Health TrialsbyHealthPanel

A Study Evaluating the Efficacy and Safety of Momelotinib in Participants With Vacuoles, E1-enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome

ConditionVEXAS Syndrome
Trial statusNot yet recruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorGlaxoSmithKline

About this trial

This study will assess the efficacy and safety of momelotinib in participants with a diagnosis of VEXAS.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age greater than equal to (>=)18 years OR of legal age of consent in the jurisdiction in which the study is taking place, at the time of signing the Informed Consent Form.

Documented evidence of a canonical, pathogenic Ubiquitin-like modifier activating enzyme 1 (UBA1) mutation

Inflammatory manifestations: current or documented past involvement within 6 months of at least one organ system

Receiving glucocorticoid (GC) treatment (prednisone/prednisolone) for >=4 consecutive weeks for >=10 days prior to randomization.

Disqualifiers

More than 1 prior admission to an intensive care unit due to a VEXAS flare within the 6 months prior to randomization.

History of severe corticosteroid toxicity: uncontrolled concomitant cardiovascular, nervous system, pulmonary (including obstructive pulmonary disease), renal, hepatic, endocrine (including uncontrolled diabetes mellitus), psychiatric, osteoporosis/osteomalacia, glaucoma, corneal ulcers/injuries, nausea or vomiting or gastrointestinal disease.

High risk/very high risk Myelodysplastic syndrome (MDS), according to the Revised International Prognostic Scoring System (IPSS-R) with overall risk score >3.5.

Peripheral blood blast counts >=10%.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Momelotinib

    Drug

    Momelotinib will be administered

  • Glucocorticoids

    Drug

    Glucocorticoids (prednisone or prednisolone) will be administered

  • Placebo

    Drug

    Placebo will be administered

Treatment groups

136 Participants
are divided into 3 treatment groups
Group A: Momelotinib Dose level 1 + GlucocorticoidsExperimental treatment 2 interventions
Group B: Momelotinib Dose level 2 + GlucocorticoidsExperimental treatment 2 interventions
Group C: Placebo + GlucocorticoidsPlacebo comparator 2 interventions

Trial outcomes

Primary outcomes

1

ORR (Objective response rate) at Week 26

ORR is defined as the proportion of participants who have achieved complete response (CR) or partial response (PR) during the 26-week Primary Treatment Period.

Time frame
At Week 26

Secondary outcomes

1

Phase 2: Percentage of participants with partial response (PR) or complete response (CR) at Week 26

Percentage of participants with PR or CR at Week 26 to support identification of the Recommended Phase 3 dose (RP3D)

Time frame
At Week 26
2

Phase 2: Number of participants with adverse events and clinically significant changes in laboratory parameters, and vital signs to support identification of the RP3D

Time frame
Up to 26 Weeks
3

Phase 2: Plasma concentrations of momelotinib and metabolite of momelotinib 21 (M21) to support identification of the RP3D

Time frame
Up to 26 Weeks
4

Number of flare-free days

Flare-free days are calendar days without clinical evidence of VEXAS flare and without treatment escalation. Total number of flare-free days is defined as the cumulative number of consecutive and non-consecutive flare-free days.

Time frame
Up to 26 Weeks

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

GlaxoSmithKline

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: