Ignite Creation Date:
2024-10-26 @ 3:40 PM
Last Modification Date:
2024-10-26 @ 3:40 PM
Study NCT ID:
NCT06607367
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-09-15
Brief Title:
REscuing Bone Marrow Function in Patients with AplaStic AnaEmia and Bone Marrow FaiLure Post AllogEneiC Transplantation 2
Sponsor:
None
Organization:
None
Study Overview
Official Title:
REscuing Bone Marrow Function in Patients with AplaStic AnaEmia and Bone Marrow FaiLure Post AllogEneiC Transplantation 2
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
RESELECT2
Brief Summary:
Allogeneic stem cell transplantation involves the transplanting of donor blood stem cells into a recipient this is performed mainly for the treatment of blood cancers The bone marrow is the organ that produces all blood cells and allogeneic stem cell transplantation results in the replacement of abnormal recipient bone marrow with donor blood cells as well as the production of donor immune cells from the donor bone marrow The production of donor immune cells will hopefully lead to an immune response directed at any persisting cancerous cells leading to their eradication As such one of the key measures of success of a transplant is establishment of donor engraftment
Engraftment is considered successful when the patient has normal blood cell counts on routine laboratory testing as well as confirmation that the blood cells are being produced by donor bone marrow cells Confirming donor blood cell production is done by a process called chimerism Poor graft function PGF is a complication of allogeneic stem cell transplantation related to engraftment manifested by low blood counts despite complete donor chimerism This has significant consequences for the patient leaving them susceptible to infection because of low white blood cells and bleeding because of low platelets the cell components that are important for blood clotting There is currently no established treatment for this condition and patients with this condition who do not recover have a poor survival
Aplastic anaemia AA is a rare autoimmune condition that results in a patients own immune system attacking important components of their bone marrow resulting in low blood counts The current treatments for AA include suppressing the immune system or a bone marrow transplant however long term survival for patients who do not respond to these treatments or relapse is poor and more effective treatments are required
There is emerging evidence that demonstrates that the components of the immune system are dysfunctional and result in excessive immune activation resulting in suppression of the bone marrow characteristic of PGF Similar features of immune dysfunction has been demonstrated in AA Ruxolitinib is a drug that may be able to reduce this excessive immune activation Eltrombopag is a drug that has been shown to stimulate the production of blood cells The aim of this study is to evaluate whether the combination of ruxolitinib and eltrombopag is safe and effective in the treatment of PGF and AA
Engraftment is considered successful when the patient has normal blood cell counts on routine laboratory testing as well as confirmation that the blood cells are being produced by donor bone marrow cells Confirming donor blood cell production is done by a process called chimerism Poor graft function PGF is a complication of allogeneic stem cell transplantation related to engraftment manifested by low blood counts despite complete donor chimerism This has significant consequences for the patient leaving them susceptible to infection because of low white blood cells and bleeding because of low platelets the cell components that are important for blood clotting There is currently no established treatment for this condition and patients with this condition who do not recover have a poor survival
Aplastic anaemia AA is a rare autoimmune condition that results in a patients own immune system attacking important components of their bone marrow resulting in low blood counts The current treatments for AA include suppressing the immune system or a bone marrow transplant however long term survival for patients who do not respond to these treatments or relapse is poor and more effective treatments are required
There is emerging evidence that demonstrates that the components of the immune system are dysfunctional and result in excessive immune activation resulting in suppression of the bone marrow characteristic of PGF Similar features of immune dysfunction has been demonstrated in AA Ruxolitinib is a drug that may be able to reduce this excessive immune activation Eltrombopag is a drug that has been shown to stimulate the production of blood cells The aim of this study is to evaluate whether the combination of ruxolitinib and eltrombopag is safe and effective in the treatment of PGF and AA
Detailed Description:
Study Overview
This is a multi-centre single active arm with historic control arm phase III study that will assess the safety and efficacy of ruxolitinib and eltrombopag in patients with relapserefractory AA and PGF post alloSCT
Efficacy will be evaluated using the study endpoints The study will consist of screening and enrolment up to 28 days treatment 12 weeks and follow up up to 9 months post enrolment Therefore the maximum time on study for a single patient will be a maximum of 12 months
There will be an initial safety lead in phase of 10 participants Adverse events will be evaluated by the SMC who will then decide whether the study will proceed to the complete enrolment of 20 participants based on the safety analysis
Treatment
Upon enrolment patients in the intervention arm will commence the following treatment Ruxolitinib at a dose of 10mg BD and Eltrombopag at a dose of 50mg
Dose adjustments may be required based on lack of efficacy or thrombocytopenia
This is a multi-centre single active arm with historic control arm phase III study that will assess the safety and efficacy of ruxolitinib and eltrombopag in patients with relapserefractory AA and PGF post alloSCT
Efficacy will be evaluated using the study endpoints The study will consist of screening and enrolment up to 28 days treatment 12 weeks and follow up up to 9 months post enrolment Therefore the maximum time on study for a single patient will be a maximum of 12 months
There will be an initial safety lead in phase of 10 participants Adverse events will be evaluated by the SMC who will then decide whether the study will proceed to the complete enrolment of 20 participants based on the safety analysis
Treatment
Upon enrolment patients in the intervention arm will commence the following treatment Ruxolitinib at a dose of 10mg BD and Eltrombopag at a dose of 50mg
Dose adjustments may be required based on lack of efficacy or thrombocytopenia
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None