Dysautonomia International

Pivotal Open-label Phase 3 Clinical Study of QTX-2101 in Adult Patients With Acute Promyelocytic Leukemia

Purpose

This Phase 3 study in adult participants with newly diagnosed low-risk APL will evaluate the efficacy, safety, and PK of an oral capsule formulation of ATO, in combination with ATRA.

Conditions

Acute Promyelocytic Leukemia (APL)
Acute Promyelocytic Leukaemia
Acute Promyelocytic Leukemia With PML-RARA
Acute Promyelocytic Leukemia With t(15;17)(q24.1;q21.2); PML-RARA
Acute Promyelocytic Leukemia

Eligibility

Eligible Ages: Between 18 Years and 71 Years
Eligible Sex: All
Accepts Healthy Volunteers: No

Inclusion Criteria

Informed Consent 2. Participants must be between 18 and under 71 years of age 3. Participants must have a confirmed diagnosis of APL proven by standard genetic testing (t(15;17) or PML-RARA) 4. Participants must be classified as low- or intermediate-risk APL 5. Participants must be willing and able to comply with the scheduled study visits, treatment plans, laboratory tests, contraception guidance, and other procedures

Exclusion Criteria

Participants who have significant heart rhythm problems including long QT syndrome, serious arrhythmias, very slow heart rate, or prolonged QTc on ECG 2. Participants who have central nervous system leukemia 3. Participants having serious ongoing medical conditions or infections including uncontrolled infections, severe organ disease, or conditions that make study participation unsafe 4. Participants who are pregnant, breastfeeding, or unwilling to use contraception 5. Participants who are unable to safely take study medication, including severe neuropathy, inability to swallow oral medication, malabsorption issues, or known allergy to ATO or ATRA

Study Design

Phase: Phase 3
Study Type: Interventional
Allocation: Randomized
Intervention Model: Parallel Assignment
Primary Purpose: Treatment
Masking: None (Open Label)