Trial · NCT04054245
PAT-1251 in Treating Patients With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocytosis Myelofibrosis
NCT04054245, Phase 2, Withdrawn, M.D. Anderson Cancer Center
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. PAT-1251 in Treating Patients With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocytosis Myelofibrosis. Trials, record NCT04054245. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04054245
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- PAT-1251 in Treating Patients With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocytosis Myelofibrosis
- NCT number
- NCT04054245
- Status
- Withdrawn
- Phases
- Phase 2
- Enrollment
- 0 participants, actual
- Lead sponsor
- M.D. Anderson Cancer Center
- Conditions
- Myelofibrosis Transformation in Essential Thrombocythemia, Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase, Primary Myelofibrosis
- Interventions
- LOXL2 Inhibitor PAT-1251, Quality-of-Life Assessment, Questionnaire Administration
- First posted
- 2019-08-13
- Last updated
- 2019-08-13
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Objective response rate (ORR) | Up to 6 cycles, each cycle is 28 days. | Will be defined as complete remission (CR) + partial remission (PR) + clinical improvement (CI). Responses will be categorized according to the revised International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) and European Leukemia Net (ELN) consensus criteria for myelofibrosis. Will be estimated along with the exact 95% confidence interval. |
| Secondary | Incidence of adverse events (AEs) | Up to 2 years | Toxicity is defined as grade 3-4 clinically relevant non-hematologic toxicity or a serious adverse event that is at least possibly drug related (Common Terminology Criteria for Adverse Events version 4.03) and occurs anytime during the treatment. The method of Thall, Simon and Estey will be used for toxicity monitoring. Safety data will be summarized by category, severity and frequency. The proportion of patients with AEs will be estimated, along with the Bayesian 95% credible intervals. |
| Secondary | Time to response | From date of first treatment to the first date at which the subject's objective status was classified as a response (CR, PR, or CI), assessed up to 2 years | The distribution will be estimated by Kaplan-Meier curves. |
| Secondary | Duration of response | From the date at which the patient's objective status is first noted to be a CR, PR, or CI to the date of progression (no longer meeting criteria for either CR, PR, or CI) is documented (if one has occurred), assessed up to 2 years | The distribution will be estimated by Kaplan-Meier curves. |
| Secondary | Changes in symptom burden | Baseline up to 2 years | Will be assessed by Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score. |
| Secondary | The Descriptive statistics in the improvement of anemia | Baseline up to 2 years | Will be used to explore improvements in anemia and the following outcomes will be summarized: mean changes in hemoglobin at monthly intervals. |
| Secondary | The Descriptive statistics in Improvements of transfusion dependence. | Baseline up to 2 years | Will be used to explore improvements in the transfusion dependence. The following outcomes will be summarized: mean changes in hemoglobin at monthly intervals. |
| Secondary | The Transfusion of independent patients | Baseline up to 2 years | Will be summarized if the patient is not requiring transfusions on the study who experience an increase of 2 g/dL in their hemoglobin. |
| Secondary | Proportion of transfusion dependent patients who become transfusion independent | Baseline up to 2 years | The proportion of transfusion dependent patients (defined as requiring a transfusion of 2 units packed red blood cells (PRBCs) monthly for 3 months (12 weeks) prior to starting the trial) who become transfusion independent (not requiring a transfusion of PRBCs over a period of 3 months (12 weeks) while on study) will be summarized. |
| Secondary | Proportion of transfusion dependent patients who become transfusion independent and have a 1 g/dL increase in hemoglobin | Baseline up to 2 years | Will be summarized. |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| P30CA016672 | The University of Texas MD Anderson Cancer Center Support Grant (CCSG) | KOONG, ALBERT | UNIVERSITY OF TX MD ANDERSON CAN CTR | NATIONAL CANCER INSTITUTE | 1985 to 2026 |
Source NIH RePORTER
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04054245
GET https://conductscience.org/api/trials/v1/NCT04054245/registered