REscuing bone marrow function in patients with aplaStic anaEmia and bone marrow faiLure post allogEneiC Transplantation (RESELECT)Phase I/II single arm with historical control study assessing the efficacy and safety of Atorvastatin and N-Acetyl Cysteine in the treatment of Poor Graft Function post allogeneic transplantation and relapsed/refractory aplastic anaemia

Phase 1

Recruiting

• Conditions: One of the health conditions being studied in this trial will be Poor Graft Function; One of the health conditions being studied in this trial will be Aplastic Anaemia; Blood - Haematological diseases

• Registration Number: ACTRN12620001339943
• Lead Sponsor: The Royal Melbourne Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-12-11
• Last Update Posted: 29 November 2021

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

1. Poor Graft function OR Relapsed/refractory AA defined as the following
Poor Graft Function:
Two Lineage cytopenias defined as
-Thrombocytopenia
i)Less than or equal to 30x10^9 /L from D40-D60 OR
ii)Less than or equal to 50 x10 ^9/L from D60 onwards
-Neutropenia requiring filgrastim support at any time post D40
-Hb less than or equal to 80g/L
Relapsed /Refractory AA:
Relapse after stem cell transplant OR relapsed post/refractory to 1st line immunosuppression without an unrelated donor identified.
2. Age greater than or equal to 17 years
3. ECOG performance status 0-1
4. Life expectancy > 6 months
5. Patient’s written informed consent

Exclusion Criteria

1. Active Grade 3-4 acute GVHD
2. Relapsed or progressive disease on screening bone marrow biopsy or most recent PET imaging.
3. Active second malignancy currently requiring treatment
4. Human Immuno-deficiency Virus (HIV) infection.
5. Any coexisting medical or psychological condition that would preclude participation in the required study procedures.
6. Female patients who are both lactating and breast-feeding or have a positive serum pregnancy test during the screening period or a positive pregnancy test on Day 1 before first dose of study drug
7. Prior history of statin induced myopathy
8. Prior history of severe asthma

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Complete Response (CR) defined as neutrophils greater than or equal to 1.5x10^9 and platelets greater than or equal to 100x10^9 without use of transfusions or cytokine support at 12 weeks <br>This outcome will be measured by a full blood examination performed on a blood sample at 12 weeks.<br><br>[The primary timepoint will be measured at 12 weeks post trial enrollment ]; Incidence of non haematological Adverse Events (AEs) of Grade 2 severity as measured by version 5 of the National Institute of Health common terminology criteria of adverse events (NIH-CTCAE) directly attributed to the trial medication as measured at 12 weeks. [12 weeks]