Myopathy clinical trials at University of California Health
21 in progress, 10 open to eligible people
Anitocabtagene Autoleucel for the Treatment of Subjects With Non-oncology Plasma Cell-related Diseases
open to eligible people ages 18 years and up
A Phase 1 dose-escalation study designed to evaluate the safety, tolerability, and preliminary efficacy of anito-cel in subjects with generalized myasthenia gravis (GMG). Anitocabtagene autoleucel (anito-cel) is a BCMA-directed CAR-T cell therapy.
at UC Irvine UCLA
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)
at UCSF
Understand How the Study Medicine (PF-06823859) Works in People With Active Idiopathic Inflammatory Myopathies [Dermatomyositis (DM) and Polymyositis (PM)]
open to eligible people ages 18 years and up
The purpose of the study is to understand how the study medicine PF-06823859 works in people with idiopathic inflammatory myopathies (DM and PM). These disorders cause inflammation that weakens the muscles that are important for movement and may also cause skin rash in people with DM. This study is seeking participants who: - Are 18 years of age or older or minimum legal adult age as defined per local regulation, whichever is greater - Have active DM or active PM. - Are receiving a stable dose of 1 corticosteroid taken by mouth and/or 1 traditional immunosuppressant. - Note: Corticosteroids and immunosuppressants are medicines that help reduce inflammation and may signal to the immune system not to attack the body. Dermatomyositis (DM) is a rare disease that causes muscle inflammation that results in muscle weakness and low muscle stamina. Patients with DM have a characteristic skin rash. Polymyositis (PM) is a rare disease that involves mainly muscle inflammation resulting in muscle weakness, that can sometimes be painful. Patients with DM and PM may have trouble going up the steps, walking or getting to a standing position. Some of the participants will receive the study medicine (PF-06823859) and some will receive placebo (which is similar to study medicine but contains no medicine in it). The study medicine or placebo will be given as an intravenous (IV) infusion (directly into the veins), which takes about1 hour; every 4 weeks from Day 1 to Week 48 of the study. Both PF-06823859 and placebo and will be given at the study site. The study will compare the experiences of people receiving study medication to those of the people who do not. This will help to see if PF-06823859 is safe and effective. Participants will take part in this study for about 13 months. During this time, participants will have 15 study visits. These visits will be performed at the study site.
at UC Irvine UCLA
RAY121 in Immunological Diseases (RAINBOW Trial)
open to eligible people ages 18-85
This Phase 1b basket trial will investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity and preliminary efficacy of RAY121, a inhibitor of classical complement pathway, after multiple dose administration in patients with immunological diseases such as antiphospholipid syndrome (APS), bullous pemphigoid (BP), Behçet's Syndrome (BS), dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM) and immune thrombocytopenia (ITP).
at UC Irvine
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
at UC Irvine UCSF
PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
open to eligible people ages 18-60
The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).
at UC Irvine
Characterization of Familial Myopathy and Paget Disease of Bone
open to eligible people ages 18 years and up
The researcher wants to explore the genetic causes of muscle disease. The researcher is particularly interested in muscle disorders that occur in combination with diseases of bone that appear to be passed on from generation to generation. Diffuse Optical Spectroscopy will measure the concentrations of blood, water, and lipids (fats, for example) in your tissues. This device essentially measures the color of tissues in order to determine tissue physiology (its physical and chemical processes).
at UC Irvine
Characterization of Inclusion Body Myopathy Associated With Paget's Disease of Bone and Frontotemporal Dementia (IBMPFD)
open to eligible people ages 18 years and up
The investigators are researching families with inherited inclusion body myopathy (IBM) and/or Paget disease of bone (PDB) and/or dementia (FTD) which is also called IBMPFD. IBMPFD is caused by mutations in the VCP gene. Our main goal is to understand how changes in the VCP gene cause the muscle, bone and cognitive problems associated with the disease. The investigators are collecting biological specimen such as blood and urine samples, family and medical histories, questionnaire data of patients with a personal or family history of VCP associated disease. Participants do not need to have all symptoms listed above in order to qualify. A select group of participants may be invited to travel to University of California, Irvine for a two day program of local procedures such as an MRI and bone scan. Samples are coded to maintain confidentiality. Travel is not necessary except for families invited for additional testing.
at UC Irvine
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.
at UCSF
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
open to all eligible people
The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders.
