Brief Summary
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.
Brief Title
Pivotal Open-label Phase 3 Clinical Study of QTX-2101 in Adult Patients With Acute Promyelocytic Leukemia
Categories
Completion Date
Completion Date Type
Estimated
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 Criteria
Inclusion Criteria:
1. 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
1. 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
1. 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
1. 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
Inclusion Criteria
Inclusion Criteria:
1. 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
1. 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
Gender
All
Gender Based
false
Keywords
APL
Acute Promyelocytic Leukemia
Hematology
Oncology
Leukemia
Hematologic Disease
Drug Therapy
Therapeutics
Blood Cancer
Heme Malignancy
Arsenic
low-risk APL
standard-risk APL
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
71 Years
Minimum Age
18 Years
NCT Id
NCT07504458
Org Class
Industry
Org Full Name
Quetzal Therapeutics
Org Study Id
QTX-2101-301
Overall Status
Recruiting
Phases
Phase 3
Primary Completion Date
Primary Completion Date Type
Estimated
Official Title
A Pivotal Open-label Phase 3 Clinical Study Evaluating the Efficacy and Safety of QTX-2101 in Combination With All-trans Retinoic Acid in Newly Diagnosed, Low-risk Acute Promyelocytic Leukemia
Primary Outcomes
Outcome Description
Cmax is defined as the maximum observed plasma concentration following administration of \[investigational product\], determined from plasma concentration-time data.
Outcome Measure
Maximum observed plasma (concentration (Cmax) of QTX-2101 for ASIII
Outcome Time Frame
Up to 1 cycle of consolidation therapy (each cycle is 8 weeks)
Outcome Description
mCR is defined as the absence of detectable PML-RARA fusion transcript in bone marrow assessed by a validated quantitative reverse transcription polymerase chain reaction (RT-qPCR) assay .The mCR rate is defined as the proportion of participants achieving molecular remission at the specified assessment time point following induction and consolidation therapy.
Outcome Measure
Molecular complete remission (molecular CR) rate
Outcome Time Frame
Up to 60 days of induction and 3 8-week cycles of consolidation treatment
Secondary Ids
Secondary Id
2025-524810-28-00
Secondary Outcomes
Outcome Description
Treatment emergent adverse events
Outcome Time Frame
Throughout approximately 10 months of study treatment
Outcome Measure
To characterize the safety and tolerability of QTX-2101/ATRA and IV ATO/ATRA
Outcome Time Frame
Assessed for up to 3 years after the first dose of treatment, or until treatment failure (disease progression), death, or study completion, whichever occurs first
Outcome Measure
To characterize the event-free survival (EFS) of QTX-2101/ATRA
Outcome Description
AUC is defined as the area under the plasma concentration-time curve from time zero to the last measurable concentration (AUC₀-t) and/or extrapolated to infinity (AUC₀-∞), calculated using noncompartmental methods.
Outcome Time Frame
Up to 10 months
Outcome Measure
Area under the plasma concentration-time curve (AUC) of QTX-2101 for ASIII
Outcome Description
Time-matched difference in change from baseline in QTcF interval between active treatment and placebo, derived from triplicate 12-lead ECGs at each post-dose time point
Outcome Time Frame
Up to 10 months
Outcome Measure
To complete a model-based concentration QT relationship evaluation
Outcome Description
Change from baseline in EORTC QLQ-C30 global health status (0-100 scale)
Outcome Time Frame
Up to 10 months
Outcome Measure
EORTC QLQ-C30 domain unit of measure and measurement tool
Outcome Description
Change from baseline in EQ-5D-5L index score (0-100)
Outcome Time Frame
Up to 10 months
Outcome Measure
EQ-5D-5L domain unit of measure and measurement tool
Outcome Description
OS is defined as the time from randomization to death from any cause. Participants who are alive at the time of analysis will be censored at the date last known to be alive.
Outcome Time Frame
Assessed for up to 3 years after the first dose of treatment, or until treatment failure (disease progression), death, or study completion, whichever occurs first
Outcome Measure
Overall Survival
Outcome Description
EFS is defined as the time from randomization to the first occurrence of any of the following events: failure to achieve hematologic or molecular remission, relapse (hematologic or molecular), or death from any cause. Participants without an event at the time of analysis will be censored at the date of last adequate disease assessment.
Outcome Time Frame
Assessed for up to 3 years after the first dose of treatment, or until treatment failure (disease progression), death, or study completion, whichever occurs first
Outcome Measure
Event Free Survival (EFS)
Start Date
Start Date Type
Estimated
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
71
Minimum Age Number (converted to Years and rounded down)
18
Investigators
Investigator Type
Principal Investigator
Investigator Name
Aditi Shastri
Investigator Email
ASHASTRI@montefiore.org
Investigator Phone
718-920-4826
Investigator Department
Medicine
Investigator Division
Oncology
Investigator Sponsor Organization
External
Study Department
Oncology (Medical/Hematologic)
Study Division
Medical and Hematologic Oncology
Categories Mesh Debug
Cancer --- NEOPLASMS
Cancer --- LEUKEMIA
Blood & Bone Marrow Cancers --- LEUKEMIA
Blood Disorders --- HEMATOLOGIC DISEASES
Blood & Bone Marrow Cancers --- HEMATOLOGIC NEOPLASMS
Blood & Bone Marrow Cancers --- LEUKEMIA, MYELOID, ACUTE
Blood & Bone Marrow Cancers --- LEUKEMIA, MYELOID
Cancer --- NEOPLASMS BY SITE
MeSH Terms
LEUKEMIA, PROMYELOCYTIC, ACUTE
NEOPLASMS
LEUKEMIA
HEMATOLOGIC DISEASES
HEMATOLOGIC NEOPLASMS
LEUKEMIA, MYELOID, ACUTE
LEUKEMIA, MYELOID
NEOPLASMS BY HISTOLOGIC TYPE
HEMIC AND LYMPHATIC DISEASES
NEOPLASMS BY SITE
TRETINOIN
ARSENIC TRIOXIDE
VITAMIN A
RETINOIDS
CAROTENOIDS
POLYENES
ALKENES
HYDROCARBONS, ACYCLIC
HYDROCARBONS
ORGANIC CHEMICALS
CYCLOHEXENES
CYCLOHEXANES
CYCLOPARAFFINS
HYDROCARBONS, ALICYCLIC
HYDROCARBONS, CYCLIC
TERPENES
DITERPENES
PIGMENTS, BIOLOGICAL
BIOLOGICAL FACTORS
ARSENICALS
INORGANIC CHEMICALS
OXIDES
OXYGEN COMPOUNDS