Acute Promyelocytic Leukemia (APL)Phase 3QUATRO-APLNCT07504458
Pivotal Open-label Phase 3 Clinical Study of QTX-2101 in Adult Patients With Acute Promyelocytic Leukemia
A Phase 3 study testing QTX-2101 + ATRA and IV arsenic trioxide (ATO) + ATRA in people with Acute Promyelocytic Leukemia (APL) and Acute Promyelocytic Leukaemia, run by Quetzal Therapeutics at 35 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 71 years
- Conditions the study is for
- Acute Promyelocytic Leukemia (APL), Acute Promyelocytic Leukaemia, Acute Promyelocytic Leukemia With PML-RARA
- What is being tested?
- QTX-2101 + ATRA and IV arsenic trioxide (ATO) + ATRA
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 150 participants
- Where
- 35 sitesin 5 countries
- Study dates
- 8 Jun 2026to Dec 2030
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 71 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Informed Consent
- Participants must be between 18 and under 71 years of age
- Participants must have a confirmed diagnosis of APL proven by standard genetic testing (t(15;17) or PML-RARA)
- Participants must be classified as low- or intermediate-risk APL
- Participants must be willing and able to comply with the scheduled study visits, treatment plans, laboratory tests, contraception guidance, and other procedures
Who cannot join
- Participants who have significant heart rhythm problems including long QT syndrome, serious arrhythmias, very slow heart rate, or prolonged QTc on ECG
- Participants who have central nervous system leukemia
- Participants having serious ongoing medical conditions or infections including uncontrolled infections, severe organ disease, or conditions that make study participation unsafe
- Participants who are pregnant, breastfeeding, or unwilling to use contraception
- 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
Trial locations and contacts
35 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
35 sites in 5 countries
United States18 sites, 18 recruiting
- Recruiting
Quetzal Site 22
Ann Arbor, Michigan, 48109
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
Quetzal Therapeutics Investigator, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 13
Augusta, Georgia, 30912
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 23
Baltimore, Maryland, 21201
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
Quetzal Therapeutics Investigator, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 34
Baltimore, Maryland, 21231
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 16
Birmingham, Alabama, 35233
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
Quetzal Therapeutics Investigator, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 4
Buffalo, New York, 14203
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 27
Chapel Hill, North Carolina, 27514
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 3
Charlottesville, Virginia, 22908
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 8
Columbus, Ohio, 43210
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 1
Duarte, California, 91010
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 6
Houston, Texas, 77030
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 31
Irvine, California, 92612
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 18
Lexington, Kentucky, 40536
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
Quetzal Therapeutics Investigator, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 7
Los Angeles, California, 90033
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 12
Nashville, Tennessee, 37203
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 32
Orange, California, 92868
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 5
Stanford, California, 94305
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 2
The Bronx, New York, 10467
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
Spain8 sites, 8 recruiting
- Recruiting
Quetzal Site 21
Badalona, 08916
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 28
Barcelona, 08035
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 15
Barcelona, 08041
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 10
Cáceres, 10003
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 30
Málaga, 29010
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 20
Oviedo, 33011
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 11
Palma de Mallorca, Balearic Islands, 07120
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 9
Valencia, 46026
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
France7 sites, 7 recruiting
- Recruiting
Quetzal Site 17
Bordeaux, 33000
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 25
La Tronche, 38700
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 33
Nancy, 54500
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 29
Nice, 06202
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 14
Paris, 75010
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 35
Saint-Priest-en-Jarez, 42270
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
- Recruiting
Quetzal Site 24
Strasbourg, 67000
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
Italy1 site, 1 recruiting
- Recruiting
Quetzal Site 26
Vicenza, 36100
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
Romania1 site, 1 recruiting
- Recruiting
Quetzal Site 19
Cluj-Napoca, 400015
Quetzal Therapeutics Investigator, +13126800501, ClinicalTrials@QuetzalTx.com
About this study
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
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
- Being tested
- QTX-2101 + ATRA, IV arsenic trioxide (ATO) + ATRA
- Keywords
- APL, Acute Promyelocytic Leukemia, Hematology, Oncology, Leukemia, Hematologic Disease, Drug Therapy, Therapeutics, Blood Cancer, Heme Malignancy, Arsenic, low-risk APL, standard-risk APL
- Medical subject headings
- Leukemia, Promyelocytic, Acute, Neoplasms, Leukemia, Hematologic Diseases, Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QTX-2101 + ATRADrug
The experimental regimen consists of IV ATO administered once daily during induction, given continuously, for up to a maximum of 60 days. During consolidation, QTX-2101 is administered once daily, per investigator's protocol. ATRA is administered orally in two divided daily doses during induction, given continuously until bone marrow remission (not exceeding 60 days). During consolidation, ATRA is taken orally in two divided daily doses following a 2-weeks-on / 2-weeks-off schedule within each 8-week cycle.
IV arsenic trioxide (ATO) + ATRADrug
The comparator regimen consists of IV ATO administered once daily during induction, given continuously, for up to a maximum of 60 days. During consolidation, IV ATO is administered once daily, per investigator's protocol. ATRA is administered orally in two divided daily doses during induction, given continuously until bone marrow remission (not exceeding 60 days). During consolidation, ATRA is taken orally in two divided daily doses following a 2-weeks-on / 2-weeks-off schedule within each 8-week cycle.
| Study group | Role | What they receive |
|---|---|---|
| QTX-2101 | Experimental | QTX-2101 (oral arsenic trioxide; ATO) All-trans-retinoic-acid (ATRA; oral) Receives: Drug: QTX-2101 + ATRA |
| IV ATO | Active comparator | IV Arsenic Trioxide (ATO) All-trans-retinoic-acid (ATRA; oral) Receives: Drug: IV arsenic trioxide (ATO) + ATRA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum observed plasma (concentration (Cmax) of QTX-2101 for ASIII
Cmax is defined as the maximum observed plasma concentration following administration of [investigational product], determined from plasma concentration-time data.
Measured: Up to 1 cycle of consolidation therapy (each cycle is 8 weeks)
Molecular complete remission (molecular CR) rate
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.
Measured: Up to 60 days of induction and 3 8-week cycles of consolidation treatment
Secondary outcomes (8)
To characterize the safety and tolerability of QTX-2101/ATRA and IV ATO/ATRA
Treatment emergent adverse events
Measured: Throughout approximately 10 months of study treatment
To characterize the event-free survival (EFS) of QTX-2101/ATRA
Measured: 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
Area under the plasma concentration-time curve (AUC) of QTX-2101 for ASIII
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.
Measured: Up to 10 months
To complete a model-based concentration QT relationship evaluation
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
Measured: Up to 10 months
EORTC QLQ-C30 domain unit of measure and measurement tool
Change from baseline in EORTC QLQ-C30 global health status (0-100 scale)
Measured: Up to 10 months
EQ-5D-5L domain unit of measure and measurement tool
Change from baseline in EQ-5D-5L index score (0-100)
Measured: Up to 10 months
Overall Survival
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.
Measured: 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
Event Free Survival (EFS)
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.
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Mar 2026
- Study start8 Jun 2026
- Primary completionMar 2027 (estimated)
- Study completionDec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Promyelocytic Leukemia (APL)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Quetzal Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07504458
- Sponsor's protocol ID
- QTX-2101-301
- Other IDs
- 2025-524810-28-00, 2025
- Acronym
- QUATRO-APL
- Status last verified
- Jun 2026
- First submitted
- 16 Mar 2026
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No