Combining oral arsenic and ATRA for treating newly diagnosed acute promyelocytic leukemia

Combination of Oral Arsenic With ATRA and Minimal-Dose Chemotherapy for Newly Diagnosed Patients With Acute Promyelocytic Leukemia: a Study by the International Consortium on APL

Phase 2 Interventional Instituto do Cancer do Estado de São Paulo · NCT06982274

This study is testing if a new treatment combining oral arsenic and ATRA can help newly diagnosed patients with acute promyelocytic leukemia live longer and feel better, especially those at high risk.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment115 (estimated)
Ages18 Years to 75 Years
SexAll
SponsorInstituto do Cancer do Estado de São Paulo Academic / other
Drugs / interventionschemotherapy
Locations1 site (São Paulo, São Paulo)
Trial IDNCT06982274 on ClinicalTrials.gov

What this trial studies

This multicenter, prospective study aims to treat newly diagnosed patients with acute promyelocytic leukemia (APL) using a combination of oral arsenic and all-trans-retinoic acid (ATRA). For high-risk patients, defined as having a white blood cell count above 10x10⁹/L, low-dose chemotherapy will also be administered. The primary goal is to assess the 2-year overall survival rate in these patients compared to historical controls treated with ATRA and chemotherapy. Secondary objectives include evaluating response rates, disease-free survival, and toxicity profiles, among others.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 75 with a new diagnosis of acute promyelocytic leukemia confirmed by cytomorphology and molecular analysis.

Not a fit: Patients with other active malignancies or those who are not eligible for chemotherapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment approach could significantly improve survival rates for patients with acute promyelocytic leukemia.

How similar studies have performed: Previous studies have shown promising results with ATRA in treating APL, but the combination with oral arsenic is a novel approach.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Informed consent
* New diagnosis of APL by cytomorphology, confirmed for molecular analysis
* Age ≥18 and ≤75 years
* Serum total bilirubin ≤ 3.0 mg/dl (≤ 51 μmol/l)
* Serum creatinine ≤ 3.0 mg/dl (≤ 260 μmol/l)
* Women must meet at least one of the following criteria to be eligible for inclusion in the study: Postmenopausal (12 months of amenorrhea or 6 months of amenorrhea with serum FSH \> 40 U/ml); After undergoing hysterectomy or bilateral oophorectomy; Continuous and correct use of a contraceptive method with a Pearl Index \<1% (e.g., implants, oral contraceptives, intrauterine devices); Sexual abstinence; Vasectomy of sexual partner.

Exclusion Criteria:

* High-risk patients who are not eligible for chemotherapy according to the judgment of the treating physician;
* Age \<18 or \>75 years
* Other active malignancy at the time of study entry
* Lack of diagnostic confirmation at the genetic level
* Significant arrhythmias, ECG abnormalities, or neuropathy: Congenital long QT syndrome; History or presence of significant ventricular or atrial tachyarrhythmia; Clinically significant resting bradycardia (\<50 beats per minute); QTc \> 500 ms on ECG screening for both sexes; Right bundle branch block with left anterior hemiblock or bifascicular block
* High-risk patients with other cardiac contraindications for intensive chemotherapy (LVEF \< 50%)
* Uncontrolled and potentially fatal infections
* Severe uncontrolled pulmonary or cardiac disease
* Severe hepatic or renal dysfunction
* Known HIV and/or hepatitis C infection
* Pregnant or breastfeeding women
* Allergy to the study drug or excipients in the study medication
* Substance abuse, medical, psychological, or social conditions that may interfere with the patient's participation in the study or the assessment of study outcomes
* Use of other investigational drugs at the time of enrollment or within 30 days before study entry.

Where this trial is running

São Paulo, São Paulo

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Acute Promyelocytic LeukemiaAcute promyelocytic leukemiaOral arsenicSurvivalAll trans-retinoic acid
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.