Trial · NCT07098936
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 14 of 14
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Determination of the maximum tolerated dose of momelotinib | First 4-week cycle of momelotinib treatment | The maximum tolerated dose (MTD) is defined by a target dose-limiting toxicities (DLT) rate of 30%, assessed during the observation window by a 3+3 design. The 3+3 design will enroll a first cohort of 3 patients at the starting dose of 200 mg/d: * If 0 of the 3 patients at the dose of 200 mg/d experienced a DLT during the first 4-week cycle of momelotinib, the dose will escalate to 300 mg/d for the next cohort. * If 1 of the 3 patients at the dose of 200 mg/d has a DLT, a cohort of three additional patients will be treated at the same dose of 200 mg/d. * If \> 1 patient of the 3 patients at the dose of 200 mg/d have a DLT, the dose will de-escalate to 150 mg/d for the next cohort. The same process will be repeated until reaching the MTD. In total, between 6 and 18 patients will be enrolled during this safety run-in phase. |
| Primary | Clinical efficacy | During the 24 first weeks of momelotinib treatment | Determination of overall clinical response rate at 24 weeks after momelotinib initiation on VEXAS related symptoms (including complete (CR) or partial response (PR)) : * CR includes complete disappearance of symptoms related to systemic inflammation according to treating physician and daily dose of steroids ≤ 10 mg/d (equivalent prednisolone). * PR includes complete disappearance of symptoms related to systemic inflammation according to treating physician and reduction of at least 50% of daily dose steroids compared to baseline and/or daily dose of steroids \> 10 mg/d. |
| Secondary | Pharmacokinetic | At cycle 1 Day 8-9 and cycle 13 Day 8-9 | Concentrations of momelotinib and its major metabolite M21 will be determined in plasma samples using the currently approved bioanalytical methodology by Frontage Laboratories Inc. Pharmacokinetic sampling will be only performed for patients in safety run-in phase. Samples should be obtained at cycle 1 Day 8 and cycle 13 Day 8, at times following : Pre-dose then at 0.5 h, 1 h, 1.5 h, 2 h, 3 h, 4 h, 6 h, 8h and at 24 h post Day 8 dose. |
| Secondary | Incidence of Treatment-Emergent Adverse Events | From enrollment to the end of treatment at 48 weeks | Collection of Adverse events (AE), serious AE (SAE) and toxicities as measured by NCI CTCAE (National Cancer Institute Common Terminology Criteria for Adverse Events) v5.0 |
| Secondary | Response | After 4, 12, 24 and 48 weeks of momelotinib treatment | Determination of overall clinical response rates (including Complete Response or Partial Response) and biological response rates (complete or partial) |
| Secondary | Hematological response | After 16 weeks of momelotinib treatment | Erythroid hematological improvement will be assessed by the investigators according to IWG 2018 criteria through collection of transfusion records and hematology parameters. |
| Secondary | Steroids dose reduction | After 24 weeks of momelotinib treatment | Steroids dose reduction compared to baseline and/or steroids withdrawal rates |
| Secondary | Overall survival | From enrollment to the end of treatment at 12 months | Overall survival assessment |
| Secondary | Myelodysplastic syndrome (MDS) evolution | From baseline at 12 and 24 weeks of momelotinib treatment | Changes in the underlying MDS from baseline, at 12 and 24 weeks, including MDS progression based on hematological, cytogenetic and molecular analysis |
| Secondary | Duration of response | From enrollment to the end of treatment at 48 weeks | Duration of response on VEXAS symptoms defined as the time from the date of initial documentation of a clinical response (Complete Response or Partial Response) to the date of first documented evidence of relapse or death |
| Secondary | Best clinical response | From enrollment to the end of treatment at 48 weeks | Determination of time to the first and best clinical response |
| Secondary | RBC independency | From enrollment to the end of treatment at 48 weeks | Duration of Red Blood Count (RBC) independency in patients with RBC dependency at time of inclusion, according to IWG 2018 criteria |
| Secondary | UBA1 VAF | From enrollment to the end of treatment at 24 weeks | Evolution of UBA1 (Ubiquitin Like Modifier Activating Enzyme 1) VAF (Variant Allelic Frequency) from baseline to W4, W12 and W24 after momelotinib treatment |
| Other | Validation of the VEXAS personal scoring system (VPSS) | After 4, 12 and 24 weeks of momelotinib treatment | Measurement of VPSS to validate the score currently under development which would allow objectively quantifying the impact of VEXAS-related symptoms on patients' 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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