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Acute Lymphoblastic LeukemiaPhase 2/3NCT04145531

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

A Phase 2/3 study testing IM JZP-458 and IV JZP-458 in people with Acute Lymphoblastic Leukemia and Lymphoblastic Leukemia, run by Jazz Pharmaceuticals at 74 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

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). Read more about this study

Who is it for?
Any age
What is being tested?
IM JZP-458 and IV JZP-458
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
229 participants
Where
74 sitesin 2 countries
Study dates
27 Dec 2019to 13 Jul 2022
Phase
Phase 2/3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

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

Who cannot join

  • Have previously received asparaginase Erwinia chrysanthemi or JZP-458.
  • Have relapsed ALL or LBL.
  • 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.
  • Have a history of ≥ Grade 3 pancreatitis.
  • Prior history of asparaginase-associated ≥ Grade 3 hemorrhagic event or asparaginase-associated thrombus requiring anticoagulation therapy, excluding catheter-related thrombotic events.

Trial locations and contacts

74 sites in 2 countries.

74 sites in 2 countries

United States67 sites

  • Kaiser Permanente - Orange County - Anaheim Medical Center

    Anaheim, California, 92806

  • C.S. Mott Children's Hospital

    Ann Arbor, Michigan, 48109

  • Scottish Rite Hospital

    Atlanta, Georgia, 30342

  • Children's Blood and Cancer Center

    Austin, Texas, 78723

  • Children's of Alabama

    Birmingham, Alabama, 35233

  • Dana-Farber/Boston Children's Cancer and Blood Disorders Center

    Boston, Massachusetts, 02215

  • Ann and Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205

  • Neurology Clinic, P.C

    Cordova, Tennessee, 38018

  • Medical City Dallas Hospital

    Dallas, Texas, 75230

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390

  • Kaiser Permanente - Downey Medical Center

    Downey, California, 90242

  • Inova Medical Group - Fairfax Hospital

    Falls Church, Virginia, 22042

  • Kaiser Permanente - Fontana Medical Center

    Fontana, California, 92335

  • Golisano Children's Hospital of Southwest Florida

    Fort Myers, Florida, 33908

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

  • Connecticut Children's Medical Center

    Hartford, Connecticut, 06106

  • Memorial Medical Office Centre

    Hollywood, Florida, 33021

  • Kapi'olani Medical Center for Women and Children

    Honolulu, Hawaii, 96826

  • Texas Children's Hospital

    Houston, Texas, 77030

  • University of Mississippi Medical Center

    Jackson, Mississippi, 39216

  • Nemours Children's Specialty Care Jacksonville

    Jacksonville, Florida, 32207

  • East Tennessee Children's Hospital

    Knoxville, Tennessee, 37913

  • Alliance for Childhood Diseases

    Las Vegas, Nevada, 89135

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202

  • Loma Linda University Medical Center

    Loma Linda, California, 92350

  • MemorialCare Miller Children's and Women's Hospital Long Beach

    Long Beach, California, 90806

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027

  • Kaiser Permanente- Los Angeles Medical Center

    Los Angeles, California, 90027

  • Norton Children's Hospital

    Louisville, Kentucky, 40202

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105

  • Children's Minnesota

    Minneapolis, Minnesota, 55404

  • Masonic Cancer Center

    Minneapolis, Minnesota, 55455

  • Morristown Medical Center

    Morristown, New Jersey, 07960

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • Rutgers Cancer Institute of New Jersey

    New Brunswick, New Jersey, 08903

  • Smilow Cancer Hospital - New Haven

    New Haven, Connecticut, 06511

  • Cohen Children's Medical Center

    New Hyde Park, New York, 11040

  • NYU - Stephen D. Hassenfeld Children's Center for Cancer and Blood Disorders

    New York, New York, 10016

  • Herbert Irving Comprehensive Cancer Center

    New York, New York, 10032

  • Memorial Sloan-Kettering Cancer Center - New York

    New York, New York, 10065

  • Children's Hospital of The King's Daughters

    Norfolk, Virginia, 23507

  • Benioff Children's Hospital Oakland

    Oakland, California, 94609

  • Kaiser Permanente - Oakland Medical Center

    Oakland, California, 94611

  • The Children's Hospital at Oklahoma University Medical Center

    Oklahoma City, Oklahoma, 73104

  • Children's Hospital of Orange County Main Campus - Orange

    Orange, California, 92868

  • AdventHealth Orlando

    Orlando, Florida, 32803

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016

  • University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224

  • Oregon Health and Science University

    Portland, Oregon, 97201

  • Massey Cancer Center

    Richmond, Virginia, 23220

  • Kaiser Permanente - Roseville Medical Center

    Roseville, California, 95661

  • University of California Davis Comprehensive Cancer Center

    Sacramento, California, 95817

  • Primary Children's Hospital

    Salt Lake City, Utah, 84113

  • Methodist Hospital - San Antonio

    San Antonio, Texas, 78229

  • Kaiser Permanente - San Diego Medical Center

    San Diego, California, 92120

  • University of California San Francisco Benioff Children's Hospital - Mission Bay

