Clinical trials: Atrophy
Muscular Dystrophy, Duchenne, Muscular Dystrophies
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren…
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT02090959
- Testing
- Ataluren
- Where
- 58 sites in United States, Australia and 17 more
- Volunteers
- 219 people
- Sponsor
- PTC Therapeutics
Neovascular Age-related Macular Degeneration
Efficacy and Safety of Ranibizumab 0.5 mg Administered as Two Alternative Dosing Regimens in Chinese Patients With nAMD (Age Related Macular Degeneration)
The study evaluated the efficacy and safety of two different dosing regimens of ranibizumab (either monthly injections or injections as-needed based on the stability of a patient's vision) in Chinese patients with wet age-related…
- Trial name
- DRAGON (NCT01775124)
- Testing
- Ranibizumab
- Where
- 23 sites in China
- Volunteers
- 332 people
- Sponsor
- Novartis Pharmaceuticals
Geographic Atrophy, Dry Age-Related Macular Degeneration
Zimura in Participants With Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration
The objectives of this study were to evaluate the safety and efficacy of Zimura intravitreal (IVT) administration when administered in participants with geographic atrophy (GA) secondary to dry age-related macular degeneration…
- Trial name
- NCT02686658
- Testing
- Avacincaptad Pegol
- Where
- 78 sites in United States, Croatia and 5 more
- Volunteers
- 286 people
- Sponsor
- IVERIC bio, Inc.
Muscular Atrophy, Spinal
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
Multi-center, randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of Risdiplam in adult and pediatric participants with Type 2 and Type 3 SMA.
- Trial name
- SUNFISH (NCT02908685)
- Testing
- Risdiplam
- Where
- 45 sites in United States, Belgium and 14 more
- Volunteers
- 231 people
- Sponsor
- Hoffmann-La Roche
Facioscapulohumeral Muscular Dystrophy
Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension
The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the treatment of adult participants with molecularly defined genetic muscular dystrophies.
- Trial name
- NCT02531217
- Testing
- ATYR1940
- Where
- 3 sites in United States, Italy and 1 more
- Volunteers
- 9 people
- Sponsor
- aTyr Pharma, Inc.
Geographic Atrophy
A Multicenter, Proof-Of-Concept Study Of Intravitreal AL-78898A In Patients With Geographic Atrophy (GA) Associated With Age-Related Macular Degeneration (AMD)
The purpose of this study was to to demonstrate superiority of AL-78898A intravitreal (IVT) injections compared to sham injections by assessing mean geographic atrophy (GA) lesion size change from baseline at Month 12.
- Trial name
- NCT01603043
- Testing
- Al-78898A
- Volunteers
- 10 people
- Sponsor
- Alcon Research
Muscular Dystrophies
First Time in Human (FTIH) Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Repeat Doses of GSK3439171A in Healthy Subjects and to Assess Food Effect
The FTIH study with GSK3439171A will evaluate the safety of GSK3439171A in healthy subjects in order to avoid confounding factors due to the disease or concomitant drugs in patients.
- Trial name
- NCT03627494
- Testing
- GSK3439171A
- Where
- United States
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Neurogenic Orthostatic Hypotension, Non-Diabetic Autonomic Neuropathy
Open-Label Clinical Study of Droxidopa in Patients With Neurogenic Orthostatic Hypotension (NOH)
The purpose of this study is to assess the durability of effect of Droxidopa in treating symptoms of neurogenic orthostatic hypotension in patients with Primary Autonomic Failure (Pure Autonomic Failure, Multiple System Atrophy…
- Conditions
- Neurogenic Orthostatic Hypotension, Non-Diabetic Autonomic Neuropathy, Multiple System Atrophy, Dopamine Beta Hydroxylase Deficiency
- Trial name
- NOH303 (NCT00738062)
- Testing
- Droxidopa
- Where
- 54 sites in United States, Australia and 2 more
- Volunteers
- 103 people
- Sponsor
- Chelsea Therapeutics
Duchenne Muscular Dystrophy
An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT03532542
- Testing
- Casimersen, Golodirsen
- Where
- 50 sites in United States, Belgium and 11 more
- Volunteers
- 171 people
- Sponsor
- Sarepta Therapeutics, Inc.
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.
