No registrations found.
ID
Source
Brief title
Health condition
Myelofibrosis, primary, post-ET, post PV-MF
Allogeneic stem cell transplantation
Myelofibrose
Allogene stamceltransplantatie
Sponsors and support
VU University Medical Center,<br>
P.O.Box 7057<br>
1007 MB Amsterdam<br>
The Netherlands<br>
tel: +31 20 4442124<br>
tel: +31 20 4449086<br>
Fax: +31 20 4443566
Intervention
Outcome measures
Primary outcome
Primary endpoint
♦ Proportion of patients receiving allo-SCT, with failure within or
at day 180 post-transplant. Events that are considered a failure
are:
o Primary graft failure
o Acute graft versus host disease grade 3-4
o Secondary graft failure
o Death, from any cause
Secondary outcome
Secondary endpoints
♦ Adverse events
♦ Proportion of patients receiving allo-SCT
♦ Response rate (≥ PR) (see appendix B)
♦ Progression free survival (PFS, i.e. time from either
registration or allo-SCT until progression/relapse or death from
any cause, whichever comes first)
♦ Overall survival (OS) calculated from either registration or allo-
SCT. Patients still alive or lost to follow up are censored at the
date they were last known to be alive
♦ Relapse mortality (RM), i.e. death due to the disease or after
progression
♦ Non-relapse mortality (NRM)
♦ Quality of Life during/after treatment
Background summary
Study design: Phase II, single arm, multicentre
Study objectives: 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.
Rationale: The only curative treatment for patients with
myelofibrosis (MF) is allogeneic stem cell transplantation (SCT).
Treatment with JAK2 inhibitors like pacritinib improves condition
of MF patients, decreases spleen size and might diminish graft-
versus-host disease (GvHD), thereby improving the outcome of
SCT.
Study design
Time of clinical evaluations
♦ Before enrollment: within 6 weeks before registration
♦ During pacritinib treatment biweekly in the first month and
thereafter monthly
♦ Before allo-SCT: 1 or 2 weeks before allo-SCT
♦ After allo-SCT: 1, 2, 3, 4, 6, 9, and 12 months after allo-SCT
Intervention
Induction with 3-4 cycles pacritinib, followed by allo-SCT if suitable donor available. All patients will receive the same treatment.
’s Gravendijkwal 230
Rotterdam 3015 CE
The Netherlands
+31 10 7033123
p.teboekhorst@erasmusmc.nl
’s Gravendijkwal 230
Rotterdam 3015 CE
The Netherlands
+31 10 7033123
p.teboekhorst@erasmusmc.nl
Inclusion criteria
- Patients with a confirmed diagnosis of post-ET, post-PV or primary myelofibrosis (Appendix A)
- Intermediate-2 or high-risk according to DIPSS plus
(Appendix E)
- Age 18-70 years inclusive
- WHO performance status 0-2 (Appendix C)
- At least 1 week since prior treatment (most recent dose)
with a potent cytochrome P450 3A4 (CYP3A4) inhibitor
- All men and women of childbearing potential must agree
to use adequate contraception during the study
- Written informed consent
- Patient is capable of giving informed consent
Exclusion criteria
- Previous treatment with JAK2 inhibitors within 2 weeks
of study inclusion. Patients who have been treated with
pacritinib as their previous JAK2 inhibitor treatment
cannot participate in this study
- Any GI or metabolic condition that could interfere with
absorption of oral medication
- Severe cardiac dysfunction (arrhythmias requiring
chronic treatment, congestive heart failure or
symptomatic ischemic heart disease)
- Experimental treatment within four weeks before
inclusion for PMF, Post-PV, or Post-ET MF
- Severe pulmonary dysfunction (CTCAE grade III-IV, see
appendix D)
- Significant hepatic dysfunction (total bilirubin ≥ 30 μmol/l
or transaminases ≥ 3 times normal level, unless
disease-related)
- Severe neurological or psychiatric disease
- Severe renal impairment (creatinine clearance < 40 ml/min)
- Patients with active, uncontrolled infections
- Patients known to be HIV(human immunodeficiency
virus)-positive
- Active hepatitis A, B or C
- History of active malignancy during the past 3 years,
except basal carcinoma of the skin or stage 0 cervical
carcinoma
- Concurrent severe and/or uncontrolled medical condition
(e.g. uncontrolled diabetes, infection, hypertension,
cancer, etc.)
- Pregnant or breastfeeding women
- Any psychological, familial, sociological and
geographical condition potentially hampering
compliance with the study protocol and follow-up
schedule
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL6578 |
| NTR-old | NTR6751 |
| Other | : MEC-2015- 750 |