Pacritinib in CMML
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT06159491 |
Recruitment Status :
Not yet recruiting
First Posted : December 6, 2023
Last Update Posted : December 6, 2023
|
Tracking Information | |||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|
First Submitted Date ICMJE | November 28, 2023 | ||||||||||
First Posted Date ICMJE | December 6, 2023 | ||||||||||
Last Update Posted Date | December 6, 2023 | ||||||||||
Estimated Study Start Date ICMJE | January 2, 2024 | ||||||||||
Estimated Primary Completion Date | July 2025 (Final data collection date for primary outcome measure) | ||||||||||
Current Primary Outcome Measures ICMJE |
|
||||||||||
Original Primary Outcome Measures ICMJE | Same as current | ||||||||||
Change History | No Changes Posted | ||||||||||
Current Secondary Outcome Measures ICMJE |
|
||||||||||
Original Secondary Outcome Measures ICMJE | Same as current | ||||||||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||||||
Descriptive Information | |||||||||||
Brief Title ICMJE | Pacritinib in CMML | ||||||||||
Official Title ICMJE | Pacritinib in Combination With Azacitidine in Patients With Chronic Myelomonocytic Leukemia | ||||||||||
Brief Summary | This is a phase 1/2 trial of pacritinib in combination with azacitidine in patients with Chronic Myelomonocytic Leukemia (CMML). Patients will be newly diagnosed or previously treated but could not have received a prior JAK inhibitor. Patients who have previously been treated with a hypomethylating agent (HMA) must have received ≤ 1 cycle. Pacritinib will be initially tested at a dose of 200mg twice daily (dose level 0) in combination with azacitidine 75mg/m2, which can be administered subcutaneously or intravenously, for 7 days in a 28-day cycle. If there are 2 DLTs in the first 6 patients, there will be a dose escalation to pacritinib 100mg twice daily (dose level -1) and an additional 6 patients will be enrolled. Based on the phase 1, 3+3 dose de-escalation design, 6-12 patients will be enrolled in the phase 1 portion. After the completion of phase 1 and identification of the recommended phase 2 dose (RP2D), the trial will then proceed to phase 2 which will employ a Simon two stage design. This portion will include the 6 patients enrolled during the phase 1 portion at the MTD. An interim analysis for futility will occur. If 3 or fewer patients have had a clinical benefit (CB) or better, as defined by 2015 MDS/MPN IWG criteria, the PI and DSMC will meet to discuss the totality of the evidence and determine if the trial shall proceed. In the second stage, an additional 12 patients will be enrolled. | ||||||||||
Detailed Description | Not Provided | ||||||||||
Study Type ICMJE | Interventional | ||||||||||
Study Phase ICMJE | Phase 1 Phase 2 |
||||||||||
Study Design ICMJE | Allocation: N/A Intervention Model: Sequential Assignment Intervention Model Description: Phase I will follow a 3+3 design in phase I with starting dose of 200mg with possibility of de-escalation if more than 1/3 or 1/6 experience DLT. Phase I will require 6-12 patients to select the MTD. Phase II, if MTD selected in Phase I, will follow a Simon's two stage design. The null hypothesis that the true response rate is 55% will be tested against a one-sided alternative that the response rate is 80%. In the first stage, 6 patients treated at the MTD from Phase I will be included. If there are 3 or fewer responses in these 6 patients, the study may be stopped. Otherwise, 12 additional patients will be accrued for a total of 18. The null hypothesis will be rejected if 13 or more responses are observed in 18 patients. This design yields a type I error rate of 9% and power of 82% when the true response rate is 80%. Primary Purpose: Treatment |
||||||||||
Condition ICMJE | Chronic Myelomonocytic Leukemia | ||||||||||
Intervention ICMJE |
|
||||||||||
Study Arms ICMJE | Experimental: Pacritinib in combination with Azacitidine
Participants will take pacritinib 200 mg BID for each 28 day cycle, azacitidine 75mg/m2 will be administered IV or SQ QD D1-7 of each 28 day cycle
Interventions:
|
||||||||||
Publications * | Not Provided | ||||||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
|||||||||||
Recruitment Information | |||||||||||
Recruitment Status ICMJE | Not yet recruiting | ||||||||||
Estimated Enrollment ICMJE |
26 | ||||||||||
Original Estimated Enrollment ICMJE | Same as current | ||||||||||
Estimated Study Completion Date ICMJE | May 2027 | ||||||||||
Estimated Primary Completion Date | July 2025 (Final data collection date for primary outcome measure) | ||||||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
|
||||||||||
Sex/Gender ICMJE |
|
||||||||||
Ages ICMJE | 18 Years and older (Adult, Older Adult) | ||||||||||
Accepts Healthy Volunteers ICMJE | No | ||||||||||
Contacts ICMJE |
|
||||||||||
Listed Location Countries ICMJE | United States | ||||||||||
Removed Location Countries | |||||||||||
Administrative Information | |||||||||||
NCT Number ICMJE | NCT06159491 | ||||||||||
Other Study ID Numbers ICMJE | GCO 23-1514 | ||||||||||
Has Data Monitoring Committee | Yes | ||||||||||
U.S. FDA-regulated Product |
|
||||||||||
IPD Sharing Statement ICMJE |
|
||||||||||
Current Responsible Party | Douglas Tremblay, Icahn School of Medicine at Mount Sinai | ||||||||||
Original Responsible Party | Same as current | ||||||||||
Current Study Sponsor ICMJE | Douglas Tremblay | ||||||||||
Original Study Sponsor ICMJE | Same as current | ||||||||||
Collaborators ICMJE | Sobi, Inc. | ||||||||||
Investigators ICMJE |
|
||||||||||
PRS Account | Icahn School of Medicine at Mount Sinai | ||||||||||
Verification Date | November 2023 | ||||||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |