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

A Single-arm, Non-blind, Single-center Study to Evaluate the Safety and Tolerability of Rituximab Down-regulating Immunoglobulin (Ig) Treatment in Children With Duchenne Muscular Dystrophy (DMD) Who Can Walk

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. 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. 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. 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.

Study Overview

• Status: Not yet recruiting
• Conditions: Duchenne Muscular Dystrophy (DMD)
• Intervention / Treatment: Drug: Rituxan treatment

• Study Type: Interventional
• Enrollment (Estimated): 5
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Yi Dai, Doctor; Phone Number: 010-69151360; Email: pumchdy@sina.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

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

Exclusion Criteria:

1. 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.
2. 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.
3. Serological tests revealed infections of HIV, HBV, HCV and syphilis.
4. Severe infections (such as pneumonia, pyelonephritis or meningitis) occurred within 8 weeks before the start of treatment with investigational product.
5. There are clear symptoms of cardiomyopathy, and echocardiography shows that the left ventricular ejection fraction is less than 50%.
6. Continuous or intermittent assistance support from a ventilator is required.
7. 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.
8. 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.
9. Participants have any taboos on immunosuppressive therapy.
10. Other comorbid diseases or conditions that the principal investigators considered unsuitable for participation in clinical trials.
11. The families of the participants do not wish to publicly disclose the patients' research participation to the attending physician and other medical providers.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Other: Rituxan treatment in Duchenne Muscular Dystrophy; 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; Rituxan treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
motor function assessment after using IP 200-day and 360-day	10-meter Run/Walk Time Test (TTRW) measured in meter;	From enrollment to the end of trial at one year；
motor function assessment after using IP 200-day and 360-day	6-minute walk distance test (6MWT) measured in meter;	From enrollment to the end of trial at one year；
motor function assessment after using IP 200-day and 360-day	Time to get up from a supine position measured in second;	From enrollment to the end of trial at one year；
motor function assessment after using IP 200-day and 360-day	Time Test for Climbing Four Flights of Stairs (TTCLIMB) measured in second	From enrollment to the end of trial at one year；
motor function assessment after using IP 200-day and 360-day	Measure muscle strength (elbow extension, elbow flexion, knee extension and knee flexion) with a handheld dynamometer measured in Newton;	From enrollment to the end of trial at one year；
motor function assessment after using IP 200-day and 360-day	Upper limb Function Test (PUL2.0 Scale) measured in Newton;	From enrollment to the end of trial at one year；

Collaborators and Investigators

• Sponsor: Peking Union Medical College Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): March 23, 2026
• Primary Completion (Estimated): May 30, 2026
• Study Completion (Estimated): April 23, 2027

Study Registration Dates

• First Submitted: March 2, 2026
• First Submitted That Met QC Criteria: March 12, 2026
• First Posted (Actual): March 16, 2026

Study Record Updates

• Last Update Posted (Actual): March 16, 2026
• Last Update Submitted That Met QC Criteria: March 12, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Muscular Dystrophies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: CD20-RTX-DMD-IIT

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No