An Open-Label Study of JZP-458 (RC-P) in Patients With Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic Lymphoma (LBL)

Jazz Pharmaceuticals

Status and phase: Completed; Phase 3; Phase 2

Conditions: Lymphoblastic Leukemia; Acute Lymphoblastic Leukemia

Treatments: Drug: IM JZP-458; Drug: IV JZP-458

Study type: Interventional

Funder types: Industry; Other

Identifiers

NCT04145531

AALL1931 (Other Identifier)

JZP458-201

Details and patient eligibility

About

This is an open-label, multicenter, dose confirmation, and PK study of JZP-458 in patients (of any age) with ALL/LBL who are hypersensitive to E. coli-derived asparaginases (allergic reaction or silent inactivation). This study is designed to assess the tolerability and efficacy of JZP-458 (only in patients who develop hypersensitivity to an E. coli-derived asparaginase), as measured by asparaginase activity.

Enrollment: 229 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Pediatric and adult patients with a diagnosis of ALL or LBL.
2. Have had an allergic reaction to a long-acting E. coli-derived asparaginase OR have silent inactivation.
3. Have 1 or more courses of E. coli-derived asparaginase remaining in his/her treatment plan.
4. Patients must have, in the opinion of the Investigator, fully recovered from their prior allergic reaction to E. coli-derived asparaginase.

Exclusion criteria

1. Have previously received asparaginase Erwinia chrysanthemi or JZP-458.
2. Have relapsed ALL or LBL.
3. Are concurrently receiving another investigational agent and/or treated with an investigational device at the same time as JZP-458 (within 48 hours) during Course 1 of JZP-458.
4. Have a history of ≥ Grade 3 pancreatitis.
5. Prior history of asparaginase-associated ≥ Grade 3 hemorrhagic event or asparaginase-associated thrombus requiring anticoagulation therapy, excluding catheter-related thrombotic events.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

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229 participants in 1 patient group

JZP-458

Experimental group

Description:

Part A (IM JZP-458) of the study will have 2 IM cohorts: * Cohort 1: a JZP-458 repeat dose/confirmatory cohort; a final IM JZP-458 dose level will be selected, and * Cohort 2: an expansion cohort to confirm the efficacy and safety of the final IM JZP-458 dose level and schedule Part B (IV JZP-458 Dose Confirmation) will be conducted to define the optimal dose of the IV administration of JZP-458 for further study in ALL/LBL patients as a repeated dose. Additional courses of JZP-458 (IM or IV depending on patient's allocation at study enrollment) will be administered based on each patient's original treatment plan for as long as the patient derives clinical benefit.

Treatment:

Drug: IM JZP-458

Drug: IV JZP-458