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GRASPA (Erythrocytes Encapsulating L-asparaginase) in Patients With Relapse of Acute Lymphoblastic Leukemia

Phase 2
Completed
Conditions
Acute Lymphoblastic Leukemia, in Relapse
Interventions
Registration Number
NCT01518517
Lead Sponsor
ERYtech Pharma
Brief Summary

Asparaginase is a cornerstone in the treatment of ALL, but its utility is limited by toxicities including hypersensitivity. Clinical allergy is associated with inactivation of asparaginase by antibodies (A-Abs), which can also neutralize asparaginase without any clinical signs of hypersensitivity (silent inactivation). GRASPA improves pharmacokinetics, tolerability and maintain circulating asparaginase activity due to the protective barrier of the erythrocyte membrane.

This study is run to confirm the benefit/risk profile of GRASPA at 150 IU/kg in combination with the COOPRALL regimen in adults and children patients with relapsed ALL, with or without known hypersensitivity to L-asparaginase.

Detailed Description

This open, randomized international Phase 2/3 study will enrol patients with relapsed ALL. The co-primary endpoints were the duration of asparagine depletion \< 2µmol/L and the incidence of asparaginase hypersensitivity during induction. Key secondary endpoints are complete remission (CR), minimal residual disease (MRD), event free survival (EFS) and overall survival (OS).The study was powered to detect 3-fold difference in the incidence of allergic reactions between treatments. patients will be randomized to GRASPA or to Reference L-asparaginase. Patients with history of hypersensitivity to previous L-asparaginase treatment will be treated with GRASPA (exploratory arm)

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
85
Inclusion Criteria
  • Patient from 1 to 55 years old (Children and adolescents from 1 to 17 years/ Adults from 18 to 55 years)
  • Patients with 1st ALL relapse, which could be either isolated bone marrow relapse, or combined (medullary and extra-medullary) relapse, or extra-medullary isolated relapse; or lymphoblastic lymphoma (excepted Burkitt lymphoma) OR Failure to ALL first line treatment (no complete remission obtained)
  • Patient previously treated with free E.Coli L-asparaginase form or pegylated one
  • Performance Status ≤ 2 (WHO score)
  • Patient informed and consent provided (the 2 parents need to consent when children are below 18)
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Exclusion Criteria
  • ALL t(9;22) and/or BCR-ABL positive (Philadelphia chromosome positive)
  • Patient with 2nd relapse and over
  • Women of childbearing potential without effective contraception as well as pregnant or breast feeding women
  • Patient unable to receive treatments used in global chemotherapy protocols, due to general or visceral conditions such as:Severe cardiac impairment (NYHA grade 3 or 4 cardiomyopathy)/Serum creatinine 2 x ULN unless related to ALL /ALT or AST 5 x ULN unless related to ALL /Pancreatitis history /Other malignancy that ALL / Severe Infection, HIV positive, active hepatitis related to B or C virus infection / Trisomy 21 / Other serious conditions according to investigator's opinion
  • Known grade 4 allergic reaction to E.Coli L-asparaginase (according NCI-CTCAE, Version 3.0)
  • History of grade 3 transfusional incident
  • Presence of specific anti-erythrocyte antibodies preventing from getting a compatible erythrocyte concentrate for the patient
  • Patient under concomitant treatment likely to cause hemolysis
  • Patient undergoing yellow fever vaccination
  • Patient under phenytoin treatment
  • Patient included in previous clinical study less than 6 weeks ago
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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
GRASPAGRASPAEach patient randomized in GRASPA® group is to receive at least 2 and up to 10 administration of GRASPA® 150 IU/kg, in combination with standard chemotherapy (COOPRALL). GRASPA® administration takes place as below: * for induction phase: at Day 4 and D18 (F1-F2 induction ) or at D6 if Vanda induction applies (according disease severity) * for consolidation phase: at Day 6 of R2 / R1 blocks, each time block of chemotherapy is given (up to 8 cycles)
reference L-asparaginaseL-asparaginaseFor patient randomized in control group, reference L-asparaginase 10,000 IU/m² will be administered every 3 days intravenously, in combination with standard chemotherapy (COOPRALL). •for induction phase:at Day 4 , D7, D10, D13 (F1 block ) then at Day 18, D21, D24, D27 (of F2 Blocks). NB: administrations take place at D6, D9, D12 and D15 in case of F1-F2 Induction is replaced by VANDA (according disease severity) •for consolidation phase: at D6, D9, D12 ofR2/R1 blocks, each time block of chemotherapy is given (up to 8 cycles).
Primary Outcome Measures
NameTimeMethod
Duration of Asparaginase Activity >100 U/L During InductionInduction treatment period (i.e. 28 days)

