Trial purpose
Cancer treatment
Tumor type
Haematological
Age
17+
Clinical summary
Summary
This clinical trial is exploring a new treatment approach for people who have trouble producing healthy blood cells due to two conditions:
- Poor Graft Function (PGF) after a stem cell transplant
- Relapsed or treatment-resistant Aplastic Anaemia (AA)
Both conditions affect the bone marrow—the part of the body that makes blood cells—and can lead to low levels of red cells, white cells, and platelets.
Researchers believe that problems in the bone marrow’s environment may be contributing to these conditions. Two medications, atorvastatin (commonly used for high cholesterol) and N-acetyl-cysteine (NAC) (used as an antioxidant), may help improve this environment and support better blood cell production.
What the Study Involves
Participants will take:
- Atorvastatin: 10 mg once daily
- NAC: 600 mg twice daily
Both medications are taken by mouth for 12 weeks.
The study will monitor how well these medications work and how safe they are. Researchers will check blood tests regularly and ask participants to return medication bottles to help track how consistently the treatment is taken.
Conditions
This trial is treating people with poor graft function or relapsed or refractory aplastic anaemia
Eligibility
Inclusion
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
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
Inclusion
- You have had treatment, but your cancer has come back (relapsed or recurrent).
- You have had treatment but your cancer has gotten worse or has not responded to the treatment you have been given.
- You have the type of cancer, symptoms, or health risks that this clinical trial is focused on.
Exclusion
- You have been diagnosed with a prior or secondary type of cancer.
- You have certain types of non-cancer medical conditions.
Clinical trials have complex eligibility criteria, and other criteria may apply for this trial. Ask your doctor about whether this trial could be right for you.
More information
Trial Identifiers
Information on this page is partially produced from ANZCTR *. View further details about this trial on the registry via the links below:
Trial sponsor
The Royal Melbourne Hospital
Scientific Title
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
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