    San Francisco, California, 94158

  • Kaiser Permanente - Santa Clara Medical Center

    Santa Clara, California, 95051

  • Seattle Children's Hospital

    Seattle, Washington, 98105

  • Providence Sacred Heart Medical Center

    Spokane, Washington, 99204

  • Washington University School of Medicine in Saint Louis

    St Louis, Missouri, 63110

  • Johns Hopkins All Children's Hospital

    St. Petersburg, Florida, 33701

  • Children's National Health System

    Washington D.C., District of Columbia, 20010

  • Nemours Alfred I. Dupont Hospital for Children

    Wilmington, Delaware, 19803

  • Wake Forest Baptist Health

    Winston-Salem, North Carolina, 27157

Other trials in United States

Canada7 sites

  • McMaster Children's Hospital

    Hamilton, Ontario, L8N 3Z5

  • Victoria Hospital & Children's Hospital

    London, Ontario, N6C 2V5

  • Centre Hospitalier Universitaire Sainte-Justine

    Montreal, Quebec, H3T 1C5

  • The Montreal Children's Hospital

    Montreal, Quebec, H4A 3J1

  • Children's Hospital of Eastern Ontario

    Ottawa, Ontario, K1H 8L1

  • SickKids - The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8

  • CancerCare Manitoba - McDermot and Urgent Care Site

    Winnipeg, Manitoba, R3E 0V9

Other trials in Canada

About this study

Official title: An Open-Label, Multicenter Study of Recombinant Crisantaspase Produced in Pseudomonas Fluorescens (RC-P) in Patients With Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic Lymphoma (LBL) Following Hypersensitivity to E. Coli-derived Asparaginases

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.

Being tested
IM JZP-458, IV JZP-458
Keywords
ALL, LBL, Asparaginase, Leukemia, Childhood acute lymphoblastic leukemia
Medical subject headings
Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • IM JZP-458Drug

    IM JZP-458 will be administered in Part A, Cohorts 1 \& 2

  • IV JZP-458Drug

    IV JZP-458 will be administered in Part B

Study groups in NCT04145531
Study groupRoleWhat they receive
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.
Receives: Drug: IM JZP-458; Drug: IV JZP-458

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Response Rate During the First Course of JZP-458 Administration

    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.

    Measured: Baseline up to 2 weeks

  2. Number of Participants With Any Treatment-emergent Adverse Events (TEAEs)

    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.

    Measured: Date of written informed consent up to 30 days after last dose of last course, up to approximately 2 years 7 months

Secondary outcomes (4)

  1. Number of Participants With Last 48-hour NSAA Level ≥ 0.1 IU/mL During The First Course (6 Doses) of JZP-458 Administration

    Blood samples were collected for serum asparaginase activity level determination.

    Measured: Baseline up to 2 weeks

  2. Number of Participants With Last NSAA Levels ≥ 0.4 IU/mL During The First Course (6 Doses) of JZP-458 Administration

    Blood samples were collected for serum asparaginase activity level determination.

    Measured: Baseline up to 2 weeks

  3. Mean Serum Asparaginase Activity Levels in First Course of JZP-458 Administration

    Serum asparaginase levels serve as a surrogate marker for asparagine depletion. Mean serum asparaginase activity levels in Course 1 are reported.

    Measured: Up to 2 weeks (6 doses)

  4. Number of Participants Who Are Anti-drug Antibody Positive or Negative Against JZP-458

    Blood samples were collected for immunogenicity analysis. Anti-drug antibody positive (ADA+) participants were those with a positive result on the first test and also a positive result on the confirmatory test. Anti-drug antibody negative (ADA-) participants had a negative result on the first test.

    Measured: Baseline up to 30 days (ADA- samples) after last dose of last course and up to 6 months (ADA+ samples) after last dose of last course, up to approximately 2 years 7 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Oct 2019
  2. Study start27 Dec 2019
  3. Primary completion13 Jul 2022
  4. Study completion13 Jul 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Masking
None (open label)
Enrolment
229 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04145531
Sponsor's protocol ID
JZP458-201
Other IDs
AALL1931
Status last verified
Nov 2023
First submitted
28 Oct 2019
Last updated
18 Nov 2023
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Maese L, Loh ML, Choi MR, Lin T, Aoki E, Zanette M, Agarwal S, Iannone R, Silverman JA, Silverman LB, Raetz EA, Rau RE. Recombinant Erwinia asparaginase (JZP458) in acute lymphoblastic leukemia: results from the phase 2/3 AALL1931 study. Blood. 2023 Feb 16;141(7):704-712. doi: 10.1182/blood.2022016923.

    PubMed 36108304

  • Maese L, Loh ML, Choi MR, Agarwal S, Aoki E, Liang Y, Lin T, Girgis S, Chen C, Roller S, Chandrasekaran V, Iannone R, Silverman LB, Raetz EA, Rau RE. Recombinant Erwinia asparaginase (JZP458) in ALL/LBL: complete follow-up of the Children's Oncology Group AALL1931 study. Blood Adv. 2025 Jan 14;9(1):66-77. doi: 10.1182/bloodadvances.2024013346.

    PubMed 39454281

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing IM JZP-458 and IV JZP-458 in people with Acute Lymphoblastic Leukemia and Lymphoblastic Leukemia, run by Jazz Pharmaceuticals at 74 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 18 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 74 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 18 Nov 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Response Rate During the First Course of JZP-458 Administration (time frame: Baseline up to 2 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04145531, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Nov 2023.

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