Clinical trial NCT00930098: A Phase II, Open-Label Study of Clofarabine in Paediatric Patients With Refractory/Relapsed Acute Lymphoblastic Leukaemia
Official title: A Phase II, Open-Label Study of Clofarabine in Paediatric Patients With Refractory/Relapsed Acute Lymphoblastic Leukaemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Acute Lymphoblastic Leukemia
Keywords: acute lymphoblastic leukemia
clolar
evoltra
clofarabine
refractory/relapsed acute leukemia
pediatric acute leukemia
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
Collaborators: name: Bioenvision
First posted: 2009-06-30
Enrollment: 74
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study is for a population of patients with few or no alternative options that was conducted to determine the response rate to clofarabine. Additionally the study will provide information on the safety profile, impact of overall survival, and impact on remission duration with clofarabine. It is a single arm study and has no comparator.

Detailed description: Note: This clinical trial was conducted by Bioenvision Ltd. Bioenvision Ltd. was acquired by Genzyme Corporation Oct 2007.

Eligibility criteria: Inclusion Criteria:

* Have a diagnosis of ALL and confirmed by pathologic assessment.
* Be ≤ 21 years old at time of initial diagnosis.
* Eligible patients must have: Primary refractory disease; OR relapsed or refractory disease after a minimum of 2 prior blocks of treatment.
* Must not be eligible for therapy of higher curative potential.
* Have a Karnofsky Performance Status of ≥ 50 or Lansky Performance Scale of ≥ 30.
* Provide a signed, written informed consent from parent or guardian or young adult patients.
* Be able to comply with study procedures and follow-up examinations.
* Have adequate cardiac function without treatment.
* Have adequate organ function as indicated by the laboratory values for serum creatinine, serum bilirubin, AST and ALT, obtained within 7 days prior to registration.

Exclusion Criteria:

* Received previous treatment with clofarabine.
* Patients with isolated extramedullary disease.
* Have received prior BMT or PBSCT within the last 6 months.
* Have received prior BMT or PBSCT more than 6 months ago, but now has compromised organ function.
* Have an active, uncontrolled systemic infection.
* Are pregnant or lactating. Male and female patients who are fertile must agree to use an effective means of birth control to avoid pregnancy.
* Have a psychiatric disorder that would interfere with consent, study participation, or follow-up.
* Have received any other chemotherapy within the previous 2 weeks and must have recovered from acute toxicity oa all previous therapy prior to enrollment.
* Have any other severe concurrent disease.
* Have recent history of significant renal, hepatic or pulmonary dysfunction, or cardiac dysfunction or on treatment to support cardiac function.
* Have CNS disease.

Primary outcomes:
measure: Overall response rate after 1 course or more
timeFrame: minimum of 1 course and maximum of 12 courses
Secondary outcomes:
measure: Overall response rate after 2 courses or more
timeFrame: minimum of 2 courses and maximum of 12 courses
measure: Rate of response (complete, complete with incomplete blood count recovery, partial) after 1 course or more
timeFrame: minimum of 1 course and maximum of 12 courses
measure: Time-to-event parameters including duration of remission and overall survival
timeFrame: Until death of patient or until end of study treatment for last enrolled patient
measure: Safety and tolerability
timeFrame: Until death of patient or until end of study treatment for last enrolled patient
measure: Number of patients received bone marrow or peripheral blood stem cell transplantation and the time to transplant following commencement of Clofarabine
timeFrame: Until death of patient or until end of study treatment for last enrolled patient
measure: Determine pharmacokinetic profile and intracellular triphosphate and levels of clofarabine
timeFrame: every day during the first course of treatment
measure: Document the metabolic pathways that may predict Clofarabine sensitivity or resistance
timeFrame: day 1, day 5, day 6 and day 7 of the first course
Other outcomes:
Locations:
city: Vienna
country: Austria
city: Besancon
country: France
city: Bordeaux
country: France
city: Lille
country: France
city: Lyon
country: France
city: Marseille
country: France
city: Nantes
country: France
city: Paris
country: France
city: Toulouse
country: France
city: Vandoeuvre-les-Nancy
country: France
city: Berlin
country: Germany
city: Dusseldorf
country: Germany
city: Frankfurt
country: Germany
city: Hamburg
country: Germany
city: Hannover
country: Germany
city: Kiel
country: Germany
city: Muenster
country: Germany
city: Munich
country: Germany
city: Stuttgart
country: Germany
city: Monza
country: Italy
city: Pavia
country: Italy
city: Amsterdam
country: Netherlands
city: Groningen
country: Netherlands
city: Leiden
country: Netherlands
city: Rotterdam
country: Netherlands
city: Utrecht
country: Netherlands
city: Birmingham
country: United Kingdom
city: Bristol
country: United Kingdom
city: Glasgow
country: United Kingdom
city: Leeds
country: United Kingdom
city: Liverpool
country: United Kingdom
city: London
country: United Kingdom
city: Manchester
country: United Kingdom
city: Newcastle upon Tyne
country: United Kingdom
city: Sheffield
country: United Kingdom
city: Sutton
country: United Kingdom
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Medical Monitor
role: STUDY_DIRECTOR
References: