Bomedemstat vs BAT for Essential Thrombocythemia

JRCT ID: jRCT2031230658

Registered date:27/02/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: essential thrombocythemia
• Date of first enrollment: 29/05/2024
• Target sample size: 17
• Countries of recruitment: Argentina,Japan,Australia,Japan,Belgium,Japan,Canada,Japan,China,Japan,Colombia,Japan,France,Japan,Germany,Japan,Hong Kong,Japan,Hungary,Japan,Israel,Japan,Italy,Japan,South Korea,Japan,New Zealand,Japan,Poland,Japan,Portugal,Japan,Spain,Japan,Sweden,Japan,Netherlands,Japan,Taiwan,Japan,Turkey,Japan,United Kingdom,Japan,United States,Japan
• Study type: Interventional
• Intervention(s): After the screening phase, each participant will be randomly assigned to receive either bomedemstat or BAT until one of the conditions for discontinuation of intervention is met.

Outcome(s)

• Primary Outcome: DCHR

Secondary Outcome

Change in fatigue score from baseline as measured on the MFSAF v4.0 fatigue symptom item.
Change in total fatigue score from baseline as measured on the PROMIS Fatigue SF-7a.
Change in total symptom score from baseline as measured on the MFSAF v4.0.
Duration of clinicohematologic response
Duration of hematologic remission
Thrombotic event
Major hemorrhagic event
Disease progression: Transformation to post-ET MF or MDS/AML
EFS: The time from randomization to the first documented occurrence of death due to any cause or one of the following as assessed by the adjudication committee, whichever occurs first:
- Thrombotic event
- Major hemorrhagic event
- Disease progression to MF or MDS/AML
AE
Discontinuing study intervention due to an AE.

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

- Has a diagnosis of ET per WHO 2016 diagnostic criteria for myeloproliferative neoplasms
- Has a bone marrow fibrosis score of Grade 0 or Grade 1, as per a modified version of the European Consensus Criteria for Grading Myelofibrosis
- Has a history of inadequate response to or intolerance of hydroxyurea per at least 1 of the following criteria, based on modified European LeukemiaNet (ELN) criteria for hydroxyurea resistance or intolerance: hydroxyurea resistance (or inadequate response) or hydroxyurea Intolerance
- Has an inadequate or loss of response to their most recent prior ET therapy, requiring a change of cytoreductive therapy
- Has a platelet count > 450 x 109/L (450k /uL) assessed up to 72 hours before first dose of study intervention
- Has an absolute neutrophil count (ANC) >=0.75 x 109/L assessed up to 72 hours before first dose of study intervention

Exclude criteria

- Known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to bomedemstat or lysine demethylase or monoamine oxidase inhibitor (LSDi or MAOi) that contraindicates participation
- History of any illness/impairment of GI function that might interfere with drug absorption (eg, chronic diarrhea or history of gastric bypass surgical procedure), confound the study results or pose an additional risk to the individual by participation in the study
- Evidence at the time of Screening of increased risk of bleeding
- History of a malignancy, unless potentially curative treatment has been completed with no evidence of malignancy for 2 years.
Note: The time requirement does not apply to participants who underwent successful definitive resection of basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ, excluding carcinoma in situ of the bladder
- Human immunodeficiency virus (HIV)-infected participants with a history of Kaposi's sarcoma and/or Multicentric Castleman's Disease

Related Information

• Primary Sponsor: Tanaka Yoshiyuki
• Secondary ID(s): NCT06079879