Overview
Sponsor-declared trial summary
This is a prospective, French, multicenter, open-label, design, stratified on the immunophenotypic characterization (B- or T- lineage) and the patient risk group, that aims at evaluating the efficacy and the tolerance of different schedules of pegaspargase in patients from 12 months to less than 18 years newly diagnosed with standard/medium-risk ALL
1. For children and adolescents with standard or medium risk ALL, the study has two primary objectives: 1) to assess the superiority in terms of PK at D33 of the fractionated scheme; 2) to assess the equivalence in the tolerance of the 2 schemes (from D12 of induction to D49) 2. In the High/Very High Risk group two pri…
Key facts
- Sponsor
- Assistance Publique Hopitaux De Paris
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Immune System Diseases [C20]
- Decision date (initial)
- 2024-10-10
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2024-514243-29-01
- EudraCT number
- 2015-002734-41
- ClinicalTrials.gov
- NCT02716233
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy
1. For children and adolescents with standard or medium risk ALL, the study has
two primary objectives: 1) to assess the superiority in terms of PK at D33 of
the fractionated scheme; 2) to assess the equivalence in the tolerance of the
2 schemes (from D12 of induction to D49)
2. In the High/Very High Risk group two primary objectives have been defined:
1) to assess the PK at D33; 2) to assess the toxicity of the intensified scheme
from D12 of induction to D49
Secondary objectives 1
- - to evaluate the incidence of rare subgroups of ALL and their prognostic value e.g. so-called “B-other” subgroup: BCR-ABL like (including EBF1-PDGFRB), MEF2D-X, ZNF384-X, TCF3-HLF… - 5 year EFS, DFS and OS of the rare patients with suboptimal response to therapy (induction failure or MRDTP1 ≥ 10-3) and ABL-class fusions ALLs treated with imatinib - Imatinib related adverse events (immediate and long term, cf appendix 9) in the rare patients with suboptimal response to therapy (induction failure or MRDTP1 ≥ 10-3) and ABL-class fusions ALLs treated with imatinib
Conditions and MedDRA coding
This is a prospective, French, multicenter, open-label, design, stratified on the immunophenotypic characterization (B- or T- lineage) and the patient risk group, that aims at evaluating the efficacy and the tolerance of different schedules of pegaspargase in patients from 12 months to less than 18 years newly diagnosed with standard/medium-risk ALL
Regulatory references
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2024-514243-29-00 | CAALL-F01: a French protocol for the treatment of acute lymphoblastic leukemia (ALL) in children and adolescents | Assistance Publique Hopitaux De Paris |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 1
- · Children and adolescents Age > 12 months but < 18 yearsB-lineage or T- lineage ALL · Written informed consent obtained before day 8 of treatment
Exclusion criteria 1
- · Ph+/BCR-ABL ALL (ESPhALL protocol) · CNS thrombosis before D12
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- o Adequate asparaginase activity (>100 IU/L) at D33 of induction o Toxicity: Incidence of severe toxicities (Grade ≥ 3) directly asparaginaserelated (CNS thrombosis, pancreatitis, anaphylaxia, and hyperbilirubinemia) between D12 and D49 of treatment and anyway before D8 of consolidation
Secondary endpoints 1
- - to evaluate the incidence of rare subgroups of ALL and their prognostic value e.g. so-called “B-other” subgroup: BCR-ABL like (including EBF1-PDGFRB), MEF2D-X, ZNF384-X, TCF3-HLF… - 5 year EFS, DFS and OS of the rare patients with suboptimal response to therapy (induction failure or MRDTP1 ≥ 10-3) and ABL-class fusions ALLs treated with imatinib - Imatinib related adverse events in the rare patients with suboptimal response to therapy (induction failure
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
SCP30502979 · ATC
- Active substance
- Pegaspargase
- Substance synonyms
- PEG-Asparaginase, PEG-L-Asparaginase
- Route of administration
- INJECTION
- Authorisation status
- Authorised
- ATC code
- L01XX24 — PEGASPARGASE
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Assistance Publique Hopitaux De Paris
- Sponsor organisation
- Assistance Publique Hopitaux De Paris
- Address
- Porte 23, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux
- City
- Paris Cedex 10
- Postcode
- 75475
- Country
- France
Scientific contact point
- Organisation
- Assistance Publique Hopitaux De Paris
- Contact name
- Pr BARUCHEL Andre
Public contact point
- Organisation
- Assistance Publique Hopitaux De Paris
- Contact name
- Pr BARUCHEL Andre
Locations
1 EU/EEA country · 28 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Authorised, recruitment pending | 1,636 | 28 |
| Rest of world | — | 0 | — |
Investigational sites
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-09-19 | France | Acceptable 2024-10-10
|
2024-10-10 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2026-03-04 | France | Acceptable | 2026-04-07 |