A Phase 2 interventional study of Oral THU-decitabine in Myelofibrosis, MPN - Accelerated or Blast Phase and Chric Phase MPN, sponsored by John Mascarenhas. Not yet recruiting at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-14.
Sponsored by John Mascarenhas · Phase 2, Interventional, and Treatment
This is a phase II, multicenter, open-label study evaluating the feasibility, safety, and efficacy of oral tetrahydrouridine-decitabine (THU-decitabine) in patients with advanced myeloproliferative neoplasms (MPN).
This is a phase 2, multicenter trial evaluating the feasibility of administering oral THU-Dec with or without JAK2 inhibitor in patients with advanced MPNs. Eligible subjects will receive oral THU-Dec orally once weekly on a four week (28 day) cycle for a core study period of 6 cycles (24 weeks). The primary endpoint will be assessed after 6 cycles of treatment. Feasibility will be assessed as the treatment completion rate, defined as the proportion of patients who receive ≥75% of planned THU-Decitabine doses for at least 6 cycles or who discontinue treatment to receive allogeneic hematopoietic stem cell transplantation. After 24 weeks, subjects who demonstrate any response as per modified Cheson Criteria or stable disease may continue to receive treatment until loss of response, disease progression, unacceptable toxicity, subject/physician withdrawal, or the study is terminated.
431 studies on the registry are indexed under Primary Myelofibrosis; 118 are open to participants now.
This study's planned enrollment of 20 is below the median of 44 across 355 interventional studies indexed under Primary Myelofibrosis.
Browse Primary Myelofibrosis studies →John Mascarenhas is the lead sponsor of 13 studies on the registry; 3 are open to participants now.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Study participants will receive oral THU-decitabine once weekly (on the same day each week ±1 day). Oral THU-Dec 8-12/0.16-0.24 mg/kg (\~10/0.2 mg/kg) (THU ingested 1 hour before decitabine). THU and decitabine are supplied as capsules: each THU capsule contains THU 250 mg; each decitabine capsule contains decitabine 5 mg. Since this trial utilizes capsules, there has to be rounding of dosages.
Drug: Oral THU-decitabine
Combination of Tetrahydrouridine and Decitabine oral treatment. Treatment is ingested weekly without interruption. For convenience of communication and planning, 4 weeks of treatment (28 days) is considered a treatment cycle. The first 2 cycles of therapy (week 1-8 of therapy) is considered an induction phase. After the induction phase (Cycle 3+): Treatment continues weekly.
Treatment Completion Rate
Proportion of patients who receive ≥75% of planned THU-Dec doses for at least 6 cycles (24 weeks) OR discontinue treatment to receive allogeneic hematopoietic stem cell transplantation
Time frame: 6 Cycles (24 Weeks)
Overall Survival
The median overall survival of advanced phase MPN patients treated with oral THU-Dec ± JAK2i
Time frame: 48 Months
Complete Blast Response
No circulating blasts in peripheral blood and less than 5% in bone marrow.
Time frame: 48 Months
Partial Blast Response
A 50% reduction in peripheral blood or bone marrow blasts.
Time frame: 48 Months
Chronic Phase BF Blast
The proportion of treated patients with chronic phase MF and 4%-9% circulating blasts that achieve a response (complete response, partial response, clinical improvement, anemia, spleen, symptom response, cytogenetic or molecular remission).
Time frame: 6 Cycles (24 Weeks)
MFSAF TSS
Change in MPN-SAF TSS from baseline after 6 and 12 cycles; symptom response will be determined in participants with baseline MPN-SAF TSS ≥ 10. Symptom improvement response is defined as a number of participants with improvement in symptoms response as defined by Myeloproliferative Neoplasms Symptoms Assessment Form (MPN-SAF). The MPN-SAF is a questionnaire that measures certain symptoms and how they improve throughout the study, from baseline. The questionnaire is divided into 4 sections - 1: 16 questions about the most common symptoms in these disease types, rated from 0 (lowest impact) to 10 (biggest impact); 2: Highest grade of fever; 3: Unintentional weight loss; 4. Quality of life, rated from 0 (as good as it can be) to 10 (as bad as it can be). MPN-SAF is an 18-item instrument. Each item score 0-10 averaged, total scale from 0-10, with higher score indicating more symptoms.
Time frame: 48 Months
EORTC-QLC-30
This is a 30-question tool used to assess the overall QoL in cancer participants. It consisted of 15 domains: 1 GHS/QoL scale, 5 functional scales (Physical, role, cognitive, emotional, social), and 9 symptom scales/items (Fatigue, nausea and vomiting, pain, dyspnea, sleep disturbance, appetite loss, constipation, diarrhea, financial impact). Most items are scored 1 ("not at all") to 4 ("very much") except for the items contributing to the GHS/QoL, which are scored 1 ("very poor") to 7 ("excellent"). A linear transformation was applied to the raw scores so that all transformed scores lie between 0 to 100. For the GHS/QoL and 5 functional scales a high score indicates better global health status/functioning and a negative change from baseline indicated less improvement. For the symptom scales, a high score indicates a higher level of symptoms, and a negative change from baseline indicated an improvement in symptoms.
Time frame: 48 Months
PGIC Score
The Patient's Global Impression of Change (PGIC) will be used to assess self-reported relieving effect. It will evaluate pain from no change (score 0-1), minimally improved (score 2-3), much improved (score 4-5), and very much improved (score 6-7). The patients will answer the following question: "Since beginning treatment at this program, how would you describe the change (if any) in activity limitations, symptoms, emotions, and overall quality of life related to your condition?". Full score from 0-7, with higher score indicating more improvement. Change in score at Week 2, Week 10 and Week 14 as compared to Baseline.
Time frame: 48 Months
Allogeneic Stem Cell Transplant
Rate of allogeneic stem cell transplant occurrence among those participating in the study.
Time frame: 48 Months
Time to Treatment Failure (TTF)
TTF is defined as time from enrollment to discontinuation of treatment for any reason.
Time frame: 48 Months
Dose Delays
Frequency of THU-Dec dose delays
Time frame: 48 Months
Dose Interruptions
Frequency of THU-Dec dose interruptions
Time frame: 48 Months
Rate of Hospitalization
Rate of hospitalizations related to complications of treatment or disease.
Time frame: 48 Months
Plan to share: No — The completed dataset is the sole property of the Sponsor-Investigator's institution and should not be exported to third parties, except for authorized representatives of appropriate Health/Regulatory Authorities, without permission from the Sponsor-investigator and their institution.
No publications or documents are linked to this record.
Start is estimated for Jan 2027; primary completion for Jul 2029, 30 months later.
Of 60 non-industry phase 2 interventional studies under Primary Myelofibrosis, half reached primary completion within 89 months of starting (the median), counting those still running; a quarter within 42 and three quarters within 119. This study's planned 30 months is below that.
Counting every sponsor, half of 90 studies reached it within 66 months; a quarter within 32 and three quarters within 111.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is not yet recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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John Mascarenhas