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

Primary Purpose

Acute Lymphoblastic Leukemia, Lymphoblastic Leukemia

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
IM JZP-458
IV JZP-458
Sponsored by
Jazz Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Acute Lymphoblastic Leukemia focused on measuring ALL, LBL, Asparaginase, Leukemia, Childhood acute lymphoblastic leukemia

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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.

Sites / Locations

  • Children's of Alabama
  • Phoenix Children's Hospital
  • Arkansas Children's Hospital
  • Kaiser Permanente - Orange County - Anaheim Medical Center
  • Kaiser Permanente - Downey Medical Center
  • Kaiser Permanente - Fontana Medical Center
  • Loma Linda University Medical Center
  • MemorialCare Miller Children's and Women's Hospital Long Beach
  • Children's Hospital Los Angeles
  • Kaiser Permanente- Los Angeles Medical Center
  • Benioff Children's Hospital Oakland
  • Kaiser Permanente - Oakland Medical Center
  • Children's Hospital of Orange County Main Campus - Orange
  • Kaiser Permanente - Roseville Medical Center
  • University of California Davis Comprehensive Cancer Center
  • Kaiser Permanente - San Diego Medical Center
  • University of California San Francisco Benioff Children's Hospital - Mission Bay
  • Kaiser Permanente - Santa Clara Medical Center
  • Connecticut Children's Medical Center
  • Smilow Cancer Hospital - New Haven
  • Nemours Alfred I. Dupont Hospital for Children
  • Children's National Health System
  • Golisano Children's Hospital of Southwest Florida
  • Memorial Medical Office Centre
  • Nemours Children's Specialty Care Jacksonville
  • AdventHealth Orlando
  • Johns Hopkins All Children's Hospital
  • Scottish Rite Hospital
  • Kapi'olani Medical Center for Women and Children
  • Ann and Robert H. Lurie Children's Hospital of Chicago
  • Norton Children's Hospital
  • Dana-Farber/Boston Children's Cancer and Blood Disorders Center
  • C.S. Mott Children's Hospital
  • Children's Minnesota
  • Masonic Cancer Center
  • University of Mississippi Medical Center
  • Washington University School of Medicine in Saint Louis
  • Alliance for Childhood Diseases
  • Hackensack University Medical Center
  • Morristown Medical Center
  • Rutgers Cancer Institute of New Jersey
  • Cohen Children's Medical Center
  • NYU - Stephen D. Hassenfeld Children's Center for Cancer and Blood Disorders
  • Herbert Irving Comprehensive Cancer Center
  • Memorial Sloan-Kettering Cancer Center - New York
  • Wake Forest Baptist Health
  • Cincinnati Children's Hospital Medical Center
  • Nationwide Children's Hospital
  • The Children's Hospital at Oklahoma University Medical Center
  • Oregon Health and Science University
  • Children's Hospital of Philadelphia
  • University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
  • Neurology Clinic, P.C
  • East Tennessee Children's Hospital
  • St. Jude Children's Research Hospital
  • Vanderbilt University Medical Center
  • Children's Blood and Cancer Center
  • Medical City Dallas Hospital
  • University of Texas Southwestern Medical Center
  • Texas Children's Hospital
  • Methodist Hospital - San Antonio
  • Primary Children's Hospital
  • Inova Medical Group - Fairfax Hospital
  • Children's Hospital of The King's Daughters
  • Massey Cancer Center
  • Seattle Children's Hospital
  • Providence Sacred Heart Medical Center
  • CancerCare Manitoba - McDermot and Urgent Care Site
  • McMaster Children's Hospital
  • Victoria Hospital & Children's Hospital
  • Children's Hospital of Eastern Ontario
  • SickKids - The Hospital for Sick Children
  • Centre Hospitalier Universitaire Sainte-Justine
  • The Montreal Children's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

JZP-458

Arm 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.

Outcomes

Primary Outcome Measures

Response Rate During the First Course of IM JZP-458
The response rate is defined as the proportion of patients with the last 72-hour NSAA level ≥ 0.1 IU/mL during the first course of IM JZP-458.
Occurrence of treatment-emergent adverse events (TEAEs)

Secondary Outcome Measures

Proportion of patients with the last 48-hour NSAA level ≥ 0.1 IU/mL during the first course of IM administration of JZP-458
For patients in Part A
Proportion of patients with the last 48-hour NSAA level ≥ 0.4 IU/mL during the first course of IM administration of JZP-458
For patients in Part A
Proportion of patients with the last 72-hour NSAA level ≥ 0.4 IU/mL during the first course of IM administration of JZP-458
For patients in Part A
JZP-458 Pharmacokinetics
For patients in Part A. Serum asparaginase activity levels after Course 1 (6 doses), then after each subsequent Courses during a patient's treatment plan.
Incidence of anti-drug antibody formation against JZP-458
For patients in Part A

