Hungary and 1 more
- Volunteers
- 40 people
- Sponsor
- Solstice Neurosciences, LLC, a subsidiary of MDD US Operations, LLC
Mitochondrial Diseases, Mitochondrial Myopathies
The KHENERGY Study
Mitochondrial Diseases are rare, progressive, multi-system, often-early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial…
- Conditions
- Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS
- Trial name
- KHENERGY (NCT02909400)
- Testing
- KH176
- Where
- Netherlands
- Volunteers
- 20 people
- Sponsor
- Khondrion BV
Fibromyalgia
Phase 2 Study of TD-9855 to Treat Fibromyalgia
The purpose of this study study is to determine whether TD-9855 is effective in treating patients with fibromyalgia.
- Trial name
- NCT01693692
- Testing
- TD-9855 Group 1, TD-9855 Group 2
- Where
- 34 sites in United States
- Volunteers
- 392 people
- Sponsor
- Theravance Biopharma
Muscle Weakness
Magnetic Muscle Stimulation of Abdominal Muscle
Evaluate the safety and efficacy of magnetic muscle stimulation (MMS) of abdominal muscle.
- Trial name
- MMS (NCT04199312)
- Testing
- Magnetic Muscle Stimulation (MMS) device
- Where
- 3 sites in United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Zeltiq Aesthetics
PreDiabetes, Older Adult
Examining the Effects of Vivo on Physical Function and Glycemic Level in Sedentary Older With Prediabetes.
This is a 12-week randomized, controlled trial that seeks to examine the effect of Vivo on 1) change in lower extremity strength defined as number of chair stands done in 30 seconds and 2) change in average glycemic level (HbA1c)…
- Conditions
- PreDiabetes, Older Adult, Muscle Loss, Muscle Weakness
- Trial name
- NCT06458530
- Testing
- Vivo
- Where
- United States
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- Impactiv, Inc.
Duchenne Muscular Dystrophy
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT04906460
- Testing
- WVE-N531
- Where
- 6 sites in United States, Jordan and 1 more
- Volunteers
- 25 people
- Sponsor
- Wave Life Sciences USA, Inc.
Moderate Hepatic Impairment
Study of Pexidartinib in Participants With Moderate Hepatic Impairment Compared With Healthy Participants
The pharmacokinetics of a single dose of pexidartinib was investigated in participants with impaired hepatic function and compared with healthy control participants with normal hepatic function.
- Trial name
- NCT04223635
- Testing
- Pexidartinib
- Where
- 2 sites in United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Daiichi Sankyo
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55.
- Trial name
- NCT07429240
- Testing
- PBGENE-DMD (IV)
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Precision BioSciences, Inc.
Fibromyalgia
A Multicentre Trial to Determine the Efficacy and Safety of AD-337 in the Treatment of Fibromyalgia
This is a multicentre, randomized, double-blind, placebo controlled, parallel group exploratory study to investigate the efficacy, safety and tolerability of AD 337 in the treatment of fibromyalgia in female subjects.
- Trial name
- NCT00377039
- Testing
- AD 337
- Where
- United Kingdom
- Volunteers
- 100 people
- Sponsor
- Sosei
Refractory Autoimmune Diseases
A Study of Healthy Donor CD19-targeted Allogeneic CAR T Cells in Participants With Severe, Refractory Autoimmune Diseases
The purpose of this study is to determine the safety, tolerability, optimal dose, and preliminary efficacy of BMS-986515, a healthy donor (HD) allogeneic CD19-targeted CART cell product, in participants with severe, refractory…
- Trial name
- NCT07115745
- Testing
- BMS-986515, Fludarabine, Cyclophosphamide
- Where
- 28 sites in United States, Australia and 8 more
- Volunteers
- 125 people
- Sponsor
- Juno Therapeutics, Inc., a Bristol-Myers Squibb Company
Osteoporosis
1 Year Open-label Extension to CZOL446H2337 Safety and Efficacy Trial of Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids
This 1-year open-label extension to CZOL446H2337 is designed to evaluate the safety and efficacy of zoledronic acid twice yearly in osteoporotic children treated with glucocorticoids.
- Trial name
- NCT01197300
- Testing
- Zoledronic acid
- Where
- 10 sites in Australia, Canada and 4 more
- Volunteers
- 25 people
- Sponsor
- Novartis Pharmaceuticals
Amyotrophic Lateral Sclerosis (ALS), Angina, Stable
Quest to Analyze One Thousand Humans Meditating
This study will be focused on assessing the molecular, physiological, and emotional correlates of an intensive meditation experience in the context of a retreat setting in a large 2000 plus-person cohort comprised of healthy and…
- Conditions
- Amyotrophic Lateral Sclerosis (ALS), Angina, Stable, Anxiety, Asthma
- Trial name
- QUANTUM (NCT06583395)
- Testing
- Advanced multi-component meditation practice
- Where
- United States
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Tobias Moeller-Bertram