Clinical trials: Neuromuscular Spinal Deformity
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.
Alzheimer's Disease, Multiple Sclerosis
A Biospecimen and Clinical Data Study on Patients for Drug & Biomarker Discovery
The purpose of this research project is to collect and store blood samples and clinical data. Researchers can then use the stored samples in future studies.
- Conditions
- Alzheimer's Disease, Multiple Sclerosis, Parkinson's Disease, Huntington's Disease
- Trial name
- NCT01592552
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Increased Lordosis/Scoliosis, Hyporeflexia
Biomarker for Duchenne Muscular Dystrophy
International, multicenter, observational, longitudinal study to identify biomarker/s for Duchenne Muscular Dystropy (DMD) and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s.
- Conditions
- Increased Lordosis/Scoliosis, Hyporeflexia, Duchenne Muscular Dystrophy, Red-Green Color Blindness
- Trial name
- BioDuchenne (NCT02994030)
- Where
- 12 sites in Albania, Egypt and 6 more
- Volunteers
- 103 people
- Sponsor
- CENTOGENE GmbH Rostock
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 1
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS-101 as a treatment of spinal muscular atrophy Type 1 (SMN1).
- Trial name
- NCT02122952
- Testing
- AVXS-101
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Novartis Gene Therapies
Spinal Muscular Atrophy Type 3
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.
- Trial name
- SMA3 (NCT03819660)
- Testing
- Amifampridine Phosphate 10 MG Oral Tablet
- Where
- Italy
- Volunteers
- 13 people
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients
This is a phase 1/2a randomized, double-blind study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of study drug AJ201 in subjects with Spinal and Bulbar Muscular Atrophy (SBMA).
- Trial name
- NCT05517603
- Testing
- AJ201
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- AnnJi Pharmaceutical Co., Ltd.
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.
Degenerative Disc Disease(DDD)
Evaluation of the Safety and Effectiveness of the CORUS™ Posterior Cervical Stabilization System 3D (PCSS 3D) for the Treatment of 2-level Cervical Degenerative Disease
The objective of this clinical trial is to evaluate the treatment of patients with two-level cervical degenerative disease using a combination of Anterior Cervical Discectomy and Fusion (ACDF) and Posterior Cervical Fusion (PCF)…
- Trial name
- SECOND (NCT07721493)
- Testing
- Circumferential Cervical Fusion (CCF), Anterior Cervical Discectomy and Fusion
- Where
- United States
- Volunteers
- 225 people
- Sponsor
- Providence Medical Technology, Inc.
Friedreich's Ataxia (FA)
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA).
- Trial name
- FALCON (NCT07180355)
- Testing
- SGT-212
- Where
- 3 sites in United States
- Volunteers
- 10 people
- Sponsor
- Solid Biosciences Inc.
Type I Spinal Muscular Atrophy
Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I
The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy.
- Trial name
- NCT06576388
- Testing
- EXG001-307 injection
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Guangzhou Jiayin Biotech Ltd
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Spinal Muscular Atrophy, SMA
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones…
- Conditions
- Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- OPAL (NCT07047144)
- Testing
- Apitegromab, Nusinersen, Risdiplam
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 52 people
- Sponsor
- Scholar Rock, Inc.
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 Type I, Spinal Muscular Atrophy Type II
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi.
- Conditions
- Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
- Trial name
- NCT04042025
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 31 sites in United States, Australia and 7 more
- Volunteers
- 85 people
- Sponsor
- Novartis Gene Therapies
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
Metastatic Castration Resistant Prostate Cancer
A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1)
Himalayas-1 is a randomized, open-label, global, multicenter phase 3 study evaluating whether the combination of rinzimetostat with darolutamide is more effective compared to physician's choice of control; ARPI (darolutamide or…
- Trial name
- Himalayas-1 (NCT07723248)
- Testing
- Rinzimetostat, Darolutamide, Enzalutamide
- Where
- 12 sites in United States, Puerto Rico
- Volunteers
- 600 people
- Sponsor
- ORIC Pharmaceuticals
Spinal Muscular Atrophy 1
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi.
- Trial name
- START (NCT03421977)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Novartis Gene Therapies
Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis
European Home Mechanical Ventilation Registry
The European Home Mechanical Ventilation Registry (EHMVR) will enable a thorough evaluation of HMV by documenting the characteristics of HMV patients and their treatment.
- Conditions
- Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis, Spinal Cord Injury, Muscular Dystrophies
- Trial name
- EHMVR (NCT02315339)
- Where
- Germany
- Volunteers
- 37 people
- Sponsor
- ResMed
Prostate Cancer
An Open Label Prostate Cancer Study in Japanese Patients
The primary aim of study is to gain an initial assessment of safety and tolerability of AZD3514 in Japanese patients together with assessing Pharmacokinetics (PK) and gaining a preliminary assessment of anti-tumour action.
- Trial name
- NCT01351688
- Testing
- AZD3514
- Where
- 2 sites in Japan
- Volunteers
- 13 people
- Sponsor
- AstraZeneca