RecruitingPhase 2ACTRN12620001339943

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

Sponsor

The Royal Melbourne Hospital

Enrollment

20 participants

Start Date

Sep 28, 2021

Study Type

Interventional

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

Summary

Allogeneic Stem Cell transplant (alloSCT) is a curative procedure for many blood disorders and involves the transplantation of donor blood cells into a compatible recipient. Engraftment is a key milestone of success of alloSCT and is measured by standard blood tests to ensure normal levels of blood cells as well as a specialised blood test to confirm this blood is being produced by donor tissue. Poor graft function (PGF) is a significant complication of alloSCT and results in poor blood cell production by engrafted donor tissue. Aplastic anaemia(AA) is an autoimmune disease that results in the body's own immune cells attacking the bone marrow (the organ that produces blood cells) resulting in decreased production of blood cells. The mechanisms leading to PGF and AA are similar. There is evidence that the bone marrow microenvironment, the ecosystem in which blood cells reside, is abnormal in both PGF and AA. Atorvastatin is an old drug that has been used to treat high cholestrol and N-acetyl-cysteine (NAC) has been used as an antioxidant to treat paracetamol overdose. There is evidence that atorvastatin and NAC may be able to reverse this microenvironment dysfunction in PGF and AA. This study is aimed at testing the efficacy and safety of atorvastatin and NAC in the treatment of PGF and relapsed/refractory Aplastic anaemia.

Eligibility

Sex: Both males and femalesMin Age: 17 Yearss

Plain Language Summary

Simplified for easier understanding

After a bone marrow or stem cell transplant, it's essential that the donor cells "take root" and begin producing healthy blood cells. When this doesn't happen well — a problem called "poor graft function" — patients develop dangerously low blood counts and face serious risks. A related condition, aplastic anaemia, involves the body's immune system attacking its own bone marrow, leading to similar outcomes. Both conditions are hard to treat once standard approaches fail. This trial is testing a combination of two repurposed medications: atorvastatin (commonly used to lower cholesterol) and N-acetylcysteine (NAC, used to treat paracetamol overdose). Lab research suggests these drugs may help restore the bone marrow's microenvironment — the biological ecosystem that blood-producing cells depend on — which appears to be abnormal in both poor graft function and aplastic anaemia. You may be eligible if you are 17 or older with poor graft function after a stem cell transplant or relapsed/refractory aplastic anaemia, have reasonable physical function, and do not have active severe graft-versus-host disease or other active cancers. The study will measure whether blood counts improve over the treatment period, and whether the drugs are safe in this patient group.

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Interventions

This is a Phase I/II single arm, with historical cohort study assessing the efficacy and safety of: Atorvastatin tablet 10mg daily orally N-Acetyl Cysteine capsule 600mg twice a day orally Both

This is a Phase I/II single arm, with historical cohort study assessing the efficacy and safety of: Atorvastatin tablet 10mg daily orally N-Acetyl Cysteine capsule 600mg twice a day orally Both taken for 12 weeks in total In the treatment of Poor Graft Function post allogeneic transplant and relapsed refractory Aplastic Anaemia. Adherence will be assessed by reviewing bottle returns

Locations(1)

NSW,WA,VIC, Australia

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ACTRN12620001339943