A Study for Improving the Outcome of Childhood Acute Promyeloid Leukemia
Conditions
Childhood Acute Promyelocytic Leukemia
Conditions: official terms
Leukemia - Leukemia, Promyelocytic, Acute
Study Type
Interventional
Study Phase
Phase 4
Study Design
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Intervention
Name: ATO Type: Drug
Name: RIF Type: Drug
Name: ATRA Type: Drug
Name: mitoxantrone Type: Drug
Name: Ara-C Type: Drug
Name: MTX Type: Drug
Name: 6MP Type: Drug
Name: intrathecal injection Type: Other
Overall Status
Recruiting
Summary
Outcome of acute promyelocytic leukemia (APL) has greatly improved since the introduction of all-trans-retinoic acid (ATRA). Treatment with ATRA and anthracycline-based chemotherapy (ATRA + chemotherapy) decreases relapses of the disease as well as early hemorrhagic deaths. Nowadays patients with APL have an event-free survival (EFS) of up to 80%. However, there remains a subset of the patients in whom the disease relapses. Recently, a randomized prospective study showed that the addition of ATO to "ATRA + chemotherapy" treatment protocol had a significantly higher EFS in patients with APL than those treated with "ATRA + chemotherapy" protocol. The patients treated with "ATO + ATRA + chemotherapy" had a five years EFS of 89.2%. Moreover, a recent study showed that Indigo naturalis formula (RIF), a traditional Chinese medicine with tetraarsenic tetrasulfide (As4S4), indirubin, and tanshinone IIA as major active ingredients, yielded synergy in the treatment of a murine APL model in vivo and in the induction of APL cell differentiation in vitro . It is about 20 years since RIF was used to treat ALP in China. Clinical studies showed that this agent was effective against APL. Compared to ATO, RIF is relatively inexpensive and can be taken orally, resulting in reducing the number of hospital days and the treatment cost. However, there is no report comparing treatment outcomes of "ATO + ATRA + chemotherapy" and "RIF + ATRA + chemotherapy" protocols in children with APL so far. For this purpose, therefore, investigators are going to conduct a multicenter and randomized prospective study in children with APL.
Detailed Description
OBJECTIVES:

- Determine the safety and efficacy of "ATO/RIF + ATRA + less intensive chemotherapy" protocol in children with acute promyelocytic leukemia (APL).

- Compare the safety,efficacy and treatment cost of "RIF + ATRA + less intensive chemotherapy" with "ATO + ATRA + less intensive chemotherapy" protocol in children with APL. Determine if ATO can be substituted by RIF.

OUTLINE: This is a multicenter and randomized prospective study.

PROJECTED ACCRUAL: A total of 240 patients will be accrued for this study.
Criteria for eligibility
Healthy Volunteers: No
Maximum Age: 16 Years
Minimum Age: N/A
Gender: Both
Criteria: Inclusion Criteria:

- Patients less than 16 years old with newly diagnosed PML-RARa positive acute promyelocytic leukemia.

Exclusion Criteria:

- Patients who have coma, convulsion or paralysis due to intracranial hemorrhage or central nervous system leukemia at diagnosis.
Location
The First Affiliated Hospital of Sun Yat-Sen University
Guangzhou, Guangdong, China
Status: Recruiting
Contact: Xue-Qun Luo, professor - L-xuequn@126.com
Start Date
September 2011
Completion Date
September 2022
Sponsors
South China Children's Leukemia Group
Source
South China Children's Leukemia Group
Record processing date
ClinicalTrials.gov processed this data on July 28, 2015
ClinicalTrials.gov page