Duchenne Muscular Dystrophy (DMD)
A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD
The primary objective of Parts 1 and 2 of the study were to establish the histologic effects of givinostat administered chronically at the selected daily dose.
- Trial name
- NCT01761292
- Testing
- Givinostat
- Where
- 4 sites in Italy
- Volunteers
- 20 people
- Sponsor
- Italfarmaco
Duchenne Muscular Dystrophy
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.
- Trial name
- NCT04060199
- Testing
- Viltolarsen
- Where
- 40 sites in United States, Australia and 17 more
- Volunteers
- 77 people
- Sponsor
- NS Pharma, Inc.
Menopause, Vulvovaginal Atrophy
TXV13-01 Estradiol Vaginal Softgel Capsules in Treating Postmenopausal Women With Symptoms of Vulvar and Vaginal Atrophy
The study was designed to evaluate the efficacy and safety of TX-12-004-HR 10 μg in treating moderate to severe symptoms of vaginal atrophy associated with menopause after 14 days of treatment, and to estimate the effect size and…
- Conditions
- Menopause, Vulvovaginal Atrophy, Painful Intercourse, Dyspareunia
- Trial name
- NCT02449902
- Testing
- Estradiol
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- TherapeuticsMD
Geographic Atrophy
Patients With Geographic Atrophy and Their Patient Journey in the United States (US)
To quantify and describe patients with a Geographic Atrophy (GA) diagnosis in at least one eye, and the progression to neovascular Age-Related Macular Degeneration (including how many receive Anti-VEGF), or to GA (in the fellow…
- Trial name
- NCT05891275
- Where
- United States
- Volunteers
- 68,563 people
- Sponsor
- Boehringer Ingelheim
Atrophic Acne Scar
SkinPen Efficacy on Acne Scars on the Face and/or Back
This single-center, clinical trial will take place over a 90 day course followed by 1-month and 6-month post treatment visits to assess the efficacy and tolerability of the SkinPen device when used on both men and women on the…
- Trial name
- NCT02646917
- Testing
- Aesthetic Microneedling Treatment
- Where
- United States
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- Bellus Medical, LLC
Dyspareunia
Study Comparing Estradiol Vaginal Inserts 4mcg To IMVEXXY ® (Estradiol Vaginal Inerts 4 mcg) In The Treatment Of Dyspareunia in Women With Vulvur and Vaginal Atrophy
Randomized, double-blind, placebo-controlled, parallel-designed, multiple-site, bioequivalence study with clinical endpoints.
- Trial name
- NCT05617820
- Testing
- Estradiol, Imvexxy
- Where
- 48 sites in United States, El Salvador and 1 more
- Volunteers
- 1,050 people, healthy volunteers welcome
- Sponsor
- Teva Pharmaceuticals USA
Multiple System Atrophy
A Study of TAK-341 in Treatment of Multiple System Atrophy
The main aim is to see how TAK-341 works after 52 weeks in participants with multiple system atrophy as measured by the Unified Multiple System Atrophy Rating Scale Part I (UMSARS).
- Trial name
- NCT05526391
- Testing
- TAK-341
- Where
- 41 sites in United States, Austria and 8 more
- Volunteers
- 158 people
- Sponsor
- Takeda
Multiple System Atrophy, MSA
AZD3241 PET MSA Trial, Phase 2, Randomized,12 Week Safety and Tolerability Trial With PET in MSA Patients
AZD3241 myeloperoxidase (MPO) inhibitor trial is assessing safety and tolerability, randomized trial, in patients with Multiple System Atrophy.
- Trial name
- NCT02388295
- Testing
- AZD3241
- Where
- 17 sites in United States, Austria and 5 more
- Volunteers
- 59 people
- Sponsor
- AstraZeneca
Facioscapulohumeral Muscular Dystrophy (FSHD)
Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy
The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the treatment of adult participants with molecularly defined genetic muscular dystrophies
- Trial name
- NCT02239224
- Testing
- ATYR1940
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 20 people
- Sponsor
- aTyr Pharma, Inc.
Duchenne Muscular Dystrophy
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) 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
- NCT00759876
- Testing
- Ataluren
- Where
- 3 sites in United States
- Volunteers
- 36 people
- Sponsor
- PTC Therapeutics