Not Yet RecruitingPhase 1/2

Pacritinib With Aza for Upfront Myelodysplastic Syndrome

25 participantsStarted 2026-05

Plain-language summary

This study will be conducted as a phase 1/2 study of safety and preliminary efficacy of pacritinib in combination with azacitidine for IPSS-M moderate low to very high risk MDS. Phase one will be a 3 + 3 design to assess the dose for the phase two portion. The phase two portion will employ a simon min-max two-stage design whereby fifteen patients will be enrolled in the first stage then ten more if at least two patients in stage one have a response. The dosing of pacritinib for the phase two study will be based on the phase one findings. Standard dosing of azacitidine will be used. A correlative study will be conducted in conjunction with the trial where the investigators will measure whole blood collected pre-treatment and at four days post-treatment to measure intracellular flow and phosflow to detect JAK/STAT, NF-κβ, and AKT/mTOR signaling in patient samples and how treatment affects these pathways.

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Patients must have histologic evidence of intermediate to high-risk myelodysplastic syndrome defined as having an IPSS-M score of moderate low, moderate high, high or very high risk. This will be assessed based on evaluations performed prior to screening for trial. Of note, the most recent evaluation pre-trial may be used which does not have to necessarily be at diagnosis.
✓. Subjects must have recovered from the toxic effects of any prior chemotherapy to ≤ Grade 1 (except alopecia).
✓. Required screening visit laboratory values: CrCL ≥45; total bilirubin \<2xULN except for patients with known Gilbert's disease; SGPT (ALT) ≤2xULN, PTT ≤1.5xULN.
✓. Negative pregnancy test for women with child-bearing potential at screening visit.
✓. Initial screening baseline QTc ≤480ms.
✓. Patients must be able to sign consent and be willing and able to comply with scheduled visits, treatment plan and laboratory testing.
✓. Patients must have an absolute neutrophil count of ≥750 to enroll in study, this must be achieved without the addition of growth factor medication.

Exclusion criteria

What they're measuring

Optimal Dose of Pacritinib in Combination with Azacitidine - Phase 1

Timeframe: Baseline through Day 28 (Cycle 1)

Rate of Dose Limiting Toxicities (DLTs) - Phase 1

Timeframe: Baseline through Day 28 (Cycle 1)

Overall Response Rate (ORR) - Phase 2

Timeframe: Baseline through Week 16 (approximately 4 months)

Trial details

NCT IDNCT07387354

SponsorThomas Jefferson University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-01

Contact for this trial

Chetan S Jeurkar, DO

215-955-8874 Chetan.Jeurkar@jefferson.edu

View on ClinicalTrials.gov More Myelodysplastic Syndromes trials

Plain-language summary

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Patients must have histologic evidence of intermediate to high-risk myelodysplastic syndrome defined as having an IPSS-M score of moderate low, moderate high, high or very high risk. This will be assessed based on evaluations performed prior to screening for trial. Of note, the most recent evaluation pre-trial may be used which does not have to necessarily be at diagnosis.
✓. Subjects must have recovered from the toxic effects of any prior chemotherapy to ≤ Grade 1 (except alopecia).
✓. Required screening visit laboratory values: CrCL ≥45; total bilirubin \<2xULN except for patients with known Gilbert's disease; SGPT (ALT) ≤2xULN, PTT ≤1.5xULN.
✓. Negative pregnancy test for women with child-bearing potential at screening visit.
✓. Initial screening baseline QTc ≤480ms.
✓. Patients must be able to sign consent and be willing and able to comply with scheduled visits, treatment plan and laboratory testing.
✓. Patients must have an absolute neutrophil count of ≥750 to enroll in study, this must be achieved without the addition of growth factor medication.

Exclusion criteria

What they're measuring

Optimal Dose of Pacritinib in Combination with Azacitidine - Phase 1

Timeframe: Baseline through Day 28 (Cycle 1)

Rate of Dose Limiting Toxicities (DLTs) - Phase 1

Timeframe: Baseline through Day 28 (Cycle 1)

Overall Response Rate (ORR) - Phase 2

Timeframe: Baseline through Week 16 (approximately 4 months)