Myelofibrosis Treated With Pacritinib Before aSCT. (HOVON134MF) (HOVON134MF)
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|ClinicalTrials.gov Identifier: NCT03645824|
Recruitment Status : Recruiting
First Posted : August 24, 2018
Last Update Posted : August 27, 2018
|Condition or disease||Intervention/treatment||Phase|
|Myelofibrosis||Drug: Pacritinib||Phase 2|
Despite recent new therapeutic options, allogeneic Stem Cell Transplantation remains the only curative option in patients with Myelofibrosis. Therefore, optimalization of this therapy remains a major challenge. Improvement of the clinical condition of these patients, decreasing spleen size can be accomplished by JAK2 inhibitor treatment and might improve SCT outcome. In addition, selective JAK2 inhibitors might modulate GvHD which can also add to improved SCT outcome. Also, decreasing the burden/activity of the disease before allo-SCT might also improve final disease response. The first, limited, clinical data of ruxolitinib treatment before allo-SCT show controversial effects on the outcome of SCT. Therefore, additional prospective clinical trials have to be done to establish the role of JAK2 inhibition before allogeneic SCT. Since ruxolitinib has considerable myelosuppressive effects which might limit the clinical use in some MF patients, other selective JAK2 inhibitors might be useful in this setting. Pacritinib, as a JAK2/FLT3 inhibitor, has a very potent JAK2 inhibitory activity without myelosuppressive effects and might therefore be more suitable than ruxolitinib. The major possible side effects are gastro-intestinal and can be managed with medication. In several studies, pacritinib has shown to be effective in decreasing spleen size and has shown to improve clinical condition of patients. Therefore, this compound seems promising in improving the outcome of allo-SCT. Although pacritinib causes no inhibition of JAK1 activity and therefore might have limited effects in decreasing inflammatory response, this might also be of benefit since a "withdrawal syndrome" as has been described after cessation of ruxolitinib is not to be expected.
With this trial, using pacritinib treatment before allo-SCT, the issue of improvement of SCT outcome will be investigated.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||70 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Trial in Patients With Myelofibrosis (Primary, Post-ET or Post PV-MF) Treated With the Selective JAK2 Inhibitor Pacritinib Before Reduced-intensity Conditioning Allogeneic Stem Cell Transplantation|
|Actual Study Start Date :||June 4, 2018|
|Estimated Primary Completion Date :||September 2022|
|Estimated Study Completion Date :||March 2023|
Experimental: Pacritinib treatment befor allo-SCT
The effect of pacritinib treatment during 3 to 4 cycles before allo-SCT on engraftment 6 months (day +180) post allo-SCT in MF patients.
Patients receive up to 4 cycles of pacritinib before allo-SCT
- Proportion of patients receiving allo-SCT, with failure within or at day 180 post-transplant. [ Time Frame: 2 years ]
Failure can be defined by one of the following parameters:
- Primary graft failure
- Acute graft versus host disease grade 3-4
- Secondary graft failure
- Death, from any cause
- Adverse events [ Time Frame: 5 years ]Adverse events will be monitored.
- Progression free survival [ Time Frame: 5 years ]Progression free survival as time between registration or SCT until progression/relapse or death from any cause
- Overall survival [ Time Frame: 5 years ]Over all survival, calculated from either registration or SCT.
- Relapse mortality [ Time Frame: 5 years ]Death due to the disease or after progression
- Non-relapse mortality [ Time Frame: 5 years ]Death not due to disease or relapse
- Quality of life during and after treatment [ Time Frame: 5 years ]
Quality of life during and after treatment will be recorded by use of the MPN-SAF questionnaire.
From a total of 27 questions, the scores (from 0 to 10) from the following 10 questions are added and subsequently averaged to come to a total quality of life score.
- satisfction after a meal
- stomach complaints
- not able to perform activities
- concentration problems
- night sweat
- bone pain
- sudden weight loss
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03645824
|Contact: Peter AW te Boekhorst, M.D. PhD||+31 10 703 ext firstname.lastname@example.org|
|AMC||Not yet recruiting|
|Amsterdam, Noord-Holland, Netherlands|
|Erasmus Medical Center||Recruiting|
|Rotterdam, Zuid-Holland, Netherlands, 3000 CA|
|Contact: Peter AW te Boekhorst, MD|
|Principal Investigator: Peter AW te Boekhorst, MD|
|Principal Investigator:||Peter AW te Boekhorst, M.D. PhD||Erasmus MC|