Clinical trial NCT04145531: 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
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Acute Lymphoblastic Leukemia
Lymphoblastic Leukemia
Keywords: ALL
LBL
Asparaginase
Leukemia
Childhood acute lymphoblastic leukemia
Lead sponsor: Jazz Pharmaceuticals
Sponsor class: INDUSTRY
Collaborators: name: Children's Oncology Group
First posted: 2019-10-30
Enrollment: 229
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Response Rate During the First Course of JZP-458 Administration
timeFrame: Baseline up to 2 weeks
measure: Number of Participants With Any Treatment-emergent Adverse Events (TEAEs)
timeFrame: Date of written informed consent up to 30 days after last dose of last course, up to approximately 2 years 7 months
Secondary outcomes:
measure: Number of Participants With Last 48-hour NSAA Level ≥ 0.1 IU/mL During The First Course (6 Doses) of JZP-458 Administration
timeFrame: Baseline up to 2 weeks
measure: Number of Participants With Last NSAA Levels ≥ 0.4 IU/mL During The First Course (6 Doses) of JZP-458 Administration
timeFrame: Baseline up to 2 weeks
measure: Mean Serum Asparaginase Activity Levels in First Course of JZP-458 Administration
timeFrame: Up to 2 weeks (6 doses)
measure: Number of Participants Who Are Anti-drug Antibody Positive or Negative Against JZP-458
timeFrame: 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
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Children's of Alabama
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Anaheim
country: United States
facility: Kaiser Permanente - Orange County - Anaheim Medical Center
state: California
city: Downey
country: United States
facility: Kaiser Permanente - Downey Medical Center
state: California
city: Fontana
country: United States
facility: Kaiser Permanente - Fontana Medical Center
state: California
city: Loma Linda
country: United States
facility: Loma Linda University Medical Center
state: California
city: Long Beach
country: United States
facility: MemorialCare Miller Children's and Women's Hospital Long Beach
state: California
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Los Angeles
country: United States
facility: Kaiser Permanente- Los Angeles Medical Center
state: California
city: Oakland
country: United States
facility: Benioff Children's Hospital Oakland
state: California
city: Oakland
country: United States
facility: Kaiser Permanente - Oakland Medical Center
state: California
city: Orange
country: United States
facility: Children's Hospital of Orange County Main Campus - Orange
state: California
city: Roseville
country: United States
facility: Kaiser Permanente - Roseville Medical Center
state: California
city: Sacramento
country: United States
facility: University of California Davis Comprehensive Cancer Center
state: California
city: San Diego
country: United States
facility: Kaiser Permanente - San Diego Medical Center
state: California
city: San Francisco
country: United States
facility: University of California San Francisco Benioff Children's Hospital - Mission Bay
state: California
city: Santa Clara
country: United States
facility: Kaiser Permanente - Santa Clara Medical Center
state: California
city: Hartford
country: United States
facility: Connecticut Children's Medical Center
state: Connecticut
city: New Haven
country: United States
facility: Smilow Cancer Hospital - New Haven
state: Connecticut
city: Wilmington
country: United States
facility: Nemours Alfred I. Dupont Hospital for Children
state: Delaware
city: Washington
country: United States
facility: Children's National Health System
state: District of Columbia
city: Fort Myers
country: United States
facility: Golisano Children's Hospital of Southwest Florida
state: Florida
city: Hollywood
country: United States
facility: Memorial Medical Office Centre
state: Florida
city: Jacksonville
country: United States
facility: Nemours Children's Specialty Care Jacksonville
state: Florida
city: Orlando
country: United States
facility: AdventHealth Orlando
state: Florida
city: Saint Petersburg
country: United States
facility: Johns Hopkins All Children's Hospital
state: Florida
city: Atlanta
country: United States
facility: Scottish Rite Hospital
state: Georgia
city: Honolulu
country: United States
facility: Kapi'olani Medical Center for Women and Children
state: Hawaii
city: Chicago
country: United States
facility: Ann and Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Louisville
country: United States
facility: Norton Children's Hospital
state: Kentucky
city: Boston
country: United States
facility: Dana-Farber/Boston Children's Cancer and Blood Disorders Center
state: Massachusetts
city: Ann Arbor
country: United States
facility: C.S. Mott Children's Hospital
state: Michigan
city: Minneapolis
country: United States
facility: Children's Minnesota
state: Minnesota
city: Minneapolis
country: United States
facility: Masonic Cancer Center
state: Minnesota
city: Jackson
country: United States
facility: University of Mississippi Medical Center
state: Mississippi
city: Saint Louis
country: United States
facility: Washington University School of Medicine in Saint Louis
state: Missouri
city: Las Vegas
country: United States
facility: Alliance for Childhood Diseases
state: Nevada
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: Morristown
country: United States
facility: Morristown Medical Center
state: New Jersey
city: New Brunswick
country: United States
facility: Rutgers Cancer Institute of New Jersey
state: New Jersey
city: New Hyde Park
country: United States
facility: Cohen Children's Medical Center
state: New York
city: New York
country: United States
facility: NYU - Stephen D. Hassenfeld Children's Center for Cancer and Blood Disorders
state: New York
city: New York
country: United States
facility: Herbert Irving Comprehensive Cancer Center
state: New York
city: New York
country: United States
facility: Memorial Sloan-Kettering Cancer Center - New York
state: New York
city: Winston-Salem
country: United States
facility: Wake Forest Baptist Health
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Oklahoma City
country: United States
facility: The Children's Hospital at Oklahoma University Medical Center
state: Oklahoma
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
state: Pennsylvania
city: Cordova
country: United States
facility: Neurology Clinic, P.C
state: Tennessee
city: Knoxville
country: United States
facility: East Tennessee Children's Hospital
state: Tennessee
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Austin
country: United States
facility: Children's Blood and Cancer Center
state: Texas
city: Dallas
country: United States
facility: Medical City Dallas Hospital
state: Texas
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: San Antonio
country: United States
facility: Methodist Hospital - San Antonio
state: Texas
city: Salt Lake City
country: United States
facility: Primary Children's Hospital
state: Utah
city: Falls Church
country: United States
facility: Inova Medical Group - Fairfax Hospital
state: Virginia
city: Norfolk
country: United States
facility: Children's Hospital of The King's Daughters
state: Virginia
city: Richmond
country: United States
facility: Massey Cancer Center
state: Virginia
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Spokane
country: United States
facility: Providence Sacred Heart Medical Center
state: Washington
city: Winnipeg
country: Canada
facility: CancerCare Manitoba - McDermot and Urgent Care Site
state: Manitoba
city: Hamilton
country: Canada
facility: McMaster Children's Hospital
state: Ontario
city: London
country: Canada
facility: Victoria Hospital & Children's Hospital
state: Ontario
city: Ottawa
country: Canada
facility: Children's Hospital of Eastern Ontario
state: Ontario
city: Toronto
country: Canada
facility: SickKids - The Hospital for Sick Children
state: Ontario
city: Montréal
country: Canada
facility: Centre Hospitalier Universitaire Sainte-Justine
state: Quebec
city: Montréal
country: Canada
facility: The Montreal Children's Hospital
state: Quebec
Study officials:
References:
citation: 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.