Clinical trials: Atrophy
Muscular Atrophy, Spinal
A Study of the Safety of Nusinersen (Spinraza®) and Its Effects on Motor Function in Koreans Living With SMA Treated During Their Usual Medical Care
In this study, researchers will learn more about the effects of nusinersen, also known as Spinraza®, in Korean participants with spinal muscular atrophy, also known as SMA.
- Trial name
- STANDARD (NCT04317794)
- Testing
- Nusinersen Sodium Injection
- Where
- 16 sites in South Korea
- Volunteers
- 74 people
- Sponsor
- Biogen
Geographic Atrophy Secondary to Age-Related Macular Degeneration
A Study to Assess the Efficacy and Safety of RO7965032 in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration
This study will assess the safety and efficacy of RO7965032 to address a significant unmet medical need in participants with geographic atrophy (GA) secondary to age-related macular degeneration (AMD)
- Trial name
- NCT07851259
- Testing
- RO7965032
- Where
- 5 sites in United States
- Volunteers
- 321 people
- Sponsor
- Genentech, Inc.
Healthy Volunteer, Muscular Atrophy, Spinal
A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec
In this study, researchers will learn about a study drug called BIIB115 in healthy adult male volunteers and in participants with spinal muscular atrophy (SMA). This study will focus on children with SMA.
- Trial name
- NCT05575011
- Testing
- BIIB115
- Where
- 16 sites in Belgium, Canada and 7 more
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Biogen
Muscular Atrophy, Spinal
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have been diagnosed with SMA through…
- Trial name
- STELLAR-1 (NCT07221669)
- Testing
- Salanersen
- Where
- 14 sites in United States, Australia and 5 more
- Volunteers
- 30 people
- Sponsor
- Biogen
Spinal Muscular Atrophy
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S.
- Trial name
- WeSMA (NCT05232929)
- Testing
- Risdiplam
- Where
- 38 sites in United States, Puerto Rico
- Volunteers
- 403 people
- Sponsor
- Genentech, Inc.
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene…
- Trial name
- HINALEA 2 (NCT05861999)
- Testing
- Risdiplam
- Where
- 17 sites in United States, Germany and 4 more
- Volunteers
- 19 people
- Sponsor
- Hoffmann-La Roche
Geographic Atrophy of the Macula
A Phase 2 Efficacy and Safety Study of GAL-101, 2% Ophthalmic Solution in Non-foveal Geographic Atrophy Secondary to Non-neovascular AMD
Age-related macular degeneration (AMD) affects millions of elderly patients. When advanced, there is Geographic Atrophy (GA) in the retina.
- Trial name
- eDREAM (NCT06659549)
- Testing
- GAL-101
- Where
- 24 sites in United States, Armenia and 6 more
- Volunteers
- 110 people
- Sponsor
- Galimedix Therapeutics Inc
Spinal Muscular Atrophy
A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients
This study aims to assess the safety and effectiveness of onasemnogene abeparvovec intrathecal injection in spinal muscular atrophy (SMA) patients in clinical practice in Japan.
- Trial name
- NCT07767890
- Where
- 4 sites in Japan
- Volunteers
- 80 people
- Sponsor
- Novartis Pharmaceuticals
Geographic Atrophy
A Study Investigating the Efficacy and Safety of Intravitreal Injections of ANX007 in Patients With Geographic Atrophy
This study is being conducted in participants with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) to determine if intravitreal (IVT) injections of ANX007 reduce GA lesion growth rate.
- Trial name
- ARCHER (NCT04656561)
- Testing
- ANX007
- Where
- 53 sites in United States, Australia and 1 more
- Volunteers
- 270 people
- Sponsor
- Annexon, Inc.
