Clinical trials: SMA - Spinal Muscular Atrophy
Muscular Atrophy, Spinal
A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy
The aim of this study was to evaluate real-world treatment patterns and clinical outcomes in pediatric SMA patients in the Czech and Slovak Republics, using data from the Registry of muscular Dystrophy (REaDY).
- Trial name
- NCT07474311
- Where
- Czechia
- Volunteers
- 213 people
- Sponsor
- Novartis Pharmaceuticals
Spinal Muscular Atrophy
A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA
An Open-label, Long-term Follow-Up Study to Evaluate the Safety and Tolerability of Gene Therapy with EXG001-307 in SMA1 patients ,who joined the parent study (EXG001-307-102)
- Trial name
- NCT06588803
- Testing
- No intervention, only for observational studies
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Muscular Atrophy, Spinal
Pediatric Spinal Muscular Atrophy (SMA) China Registry
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) treatments among pediatric Chinese participants with spinal muscular atrophy linked to chromosome 5q…
- Trial name
- NCT05042921
- Where
- 25 sites in China
- Volunteers
- 600 people
- Sponsor
- Biogen
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
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
Muscular Atrophy, Spinal
A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)
The purpose of this open-label, single arm study is to further evaluate long-term tolerability, safety and efficacy outcomes of olesoxime in participants with Spinal Muscular Atrophy (SMA) who previously participated in one of…
- Trial name
- NCT02628743
- Testing
- Olesoxime
- Where
- 24 sites in Belgium, France and 5 more
- Volunteers
- 131 people
- Sponsor
- Hoffmann-La Roche
Prostate Cancer
A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer Drugs
ANDROMEDA is a first-in-human, Phase I/II, open-label, multicenter study of AZD9750 in participants with metastatic prostate cancer.
- Trial name
- ANDROMEDA (NCT07336446)
- Testing
- AZD9750, AZD5305
- Where
- 5 sites in United States, Australia and 2 more
- Volunteers
- 6 people
- Sponsor
- AstraZeneca
Spinal Muscular Atrophy (SMA), Carrier Screening
Multi-disease Carrier Screening Test Validation
The purpose of this study is to collect blood samples to enable validation of genetic testing for diseases within a multi-disease carrier screening panel.
- Conditions
- Spinal Muscular Atrophy (SMA), Carrier Screening, Genetic Testing
- Trial name
- NCT01663584
- Testing
- Blood draw
- Where
- United States
- Sponsor
- Natera, Inc.
Spinal Muscular Atrophy
An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy
This study will test the safety, tolerability, and pharmacokinetics of escalating doses of nusinersen (ISIS 396443) administered into the spinal fluid either two or three times over the duration of the trial, in participants with…
- Trial name
- NCT01703988
- Testing
- Nusinersen
- Where
- 4 sites in United States
- Volunteers
- 34 people
- Sponsor
- Biogen
Spinal Muscular Atrophy
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)
The primary objective of this study is to examine the safety and tolerability of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA) who previously participated in ISIS…
- Trial name
- NCT01780246
- Testing
- nusinersen
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- Biogen
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
ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)
The primary purpose is to address critical evidence in the treatment landscape for Spinal Muscular Atrophy (SMA), specifically focusing on the intrathecal formulation of onasemnogene abeparvovec-brve (ITVISMA®). U.S.
- Trial name
- NCT07448610
- Testing
- Onasemnogene Abeparvovec-brve
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
Spinal Muscular Atrophy (SMA)
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial.
- Trial name
- SPECTRUM (NCT05335876)
- Testing
- onasemnogene abeparvovec
- Where
- 32 sites in United States, Australia and 17 more
- Volunteers
- 20 people
- Sponsor
- Novartis Pharmaceuticals
Neuromuscular Disorders, Neurodevelopmental Disorders
Efficacy and Usability of the EXPLORER Exoskeleton in Children With Neurodevelopmental Disorders in Natural Environments
Neurodevelopmental disorders often lead to abnormal development of the Central Nervous System (CNS), frequently causing motor dysfunctions such as an inability to stand and walk.
- Trial name
- NCT06765070
- Testing
- EXPLORER
- Where
- 6 sites in Spain
- Volunteers
- 15 people
- Sponsor
- MarsiBionics
Muscular Atrophy, Spinal
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusinersen is given by lumbar puncture (LP).
- Trial name
- PIERRE-PK (NCT06555419)
- Testing
- Nusinersen, ThecaFlex DRx System
- Where
- 21 sites in United States, France and 5 more
- Volunteers
- 78 people
- Sponsor
- Biogen
Spinal Muscular Atrophy
An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)
An open-label, multi-part, first-in-human study of oral branaplam in infants with Type 1 spinal muscular atrophy. The purpose of this study was to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD)…
- Trial name
- NCT02268552
- Testing
- branaplam
- Where
- 14 sites in Belgium, Bulgaria and 5 more
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
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
Muscular Atrophy, Spinal
A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function
This is a multi-center, open-label, non-randomized, parallel-group, 2-part study to evaluate the effect of hepatic impairment on the PK and safety and tolerability of a single oral dose of risdiplam compared to matched healthy…
- Trial name
- NCT03920865
- Testing
- Risdiplam
- Where
- 3 sites in United States
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Cerebral Palsy, SMA II
Evaluation of the Exoskeleton ATLAS 2030 as Robot-assisted Physical Therapy to Children With Neuromuscular Diseases
This study aims to evaluate the safety, usability and acceptability of a motorized mobility assistance exoskeleton. The protocol explores the use of the motorized device during static and dynamic rehabilitation sessions focused…
- Conditions
- Cerebral Palsy, SMA II, Neuromuscular Diseases
- Trial name
- NCT04837157
- Testing
- Exoskeleton rehabilitation
- Where
- Spain
- Volunteers
- 6 people
- Sponsor
- MarsiBionics
Muscular Atrophy, Spinal
A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)
In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA).
- Trial name
- RESPOND (NCT04488133)
- Testing
- Nusinersen
- Where
- 15 sites in United States, Germany and 3 more
- Volunteers
- 46 people
- Sponsor
- Biogen