Clinical trials: Spinal Muscular Atrophy
Muscular Atrophy, Spinal
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital…
- Trial name
- DEVOTE (NCT04089566)
- Testing
- Nusinersen
- Where
- 45 sites in United States, Brazil and 16 more
- Volunteers
- 145 people
- Sponsor
- Biogen
Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 4
Spinal Cord Stimulation in Spinal Muscular Atrophy
Spinal cord stimulation (SCS) has shown remarkable efficacy in restoring motor function in people with spinal cord injury by recruiting afferent input to enhance the responsiveness of spared neural circuits to residual cortical…
- Trial name
- SCSinSMA (NCT05430113)
- Testing
- Spinal Cord Stimulator (octopolar Medtronic Vectris Leads)
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Marco Capogrosso
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
Breast Cancer
Phase II Trial of SAHA & Tamoxifen for Patients With Breast Cancer
Phase II trial to explore the efficacy of vorinostat and tamoxifen combined.
- Trial name
- NCT00365599
- Testing
- suberoylanilide hydroxamic acid (SAHA, Vorinostat), tamoxifen citrate (Tamoxifen)
- Where
- 8 sites in United States
- Volunteers
- 43 people
- Sponsor
- H. Lee Moffitt Cancer Center and Research Institute
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III
The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy types II or III; and to determine if the drug has an effect on SMN mRNA and protein levels.
- Trial name
- NPTUNE01 (NCT00439569)
- Testing
- sodium phenylbutyrate
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Westat
Spinal Muscular Atrophy
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA).
- Trial name
- NCT01839656
- Testing
- nusinersen
- Where
- 4 sites in United States, Canada
- Volunteers
- 21 people
- Sponsor
- Biogen
Spinal Muscular Atrophy
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in pre-symptomatic patients with SMA and 2 or 3 copies SMN2
- Trial name
- SPR1NT (NCT03505099)
- Testing
- onasemnogene abeparvovec-xioi
- Where
- 16 sites in United States, Australia and 4 more
- Volunteers
- 30 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy
A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically…
- Trial name
- NURTURE (NCT02386553)
- Testing
- Nusinersen
- Where
- 21 sites in United States, Australia and 5 more
- Volunteers
- 25 people
- Sponsor
- Biogen
Spinal Muscular Atrophy Type 3
Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) are one of the common physical disabilities in childhood. For SMA, progressive muscle weakness and early fatigue hamper the mobility of the sufferers.
- Trial name
- NCT03056144
- Testing
- whole body vibration therapy
- Where
- Hong Kong
- Volunteers
- 1 people
- Sponsor
- The Hong Kong Polytechnic University
Spinal Muscular Atrophy
A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants
This will be a Phase I, 2-part, open-label, non-randomized study to investigate the safety, tolerability, and pharmacokinetics (PK) of a multiple-dosing regimen of risdiplam (Part 1) and the effect of risdiplam on the PK of…
- Trial name
- NCT03988907
- Testing
- Risdiplam, Midazolam
- Where
- United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Muscular Dystrophy, Arthrogryposis
WREX Outcome Study
This study is to evaluate how the Wilmington Robotic Exoskeleton (WREX) is working for children who are using the WREX, or have used it in the past.
- Conditions
- Muscular Dystrophy, Arthrogryposis, Spinal Muscular Atrophy
- Trial name
- NCT02218593
- Testing
- WREX orthosis
- Volunteers
- 25 people
- Sponsor
- Nemours Children's Clinic
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy
The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- TOPAZ (NCT03921528)
- Testing
- SRK-015
- Where
- 16 sites in United States, Italy and 2 more
- Volunteers
- 58 people
- Sponsor
- Scholar Rock, Inc.
SMA - Spinal Muscular Atrophy, Gene Therapy
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
- Trial name
- STR1VE (NCT03306277)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 16 sites in United States
- Volunteers
- 22 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA)…
- Trial name
- Jewelfish (NCT03032172)
- Testing
- Risdiplam
- Where
- 24 sites in United States, Belgium and 7 more
- Volunteers
- 174 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy
A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).
The primary objective of Part 1 of this study is to assess the safety and tolerability of Nusinersen in participants with SMA who are not eligible to participate in the clinical studies ISIS 396443-CS3B (NCT02193074) or ISIS…
- Trial name
- EMBRACE (NCT02462759)
- Testing
- Nusinersen
- Where
- 7 sites in United States, Germany
- Volunteers
- 21 people
- Sponsor
- Biogen
SMA
Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in patients with spinal muscular atrophy (SMA) Type 1 who meet enrollment criteria and are genetically defined by a…
- Trial name
- STRIVE-EU (NCT03461289)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 10 sites in Belgium, France and 2 more
- Volunteers
- 33 people
- Sponsor
- Novartis Gene Therapies
Duchenne Muscular Dystrophy, Spinal Muscular Atrophy
Flu Vaccine Study in Neuromuscular Patients 2011
The purpose of the study is to compare the immune response of two different injection methods (Intramuscular V.S. Subcutaneous) of the 2011-2012 seasonal Influenza (Flu) vaccine among patients with neuromuscular conditions who…
- Conditions
- Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Congenital Muscular Dystrophy
- Trial name
- NCT01422200
- Testing
- 2011-2012 seasonal flu vaccine Subcutaneous, 2011-2012 seasonal flu vaccine Intramuscular
- Volunteers
- 22 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Small Cell Neuroendocrine Carcinoma, Prostate Cancer
Apalutamide Plus Cetrelimab in Patients With Treatment-Emergent Small Cell Neuroendocrine Prostate Cancer
Despite the low androgen receptor (AR) transcriptional activity of treatment-emergent small cell neuroendocrine prostate cancer, there is persistent AR expression observed in the majority of treatment-emergent small-cell…
- Conditions
- Small Cell Neuroendocrine Carcinoma, Prostate Cancer, Small Cell Carcinoma
- Trial name
- NCT04926181
- Testing
- Apalutamide, Cetrelimab
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Rahul Aggarwal
Muscular Atrophy, Spinal
A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Type I Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Type I SMA; and to detect the…
- Trial name
- NCT00568698
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Stanford University
Muscular Atrophy, Spinal
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)
This was a phase IV Open-label, single-arm, single-dose, multicenter study, to evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with SMA with bi-allelic mutations in…
- Trial name
- OFELIA (NCT05073133)
- Testing
- OAV101
- Where
- 5 sites in Argentina, Brazil
- Volunteers
- 16 people
- Sponsor
- Novartis Pharmaceuticals