Showing 1 - 9 of 9 Muscle, Bone, and Cartilage Diseases & Disorders
A prospective, multi-center, observational study of the safety, tolerability and effectiveness of SPINRAZA in adult patients with Spinal Muscular Atr

Status: Open Not Enrolling

Investigator: Bing Liao

Study Coordinator: KaDeesia Brown

Phone: 713.441.9120

The purpose of this study is to measure the safety and effectiveness of Spinraza in adults with SMA. ... Read more >

A Study to Evaluate CC-92480 in Combination With Carfilzomib and Dexamethasone (480Kd) Versus Carfilzomib and Dexamethasone (Kd) in Participants With Relapsed or Refractory Multiple Myeloma (SUCCESSOR-2)

Status: Enrolling

Investigator: Carrie Yuen

Study Coordinator: Danielle Sewall

Phone: 346.238.2674

The purpose of the study is to compare CC-92480 (BMS-986348) with carfilzomib and dexamethasone (480Kd) against carfilzomib and dexamethasone (Kd) in the treatment of RRMM. ... Read more >

Clinical Trial Readiness for SCA1 and SCA3

Status: Open Not Enrolling

Investigator: Tetsuo Ashizawa

Study Coordinator: Valerie Flores

Phone: 713.363.9803

The investigators plan to fill the gap between the current state of clinical trial readiness and the optimal one for SCA1 and SCA3, which are fatal rare diseases with no treatments. Through US-European collaborations, the investigators will esta ... Read more >

Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1: END-DM1

Status: Open Not Enrolling

Investigator: Ericka Greene

Study Coordinator: Aramide Balogun

Phone: 713.441.6955

Provide natural history data, biomarkers, and biological understanding of the phenotypic heterogeneity of myotonic dystrophy type 1 (DM1). ... Read more >

Global Study of Del-desiran for the Treatment of DM1 (HARBOR)

Status: Enrolling

Investigator: Ericka Greene

Study Coordinator: Aramide Balogun

Phone: 713.441.6955

The study consists of a Screening Period of up to 6 weeks and 54-week Treatment Period. The anticipated duration is approximately 60 weeks. Participants will be randomized to receive an intravenous infusion of either del-desiran or placebo at ... Read more >

Long-Term, Observational, Registry of Patients With Generalized Myasthenia Gravis Who Have Received Treatment With Complement C5 Inhibition Therapies

Status: Enrolling

Investigator: Ericka Greene

Study Coordinator: KaDeesia Brown

Phone: 713.441.9120

Complement component 5 (C5) inhibition therapies have been available for patients with generalized Myasthenia Gravis (gMG) in the United States since the introduction of Soliris® in late 2017. Alexion is developing potential new therapies that ... Read more >

Myotonic Dystrophy Compilation of Clinical Findings

Status: Enrolling

Investigator: Ericka Greene

Study Coordinator: Aramide Balogun

Phone: 713.441.6955

The purpose of this protocol is to make available clinical findings, including blood pressure, pulmonary function tests, cardiac function tests, clinical evaluations, cognitive evaluations, performance tests, medications, routine blood work eval ... Read more >

Neuromuscular Observational Research (MOVR) Data Hub Protocol

Status: Open Not Enrolling

Investigator: Ericka Greene

Study Coordinator: Aramide Balogun

Phone: 713.441.6955

This is a registry of people with neuromuscular diseases. It is the intent to follow patients with ALS and other neuromuscular diseases to determine best practices for care delivery. ... Read more >

Protective Genetic Factors Against Neurological Diseases

Status: Open Not Enrolling

Investigator: Jun Li

Study Coordinator: Aramide Balogun

Phone: 713.441.6955

This study aims to look at the genetic differences between individuals with neurological disorders and healthy controls, and whether these genetic differences contribute to nerve conduction speeds. We expect that this study will identify a small ... Read more >