Myopathy clinical trials at UCSF
8 in progress, 3 open to eligible people
Andecaliximab in Participants With Fibrodysplasia Ossificans Progressiva (FOP)
open to eligible people ages 2 years and up
This study is researching an experimental drug called andecaliximab. The study will include pediatric and adult patients with fibrodysplasia ossificans progressiva (FOP). The study will evaluate how safe and effective andecaliximab is in patients with FOP. The study is looking at several research questions, including: - Safety of andecaliximab in participants with FOP - Whether andecaliximab reduces the number of new heterotopic bone lesions (Heterotopic Ossification; HO) - Whether andecaliximab reduces the number or severity of flare-ups - Pharmacokinetics/pharmacodynamics (PK/PD): How much study drug is in your blood at different times and its impact on blood biomarker(s) - Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)
San Francisco 5391959, California 5332921 and other locations
RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
open to eligible people ages 6-75
RESET-Myositis: Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects with Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
San Francisco 5391959, California 5332921 and other locations
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
open to all eligible people
The primary objective of this study is to assess the long-term safety, including pregnancy, infant, and lactation outcomes, of patients with LC-FAOD who are enrolled in the DMP.
San Francisco 5391959, California 5332921 and other locations
Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Active Idiopathic Inflammatory Myopathy
Sorry, accepting new patients by invitation only
The purpose of this study is to measure the long-term safety and tolerability of efgartigimod PH20 SC in adult participants with IIM who previously participated in ARGX-113-2007. Secondary objectives include efficacy measures of efgartigimod PH20 SC in participants with IIM.
San Francisco 5391959, California 5332921 and other locations
ABC008 for Inclusion Body Myositis
Sorry, in progress, not accepting new patients
A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body Myositis
San Francisco 5391959, California 5332921 and other locations
Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy.
Sorry, in progress, not accepting new patients
This study's purpose is to measure the treatment response from efgartigimod PH20 SC compared with placebo in participants with Idiopathic Inflammatory Myopathy (IIM). Participants with the IIM subtypes of dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM), or certain other subtypes of polymyositis (PM; including antisynthetase syndrome [ASyS]) will be included in the study. Treatment response will be measured by Total improvement score (TIS). Additional information can be found on https://myositis-study.com/.
San Francisco 5391959, California 5332921 and other locations
Encaleret Compared to Standard of Care in Participants With ADH1
Sorry, in progress, not accepting new patients
The primary purpose of the study is to understand the effectiveness, safety, and tolerability of encaleret when compared to standard of care (SoC) treatment in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1).
Oakland 5378538, California 5332921 and other locations
ADH1 and ADH2 Disease Monitoring Study (DMS)
Sorry, in progress, not accepting new patients
A global, multi-center, Disease Monitoring Study (DMS) in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1) or Autosomal Dominant Hypocalcemia Type 2 (ADH2) designed to characterize ADH1 and ADH2 disease presentation and progression through retrospective (past) and longitudinal prospective (over time into the future) data collection.
Oakland 5378538, California 5332921 and other locations
Our lead scientists for Myopathy research studies include Susan Kim, MD, MMSc Edward Hsiao.
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