Study of the safety and efficacy of asciminib in pediatric and young adult participants with r/r Ph+ or ABL-class Ph-like ALL
Key facts
- Where
- Bordeaux, Lyon, Marseille, Nantes, Paris, Pessac és Rennes Cedex 9, FranciaországSee the 8 sites
- Phase
- 1/2. fázis
Where in Franciaország
- Centre hospitalier Universitaire de BordeauxBordeaux, FranciaországToborzás még nem indult
- Hospices Civils de LyonLyon, FranciaországToborzás még nem indult
- Centre Hospitalier Regional De MarseilleMarseille, FranciaországToborzás még nem indult
- Hospital Hotel DieuNantes, FranciaországToborzás még nem indult
- Hopital Saint LouisParis, FranciaországToborzás még nem indult
- Robert Debre University HospitalParis, FranciaországToborzás még nem indult
- Centre Hospitalier Universitaire de BordeauxPessac, FranciaországToborzás még nem indult
- Centre Hospitalier Universitaire De RennesRennes Cedex 9, FranciaországToborzás még nem indult
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Novartis Pharma AG
- Trial code
- EU 2025-522019-40-00
- Record updated
- 2026. október 6.
- Original description
The primary objective for Part 1 dose escalation is to determine the RP2D and the primary endpoints will be the incidence of DLTs and incidence and severity of AEs and laboratory safety findings during cycle 1. The primary objective for Part 2 dose expansion will be to assess complete remission rates at the end of cycle 1 and the primary endpoint will be proportion of CR evaluable Ph+ participants achieving CR at the end of cycle 1Read full original descriptionShow less
A multi-center, open-label, phase I/II study to evaluate the safety and efficacy of asciminib with chemotherapy followed by blinatumomab in pediatric, adolescent, and young adult patients with relapsed or refractory BCR::ABL1-positive (Ph+) or ABL-class Ph-like Acute Lymphoblastic Leukemia (ALL)
The primary objective for Part 1 dose escalation is to determine the RP2D and the primary endpoints will be the incidence of DLTs and incidence and severity of AEs and laboratory safety findings during cycle 1. The primary objective for Part 2 dose expansion will be to assess complete remission rates at the end of cycle 1 and the primary endpoint will be proportion of CR evaluable Ph+ participants achieving CR at the end of cycle 1
Inclusion: Male or female participants ≥1 year to ≤30 years of age at screening; Participants with documented history of either Ph+ ALL or ABL-class Ph-like ALL with ABL1 or ABL2 rearrangements. Genetic testing will be performed locally and eligible alterations confirmed by treating investigators; central confirmation will not be performed as part of the study. (Of note, participants with known T315I mutations are eligible for inclusion in a separate cohort); Active B-Cell ALL at screening defined by MFC or IG/TCR PCR of ALL blasts >0.01% in participants with either: a. Primary refractory disease (>0.01% ALL blasts present at the end of induction and 1 consolidation cycle) OR b. Relapsed ALL with evidence of involvement of BM with ALL (MFC or IG/TCR PCR>0.01%) after at least one line of therapy; Participants with CNS1, CNS2, CNS3a or CNS3b at screening; Documented history of CD19 expressing B-cell ALL (in peripheral blood or bone marrow by flow cytometry) a. For participants who received anti-CD19 targeted therapy (e.g CD19 CAR T cells or blinatumomab), CD19 expressing B-cell ALL must be documented after anti-CD19 therapy completion prior to cycle 1 day 1.
Exclusion: History of hematopoietic stem cell transplant within the prior 12 weeks; Presence of active acute or chronic graft-versus-host disease (GVHD). Hematopoietic stem cell transplant recipients receiving any agent to treat or prevent GVHD within 4 weeks are not eligible for this trial; Extramedullary disease (non-CNS and/ or isolated CNS disease) at screening; Participants with CNS3c at screening (Clinical signs of CNS leukemia (such as facial nerve palsy, brain/eye involvement or hypothalamic syndrome); Participants with >3 relapses of ALL at screening.