Trial · NCT04054245
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 13 of 13
Download this page as JSON| 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. |
| Secondary | Proportion of transfusion independent patients requiring a transfusion | Baseline up to 2 years | Will be summarized. |
| Other | The Correlative Studies | Baseline up to 2 years | Descriptive statistics including plots, mean, median and standard deviations will be used to summarize data. For continuous outcomes, t-test and analysis of variance (ANOVA) will be used to compare outcome measures across patient characteristics. Dunnett's and Tukey's test that properly adjust for multiplicity in multiple tests will be implemented. Pair-wise comparisons will be performed using pre- and post-therapy samples from each patient. The chi-square test or Fisher's exact test will be used to test the association between two categorical variables such as disease state and performance status. |
| Other | Percent target engagement after treatment | Baseline up to 2 years | The percent target engagement based on a plasma target engagement assay after treatment with LOXL2 inhibitor PAT-1251 will be determined. Descriptive statistics including plots, mean, median and standard deviations will be used to summarize data. For continuous outcomes, t-test and ANOVA will be used to compare outcome measures across patient characteristics. Dunnett's and Tukey's test that properly adjust for multiplicity in multiple tests will be implemented. Pair-wise comparisons will be performed using pre- and post-therapy samples from each patient. The chi-square test or Fisher's exact test will be used to test the association between two categorical variables such as disease state and performance status. |
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.
For software
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