A Study to Evaluate the Safety and Tolerability of Rituxan in Duchenne Muscular Dystrophy

NCT07475754 | Not Yet Recruiting

• 1 other identifier: CD20-RTX-DMD-IIT
• Study Type: interventional
• Target: 5
• Locations: 0 countries
• Sites: N/A

Brief Summary

1. 1.Study population:It is applicable to male participants with genetically confirmed and clinically confirmed Duchenne muscular dystrophy (DMD), aged between 6 and 10 years.
2. 2.Research period:The main research period of this clinical study is one year. Participants were tested during the baseline period and were followed up on days 0, 7, 14, 21, 60, 120, 200, and 360.
3. 3.Exploratory indicators：MR Of both thighs, quantitatively calculating the muscle fat replacement indicators of the buttocks and proximal thighs；Patient Self-Rating Scale, Caregiver Self-Rating Scale.
4. 4.Safety assessment：The safety assessment population will include all participants who have received the drug dose and have at least one post-drug safety assessment. Adverse events (AE) collected from the participants signed informed consent, all the way to the main study period at the end of the last follow-up. Safety laboratory evaluation, laboratory safety monitoring, including hematology, blood biochemistry, urine analysis (including troponin I, CK and CK - MB) and blood coagulation function, as well as complement. All common medication will be recorded. All adverse events, including abnormal complete blood cell count results, will be continuously tracked until they are resolved or stabilized. Only treatment-related adverse events (TEAE) will be summarized. AEs will be based on MedDRA and organ systems are recorded and archived. The classification and terminology related to AEs will be described according to the version of CTCAE v6.0.

Conditions

Duchenne Muscular Dystrophy (DMD)

Outcome Measures

Primary Outcomes (1)

• The incidence of adverse events (AEs)；

  The incidence of adverse events (AEs) will be classified according to version 6.0 of the General Terminology Standard for Adverse Events (CTCAE)；

  1 year,

Secondary Outcomes (6)

• motor function assessment after using IP 200-day and 360-day

  From enrollment to the end of trial at one year；

• motor function assessment after using IP 200-day and 360-day

  From enrollment to the end of trial at one year；

• motor function assessment after using IP 200-day and 360-day

  From enrollment to the end of trial at one year；

• motor function assessment after using IP 200-day and 360-day

  From enrollment to the end of trial at one year；

• motor function assessment after using IP 200-day and 360-day

  From enrollment to the end of trial at one year；

Study Arms (1)

Rituxan treatment in Duchenne Muscular Dystrophy

OTHER

Dosage form:injection,intravenous drip； Dosage:The dosage was calculated based on the body surface area. For the first and second administrations, 375mg/m2 BSA was used. For the third and fourth administrations, the dose was halved to 187.5mg/m2 BSA； Frequency of administration:administered intravenously for 4 weeks, once a week, for a total of 4 times；

Drug: Rituxan treatment

Interventions

Rituxan treatment DRUG

Rituxan treatment

Rituxan treatment in Duchenne Muscular Dystrophy

Eligibility Criteria

• Age: 6 Years - 10 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participant age: 6-10 years old (including 10 years old).
• Gender: male.
• Gene diagnosis of DMD gene type, conform to the DMD phenotypes and clinical manifestations.
• There has been a muscle weakness, symptoms of motor function decline.
• Patients could walk independently, able to complete 10 meters walk test. Lie on your back up,
• patients can complete independently and got up time is less than 30 seconds.
• Participants cognitive assessment and mental state can cooperate movement.
• Never accept oral hormone therapy or stop using hormone therapy more than six months.
• Willing to accept and be able to cooperate with more than one muscle biopsies.

You may not qualify if:

• The participants are in active virus infection or other pathogen infection, including but not limited to, the TORCH virus, Epstein - Barr virus, the new crown virus, bacteria, fungi, etc.
• Having received a live attenuated vaccine or systemic antiviral, anti-infective and/or interferon therapy within 3 months prior to the treatment with investigational product.
• Serological tests revealed infections of HIV, HBV, HCV and syphilis.
• Severe infections (such as pneumonia, pyelonephritis or meningitis) occurred within 8 weeks before the start of treatment with investigational product.
• There are clear symptoms of cardiomyopathy, and echocardiography shows that the left ventricular ejection fraction is less than 50%.
• Continuous or intermittent assistance support from a ventilator is required.
• Laboratory biochemical tests the following indices abnormal: gamma GGTP (gamma glutamyl transpeptidase) 2 times higher than upper limit (GGT), total bilirubin is higher than 1.5 times the upper limit of the elf inhibition C (cystatin C) \> 1.27 mg/L, hemoglobin (Hgb) \< 100 g/L or \> 200 g/L; White blood cell (WBC) \<4×109/L or \>18.5×109/L or platelet ≤125×109/L.
• The patient has received any type of gene therapy (such as AAV gene therapy), cell therapy (such as stem cell transplantation), in vivo editing or in vitro editing reinfusion gene editing therapy (such as CRISPR-Cas9, TALEN), or other experimental drug treatments in the past.
• Participants have any taboos on immunosuppressive therapy.
• Other comorbid diseases or conditions that the principal investigators considered unsuitable for participation in clinical trials.
• The families of the participants do not wish to publicly disclose the patients' research participation to the attending physician and other medical providers.

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Yi Dai, Doctor

CONTACT

010-69151360; pumchdy@sina.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Deputy Director of the Neurology Department,archiater

Study Record Dates

• First Submitted: March 2, 2026
• First Posted: March 16, 2026
• Study Start: March 23, 2026
• Primary Completion: May 30, 2026
• Study Completion (Estimated): April 23, 2027
• Last Updated: March 16, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL