Treatment study for patients with acute promyelocytic leukemia under 21 years of age

Phase 3

Completed

Conditions: Acute promyelocitic leukemia
10024324

Registration Number: NL-OMON39899

Lead Sponsor: Stichting Kinderoncologie Nederland

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 5

Inclusion Criteria

- Patients with a clinical diagnosis of initial APL and subsequently confirmed to have PML-RARa, NPM1-RARa or NUMA-RARa fusion. Whilst this study is only for ATRA-sensitive APL, APL is a hematological emergency and ATRA should be commenced as soon as the diagnosis is suspected. Study entry should not wait until the diagnosis of APL has been confirmed molecularly or cytogenetically
- Less than 21 years of age at initial diagnosis
- Considered suitable for anthracycline-based chemotherapy
- Written informed consent available
- Females of childbearing age must have a negative pregnancy test and subsequently must attempt to avoid pregnancy

Exclusion Criteria

- Patients with a clinical diagnosis of APL but subsequently found to have PLZF-RARa fusion or lacking PML-RAR*, NPM-RAR* or NuMA-RAR* rearrangement should be withdrawn from the study and treated on an alternative protocol.
- Refractory/relapsed APL (the guidelines in this protocol for that subgroup are intended for patients treated from initial diagnosis according to this protocol)
- Concurrent active malignancy
- Pregnant or lactating
- Physician and patient/guardian think that intensive chemotherapy is not an appropriate treatment option
- Patients who have received alternative chemotherapy for 7 days or longer without ATRA for any reason (either APL not initially suspected or ATRA not available).

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The main endpoints will be:<br /><br>•Achievement of molecular complete remission (CRm) and reasons for failure<br /><br>•Duration of remission, rates of molecular and frank relapse and deaths in<br /><br>first CR<br /><br>•Overall survival<br /><br>•Toxicity - hematological and non-hematological<br /><br>•Supportive care requirements</p><br>

Secondary Outcome Measures