Expanded Access Protocol - Blinatumomab in Pediatric & Adolescent Subjects With Relapsed/Refractory B-precursor ALL (RIALTO)

Amgen

Status

Conditions: Relapsed/Refractory B-Precursor Acute Lymphoblastic Leukemia

Treatments: Other: Extension of LTFU as per ProtocolAmendment7 7Jun18; Drug: Blinatumomab

Study type: Expanded Access

Funder types: Industry

Identifiers

NCT02187354

20130320

2014-001700-21 (EudraCT Number)

Details and patient eligibility

About

Primary Objective:

To estimate the incidence of treatment-emergent and treatment-related adverse events during treatment with blinatumomab in pediatric and adolescent subjects with B-precursor ALL in second or later bone marrow relapse, in any marrow relapse after alloHSCT, or refractory to other treatments

Secondary Objective(s):

To describe key efficacy outcomes, including incidence of complete response (CR) within 2 cycles of blinatumomab, minimal residual disease (MRD) remission within 2 cycles of blinatumomab, relapse free survival (RFS), overall survival (OS), incidence of alloHSCT, and 100-day mortality after alloHSCT.

Hypotheses:

A formal statistical hypothesis will not be tested. The incidence of treatment-emergent and treatment-related adverse events will be estimated.

Study Endpoints:

• Incidence of treatment-emergent and treatment-related adverse events
• Incidence of CR within 2 cycles of blinatumomab
• MRD remission within 2 cycles of blinatumomab
• RFS
• OS
• Incidence of alloHSCT
• 100-day mortality after alloHSCT

Study Design:

Multi-center, open-label, single-arm expanded access protocol

Sex: All

Ages: Under 17 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria 101 Immunophenotypic evidence of CD19 positive B-precursor ALL (pro B-, pre B-, common ALL) 102 Age > 28 days and < 18 years at the time of informed consent/assent 103 Morphological or molecular evidence of relapsed/refractory disease, defined as one of the following:

• Second or later bone marrow relapse (defined as M3 marrow or M2 marrow or M1 marrow but with MRD level ≥ 10E-3), or

• Any marrow relapse after alloHSCT (defined as M3 marrow or M2 marrow or M1 marrow but with and MRD level ≥ 10E-3), or

• Refractory to other treatments:

  • For patients in first relapse: failure to achieve a CR following a full standard reinduction chemotherapy regimen
  • For patients who have not achieved a first remission: failure to achieve remission following a full standard induction regimen

• Subjects previously treated with blinatumomab may be eligible, if subject ended treatment for reason(s) other than disease progression or intolerability to blinatumomab (Note: This does not include patients who have already received blinatumomab treatment on this study, but refers only to patients outside of the 20130320 study)

Other Inclusion Criteria may apply

Exclusion Criteria 201 Any active acute Graft-versus-Host Disease (GvHD) grade 2 to grade 4 according to the Glucksberg criteria or active chronic GvHD requiring systemic treatment 202 Immunosuppresive agents to prevent or treat GvHD within 2 weeks prior to blinatumomab treatment (except for topical corticosteroids) 203 Active (overt) ALL in the CNS (confirmed by cerebrospinal fluid [CSF] analysis) or in testes

Other Exclusion Criteria may apply