Co-primary efficacy endpoint: duration in days of asparaginase activity \>100 U/L in whole blood during the induction treatment phase: last available date/time of activity \>100 UI/L before activity drops below 100 U/L - date/time of first activity \>100 UI/L. Asparaginase activity is compared for GRASPA versus native ASNase to demonstrate the non-inferiority of GRASPA.

Allergic Reaction During Induction PhaseInduction treatment period (i.e. 28 days)

Co-primary safety endpoint: allergic reaction regardless of grade during induction phase. Only those reactions that were reported in relation to the treatment to which the patient was randomised were counted.

Secondary Outcome Measures
NameTimeMethod
Complete Remission (CR)Induction treatment period (i.e. 28 days)

CR is defined as, no physical evidence of leukemia, normal CBC, cytologic remission: normally regenerating bone marrow, with \<5% leukemic blasts and the absence of detectable CNS or extramedullary disease, evaluated with physical examination and CSF findings, at the end of induction

Overall Survival (OS)Overall trial period to 36 months

OS is defined as the time from randomisation or date of inclusion (allergic arm) until death due to any cause. Patients who did not die were censored at 36 months of follow-up or the date of the patient's last visit, whichever was earlier.

Event Free SurvivalOverall trial period to 36 months

EFS is defined as the time from randomisation until the first documented sign of disease relapse or death due to any cause. In line with CHMP guidance (CHMP, 2016), patients who did not achieve CR at the end of the induction period were considered to have had an event at time 0. For the patients enrolled in the GRASPA allergic arm, EFS is defined from the date of inclusion in the study.

Patients who achieved CR at the end of induction and who did not have a documented relapse or death due to any cause were censored at 36 months of follow-up or the date of the patient's last visit, whichever was earlier.

Trial Locations

Locations (27)

Hopital Des Enfants Reine Fabiola

🇧🇪

Bruxelles, Belgium

Chr de La Citadelle

🇧🇪

Liege, Belgium

Hopital Saint Jacques

🇫🇷

Besancon, France

Chu D'Angers

🇫🇷

Angers, France

Hopital Pellegrin Enfants

🇫🇷

Bordeaux, France

Chu Estaing

🇫🇷

Clermont Ferrand, France

Hopital Henri Mondor

🇫🇷

Creteil, France

Chu Grenoble

🇫🇷

Grenoble, France

Chru Lille - Hop Jeanne de Flandres

🇫🇷

Lille, France

Hopital Mere Enfant

🇫🇷

Nantes, France

Institut Hematologie Oncologie Pediatrique

🇫🇷

Lyon, France

Hotel Dieu

🇫🇷

Valenciennes, France

Hopital de L'Archet 2

🇫🇷

Nice, France

Hopital Armand Trousseau

🇫🇷

Paris, France

Institut Paoli Calmettes

🇫🇷

Marseille, France

Hopital Robert Debre

🇫🇷

Paris, France

Hopital Saint Louis

🇫🇷

Paris, France

Hopital Haut-Leveque

🇫🇷

Pessac, France

Hopital Lyon Sud

🇫🇷

Pierre Benite, France

Centre Henri Becquerel

🇫🇷

Rouen, France

Chru Hopital Sud

🇫🇷

Rennes, France

Chu Hopital Nord

🇫🇷

Saint Etienne, France

Institut Cancerologie de La Loire

🇫🇷

Saint Priest En Jarez, France

Hopital de Hautepierre

🇫🇷

Strasbourg, France

Hopital Brabois Enfants

🇫🇷

Vandoeuvre Les Nancy, France

Chu de Toulouse Enfants

🇫🇷

Toulouse, France

Hopital de Brabois

🇫🇷

Vandoeuvre Les Nancy, France

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