Leber Congenital Amaurosis 10, Blindness
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A>G…
- Conditions
- Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders
- Trial name
- HYPERION (NCT06891443)
- Testing
- sepofarsen
- Where
- 18 sites in United States, Australia and 8 more
- Volunteers
- 32 people
- Sponsor
- Laboratoires Thea
Geographic Atrophy
A Safety and Efficacy Study of a One-time Intravitreal Injection of SAR446597 in Participants With Geographic Atrophy Secondary to Age-related Macular Degeneration
This is a sequential Phase 1/2, two-part, multicenter study on safety, tolerability, and efficacy of one-time intravitreal SAR446597 for the treatment of participants with Geographic Atrophy (GA) secondary to Age-related Macular…
- Trial name
- NCT07215234
- Testing
- SAR446597
- Where
- 35 sites in United States, Australia and 1 more
- Volunteers
- 104 people
- Sponsor
- Sanofi
Vulvo Vaginal Atrophy (VVA)
Proof of Concept Study of Vaginal AZU-101 in Vulvovaginal Atrophy in Postmenopausal Women
Study Objectives: Primary: To assess the safety, tolerability, and systemic pharmacokinetics (PK) of AZU-101 Secondary: To evaluate efficacy of daily vaginal doses of AZU-101 in postmenopausal women on vaginal epithelium
- Trial name
- NCT06197568
- Testing
- Lasofoxifene Tartrate
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Azure Biotech Inc.
Vulvar Atrophy
Evaluation of the Safety and Efficacy of Hyaluronic Acid Injection in Labia Majora Augmentation
The goal of this interventional clinical investigation using Genefill Contour® CE marked device outside of its indication is to compare with other hyaluronic acid device already marketed for labia majora augmentation in women…
- Trial name
- NCT06333782
- Testing
- Experimental- Genefill Contour®, Marketed comparator - Desirial®Plus
- Where
- 3 sites in France, Poland
- Volunteers
- 110 people, healthy volunteers welcome
- Sponsor
- Bioscience Cosmetics SL
Geographic Atrophy Secondary to Age-related Macular Degeneration
A Non-Interventional Study Observing Short-Term Progression in Geographic Atrophy (GA)
This is a non-interventional, observational study to provide insights into the short-term progression of GA secondary to AMD in participants aged ≥55 years.
- Trial name
- Pre-GAIN (NCT07144137)
- Where
- 19 sites in United States, United Kingdom
- Volunteers
- 75 people
- Sponsor
- Complement Therapeutics
Geographic Atrophy Secondary to Age-related Macular Degeneration
An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA)
This is a clinical study to evaluate the safety, tolerability and efficacy of CTx001, administered via a single subretinal injection, for GA (secondary to AMD).
- Trial name
- Opti-GAIN (NCT07392255)
- Testing
- CTx001
- Where
- 8 sites in United States, United Kingdom
- Volunteers
- 75 people
- Sponsor
- Complement Therapeutics
Muscular Atrophy, Spinal
A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec
In this study, researchers will learn more about the safety and effects of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have already been treated with…
- Trial name
- STELLAR-2 (NCT07444450)
- Testing
- Salanersen
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Biogen
Spinal Muscular Atrophy
A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia
The aim of this retrospective medical chart review is to describe the clinical outcomes, clinical characteristics, and demographics of patients with spinal muscular atrophy (SMA) type 1 treated with onasemnogene abeparvovec (OA)…
- Trial name
- SMA-REAL (NCT07737756)
- Volunteers
- 6 people
- Sponsor
- Novartis Pharmaceuticals
Geographic Atrophy
A Study to Optimize Subretinal Surgical Delivery and to Evaluate Safety and Activity of Opregen in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (GAlette); Adaptive Optics (AO) Retinal Imaging Substudy in Association With Study GR44251
This study will evaluate the success and safety of subretinal surgical delivery as well as the preliminary activity of OpRegen in participants with geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
- Trial name
- NCT05626114
- Testing
- OpRegen
- Where
- 17 sites in United States, Israel
- Volunteers
- 60 people
- Sponsor
- Genentech, Inc.
Multiple System Atrophy
A Trial of Amlenetug (Lu AF82422) in Participants With Multiple System Atrophy (MSA)
The main goal of this trial is to evaluate the efficacy and safety of amlenetug for the treatment of participants with Multiple System Atrophy (MSA).
- Trial name
- MASCOT (NCT06706622)
- Testing
- Amlenetug
- Where
- 74 sites in United States, Australia and 8 more
- Volunteers
- 401 people
- Sponsor
- H. Lundbeck A/S
Spinal Muscular Atrophy 1
A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I
This is a phase I/II clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atrophy Type I.
- Trial name
- NCT07554924
- Testing
- SKG0201 Injection
- Where
- 3 sites in China
- Volunteers
- 11 people
- Sponsor
- Lanyue Biotech (Hangzhou) Co., Ltd.