Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Catabasis Pharmaceuticals
The study includes a 104-week open-label treatment period with edasalonexent. Patients who completed CAT-1004-201 or CAT-1004-301 and eligible siblings of these boys will be enrolled in this trial.
Age
4–12
Sex
MALE
Healthy volunteers
Not accepted
You may be eligible if
For Patients who Completed CAT-1004-201 or CAT-1004-301:
Written consent/assent by patient and/or legal guardian as per regional and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements
Completion of either CAT-1004-201 or CAT-1004-301
Written consent/assent by patient and/or legal guardian as per regional and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements
A sibling of a patient who completed either CAT-1004-201 or CAT-1004-301
Diagnosis of DMD based on a clinical phenotype with increased serum creatine kinase (CK) and documentation of mutation(s) in the dystrophin gene known to be associated with a DMD phenotype
Followed by a doctor or medical professional who coordinates Duchenne care on a regular basis and willingness to disclose patient's study participation with medical professionals
You may not be if
In the Investigator's opinion, unwilling or unable for any reason to complete all study assessments and laboratory tests and comply with scheduled visits, administration of drug, and all other study procedures
For Siblings of Patients who Completed CAT-1004-201 or CAT-1004-301:
Use of oral corticosteroids at screening; use of inhaled, intranasal, and topical corticosteroids is permitted
Use of another investigational drug, idebenone, or dystrophin-focused therapy within 4 weeks. Exception: Patients who are currently on or plan to initiate treatment with approved oligonucleotide exon-skipping therapies, and expected to continue treatment throughout the study, will be eligible
Use of the following within 4 weeks prior to Day 1: immunosuppressive therapy, anticoagulants, cyclosporine, dihydroergotamine, ergotamine, fentanyl, alfentanil, pimozide, quinidine, sirolimus or tacrolimus
Use of human growth hormone within 3 months prior to Day 1
Other prior or ongoing significant medical conditions
Clareo Health | An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
