Clinical trials: Muscular Atrophy, Spinal
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
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
Muscular Atrophy, Spinal
A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies
The aim of this study was to assess real-world dosing patterns, long-term healthcare costs, and characteristics of SMA patients who received treatment with DMTs. DMTs included onasemnogene abeparvovec, nusinersen, and risdiplam.
- Trial name
- NCT07378943
- Where
- United States
- Volunteers
- 4,114 people
- Sponsor
- Novartis Pharmaceuticals
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
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved…
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- SAPPHIRE (NCT05156320)
- Testing
- Apitegromab
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 188 people
- Sponsor
- Scholar Rock, Inc.
Spinal Muscular Atrophy
Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
The purpose of this trial is to evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with Spinal Muscular Atrophy with 3 copies of SMN2 and deletion of SMN1.
- Trial name
- STRONG (NCT03381729)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 11 sites in United States
- Volunteers
- 32 people
- Sponsor
- Novartis Gene Therapies
Progressive Metastatic Prostate Cancer
An Asian Study to Evaluate Efficacy and Safety of Oral Enzalutamide in Progressive Metastatic Prostate Cancer Participants
Purpose of the study was to assess the effect of enzalutamide on time to Prostate Specific Antigen (PSA) progression as compared to placebo in chemotherapy naïve participants with progressive metastatic prostate cancer who have…
- Trial name
- NCT02294461
- Testing
- Enzalutamide
- Where
- 47 sites in China, Hong Kong and 2 more
- Volunteers
- 395 people
- Sponsor
- Astellas Pharma Inc
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
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
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
Postpoliomyelitis Syndrome
A Randomized, Double Blind, Placebo Controlled Trial L-carnitine and Piracetam in the Treatment of Weakness, Muscle Fatigue and Muscle Pain in the Postpoliomyelitis Syndrome
This protocol aims to assess of L-carnitine and piracetam to relieve weakness, muscle fatigue and muscle pain in patients with Postpoliomyelitis Syndrome.
- Trial name
- NCT01549847
- Testing
- L-carnitine and piracetam
- Where
- Brazil
- Sponsor
- Biolab Sanus Farmaceutica
Spinal Muscular Atrophy
A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
The primary objective of the study is to explore the convergent validity of smartphone-based Konectom DOAs against in-clinic standard assessments.
- Trial name
- DigiNOA (NCT05109637)
- Testing
- Konectom NMD Application
- Where
- 5 sites in Germany
- Volunteers
- 93 people
- Sponsor
- Biogen
Metastatic Breast Cancer
Efficacy Evaluation of Enobosarm Monotherapy in Treatment of AR+/ER+/HER2- Metastatic Breast Cancer
To demonstrate the efficacy of enobosarmin the treatment of androgen receptor positive (AR+) and estrogen receptor positive (ER+) metastatic breast cancer (MBC) as measured by radiographic progression free survival (rPFS).
- Trial name
- ARTEST (NCT04869943)
- Testing
- Enobosarm, Exemestane
- Where
- 54 sites in United States, Poland and 2 more
- Volunteers
- 52 people
- Sponsor
- Veru Inc.
Spinal and Bulbar Muscular Atrophy
Specified Drug-Use Survey of Leuprorelin Acetate Injection Kit 11.25 mg "All-Case Investigation: Spinal and Bulbar Muscular Atrophy (SBMA)"
The purpose of this survey is to evaluate the long-term safety and efficacy of leuprorelin acetate injection kit 11.25 mg in patients with spinal and bulbar muscular atrophy (SBMA) in the routine clinical setting.
- Trial name
- NCT03555578
- Testing
- Leuprorelin Acetate
- Where
- Japan
- Volunteers
- 1,905 people
- Sponsor
- Takeda
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- ONYX (NCT05626855)
- Testing
- Apitegromab
- Where
- 49 sites in United States, Belgium and 7 more
- Volunteers
- 238 people
- Sponsor
- Scholar Rock, Inc.
Spinal Muscular Atrophy
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
The primary objective of the study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally (IT) to participants with infantile-onset with infantile-onset spinal muscular atrophy (SMA).
- Trial name
- ENDEAR (NCT02193074)
- Testing
- nusinersen
- Where
- 31 sites in United States, Australia and 11 more
- Volunteers
- 122 people
- Sponsor
- Biogen
Muscular Dystrophies, Spinal Muscular Atrophy
Schulze Muscular Dystrophy Ability Clinical Study
The primary objective of the Schulze study is to evaluate the function of the upper limbs of subjects diagnosed with neuromuscular disorders, with and without use of the Abilitech Assist device in the clinic and home…
- Conditions
- Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT05409079
- Testing
- Abilitech Assist
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- AbiliTech Medical Inc.
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 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
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3
- Trial name
- SYNAPSE-SMA (NCT05794139)
- Testing
- NMD670
- Where
- 25 sites in United States, Belgium and 6 more
- Volunteers
- 52 people
- Sponsor
- NMD Pharma A/S