Clinical trials: Spinal Muscular Atrophy
Spinal Muscular Atrophy (SMA)
Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy
The primary objective of the study is to evaluate anxiety level during intrathecal administration (IT) under standard of care (SOC) and virtual reality (VR) conditions using a reliable self-rating scale.
- Trial name
- REALITY (NCT05354414)
- Testing
- Virtual reality, Standard of Care
- Where
- 12 sites in France
- Volunteers
- 63 people
- Sponsor
- Biogen
SMA
EAP of Apitegromab for Patients With Spinal Muscular Atrophy
The purpose of this expanded access program (EAP) is to provide access to apitegromab for eligible patients with spinal muscular atrophy (SMA) prior to approval by the local regulatory agency .
- Trial name
- NCT06877689
- Testing
- Apitegromab
- Sponsor
- Scholar Rock, Inc.
Prostatic Neoplasm
A Phase 1a/1b Study to Evaluate the Safety of EZN-4176, in Adult Patients With Castration-Resistant Prostate Cancer
This study will evaluate an experimental drug called EZN-4176 to determine the anticancer effects when it is given to patients with an advanced form of prostate cancer called castration-resistant prostate cancer (CRPC).
- Trial name
- NCT01337518
- Testing
- EZN-4176
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 150 people
- Sponsor
- Enzon Pharmaceuticals, Inc.
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi.
- Conditions
- Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
- Trial name
- NCT04042025
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 31 sites in United States, Australia and 7 more
- Volunteers
- 85 people
- Sponsor
- Novartis Gene Therapies
Metastatic Castration-resistant Prostate Cancer
A Study to Compare the Efficacy and Safety of BMS-986365 Versus the Investigator's Choice of Therapy in Participants With Metastatic Castration-resistant Prostate Cancer
The purpose of this study is to compare the efficacy and safety of BMS-986365 versus the investigator's choice of therapy in participants with Metastatic Castration-resistant Prostate Cancer.
- Trial name
- rechARge (NCT06764485)
- Testing
- BMS-986365, Enzalutamide, Abiraterone
- Where
- 227 sites in United States, Argentina and 24 more
- Volunteers
- 960 people
- Sponsor
- Celgene
Spinal Muscular Atrophy (SMA)
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
Risdiplam works by helping the body produce more survival motor neuron (SMN) protein throughout the body. This means fewer motor neurons - nerve cells that pass impulses from nerves to muscles to cause movement - are lost, which…
- Trial name
- MANATEE (NCT05115110)
- Testing
- RO7204239, Risdiplam
- Where
- 30 sites in United States, Australia and 10 more
- Volunteers
- 97 people
- Sponsor
- Hoffmann-La Roche
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
Metastatic Castration Resistant Prostate Cancer
A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1)
Himalayas-1 is a randomized, open-label, global, multicenter phase 3 study evaluating whether the combination of rinzimetostat with darolutamide is more effective compared to physician's choice of control; ARPI (darolutamide or…
- Trial name
- Himalayas-1 (NCT07723248)
- Testing
- Rinzimetostat, Darolutamide, Enzalutamide
- Where
- 12 sites in United States, Puerto Rico
- Volunteers
- 600 people
- Sponsor
- ORIC Pharmaceuticals
Spinal Muscular Atrophy 1
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi.
- Trial name
- START (NCT03421977)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Novartis Gene Therapies
Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis
European Home Mechanical Ventilation Registry
The European Home Mechanical Ventilation Registry (EHMVR) will enable a thorough evaluation of HMV by documenting the characteristics of HMV patients and their treatment.
- Conditions
- Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis, Spinal Cord Injury, Muscular Dystrophies
- Trial name
- EHMVR (NCT02315339)
- Where
- Germany
- Volunteers
- 37 people
- Sponsor
- ResMed
Prostate Cancer
An Open Label Prostate Cancer Study in Japanese Patients
The primary aim of study is to gain an initial assessment of safety and tolerability of AZD3514 in Japanese patients together with assessing Pharmacokinetics (PK) and gaining a preliminary assessment of anti-tumour action.
- Trial name
- NCT01351688
- Testing
- AZD3514
- Where
- 2 sites in Japan
- Volunteers
- 13 people
- Sponsor
- AstraZeneca
Neuromuscular Disorders, Neurodevelopmental Disorders
Safety, Usability, and Effectiveness of a Gait Exoskeleton for Children and Adolescents With Neurodevelopmental Disorders.
Neurodevelopmental disorders often result in abnormal development of the Central Nervous System (CNS), frequently causing motor dysfunctions such as the inability to stand and walk.
- Trial name
- NCT07159360
- Testing
- CLINICAL EXPLORER
- Where
- 2 sites in Mexico, Spain
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- MarsiBionics
Spinal Muscular Atrophy
Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States
This global, retrospective, non-interventional, medical chart review (MCR), descriptive study collected patient-level data in regions outside the US.
- Trial name
- NCT06178393
- Where
- United States
- Volunteers
- 162 people
- Sponsor
- Novartis Pharmaceuticals
Spinal Muscular Atrophy (SMA)
Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)
The goal of this multicenter, open-label, non-comparative, cohort study is to investigate the safety, immunogenicity, and efficacy of ANB-004 in children with spinal muscular atrophy.
- Trial name
- BLUEBELL (NCT05747261)
- Testing
- ANB-004, dose 1, ANB-004, dose 2, ANB-004, dose 3
- Where
- 7 sites in Belarus, Russia
- Volunteers
- 40 people
- Sponsor
- Biocad
Motor Neuron Disease
A Study to Assess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps.
The COMMEND Study will assess the safety and effectiveness of FLX-787 in men and women with Motor Neuron Disease [including Amyotrophic Lateral Sclerosis (ALS), Primary Lateral Sclerosis (PLS) or Progressive Muscular Atrophy…
- Trial name
- NCT03196375
- Testing
- FLX-787-ODT (orally disintegrating tablet)
- Where
- 35 sites in United States
- Volunteers
- 54 people
- Sponsor
- Flex Pharma, Inc.
Muscular Atrophy, Spinal
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2…
- Trial name
- HINALEA 1 (NCT05861986)
- Testing
- Risdiplam
- Where
- 16 sites in United States, Germany and 2 more
- Volunteers
- 28 people
- Sponsor
- Hoffmann-La Roche
Androgenetic Alopecia
Association of Polymorphisms in the Androgen Receptor Gene and Finasteride Response in Women With Androgenetic Alopecia
Previous studies of finasteride treatment in women with hair loss have failed to show positive results, yet, some women have responded anecdotally.
- Trial name
- NCT01052870
- Testing
- Finasteride
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- HairDx, LLC
Spinal Muscular Atrophy
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.
- Trial name
- NCT05824169
- Testing
- GC101
- Where
- 4 sites in China
- Volunteers
- 18 people
- Sponsor
- GeneCradle Inc
Muscular Atrophy, Spinal
A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)
This multicenter, randomized, double-blind, 12-week, placebo-controlled multiple dose study will investigate the safety and tolerability of RO6885247 in adult and pediatric patients with spinal muscular atrophy (SMA).
- Trial name
- NCT02240355
- Testing
- RO6885247
- Where
- 26 sites in United States, Canada and 7 more
- Volunteers
- 9 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam
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 individuals with SMA who are between the ages…
- Trial name
- SOLAR (NCT07444476)
- Testing
- Salanersen
- Where
- 3 sites in United States, China
- Volunteers
- 90 people
- Sponsor
- Biogen