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.
調査の概要
詳細な説明
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.
研究の種類
入学 (推定)
段階
- フェーズ2
連絡先と場所
研究連絡先
- 名前：Clinical Research Assistant
- 電話番号：81630 613 738 8400
- メール：nasaidi@ohri.ca
研究場所
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Ontario
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Ottawa、Ontario、カナダ
- The Ottawa Hospital
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-
参加基準
適格基準
就学可能な年齢
- 大人
- 高齢者
健康ボランティアの受け入れ
説明
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
研究計画
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：防止
- 割り当て：ランダム化
- 介入モデル：並列代入
- マスキング：なし（オープンラベル）
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
実験的：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.
|
|
介入なし：Control arm
No scleroderma prophylaxis (current standard of care)
|
この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Feasibility:
時間枠：18 months
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The number of participants enrolled at a single center over 18 months.
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18 months
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Safety : AEs/SAEs
時間枠：Day 90 (treatment start) , Day 180, day 270
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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
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Efficacy :
時間枠：At day 0,90,180 and 270
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-Proportion of patients showing an increase in mRSS of >7 between timepoints .
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At day 0,90,180 and 270
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Efficacy:
時間枠：At day 0,90,180 and 270
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-Proportion of patients requiring initiation of immunosuppressive therapy for progressive SSc manifestations as determined by their treating physicians.
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At day 0,90,180 and 270
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Feasibility: Consent rate:
時間枠：Both prior to AHSCT (clinic visit #1) and at day 90 (clinic visit #3) prior to randomization.
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Proportion of eligible patients who provide consent .
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Both prior to AHSCT (clinic visit #1) and at day 90 (clinic visit #3) prior to randomization.
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Feasibility: Retention rate: Proportion of participants retained
時間枠：Day 270 (clinic visit #5)
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Proportion of participants retained
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Day 270 (clinic visit #5)
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Feasibility: Study completion rate:
時間枠：Day 270
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Proportion of participants who completed proposed clinical outcomes at all pre determined time points
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Day 270
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Feasibility: Adherence rate:
時間枠：Day 180 and Day 270
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Adherence to study drug measured by proportion of prophylaxis doses received measured by pill counting at follow-up visits.
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Day 180 and Day 270
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Auto-antibody testing (clinical)
時間枠：Prior to AHSCT and Day 180 (visit# 4)
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Proportion of patients with a change in auto-antibody testing
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Prior to AHSCT and Day 180 (visit# 4)
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Clinical outcomes:
時間枠：day 0, 90, 180 and 270
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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 .
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day 0, 90, 180 and 270
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Clinical outcome:
時間枠：day 0,90,180 and 270
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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
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Clinical outcome:
時間枠：Day 0,90,180 and 270
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Proportion of patients showing a change in global score between timepoints Patient and physician global score: Visual analog scale (0 to 10)
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Day 0,90,180 and 270
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協力者と研究者
捜査官
- 主任研究者：Nancy Maltez, Dr、The Ottawa Hospital
研究記録日
主要日程の研究
研究開始 (推定)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
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