Mycophenolate After Stem Cell Transplant for Systemic Sclerosis (IMPACT-SSc)
Immunomodulatory Maintenance Therapy Post-Autologous Hematopoietic Stem Cell Transplantation for Prevention of Systemic Sclerosis Relapse: A Pilot Randomized Trial
A pilot single center randomized trial to test whether a full-scale RCT evaluating the role of post-AHSCT maintenance immunosuppression with MMF in preventing disease relapse is feasible .
We are studying whether a brief course of treatment with MMF after AHSCT will serve to prevent disease relapse.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Description détaillée
This study is performed determine the feasibility of a larger pragmatic RCT to evaluate the effectiveness of post- Autologous Hemopoietic Stem Cell Transplant (AHSCT) maintenance therapy with mycophenolate mofetil (MMF) in the prevention of Systemic sclerosis (SSc) relapse.
The study will include 6 participants who will be randomized at 1:1 ratio to treatment (MMF) or control arm (no treatment) for 6 months.
The participants will have 6 visits in total : An initial clinic visit will occur prior to stem cell collection. Post-AHSCT, in-person follow-up visits are scheduled on days 30, 90, 180, 270, and 300, with windows of +/- 1 week for days 30 and 90 and +/- 2 weeks for days 180, 270 and 300.
These visits will be coordinated at an established multidisciplinary (Rheumatology + Cell Therapy) clinic that are part of the patient's clinical care. At each visit, the participants will be asked to complete health questionnaires and laboratory tests will be performed as part of the standard of care of AHSCT recipients.
Type d'étude
Inscription (Estimé)
Phase
- Phase 2
Contacts et emplacements
Coordonnées de l'étude
- Nom: Clinical Research Assistant
- Numéro de téléphone: 81630 613 738 8400
- E-mail: nasaidi@ohri.ca
Lieux d'étude
-
-
Ontario
-
Ottawa, Ontario, Canada
- The Ottawa Hospital
-
-
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
- Adulte
- Adulte plus âgé
Accepte les volontaires sains
La description
Inclusion Criteria:
- Consenting participants 18 years of age or older who are initiating AHSCT at The Ottawa Hospital for management of SSc.
Exclusion Criteria:
- Exclusion criteria are focused on patient safety, namely inability to receive treatment with
MMF due to contraindications as determine at time of randomization (day 90 / clinic visit #3) including:
- Renal insufficiency (eGFR <25 mL/minute/1.73m2) or
- Neutropenia (ANC <1.0 x 103/mcL) or
- Thrombocytopenia (platelet count <50 000/mcL)
- Previously documented intolerance.
- Pregnant or breastfeeding - Women of childbearing potential who are not willing to use a highly effective method of contraception for the duration of study participation will be excluded
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: La prévention
- Répartition: Randomisé
- Modèle interventionnel: Affectation parallèle
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
|
Expérimental: Mycophenolate mofetil (MMF)
MMF 1000mg p.o. b.i.d.
starting at day 90 post-AHSCT.
This proposed MMF regimen is used in the management of rheumatic diseases and described in current literature and clinical guidelines.
Duration of 6 months
|
MMF 1000mg p.o. b.i.d.
starting at day 90 post-AHSCT and continued for 6 months.
|
|
Aucune intervention: Control arm
No scleroderma prophylaxis (current standard of care)
|
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Feasibility:
Délai: 18 months
|
The number of participants enrolled at a single center over 18 months.
|
18 months
|
|
Safety : AEs/SAEs
Délai: Day 90 (treatment start) , Day 180, day 270
|
The proportion of patients experiencing a serious AE OR an AE greater that grade 2 (see below) OR an AE requiring discontinuation of the study drug in a participant who has received at least one dose of the study intervention
|
Day 90 (treatment start) , Day 180, day 270
|
|
Efficacy :
Délai: At day 0,90,180 and 270
|
-Proportion of patients showing an increase in mRSS of >7 between timepoints .
|
At day 0,90,180 and 270
|
|
Efficacy:
Délai: At day 0,90,180 and 270
|
-Proportion of patients requiring initiation of immunosuppressive therapy for progressive SSc manifestations as determined by their treating physicians.
|
At day 0,90,180 and 270
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Feasibility: Consent rate:
Délai: Both prior to AHSCT (clinic visit #1) and at day 90 (clinic visit #3) prior to randomization.
|
Proportion of eligible patients who provide consent .
|
Both prior to AHSCT (clinic visit #1) and at day 90 (clinic visit #3) prior to randomization.
|
|
Feasibility: Retention rate: Proportion of participants retained
Délai: Day 270 (clinic visit #5)
|
Proportion of participants retained
|
Day 270 (clinic visit #5)
|
|
Feasibility: Study completion rate:
Délai: Day 270
|
Proportion of participants who completed proposed clinical outcomes at all pre determined time points
|
Day 270
|
|
Feasibility: Adherence rate:
Délai: Day 180 and Day 270
|
Adherence to study drug measured by proportion of prophylaxis doses received measured by pill counting at follow-up visits.
|
Day 180 and Day 270
|
|
Auto-antibody testing (clinical)
Délai: Prior to AHSCT and Day 180 (visit# 4)
|
Proportion of patients with a change in auto-antibody testing
|
Prior to AHSCT and Day 180 (visit# 4)
|
|
Clinical outcomes:
Délai: day 0, 90, 180 and 270
|
Proportion of patients showing a change between timepoints in Modified Rodnan Skin Score (mRSS): Total score range 0 to 51.
Higher scores indicate greater skin thickening and more severe skin involvement .
|
day 0, 90, 180 and 270
|
|
Clinical outcome:
Délai: day 0,90,180 and 270
|
Proportion of patients showing a change in Scleroderma Health Assessment Questionnaire (SHAQ) Disability Index between timepoints. Score range: 0 to 3. Higher scores indicate greater functional disability and worse health status. |
day 0,90,180 and 270
|
|
Clinical outcome:
Délai: Day 0,90,180 and 270
|
Proportion of patients showing a change in global score between timepoints Patient and physician global score: Visual analog scale (0 to 10)
|
Day 0,90,180 and 270
|
Collaborateurs et enquêteurs
Parrainer
Les enquêteurs
- Chercheur principal: Nancy Maltez, Dr, The Ottawa Hospital
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Estimé)
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
Autres numéros d'identification d'étude
- 20260406-01H
Plan pour les données individuelles des participants (IPD)
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Informations sur les médicaments et les dispositifs, documents d'étude
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