Study to determine the dose and safety of asciminib in pediatric patients with chronic myeloid leukemia

JRCT ID: jRCT2031210591

Registered date:01/02/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Philadelphia chromosome positive chronic myeloid leukemia in chronic phase
• Date of first enrollment: 05/07/2022
• Target sample size: 3
• Countries of recruitment: France,Japan,Germany,Japan,Hungary,Japan,Italy,Japan,Greece,Japan,Nertherlands,Japan,Poland,Japan,Russia,Japan,Korea,Japan,Thailand,Japan,Turkey,Japan,China,Japan,US,Japan
• Study type: Interventional

Intervention(s)

Drug: Asciminib Pediatric formulation group
Mini-tablets will be supplied as size 0 capsules containing 1 mg mini-tablets, taken orally:

10 mg (10x 1 mg tablets in capsule) BID 15 mg (15x 1 mg tablets in capsule) 20 mg (20x 1 mg tablets in capsule) BID 30 mg (30x 1 mg tablets in capsule) BID

Drug: Asciminib Adult formulation group
40 mg tablets BID, taken orally.
20 mg tablets BID, taken orally.

Outcome(s)

Primary Outcome

Primary Pharmacokinetic (PK) parameter: AUClast [ Time Frame: 52 weeks ]
Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).

Primary PK parameter: AUCtau [ Time Frame: 52 weeks ]
Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).

Secondary PK parameter: Cmax [ Time Frame: 52 weeks ]
Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).

Secondary PK parameter: Tmax [ Time Frame: 52 weeks ]
Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).

Secondary PK parameter: Ctrough [ Time Frame: 52 weeks ]
Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).

Key inclusion & exclusion criteria

• Age minimum: >= 1age old
• Age maximum: < 18age old
• Gender: Both

Include criteria

- Male or female participants:
a. Pediatric formulation group: >=1 and less than 18 years of age at study entry.
b. Adult formulation group: >=14 and less than 18 years of age and body weight of >= 40 kg at study entry.
- Participants must meet all of the following laboratory values at the screening visit. In the case where bone marrow blast and promyelocyte counts are available, these will be accepted if done within 56 days prior to the screening visit, to avoid unnecessary repetition of this test.
a. 15% blasts in peripheral blood and bone marrow
b. < 30% combined blasts plus promyelocytes in peripheral blood and bone marrow
c. < 20% basophils in the peripheral blood
d. Neutrophils >= 1.5 x 10E9/L (or white blood cell (WBC) >= 3 x 10E9/L if neutrophils are not available) and platelet count >= 100 x 10E9/L
e. No evidence of extramedullary leukemic involvement, with the exception of hepatosplenomegaly
- Prior treatment with a minimum of one TKI.
- Failure or intolerance to the most recent TKI therapy at the time of screening.
- Evidence of typical BCR-ABL fusion gene (BCR-ABL1) transcript [e14a2 and/or e13a2] at the time of screening which are amenable to standardized real time quantitative polymerase chain reaction (RQ-PCR) quantification.

Exclude criteria

- Known presence of the T315I mutation prior to study entry.
- Known second chronic phase of CML after previous progression to AP/BC.
- Previous treatment with a hematopoietic stem-cell transplantation.
- Patient planning to undergo allogeneic hematopoietic stem cell transplantation.
- Cardiac or cardiac repolarization abnormality.

Related Information

• Primary Sponsor: Yamauchi Kyosuke
• Secondary ID(s): NCT04925479