Full Information

First Posted
October 28, 2019
Last Updated
August 3, 2022
Sponsor
Jazz Pharmaceuticals
Collaborators
Children's Oncology Group (COG)
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1. Study Identification

Unique Protocol Identification Number
NCT04145531
Brief Title
An Open-Label Study of JZP-458 (RC-P) in Patients With Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic Lymphoma (LBL)
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
Study Type
Interventional

2. Study Status

Record Verification Date
August 2022
Overall Recruitment Status
Completed
Study Start Date
December 27, 2019 (Actual)
Primary Completion Date
July 13, 2022 (Actual)
Study Completion Date
July 13, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Jazz Pharmaceuticals
Collaborators
Children's Oncology Group (COG)

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

Brief 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). 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Lymphoblastic Leukemia, Lymphoblastic Leukemia
Keywords
ALL, LBL, Asparaginase, Leukemia, Childhood acute lymphoblastic leukemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
229 (Actual)

8. Arms, Groups, and Interventions

Arm Title
JZP-458
Arm Type
Experimental
Arm 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.
Intervention Type
Drug
Intervention Name(s)
IM JZP-458
Intervention Description
IM JZP-458 will be administered in Part A, Cohorts 1 & 2
Intervention Type
Drug
Intervention Name(s)
IV JZP-458
Intervention Description
IV JZP-458 will be administered in Part B
Primary Outcome Measure Information:
Title
Response Rate During the First Course of IM JZP-458
Description
The response rate is defined as the proportion of patients with the last 72-hour NSAA level ≥ 0.1 IU/mL during the first course of IM JZP-458.
Time Frame
2 weeks
Title
Occurrence of treatment-emergent adverse events (TEAEs)
Time Frame
Up to 30 days after last dose
Secondary Outcome Measure Information:
Title
Proportion of patients with the last 48-hour NSAA level ≥ 0.1 IU/mL during the first course of IM administration of JZP-458
Description
For patients in Part A
Time Frame
2 weeks
Title
Proportion of patients with the last 48-hour NSAA level ≥ 0.4 IU/mL during the first course of IM administration of JZP-458
Description
For patients in Part A
Time Frame
2 weeks
Title
Proportion of patients with the last 72-hour NSAA level ≥ 0.4 IU/mL during the first course of IM administration of JZP-458
Description
For patients in Part A
Time Frame
2 weeks
Title
JZP-458 Pharmacokinetics
Description
For patients in Part A. Serum asparaginase activity levels after Course 1 (6 doses), then after each subsequent Courses during a patient's treatment plan.
Time Frame
Up to 30 days after last dose
Title
Incidence of anti-drug antibody formation against JZP-458
Description
For patients in Part A
Time Frame
Up to 30 days after last dose

