台灣臨床試驗主持人資料庫|TPIDB

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Clinical Trials List

Protocol NumberCDAK539A12303

NCT Number(ClinicalTrials.gov Identfier)NCT07357727

Not yet recruiting

2026-05-18 - 2032-05-31

Phase III

Not yet recruiting2

Recruiting2

A Phase 3, Randomized, Double-blind, Active-control Study of Pelabresib (DAK539) and Ruxolitinib vs. Placebo and Ruxolitinib in Adult Patients With Myelofibrosis Who Are JAK Inhibitor Naive

Trial Applicant

NOVARTIS (TAIWAN) CO., LTD.
Sponsor
Trial scale

Multi-Regional Multi-Center
Update

2026/09/10

Investigators and Locations

Principal Investigator 李思慧無

National Taiwan University Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator 林炫聿無

CHANGHUA CHRISTIAN HOSPITAL

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator Yi-Chang Liu 無

Kaohsiung Medical Univeristy Chung-Ho Memorial Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Recruiting

Principal Investigator Tsai-Yun Chen

National Taiwan University Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Recruiting

Condition/Disease

Primary Myelofibrosis (PMF)、 Post-polycythemia Vera Myelofibrosis (PPV-MF) 、Post-essential Thrombocythemia Myelofibrosis (PET-MF)

Objectives

本試驗的目的，是針對試驗藥物 pelabresib 併用 ruxolitinib 治療骨髓纖維化患者的安全性和效果，進行更多研究。Ruxolitinib 作為單一治療已進行過研究，且已經可供醫師開立處方給骨髓纖維化患者。在本試驗中，希望評估 pelabresib 併用 ruxolitinib 相較於單獨使用 ruxolitinib 治療骨髓纖維化的效益。

Test Drug

膜衣錠

Active Ingredient

film-coated tablets

Dosage Form

116

Dosage

25 & 100 mg

Endpoints

共同主要評估指標：
• 脾臟反應，定義為依據磁振造影 (MRI)（或電腦斷層掃描 [CT]）測量，且由中央放射學判讀評估，自基準期至第 24 週達到脾臟體積減小 ≥ 35% (SVR35)
• 依據骨髓纖維化症狀評估表 (MFSAF) v4.0 測量，第 24 週時總症狀分數 (TSS) 自基準期以來的絕對變化
• 脾臟反應，定義為依據 MRI（或 CT 掃描）測量，且由中央放射學判讀評估，自基準期至第 24 週達到脾臟體積減小 ≥ 35% (SVR35)
• 依據 MFSAF v4.0 測量，第 24 週時總症狀分數 (TSS) 自基準期以來的絕對變化

Inclution Criteria

Key Inclusion Criteria:

Participants have diagnosis of primary myelofibrosis (PMF) or post-polycythemia vera myelofibrosis (post-PV MF) or post-essential thrombocythemia myelofibrosis (post-ET MF) according to the International Consensus Classification (ICC) of Myeloid Neoplasms and Acute Leukemias 2022
DIPSS risk category of intermediate-1, intermediate-2 or high-risk
Spleen volume ≥ 450 cm3 by CT or MRI scan (local read sufficient if no central read available)
Have an average TSS of ≥15 within 7 days prior to randomization, using MFSAF v. 4.0 (at least 4 out of 7 TSS assessments required for average calculation)
Participants with an Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2
Blasts <5% in peripheral blood. Assessment of blasts in peripheral blood is mandatory at screening
Platelet count ≥ 100 x 10^9/L in the absence of growth factors or transfusions for the previous 4 weeks

Exclusion Criteria

Key Exclusion Criteria:

Prior splenectomy at any time or splenic irradiation in the previous 6 months
Prior hematopoietic cell transplant or participant anticipated to receive a hematopoietic cell transplant within 24 weeks from the date of randomization
Blasts ≥ 5% in bone marrow if results available at screening or history of accelerated phase (AP) or leukemic transformation
History of a malignancy (other than MF, PPV-MF or PET-MF) in the past 3 years in need of systemic treatment
Received any approved or investigational agent other than hydroxyurea or anagrelide for the treatment of MF within 14 days of first dose of study treatment or within 5 half-lives of the approved or investigational agent, whichever is longer
Prior treatment with any JAK inhibitor or Bromodomain and extraterminal domain (BET) inhibitor

The Estimated Number of Participants

Taiwan

13 participants
Global

460 participants