Phase
Condition
N/ATreatment
GEN6050X intravenous injection
Clinical Study ID
Ages 4-10 Male
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Subject age: 4-10 years old (including 10 years old)
Gender: Male
Patients with DMD gene exon deletion types confirmed by molecular diagnosis: 8-49, 20-49, 22-49, 51, 51-53, 51-55, 51-57, 51-59, 51-60, 51-67, 51-69, 51-75 or 51-78and other mutations amenable to exon 50 skipping.
The participant is able to walk independently and completes the 10-meter walk testwithout assistance.
Participant is able to complete time to stand from supine independently in less than 30s.
The participant is able to cooperate with motor assessment testing.
Receipt of glucocorticoids for 6 months and a stable daily dose for at least 12weeks prior to study entry
Ability to tolerate muscle biopsies under anesthesia with no contraindications tothese procedures.
Exclusion
Exclusion Criteria:
Participants are in the active period of viral infection, including infections suchas TORCH virus, Epstein-Barr(EB) virus, and severe acute respiratory syndromecoronavirus 2 (SARS-COV-2).
Received a live attenuated vaccine within 3 months prior to receiving GEN6050X, orwas exposed to an influenza (or other inactivated) vaccine within 30 days prior toreceiving GEN6050X, or received systemic antiviral, anti-infective, and/orinterferon therapy.
Serological tests found HIV, Hepatitis B Virus(HBV), hepatitis C virus(HCV), andsyphilis infection.
Severe infection (e.g., pneumonia, pyelonephritis, or meningitis) within 4 weeksprior to receiving gene therapy.
With clear symptoms of cardiomyopathy, echocardiography shows that the leftventricular ejection fraction is less than 40%.
Need for continuous or intermittent assisted support from a ventilator.
Diagnosed with autoimmune disease or receiving related treatment for autoimmunedisease.
The following indicators are abnormal in laboratory biochemical testing:
γ-glutamyl transpeptidase (GGT) above the 2-fold upper limit and total bilirubinabove 1.5 times the upper limit, cystatin C (cystatin C) > 1.27 mg/L, hemoglobin (Hgb) < 100 or >200 g/L; Leukocytes (WBC) > 18.5×10^9/L or platelet ≤ 125×10^9/L.
The titer of AAV9 neutralizing antibody determined by cell suppression assay > 1:50.
Patients have received any gene therapy (e.g., adeno associated virus(AAV) genetherapy), cell therapy (e.g., stem cell transplantation), in vivo editing, or exvivo editing therapy (e.g., CRISPR-Cas9, TALEN) in the past.
Participant has any contraindication to immunosuppressive therapy.
Has a medical condition or extenuating circumstance that, in the opinion of theprincipal investigator, is unsuitable for participation in the clinical trial.
The family does not wish to disclose the patient's study participation to theattending physician and other medical providers.
Study Design
Study Description
Connect with a study center
Peking Union Medical College Hospital
Beijing, 100730
ChinaActive - Recruiting
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