Trial · NCT07387354
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 11 of 11
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Optimal Dose of Pacritinib in Combination with Azacitidine - Phase 1 | Baseline through Day 28 (Cycle 1) | "Phase 1 of the study is designed to determine the optimal dose of pacritinib in combination with azacitidine in subjects with moderate, high, and very high risk MDS. The optimal dose (recommended Phase 2 dose, RP2D) will be defined as the highest dose level associated with an acceptable rate of dose-limiting toxicities (DLTs) during Cycle 1, using a standard 3 + 3 dose-escalation design. DLTs are defined per NCI CTCAE v5.0 (Section 7.5 of protocol): * Any grade ≥ 3 non-hematologic toxicity (excluding grade 3 nausea, vomiting, or diarrhea that improves to ≤ grade 2 within 5 days); * Prolonged grade 4 hematologic toxicities not due to persistent disease; * Any grade 5 toxicity not attributable to disease progression. Safety, tolerability, and pharmacodynamic data will support RP2D selection. " |
| Primary | Rate of Dose Limiting Toxicities (DLTs) - Phase 1 | Baseline through Day 28 (Cycle 1) | "the proportion of participants experiencing dose limiting toxicities (DLTs) occurring within thirty days of first dose of study agent that meets the following qualifications and is potentially related to study medication or combination will be considered a dose limiting toxicity. DLTs are defined per NCI CTCAE v5.0 as: * Any grade ≥ 3 non-hematologic toxicity (excluding grade 3 nausea, vomiting, or diarrhea that improves to ≤ grade 2 within 5 days); * Prolonged grade 4 hematologic toxicities (e.g., neutropenia or thrombocytopenia not due to persistent disease); * Any grade 5 toxicity not attributable to disease progression." |
| Primary | Overall Response Rate (ORR) - Phase 2 | Baseline through Week 16 (approximately 4 months) | Overall response rate (ORR) = patients achieving CR (or CR equivalent), PR, CRL, CRh, or hematologic improvement (HI) ÷ total patients. ORR per IWG 2023 criteria; hematologic response per IWG 2018. Responses: CR, CR equivalent, PR, CRL, CRh, or HI (erythroid, platelet, neutrophil). IWG 2023: CR: BM \<5% blasts; Hb ≥10 g/dL; platelets ≥100×10⁹/L; ANC ≥1.0×10⁹/L; no blood blasts. CR equivalent: Baseline \<5% blasts + complete cytogenetic response. PR: CR criteria except ≥50% blast reduction (still ≥5%). CRL: \<5% blasts; no blood blasts; recovery of 1-2 lineages. CRh: \<5% blasts; no blood blasts; platelets \>50×10⁹/L; ANC \>0.5×10⁹/L. IWG 2018 HI: HI-E: Hb ↑ ≥1.5 g/dL or RBC TI ≥8 wks. HI-P: Platelets ↑ ≥30×10⁹/L or from \<20 to \>20×10⁹/L + ≥100% ↑. HI-N: ≥100% ↑ and absolute ↑ ≥0.5×10⁹/L. |
| Secondary | Duration of Overall Response Rate - Phase 2 | Up to 2 years after last patient enrolled | Time from first documented response (CR, PR, or HI per IWG criteria) until relapse, progression, or death from any cause. |
| Secondary | Overall Surival - Phase 2 | Up to 24 months | Time from study enrollment to death from any cause. |
| Secondary | Number of Treatment Emergent Adverse Events (TEAEs) | Baseline through Cycle 4, plus 30 days (Approximately 5 months) | Incidence, seriousness, severity, outcome, discontinuation or dose modifications related to TEAEs. This is separate from the primary endpoint of phase one which will be rate of DLTs. |
| Secondary | Molecular Response Rate (Phase 2) | Baseline through Week 16 (approximately 4 months) | Assessment of molecular response will be done via rate of disappearance of relevant mutations via next-generation sequencing on pre and post treatment bone marrow samples. |
| Secondary | Change in Intracellular Signaling Pathway Activation (JAK/STAT, NF-κB and AKT/mTOR | Baseline | Whole blood along with bone marrow aspirate with be collected pre and post treatment to assess intracellular flow and phosflow to detect NF-κB, JAK/STAT, and AKT/mTOR signaling within all myeloid cells. Post treatment will be compared to pre-treatment for all flow markers (intra- and extracellular) for changes in signaling and WBC maturation. Patient genetics will also be evaluated in the context of signaling response and maturation |
| Secondary | Change in Intracellular Signaling Pathway Activation (JAK/STAT, NF-κB and AKT/mTOR | After four days of azacitidine and pacritinib therapy | Whole blood along with bone marrow aspirate with be collected pre and post treatment to assess intracellular flow and phosflow to detect NF-κB, JAK/STAT, and AKT/mTOR signaling within all myeloid cells. Post treatment will be compared to pre-treatment for all flow markers (intra- and extracellular) for changes in signaling and WBC maturation. Patient genetics will also be evaluated in the context of signaling response and maturation |
| Secondary | Assessment of Quality of Life Using the quality of life in myelodysplasia scale (QUALMS) | At Baseline | To measure quality of life pre-treatment and after four cycles of treatment using the quality of life in myelodysplasia scale (QUALMS). The Quality of Life in Myelodysplasia Scale (QUALMS) is a 38-item, disease-specific questionnaire used to measure the health-related quality of life in patients with myelodysplastic syndromes (MDS). It includes three subscales for physical burden, emotional burden, and benefit finding, and a total score ranges from 0 to 100, where a higher score indicates a better quality of life. |
| Secondary | Assessment of Quality of Life Using the quality of life in myelodysplasia scale (QUALMS) | After 4 cycles of treatment (approximately 112 days) | To measure quality of life pre-treatment and after four cycles of treatment using the quality of life in myelodysplasia scale (QUALMS). The Quality of Life in Myelodysplasia Scale (QUALMS) is a 38-item, disease-specific questionnaire used to measure the health-related quality of life in patients with myelodysplastic syndromes (MDS). It includes three subscales for physical burden, emotional burden, and benefit finding, and a total score ranges from 0 to 100, where a higher score indicates a better quality of life. |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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