Clinical trials: Atrophy
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
Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.
- Trial name
- SIDEROS-E (NCT03603288)
- Testing
- idebenone 150 mg film-coated tablets
- Where
- 39 sites in United States, Austria and 7 more
- Volunteers
- 161 people
- Sponsor
- Santhera Pharmaceuticals
Geographic Atrophy
Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Geographic Atrophy
This Phase 3, multicenter, double-masked, parallel-group, placebo-controlled, randomized, fixed-dose clinical study is designed to evaluate the efficacy and safety of tinlarebant (LBS-008) in subjects diagnosed with GA.
- Trial name
- PHOENIX (NCT05949593)
- Testing
- Tinlarebant
- Where
- 49 sites in United States, Australia and 6 more
- Volunteers
- 429 people
- Sponsor
- Belite Bio, 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.
Muscle Atrophy, Hypodynamia
Safety Study of HPP593 in Subjects During and After Limb Immobilization
This is a safety and tolerability study investigating the effect of HPP593 in healthy volunteers during and after limb immobilization.
- Trial name
- NCT01524406
- Testing
- HPP593
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- High Point Pharmaceuticals, LLC.
Age Related Macular Degeneration (ARMD)
ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD)
The goal of this clinical trial is to evaluate the efficacy, safety and pharmacokinetics of elamipretide in subjects with dry age-related macular degeneration (AMD).
- Trial name
- ReNEW (NCT06373731)
- Testing
- Elamipretide
- Where
- 53 sites in United States, Czechia and 6 more
- Volunteers
- 313 people
- Sponsor
- Stealth BioTherapeutics 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
Geographic Atrophy (GA), Age - Related Macular Degeneration (AMD)
Study to Evaluate Efficacy and Safety of ONL1204 in Patients With GA Associated With AMD
The purpose of this study is to collect efficacy and safety information of intravitreal injection for 3 experimental arms, including 2 dose levels of ONL1204 and 2 treatment frequencies in patients with GA associated with AMD.
- Trial name
- NCT06659445
- Testing
- ONL1204 Opthalmic solution, Avacincaptad Pegol intravitreal solution
- Where
- 120 sites in United States, Austria and 10 more
- Volunteers
- 324 people
- Sponsor
- ONL Therapeutics
Limb-Girdle Muscular Dystrophy ; Subtypes 2A, 2B, and 2I
Limb-Girdle Video Assessment
The purpose of this study is to develop a new remote-based video assessment outcome measure for Limb-Girdle Muscular Dystrophy (LGMD) trials. The overall objectives for this study are: 1.
- Trial name
- NCT05528757
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- The Emmes Company, LLC
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.
Retinitis Pigmentosa, Stargardt Macular Dystrophy
Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD)
The Vision Research and Assessment Institute (VRAI) was established with the purpose of serving as a testing facility for efficacy endpoints for patients with Low Vision.
- Conditions
- Retinitis Pigmentosa, Stargardt Macular Dystrophy, Stargardt Disease, Geographic Atrophy From Age-related Macular Degeneration
- Trial name
- DEFINE-IRD (NCT07502664)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Ray Therapeutics, Inc.
Geographic Atrophy
A Study to Evaluate Intravitreal JNJ-81201887 (AAVCAGsCD59) Compared to Sham Procedure for the Treatment of Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD)
The purpose of this study is to evaluate change in geographic atrophy (GA) lesion growth of eyes treated with JNJ-81201887 compared to sham control.
- Trial name
- NCT05811351
- Testing
- JNJ-81201887, Prednisone, Triamcinolone
- Where
- 162 sites in United States, Australia and 15 more
- Volunteers
- 305 people
- Sponsor
- Janssen Research & Development, LLC
Menopause, Postmenopausal Vaginal Atrophy
Comparison of Two Vaginal Tablets, Produced at Different Sites, in Postmenopausal Women With Atrophic Vaginitis
This trial is conducted in Europe. The aim of this trial is to compare the absorption of estradiol after a single dose of two 25 mcg estradiol vaginal tablets, produced at two different production sites, in postmenopausal women…
- Trial name
- NCT01085877
- Testing
- estradiol, 25 mcg
- Where
- Germany
- Volunteers
- 70 people
- Sponsor
- Novo Nordisk A/S
Muscular Dystrophies
A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
This is a phase III, multicenter, open-label, uncontrolled extension study in male subjects with DMD open to eligible US and Canadian subjects who previously participated in the following studies of drisapersen: DMD114876…
- Trial name
- NCT01803412
- Testing
- Drisapersen
- Where
- 13 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- BioMarin Pharmaceutical
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
Age-Related Macular Degeneration, Geographic Atrophy
A Study of the SING IMT in an Israeli Cohort
The objective of this single center study is to determine the safety of the smaller incision, new generation (SING), implantable miniature telescope (IMT) in a small sample of patients with moderate-severe central vision loss due…
- Trial name
- NCT05941273
- Testing
- smaller incision, new generation implantable miniature telescope (SING-IMT)
- Sponsor
- VisionCare, Inc.
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
Duchenne Muscular Dystrophy
Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
This is a Phase 3, multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301.
- Trial name
- NCT04768062
- Testing
- Viltolarsen
- Where
- 31 sites in Australia, Canada and 15 more
- Volunteers
- 74 people
- Sponsor
- NS Pharma, Inc.
Duchenne Muscular Dystrophy, Exon 44
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to <15 years with DMD due to mutations amenable to exon 44 skipping.
- Conditions
- Duchenne Muscular Dystrophy, Exon 44, DMD
- Trial name
- NCT05996003
- Testing
- NS-089/NCNP-02
- Where
- 29 sites in United States, Australia and 5 more
- Volunteers
- 20 people
- Sponsor
- NS Pharma, Inc.