at UCSD
AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)
Sorry, in progress, not accepting new patients
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no therapies for this serious condition. The protein myotubularin is needed for muscle development and movement. A gene called MTM1 tells the body to make myotubularin. XLMTM is caused by changes, or mutations, in the MTM1 gene. Changes in the MTM1 gene causes low levels of myotubularin to be made, so the muscles do not work properly. XLMTM may also affect the liver, and in some cases, this can be dangerous and threaten the patient´s life. Gene therapy is a way of getting a healthy copy of a gene into the body. This allows the body's cells to make a normal protein that may reduce disease symptoms. AT132 is a gene therapy that gets a healthy MTM1 gene into the body to help improve muscle development and function in young children with the disease. AT132 does not treat liver disease, and because of the way the treatment works, it may make liver problems worse. AT132 was the gene therapy treatment given to children who participated in this study and is not available to the public. In this study, AT132 was given to children for the first time. Due to the occurrence of severe complications and fatalities associated with administration of AT132, the study has been stopped and no further participants will be enrolled. The main aim of the study is to check how long young children need machines to support breathing (ventilation support) after AT132. Due to the occurrence of severe complications and fatalities associated with administration of AT132, the study has been stopped and no further participants will be enrolled. This study included children with XLMTM under 5 years old who had breathing problems caused by XLMTM. They couldn't take part if they were born prematurely, recently had surgery, had liver disease or other condition or disease the study doctor thought was medically important. The study did enroll participants with medically significant liver disease. This is an open-label study. This means that young children and their caregivers, and clinic staff know that young children received AT132. This study was designed with 2 parts and is now in a long-term follow-up phase to collect information on the safety and improvements in muscle function in the children who received AT132. In Part 1, small groups of young children were given different doses of AT132, with one group receiving a lower dose and one group receiving a higher dose of AT132. The purpose of giving the two doses was to determine which dose was best for treating the muscle disease. After receiving AT132, a medical panel of experts reviewed each child for safety and for how their muscles responded. AT132 did not demonstrate appropriate safety at either dose. Administration of AT132 was stopped. Children who received AT132 are being monitored for 10 years for safety and to understand how their muscles function over time.
at UCLA
Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
Sorry, in progress, not accepting new patients
HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called deramiocel (CAP-1002) in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either deramiocel or placebo every 3 months for a total of 4 doses during the first 12 months of the study. All participants will be eligible to receive 4 doses of deramiocel for an additional 12 months as part of an open-label extended assessment period. After completion of the first open-label extension (Months 12-24), subjects who have completed Month 24 are eligible to continue onto a Long-Term Open-Label Extension period that will provide treatment with deramiocel until commercial availability, or until sponsor's decision to terminate the trial, or the participant withdraws consent.
at UC Davis UCSD
Nipocalimab in Participants With Active Idiopathic Inflammatory Myopathies
Sorry, in progress, not accepting new patients
The purpose of this study is to evaluate the efficacy and safety of Nipocalimab versus placebo in participants with active idiopathic inflammatory myopathies (IIM).
at UC Irvine UCLA
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.
at UC Irvine UCSF
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
at UC Irvine UCLA UCSF
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/.
at UC Irvine UCSF
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).
at UCSF
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Sorry, in progress, not accepting new patients
AOC 1001-CS2 (MARINA-OLE) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients
at UCLA
ManNAc for GNE Myopathy
Sorry, in progress, not accepting new patients
GNE myopathy is a rare genetic muscle disease characterized by progressive muscle atrophy and weakness. The disease is caused by mutations in the gene that encodes the enzyme that initiates and regulates N-acetylneuraminic acid (Neu5Ac) biosynthesis and glycan sialylation. Currently, there is no therapy available for this disease. N-Acetylmannosamine (ManNAc), an orphan drug in development for GNE myopathy, is an uncharged monosaccharide and the first committed precursor in Neu5Ac biosynthesis. In this randomized, double-blind, placebo-controlled trial the efficacy and long-term safety of ManNAc will be evaluated in subjects with GNE myopathy.
at UCLA
Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)
Sorry, in progress, not accepting new patients
SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to assess efficacy and safety of single daily subcutaneous (SC) administration of elamipretide as a treatment for subjects with primary mitochondrial myopathy associated with nuclear DNA mutations (nPMD).
at UCSD
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.
at UCSF
Our lead scientists for Myopathy research studies include Robert Naviaux, MD Virginia Kimonis, MD Christina Charles-Schoeman, MD Craig McDonald, MD Richard Haas, MD PhD Edward Hsiao Tahseen Mozaffar, MD Perry B. Shieh, MD, PhD Susan Kim, MD, MMSc.
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