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.
Aperçu de l'étude
Statut
Intervention / Traitement
Type d'étude
Inscription (Estimé)
Phase
- Phase 2
Contacts et emplacements
Coordonnées de l'étude
- Nom: wei MD, PhD
- Numéro de téléphone: +86 027-85726003
- E-mail: shiwei076@hust.edu.cn
Lieux d'étude
-
-
Hubei
-
Wuhan, Hubei, Chine, 430022
- Recrutement
- Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
-
Contact:
- Wei Shi, M.D.
- Numéro de téléphone: +86 027-85726003
- E-mail: shiwei076@hust.edu.cn
-
-
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
- Enfant
- Adulte
- Adulte plus âgé
Accepte les volontaires sains
La description
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.
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: N / A
- Modèle interventionnel: Affectation à un seul groupe
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
|
Expérimental: 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.
|
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Change in Skin and/or Joint/Fascia NIH cGVHD Scores
Délai: 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
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Overall cGVHD Response Rate
Délai: 6 months
|
Overall response rate of chronic graft-versus-host disease based on the 2014 NIH response criteria.
|
6 months
|
|
Failure-Free Survival
Délai: 2 years
|
Failure-free survival defined as survival without relapse, non-relapse mortality, or addition of new systemic therapy.
|
2 years
|
|
Non-Relapse Mortality
Délai: 2 years
|
Incidence of death without relapse of the underlying disease during follow-up.
|
2 years
|
|
Corticosteroid Dose Reduction
Délai: 6 months
|
Proportion of participants achieving at least 50% reduction or discontinuation of corticosteroid therapy.
|
6 months
|
|
Patient-Reported Outcomes
Délai: 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
Délai: Baseline to 6 months
|
Changes in Traditional Chinese Medicine symptom scores during treatment.
|
Baseline to 6 months
|
|
Safety and Adverse Events
Délai: Up to 2 years
|
Incidence and severity of adverse events assessed according to CTCAE version 5.0.
|
Up to 2 years
|
Collaborateurs et enquêteurs
Parrainer
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Réel)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
- Organisation de la pneumonie
- Maladies du système immunitaire
- Maladies des voies respiratoires
- Maladies pulmonaires
- Maladies bronchiques
- Maladies pulmonaires obstructives
- Bronchiolite oblitérante
- Bronchiolite
- Bronchite
- Syndrome de bronchiolite oblitérante
- Maladie du greffon contre l'hôte
- Administration des services de santé
- Qualité des soins de santé, accès et évaluation
- Qualité des soins de santé
- Indicateurs de qualité, soins de santé
- Standard de soins
- madecassoside
Autres numéros d'identification d'étude
- OMD-HSCT-20250817
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
Informations sur les médicaments et les dispositifs, documents d'étude
Étudie un produit pharmaceutique réglementé par la FDA américaine
Étudie un produit d'appareil réglementé par la FDA américaine
Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .