A Prospective Study of Madecassoside Tablets in Sclerotic Chronic Graft-versus-Host Disease After Allo-HSCT
A Prospective, Multicenter, Single-Arm Phase 2 Study of Madecassoside Tablets Combined With Standard Therapy for Sclerotic Chronic Graft-versus-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation
Sclerotic chronic graft-versus-host disease (scl-cGVHD) is a severe subtype of chronic GVHD after allogeneic hematopoietic stem cell transplantation (allo-HSCT), characterized by skin sclerosis, fascial involvement, and restricted joint mobility, leading to substantial functional impairment and reduced quality of life. Current standard therapies, including corticosteroids and other immunosuppressive agents, have limited efficacy in patients with refractory or steroid-dependent disease.
Madecassoside tablets, a standardized extract of Centella asiatica, have demonstrated anti-inflammatory and anti-fibrotic properties in previous studies and may provide therapeutic benefit in fibrotic diseases.
This study is a prospective, multicenter, single-arm phase 2 clinical trial designed to evaluate the efficacy and safety of madecassoside tablets combined with standard therapy in patients with scl-cGVHD after allo-HSCT. Eligible participants will receive oral madecassoside tablets (0.2 g three times daily) in addition to standard treatment for 6 months. Clinical assessments will be performed at baseline and at weeks 4, 8, 12, and 24.
The primary endpoint is the improvement rate of skin and/or joint/fascia NIH cGVHD scores at 6 months according to the 2014 NIH consensus criteria. Secondary endpoints include overall cGVHD response rate, failure-free survival, non-relapse mortality, corticosteroid dose reduction, patient-reported outcomes, and safety assessments. Peripheral blood samples will also be collected to explore changes in inflammatory cytokines and lymphocyte subsets during treatment.
Studieoversikt
Status
Intervensjon / Behandling
Studietype
Registrering (Antatt)
Fase
- Fase 2
Kontakter og plasseringer
Studiekontakt
- Navn: wei MD, PhD
- Telefonnummer: +86 027-85726003
- E-post: shiwei076@hust.edu.cn
Studiesteder
-
-
Hubei
-
Wuhan, Hubei, Kina, 430022
- Rekruttering
- Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
-
Ta kontakt med:
- Wei Shi, M.D.
- Telefonnummer: +86 027-85726003
- E-post: shiwei076@hust.edu.cn
-
-
Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Barn
- Voksen
- Eldre voksen
Tar imot friske frivillige
Beskrivelse
Inclusion Criteria:
- Ability to understand and willingness to sign written informed consent;
- Age 14 to 65 years;
- Diagnosis of sclerotic chronic graft-versus-host disease (scl-cGVHD) according to the 2014 NIH consensus criteria, including superficial or deep skin sclerosis, fasciitis, or restricted joint mobility;
Prior treatment history meeting at least one of the following:
- Received systemic corticosteroid therapy for ≥12 months and at least 1 additional systemic therapy; OR
- Received corticosteroid therapy and at least 2 additional systemic therapies;
- Karnofsky Performance Status (KPS) score ≥60;
- Absolute neutrophil count >1,000/μL;
- Platelet count ≥50,000/μL.
Exclusion Criteria:
- 1. Total bilirubin >1.5 times the upper limit of normal; 2. Creatinine clearance <30 mL/min; 3. Uncontrolled infection; 4. Uncontrolled cardiovascular or pulmonary disease; 5. Any clinical condition that, in the investigator's judgment, makes the participant unsuitable for study participation.
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Eksperimentell: Experimental: Madecassoside Tablets Plus Standard Therapy
|
Madecassoside tablets will be administered orally at a dose of 0.2 g (2 tablets) three times daily for 6 months in combination with standard therapy.
Standard therapy may include corticosteroids, calcineurin inhibitors, ruxolitinib, belumosudil, topical medications, and supportive care according to institutional guidelines and physician discretion.
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Change in Skin and/or Joint/Fascia NIH cGVHD Scores
Tidsramme: 6 months
|
The proportion of participants achieving complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD) in skin and/or joint/fascia manifestations according to the 2014 NIH chronic graft-versus-host disease response criteria after 6 months of treatment.
|
6 months
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Overall cGVHD Response Rate
Tidsramme: 6 months
|
Overall response rate of chronic graft-versus-host disease based on the 2014 NIH response criteria.
|
6 months
|
|
Failure-Free Survival
Tidsramme: 2 years
|
Failure-free survival defined as survival without relapse, non-relapse mortality, or addition of new systemic therapy.
|
2 years
|
|
Non-Relapse Mortality
Tidsramme: 2 years
|
Incidence of death without relapse of the underlying disease during follow-up.
|
2 years
|
|
Corticosteroid Dose Reduction
Tidsramme: 6 months
|
Proportion of participants achieving at least 50% reduction or discontinuation of corticosteroid therapy.
|
6 months
|
|
Patient-Reported Outcomes
Tidsramme: Baseline to 6 months
|
Changes in patient-reported outcomes including Lee Symptom Scale (LSS), Scleroderma Health Assessment Questionnaire (SHAQ), and Photographic Range of Motion (P-ROM) scores.
|
Baseline to 6 months
|
|
Traditional Chinese Medicine Symptom Score
Tidsramme: Baseline to 6 months
|
Changes in Traditional Chinese Medicine symptom scores during treatment.
|
Baseline to 6 months
|
|
Safety and Adverse Events
Tidsramme: Up to 2 years
|
Incidence and severity of adverse events assessed according to CTCAE version 5.0.
|
Up to 2 years
|
Samarbeidspartnere og etterforskere
Sponsor
Studierekorddatoer
Studer hoveddatoer
Studiestart (Faktiske)
Primær fullføring (Antatt)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
- Organisering av lungebetennelse
- Sykdommer i immunsystemet
- Sykdommer i luftveiene
- Lungesykdommer
- Bronkiale sykdommer
- Lungesykdommer, obstruktiv
- Bronchiolitis Obliterans
- Bronkiolitt
- Bronkitt
- Bronchiolitis Obliterans syndrom
- Graft vs vertssykdom
- Health Services Administration
- Helsevesenets kvalitet, tilgang og evaluering
- Kvalitet på helsehjelpen
- Kvalitetsindikatorer, helsehjelp
- Standard for omsorg
- madecassoside
Andre studie-ID-numre
- OMD-HSCT-20250817
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Studerer et amerikansk FDA-regulert enhetsprodukt
Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .