A Phase II, Open-Label Study of Clofarabine in Paediatric Patients With Refractory/Relapsed Acute Lymphoblastic Leukaemia

Sponsor

Genzyme, a Sanofi Company (Industry)

Overall Status

Completed

CT.gov ID

NCT00930098

Collaborator

Bioenvision (Industry)

Enrollment

Locations

Duration (Months)

2.1

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Acute Lymphoblastic Leukemia	Drug: clofarabine	Phase 2

Detailed Description

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

Study Design

Study Type:

Interventional

Actual Enrollment :

74 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Phase II, Open-Label Study of Clofarabine in Paediatric Patients With Refractory/Relapsed Acute Lymphoblastic Leukaemia

Study Start Date :

Dec 1, 2003

Actual Study Completion Date :

Jul 1, 2007

Outcome Measures

Primary Outcome Measures

Overall response rate after 1 course or more [minimum of 1 course and maximum of 12 courses]

Secondary Outcome Measures

Overall response rate after 2 courses or more [minimum of 2 courses and maximum of 12 courses]
Rate of response (complete, complete with incomplete blood count recovery, partial) after 1 course or more [minimum of 1 course and maximum of 12 courses]
Time-to-event parameters including duration of remission and overall survival [Until death of patient or until end of study treatment for last enrolled patient]
Safety and tolerability [Until death of patient or until end of study treatment for last enrolled patient]
Number of patients received bone marrow or peripheral blood stem cell transplantation and the time to transplant following commencement of Clofarabine [Until death of patient or until end of study treatment for last enrolled patient]
Determine pharmacokinetic profile and intracellular triphosphate and levels of clofarabine [every day during the first course of treatment]
Document the metabolic pathways that may predict Clofarabine sensitivity or resistance [day 1, day 5, day 6 and day 7 of the first course]

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 21 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	City	Country
1	Vienna	Austria
2	Besancon	France
3	Bordeaux	France
4	Lille	France
5	Lyon	France
6	Marseille	France
7	Nantes	France
8	Paris	France
9	Toulouse	France
10	Vandoeuvre-les-Nancy	France
11	Berlin	Germany
12	Dusseldorf	Germany
13	Frankfurt	Germany
14	Hamburg	Germany
15	Hannover	Germany
16	Kiel	Germany
17	Muenster	Germany
18	Munich	Germany
19	Stuttgart	Germany
20	Monza	Italy
21	Pavia	Italy
22	Amsterdam	Netherlands
23	Groningen	Netherlands
24	Leiden	Netherlands
25	Rotterdam	Netherlands
26	Utrecht	Netherlands
27	Birmingham	United Kingdom
28	Bristol	United Kingdom
29	Glasgow	United Kingdom
30	Leeds	United Kingdom
31	Liverpool	United Kingdom
32	London	United Kingdom
33	Manchester	United Kingdom
34	Newcastle upon Tyne	United Kingdom
35	Sheffield	United Kingdom
36	Sutton	United Kingdom