Childhood Acute Promyelocytic Leukemia Clinical Trial
Official title:
A Multicenter and Randomized Prospective Study for Improving the Outcome of Childhood Acute Promyeloid Leukemia
| NCT number | NCT02200978 |
| Other study ID # | 2010001 |
| Secondary ID | |
| Status | Completed |
| Phase | Phase 4 |
| First received | |
| Last updated | |
| Start date | September 2011 |
| Est. completion date | October 2021 |
| Verified date | May 2022 |
| Source | South China Children's Leukemia Group |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 176 |
| Est. completion date | October 2021 |
| Est. primary completion date | October 2021 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | N/A to 16 Years |
| Eligibility | 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. |
| Country | Name | City | State |
|---|---|---|---|
| China | The First Affiliated Hospital of Sun Yat-Sen University | Guangzhou | Guangdong |
| Lead Sponsor | Collaborator |
|---|---|
| South China Children's Leukemia Group |
China,
Hu J, Liu YF, Wu CF, Xu F, Shen ZX, Zhu YM, Li JM, Tang W, Zhao WL, Wu W, Sun HP, Chen QS, Chen B, Zhou GB, Zelent A, Waxman S, Wang ZY, Chen SJ, Chen Z. Long-term efficacy and safety of all-trans retinoic acid/arsenic trioxide-based therapy in newly diagnosed acute promyelocytic leukemia. Proc Natl Acad Sci U S A. 2009 Mar 3;106(9):3342-7. doi: 10.1073/pnas.0813280106. Epub 2009 Feb 18. — View Citation
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| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | event-free survival | 5 years | ||
| Secondary | hospitalization cost | The cost mainly includes the fees of hospital bed, drugs, therapies and blood products. Time frame: from the beginning of induction therapy to the end of maintenance treatment. | 2 years |