Clinical trials: SMA II
Spinal Muscular Atrophy Type 2
GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular Atrophy
This study explored dose escalation of single-arm, open, single intrathecal injection in patients with delayed onset type 2 SMA. The investigator plans to conduct 2 cohorts.
- Trial name
- GITOPWTSMA (NCT06772402)
- Testing
- GCB-001
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Genecombio Ltd.
Acne Vulgaris
Toxicity and Efficacy Evaluation of Topical Minoxidil in Acne Vulgaris
Patients with acne vulgaris (AV) appeared to be a chronic inflammation with a wide range in teenagers and adult. The protocol design is as follows.
- Trial name
- NCT06108193
- Testing
- topical minoxidil in Acne Vulgaris
- Where
- Taiwan
- Volunteers
- 26 people
- Sponsor
- Chang Gung Memorial Hospital
Triple Negative Breast Neoplasms
Bicalutamide and Abemaciclib in Inoperable or Metastatic Androgen Receptor-positive Triple-negative Breast Cancer
This study has as goal to evaluate the use of abemaciclib and bicalutamide in androgen receptor positive metastatic triple negative breast cancer.
- Trial name
- ABBICAR (NCT06365788)
- Testing
- Abemaciclib, Bicalutamide
- Where
- 6 sites in Belgium
- Volunteers
- 53 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
SMA II, Cerebral Palsy
Paracetamol Study in Patients With Low Muscle Mass
To investigate the safety and toxicity related to paracetamol treatment in children and adults with respectively SMA and CP.
- Trial name
- NCT03648658
- Testing
- Paracetamol 120Mg/5mL Oral Suspension
- Where
- Denmark
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Mette Cathrine Oerngreen
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Body Weight Support Harness System in Spinal Muscular Atrophy
The goal of this pilot interventional study is to learn about the use of an in-home harness system in children who have been treated for spinal muscular atrophy. The main questions it aims to answer are: 1.
- Trial name
- NCT05715749
- Testing
- In-home body weight support harness system
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Nationwide Children's Hospital
Spinal Muscular Atrophy
Monitoring to the Evolution of Motor Function in SMA Type II Adults Patients Treated With SPINRAZA®
SPINRAZA® (Nusinersen) is the first intrathecal administered drug which was approved by the FDA to treat SMA children and adults (2016).
- Trial name
- SMAII (NCT04159987)
- Testing
- Spinraza intrathecal injection
- Where
- 7 sites in France
- Volunteers
- 20 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
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, Spinal Muscular Atrophy Type 3
Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb
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…
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy 4
- Trial name
- NCT06300996
- Testing
- Spinal Cord Stimulator (Octopolar Medtronic Vectris Leads)
- Where
- United States
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- Marco Capogrosso
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).
- Trial name
- NCT06632730
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Novartis
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
SMA - Spinal Muscular Atrophy
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III
This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously received nusinersen.
- Trial name
- NCT06971094
- Testing
- GC101 adeno-associated virus injection
- Where
- 7 sites in China
- Volunteers
- 50 people
- Sponsor
- GeneCradle Inc
Muscular Atrophy, Spinal
Pilot Study of Growth Hormon to Treat SMA Typ II and III
The purpose of this study is to determine whether Growth hormon can increase strength in spinal muscular atrophy type II and III.
- Trial name
- NCT00533221
- Testing
- somatotropin
- Where
- 6 sites in Germany
- Volunteers
- 20 people
- Sponsor
- University Hospital Freiburg
Spinal Muscular Atrophy Type I
CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
This is a multi-center trial to test safety and evaluate early treatment intervention with valproic acid and carnitine in moderating SMA symptoms of Type I infants.
- Trial name
- NCT00661453
- Testing
- Valproic Acid and Levocarnitine
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 40 people
- Sponsor
- University of Utah
Breast Cancer
Bicalutamide as A Treatment in AR-positive Metastatic Triple-Negative Breast Cancer (mTNBC) Patients
This prospective, single center, phase II study is to evaluate the efficacy and safety of bicalutamide as a treatment in androgen receptor (AR)-positive metastatic triple-negative breast cancer (mTNBC) patients.
- Trial name
- NCT02348281
- Testing
- Bicalutamide
- Where
- China
- Volunteers
- 1 people
- Sponsor
- Fudan University
Spinal Muscular Atrophy Type II
Exoskeleton Impact on the Quality of Life on Patients With Spinal Muscular Atrophy
The purpose of this study is to evaluate the impact of the use of a pediatric exoskeleton on the quality of life of children, specifically in the psychological and care dimensions.
- Trial name
- NCT05416034
- Testing
- Use of the ATLAS 2025 exoskeleton at home
- Where
- Spain
- Volunteers
- 3 people
- Sponsor
- MarsiBionics
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.
Kennedy's Disease, Spinal and Bulbar Muscular Atrophy
Dutasteride to Treat Spinal and Bulbar Muscular Atrophy (SBMA)
This study will determine if the drug dutasteride can improve weakness, mobility, functioning, nerve function, and quality of life in patients with spinal and bulbar muscular atrophy (SBMA).
- Trial name
- NCT00303446
- Testing
- Dutasteride
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Spinal Muscular Atrophy
A Pilot Study of Biomarkers for Spinal Muscular Atrophy
The goal of this pilot study is to identify a marker or panel of markers in the blood or urine from a wide range of Spinal Muscular Atrophy (SMA) patients that segregates with measures of clinical severity.
- Trial name
- BforSMA (NCT00756821)
- Where
- 18 sites in United States, Canada
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- Carelon Research
Spinal Muscular Atrophy Type III, Spinal Muscular Atrophy Type II
Spinal Cord Gray Matter Imaging in Spinal Muscular Atrophy
This study aims to measure the spinal cord gray matter in patients with spinal muscular atrophy (SMA) type II and III in comparison with age- and sex-matched healthy controls (HC) using rAMIRA (radially sampled averaged…
- Trial name
- NCT06137612
- Testing
- MRI
- Where
- Switzerland
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- University Hospital, Basel, Switzerland
Spinal Muscular Atrophy
Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational Study
This study aims to characterize the clinical-epidemiological profile and baseline characteristics of patients with spinal muscular atrophy (SMA) 5q types II and III in follow-up at the Brazilian Unified Public Health System…
- Trial name
- NCT04404764
- Testing
- Nusinersen Injectable Product
- Where
- 9 sites in Brazil
- Volunteers
- 155 people, healthy volunteers welcome
- Sponsor
- Hospital Israelita Albert Einstein