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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.
Facility Information:
Facility Name
Children's of Alabama
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Phoenix Children's Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
Kaiser Permanente - Orange County - Anaheim Medical Center
City
Anaheim
State/Province
California
ZIP/Postal Code
92806
Country
United States
Facility Name
Kaiser Permanente - Downey Medical Center
City
Downey
State/Province
California
ZIP/Postal Code
90242
Country
United States
Facility Name
Kaiser Permanente - Fontana Medical Center
City
Fontana
State/Province
California
ZIP/Postal Code
92335
Country
United States
Facility Name
Loma Linda University Medical Center
City
Loma Linda
State/Province
California
ZIP/Postal Code
92350
Country
United States
Facility Name
MemorialCare Miller Children's and Women's Hospital Long Beach
City
Long Beach
State/Province
California
ZIP/Postal Code
90806
Country
United States
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Kaiser Permanente- Los Angeles Medical Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Benioff Children's Hospital Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
Kaiser Permanente - Oakland Medical Center
City
Oakland
State/Province
California
ZIP/Postal Code
94611
Country
United States
Facility Name
Children's Hospital of Orange County Main Campus - Orange
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Kaiser Permanente - Roseville Medical Center
City
Roseville
State/Province
California
ZIP/Postal Code
95661
Country
United States
Facility Name
University of California Davis Comprehensive Cancer Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Kaiser Permanente - San Diego Medical Center
City
San Diego
State/Province
California
ZIP/Postal Code
92120
Country
United States
Facility Name
University of California San Francisco Benioff Children's Hospital - Mission Bay
City
San Francisco
State/Province
California
ZIP/Postal Code
94158
Country
United States
Facility Name
Kaiser Permanente - Santa Clara Medical Center
City
Santa Clara
State/Province
California
ZIP/Postal Code
95051
Country
United States
Facility Name
Connecticut Children's Medical Center
City
Hartford
State/Province
Connecticut
ZIP/Postal Code
06106
Country
United States
Facility Name
Smilow Cancer Hospital - New Haven
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06511
Country
United States
Facility Name
Nemours Alfred I. Dupont Hospital for Children
City
Wilmington
State/Province
Delaware
ZIP/Postal Code
19803
Country
United States
Facility Name
Children's National Health System
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Golisano Children's Hospital of Southwest Florida
City
Fort Myers
State/Province
Florida
ZIP/Postal Code
33908
Country
United States
Facility Name
Memorial Medical Office Centre
City
Hollywood
State/Province
Florida
ZIP/Postal Code
33021
Country
United States
Facility Name
Nemours Children's Specialty Care Jacksonville
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32207
Country
United States
Facility Name
AdventHealth Orlando
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Facility Name
Johns Hopkins All Children's Hospital
City
Saint Petersburg
State/Province
Florida
ZIP/Postal Code
33701
Country
United States
Facility Name
Scottish Rite Hospital
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Facility Name
Kapi'olani Medical Center for Women and Children
City
Honolulu
State/Province
Hawaii
ZIP/Postal Code
96826
Country
United States
Facility Name
Ann and Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Norton Children's Hospital
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Facility Name
Dana-Farber/Boston Children's Cancer and Blood Disorders Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
C.S. Mott Children's Hospital
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Children's Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55404
Country
United States
Facility Name
Masonic Cancer Center
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
University of Mississippi Medical Center
City
Jackson
State/Province
Mississippi
ZIP/Postal Code
39216
Country
United States
Facility Name
Washington University School of Medicine in Saint Louis
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Alliance for Childhood Diseases
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89135
Country
United States
Facility Name
Hackensack University Medical Center
City
Hackensack
State/Province
New Jersey
ZIP/Postal Code
07601
Country
United States
Facility Name
Morristown Medical Center
City
Morristown
State/Province
New Jersey
ZIP/Postal Code
07960
Country
United States
Facility Name
Rutgers Cancer Institute of New Jersey
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08903
Country
United States
Facility Name
Cohen Children's Medical Center
City
New Hyde Park
State/Province
New York
ZIP/Postal Code
11040
Country
United States
Facility Name
NYU - Stephen D. Hassenfeld Children's Center for Cancer and Blood Disorders
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Herbert Irving Comprehensive Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Memorial Sloan-Kettering Cancer Center - New York
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
Wake Forest Baptist Health
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
The Children's Hospital at Oklahoma University Medical Center
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Facility Name
Oregon Health and Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97201
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Neurology Clinic, P.C
City
Cordova
State/Province
Tennessee
ZIP/Postal Code
38018
Country
United States
Facility Name
East Tennessee Children's Hospital
City
Knoxville
State/Province
Tennessee
ZIP/Postal Code
37913
Country
United States
Facility Name
St. Jude Children's Research Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38105
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Children's Blood and Cancer Center
City
Austin
State/Province
Texas
ZIP/Postal Code
78723
Country
United States
Facility Name
Medical City Dallas Hospital
City
Dallas
State/Province
Texas
ZIP/Postal Code
75230
Country
United States
Facility Name
University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Methodist Hospital - San Antonio
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
Facility Name
Primary Children's Hospital
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84113
Country
United States
Facility Name
Inova Medical Group - Fairfax Hospital
City
Falls Church
State/Province
Virginia
ZIP/Postal Code
22042
Country
United States
Facility Name
Children's Hospital of The King's Daughters
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23507
Country
United States
Facility Name
Massey Cancer Center
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23220
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
Providence Sacred Heart Medical Center
City
Spokane
State/Province
Washington
ZIP/Postal Code
99204
Country
United States
Facility Name
CancerCare Manitoba - McDermot and Urgent Care Site
City
Winnipeg
State/Province
Manitoba
ZIP/Postal Code
R3E 0V9
Country
Canada
Facility Name
McMaster Children's Hospital
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8N 3Z5
Country
Canada
Facility Name
Victoria Hospital & Children's Hospital
City
London
State/Province
Ontario
ZIP/Postal Code
N6C 2V5
Country
Canada
Facility Name
Children's Hospital of Eastern Ontario
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 8L1
Country
Canada
Facility Name
SickKids - The Hospital for Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Facility Name
Centre Hospitalier Universitaire Sainte-Justine
City
Montréal
State/Province
Quebec
ZIP/Postal Code
H3T 1C5
Country
Canada
Facility Name
The Montreal Children's Hospital
City
Montréal
State/Province
Quebec
ZIP/Postal Code
H4A 3J1
Country
Canada

12. IPD Sharing Statement

Learn more about this trial

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

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