Early use of oral Realgar-Indigo Naturalis plus all-trans retinoic acid for acute promyelocytic leukemia

Multicenter, Randomized Controlled Clinical Study on Early Application of Realgar-Indigo Naturalis Formula (RIF) for Treatment of Acute Promyelocytic Leukemia (APL)

This study will test whether starting an oral arsenic medicine called Realgar-Indigo Naturalis Formula (RIF) together with all-trans retinoic acid (ATRA) early in treatment can lower early deaths in adults with acute promyelocytic leukemia (APL).

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 224 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: First Affiliated Hospital Xi'an Jiaotong University (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 1 site (Xi'an, Shaanxi)
• Trial ID: NCT07503730 on ClinicalTrials.gov

What this trial studies

This is a multicenter, randomized controlled trial comparing early induction with oral Realgar-Indigo Naturalis Formula (RIF) plus ATRA against standard approaches that include ATO and ATRA in adults with APL. The trial enrolls newly diagnosed or highly suspected APL patients, including high-risk patients with elevated white blood cell counts. The primary outcome is early death (within 30 days), with secondary outcomes including control of coagulopathy, major bleeding events, and remission rates. Interventions include oral RIF, ATRA, and arsenic trioxide (ATO) as relevant to the assigned treatment arm, with the goal of determining whether early oral RIF can more rapidly stabilize coagulation and reduce hemorrhagic risk.

Who should consider this trial

Good fit: Adults aged 18 to 80 with newly diagnosed or strongly suspected APL, especially those with high initial white blood cell counts (>10×10^9/L), are the intended participants.

Not a fit: Patients who do not have APL, who have severe unrelated liver, kidney, or heart dysfunction, other active malignancies, or who are pregnant or breastfeeding would not be expected to benefit from this intervention.

Why it matters

Potential benefit: If successful, early RIF plus ATRA could reduce fatal bleeding in the first month of treatment and offer a more convenient oral option for initial management.

How similar studies have performed: Prior randomized and pivotal studies have shown RIF to be non-inferior to intravenous ATO for consolidation in low-to-intermediate risk APL, but early use of RIF specifically to reduce initial early deaths has been less extensively studied.

Eligibility criteria

Inclusion Criteria:

1. Age between 18 and 80 years.
2. Diagnosed with acute myeloid leukemia confirmed by bone marrow morphology and immunophenotyping, with a high clinical suspicion of acute promyelocytic leukemia (APL).

Exclusion Criteria:

1. Confirmed non-APL (M3 type) acute myeloid leukemia through cytogenetic and RT-PCR testing (PML-RARα fusion gene negative).
2. Severe liver or kidney dysfunction unrelated to APL (e.g., serum creatinine \> 2.5 times the upper limit of normal, total bilirubin ≥ 2 times the upper limit, ALT and AST \> 3 times the upper limit), or heart failure (e.g., EF \< 40%).
3. Presence of other malignancies.
4. Pregnant or breastfeeding women.

Where this trial is running

Xi'an, Shaanxi

• First Affiliated Hospital of Xi'an Jiaotong University — Xi'an, Shaanxi, China (Recruiting)

Study contacts

• Study coordinator: Huaiyu Wang

Conditions APL