MedTrace Logo

Leflunomide for Musculoskeletal GVHD After Allogeneic Stem Cell Transplant

NCT04932564 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2025-09-10

No results posted yet for this study

Summary

Graft versus host disease (GVHD) is a well-known complication of allogeneic transplant. In GVHD, the cells of the donor attack the patient's tissues and cause damage. It can affect any organ or system of the body. In a proportion of patients, it affects the joints and muscles. This is known as musculoskeletal GVHD. The standard treatment of musculoskeletal GVHD is steroids. However, these are usually needed for prolonged periods, and cause a large number of additional problems in transplant patients.

Leflunomide is a drug which has been used for several years in diseases like rheumatoid arthritis (RA). RA is an auto-immune disorder. The biological mechanisms underlying RA and musculoskeletal GVHD are quite similar. Hence it is likely that leflunomide may work in musculoskeletal GVHD also. The investigator have previously used leflunomide in a few patients with musculoskeletal GVHD and have found it to be extremely effective. Also, it was very safe (unlike steroids). Yet another advantage is that it is fairly cheap.

The purpose of the current study is to study the efficacy and safety of leflunomide in patients with musculoskeletal GVHD in a prospective way.

Conditions

  • Musculoskeletal Disease Other
  • GVHD, Chronic

Interventions

DRUG

Leflunomide tablet

Leflunomide will be given at standard dose (100 mg OD x 3 days followed by 20 mg OD) in adults and weight based dose in children. The dosing for children will be as follows: 1. \<20 kg - 100 mg x 1 day followed by 10 mg every alternate day 2. 20-40 kg - 100 mg x 2 days, followed by 10 mg daily 3. \>40 kg - Usual adult dose. This is scheduled to be continued for 1 year from the time of attaining complete response of musculoskeletal GVHD.

Sponsors & Collaborators

  • Tata Memorial Centre

    lead OTHER

Principal Investigators

  • Sachin Punatar, MD, DM · Tata Memorial Centre

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-09-18
Primary Completion
2024-06-25
Completion
2024-06-25

Countries

  • India

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04932564 on ClinicalTrials.gov