Clinical trials: SBMA
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Prostate Cancer
Gene Mutations in Patients With Advanced Prostate Cancer That Is Not Responsive to Hormone Therapy
RATIONALE: Gene mutations may make prostate cancer cells unable to attach to androgens. This may permit the growth of prostate cancer. Gene testing may improve the identification of patients with advanced prostate cancer.
- Trial name
- NCT00002924
- Testing
- mutation analysis
- Where
- 49 sites in United States
- Volunteers
- 184 people
- Sponsor
- Alliance for Clinical Trials in Oncology
Acne Vulgaris
Clascoterone for Steroid-related Acne Vulgaris in Transgender Male Patients Receiving Masculinizing Hormone Therapy
Mechanism-based acne treatment for transgender patients receiving testosterone currently does not exist and is an unmet medical need. This study explores clascoterone to treat testosterone induced acne.
- Trial name
- NCT05891795
- Testing
- Clascoterone 1% Top Cream, Vehicle
- Where
- United States
- Volunteers
- 18 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
Spinal Muscular Atrophy
Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam
This was a Phase IIIb open-label, single arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101B in participants with SMA aged 2 to <18 years after the discontinuation of treatment with nusinersen or…
- Trial name
- STRENGTH (NCT05386680)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 27 people
- Sponsor
- Novartis Pharmaceuticals
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
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
Prostate Cancer, Hormone Sensitive Prostate Cancer
Evaluation of Clinical Outcomes of Chemotherapy or Androgen-receptor Targeting Agent (Alone or Combined) or Radiotherapy on Primary Tumor in Addition to Androgen Deprivation Therapy in HOrmone-Sensitive Metastatic Prostate Cancer Patients
The aim of this observational study is to evaluate the clinical outcomes of treatment with docetaxel, ARTA (alone or in combination) or with radiotherapy on the primary tumor for mCSPC, in an unselected population, in clinical…
- Conditions
- Prostate Cancer, Hormone Sensitive Prostate Cancer, Metastatic Tumor
- Trial name
- ECHOS (NCT06473259)
- Testing
- Docetaxel, Apalutamide Oral Tablet, Enzalutamide Oral Tablet
- Where
- 3 sites in Italy
- Volunteers
- 3,000 people
- Sponsor
- Santa Chiara Hospital
Acne Vulgaris
Clascoterone Versus Clindamycin Added to Adapalene/Benzoyl Peroxide for Moderate Facial Acne
This randomised trial compares two treatments added to the same standard acne gel in people with moderate facial acne. All participants will use adapalene 0.1%/benzoyl peroxide 2.5% gel at night.
- Trial name
- CLASCO-ACNE (NCT07881315)
- Testing
- Clascoterone 1% cream, Clindamycin 1% lotion, Adapalene 0.1%/benzoyl peroxide 2.5% gel
- Where
- Pakistan
- Volunteers
- 118 people
- Sponsor
- Allied Hospital Faisalabad
Spinal Muscular Atrophy
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥…
- Trial name
- SMART (NCT04851873)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 24 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
Androgen Insensitivity Syndrome, Metabolic Parameters in AIS, CAIS, PAIS and MAIS
Androgen Receptor, Implications for Health and Wellbeing: Natural History Study of Individuals With Androgen Insensitivity
Background: Androgen effects in humans are usually (but not always) mediated by the androgen receptor which is coded for by the androgen receptor gene (AR gene).
- Conditions
- Androgen Insensitivity Syndrome, Metabolic Parameters in AIS, CAIS, PAIS and MAIS, Tumor Formation in AIS, CAIS, PAIS and MAIS, Sexual Function AIS, CAIS, PAIS and MAIS
- Trial name
- NCT04708431
- Where
- United States
- Volunteers
- 650 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Acne
Topical ASC-J9 Cream for Acne
To evaluate the safety and efficacy of topical 0.1% and 0.025% ASC-J9 creams applied twice daily for facial acne compared to vehicle control.
- Trial name
- NCT01289574
- Testing
- ASC-J9
- Where
- 7 sites in United States, Taiwan
- Volunteers
- 181 people
- Sponsor
- AndroScience Corp
Prostate Cancer
Biomarkers in Bone Marrow and Blood Samples From Patients With Prostate Cancer Treated With Ketoconazole
This research trial studies biomarkers in bone marrow and blood samples from patients with prostate cancer treated with ketoconazole.
- Trial name
- NCT01275651
- Testing
- laboratory biomarker analysis
- Where
- United States
- Sponsor
- Alliance for Clinical Trials in Oncology
Spinal Muscular Atrophy, SMA
UK SMA Patient Registry
Spinal muscular atrophy (SMA) is a form of motor neuron disease, most commonly caused by a mutation in the survival motor neuron 1 gene (SMN1) which results in a wide disease spectrum affecting children and adults.
- Trial name
- NCT04292574
- Testing
- Patient Registry
- Where
- United Kingdom
- Volunteers
- 800 people
- Sponsor
- Newcastle University
Prostate Cancer, Stereotactic Body Radiation Therapy
START-MET HS Prostate Cancer. : SbrT & Androgen Receptor Therapy METastatic HSPC
Phase III study of stereotactic body radiation therapy (SBRT) plus standard of care in castration sensitive oligometastatic prostate cancer patients, defined as androgen deprivation therapy plus radiotherapy to the primary tumor…
- Trial name
- START-MET (NCT05209243)
- Testing
- SBRT, STANDARD OF CARE
- Where
- 2 sites in Spain
- Volunteers
- 266 people
- Sponsor
- Instituto de Investigación en Oncología Radioterápica - Fundación Española de Oncología Radioterápic
Bladder Cancer, Triple Negative Breast Cancer
A Phase I/IIa Trial of HMBD-001 in Advanced HER3 Positive Solid Tumours
This clinical trial is evaluating a drug called HMBD-001 (an anti-HER3 monoclonal antibody) in participants with advanced HER3 positive solid tumours.
- Conditions
- Bladder Cancer, Triple Negative Breast Cancer, Castration-resistant Prostate Cancer, Cervical Cancer
- Trial name
- NCT05057013
- Testing
- HMBD-001, HMBD-001 and enzalutamide
- Where
- 4 sites in United Kingdom
- Volunteers
- 81 people
- Sponsor
- Cancer Research UK
Hyperandrogenism, Polycystic Ovary Syndrome
Hyperandrogenemia and Altered Day-night LH Pulse Patterns
The purpose of this study is to determine if, in mid- to late pubertal girls with hyperandrogenism, androgen-receptor blockade (spironolactone) improves the ability of progesterone to acutely reduce waking luteinizing hormone…
- Conditions
- Hyperandrogenism, Polycystic Ovary Syndrome, Puberty
- Trial name
- CRM008 (NCT03068910)
- Testing
- Micronized progesterone, Spironolactone
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- University of Virginia
Stage II Prostate Adenocarcinoma, Stage III Prostate Adenocarcinoma
Androgen Receptor Antagonist ARN-509 With or Without Abiraterone Acetate, Gonadotropin-Releasing Hormone Analog, and Prednisone in Treating Patients With High-Risk Prostate Cancer Undergoing Surgery
This randomized phase II trial studies how well androgen receptor antagonist ARN-509 works with or without abiraterone acetate, gonadotropin-releasing hormone agonist, and prednisone in treating patients with high-risk prostate…
- Trial name
- NCT02949284
- Testing
- Abiraterone Acetate, Androgen Receptor Antagonist ARN-509, Gonadotropin-releasing Hormone Analog
- Where
- 2 sites in United States
- Volunteers
- 90 people
- Sponsor
- Rutgers, The State University of New Jersey