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JZP-458

Phase 2

Acute Lymphoblastic Leukemia | Small molecule | Oncology |Jazz Pharmaceuticals plc|Last Updated: Nov 18, 2023

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment229

FDA Designations

No designations recorded

Clinical trial landscape

JZP-458 · 1 trial · 2 indications

Phase 2 1
NCT04145531An Open-Label Study of JZP-458 (RC-P) in Patients With Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic Lymphoma (LBL)Acute Lymphoblastic Leukemia
COMPLETED229 Analytics
PHASE2COMPLETED
An Open-Label Study of JZP-458 (RC-P) in Patients With Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic Lymphoma (LBL)
Acute Lymphoblastic LeukemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Response Rate During the First Course of JZP-458 Administration
Baseline up to 2 weeks

The response rate was defined as the number (proportion) of patients with the last 72-hour nadir serum asparaginase activity (NSAA) level ≥ 0.1 IU/mL during the first course of IM JZP-458. Blood samples were collected for serum asparaginase activity level determination.

Number of Participants With Any Treatment-emergent Adverse Events (TEAEs)
Date of written informed consent up to 30 days after last dose of last course, up to approximately 2 years 7 months

An adverse event (AE) is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered related to study drug. AEs were classified by the Investigator using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.

Secondary Endpoints

Number of Participants With Last 48-hour NSAA Level ≥ 0.1 IU/mL During The First Course (6 Doses) of JZP-458 Administration
Baseline up to 2 weeks
Number of Participants With Last NSAA Levels ≥ 0.4 IU/mL During The First Course (6 Doses) of JZP-458 Administration
Baseline up to 2 weeks
Mean Serum Asparaginase Activity Levels in First Course of JZP-458 Administration
Up to 2 weeks (6 doses)
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
JZP-458EXPERIMENTALPart 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.

Interventions

NameTypeDescription
IM JZP-458DRUGIM JZP-458 will be administered in Part A, Cohorts 1 \& 2
IV JZP-458DRUGIV JZP-458 will be administered in Part B
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Eligibility Criteria

SexALL
Healthy VolunteersNo
Study Sites74

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 ...

Countries:United StatesCanada
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Frequently asked questions about JZP-458

What is JZP-458 used for?

JZP-458 is an investigational small molecule being studied for the treatment of Acute Lymphoblastic Leukemia (ALL) and lymphoblastic lymphoma. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.

Who makes JZP-458?

JZP-458 is being developed by Jazz Pharmaceuticals plc, a biopharmaceutical company traded on the NASDAQ under the ticker symbol JAZZ. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Acute Lymphoblastic Leukemia.

What phase is JZP-458 in?

JZP-458 is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA or other regulatory agencies. The drug is being studied for the treatment of Acute Lymphoblastic Leukemia and lymphoblastic lymphoma.

What clinical trials is JZP-458 in?

JZP-458 has one completed clinical trial, NCT04145531, an open-label Phase 2 study in patients with Acute Lymphoblastic Leukemia and lymphoblastic lymphoma. The trial enrolled 229 participants in the United States and Canada and was not randomized or double-blinded.

Is JZP-458 the same as RC-P?

Yes, JZP-458 is also known as RC-P. The clinical trial NCT04145531 is titled 'An Open-Label Study of JZP-458 (RC-P) in Patients With Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic Lymphoma (LBL)', confirming that both names refer to the same investigational drug.