Expanded Access Protocol - Blinatumomab in Pediatric & Adolescent Subjects With Relapsed/Refractory B-precursor ALL
RIALTO
An Open-Label, Multi-center, Expanded Access Protocol of Blinatumomab for the Treatment of Pediatric and Adolescent Subjects With Relapsed and/or Refractory B-precursor Acute Lymphoblastic Leukemia (ALL)
2 other identifiers
expanded_access
N/A
7 countries
19
Brief Summary
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
Trial Health
Trial Health Score
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19 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 9, 2014
CompletedFirst Posted
Study publicly available on registry
July 11, 2014
CompletedMay 13, 2024
May 1, 2024
July 9, 2014
May 10, 2024
Conditions
Interventions
A single cycle of blinatumomab (CIVI) treatment is 6wks, 4wks of treatment followed by a 2wk treatment-free interval. Up to 5 cycles will be administered per subject. In the first cycle, for patients with an M3 bone marrow, the initial dose will be 5μg/m2/day for the first 7days, escalated to 15μg/m2/day on D8-D29. For all subsequent cycles 15μg/m2/day will be the dose for all 4wks of continuous treatment. In case of M2 bone marrow or M1 bone marrow with an MRD relapse at screening, the initial dose will start at 15μg/m2/day for the first 7days of treatment \& no dose step at D8. For all subsequent cycles the dose will remain 15μg/m2/day. A dose of 9μg/day for the initial dose (if applicable) \& 28μg/day for the escalated dose after dose step should not be exceeded.
LTFU (Long Term Follow-Up) will extend past 18 months for patients already ended the study/still on study or to be enrolled at European sites if they did not receive a transplantation after blinatumomab treatment. For subjects to be included in the additional LTFU, data will be captured until subjects are 18yrs old (every 6 months by phone contact). The following will be captured: relapse (medullary or extra-medullary relapse and its specific location), second tumor (which type), alive/died and cause of death, hospitalization and reason for hospitalization.
Eligibility Criteria
You may qualify if:
- 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)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Amgenlead
Study Sites (19)
Research Site
Aurora, Colorado, 80045, United States
Research Site
Cincinnati, Ohio, 45229, United States
Research Site
Memphis, Tennessee, 38105, United States
Research Site
Salt Lake City, Utah, 84113, United States
Research Site
Vienna, 1090, Austria
Research Site
Marseille, 13385, France
Research Site
Paris, 75019, France
Research Site
Berlin, 13353, Germany
Research Site
Frankfurt am Main, 60590, Germany
Research Site
Kiel, 24105, Germany
Research Site
München, 80337, Germany
Research Site
Münster, 48149, Germany
Research Site
Tübingen, 72076, Germany
Research Site
Würzburg, 97080, Germany
Research Site
Monza (MB), 20900, Italy
Research Site
Padua, 35128, Italy
Research Site
Roma, 00165, Italy
Research Site
Zurich, 8032, Switzerland
Research Site
Sheffield, S10 2TH, United Kingdom
Related Publications (3)
Locatelli F, Zugmaier G, Mergen N, Bader P, Jeha S, Schlegel PG, Bourquin JP, Handgretinger R, Brethon B, Rossig C, Kormany WN, Viswagnachar P, Chen-Santel C. Blinatumomab in pediatric relapsed/refractory B-cell acute lymphoblastic leukemia: RIALTO expanded access study final analysis. Blood Adv. 2022 Feb 8;6(3):1004-1014. doi: 10.1182/bloodadvances.2021005579.
PMID: 34979020BACKGROUNDLocatelli F, Zugmaier G, Mergen N, Bader P, Jeha S, Schlegel PG, Bourquin JP, Handgretinger R, Brethon B, Rossig C, Chen-Santel C. Blinatumomab in pediatric patients with relapsed/refractory acute lymphoblastic leukemia: results of the RIALTO trial, an expanded access study. Blood Cancer J. 2020 Jul 24;10(7):77. doi: 10.1038/s41408-020-00342-x. No abstract available.
PMID: 32709851BACKGROUNDQueudeville M, Stein AS, Locatelli F, Ebinger M, Handgretinger R, Gokbuget N, Gore L, Zeng Y, Gokani P, Zugmaier G, Kantarjian HM. Low leukemia burden improves blinatumomab efficacy in patients with relapsed/refractory B-cell acute lymphoblastic leukemia. Cancer. 2023 May 1;129(9):1384-1393. doi: 10.1002/cncr.34667. Epub 2023 Feb 24.
PMID: 36829303BACKGROUND
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
MD
Amgen