QTX-2101 and Arsenic Trioxide in Adult Patients With Acute Promyelocytic Leukemia

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What is this study about?

This clinical study is being done in Acute Promyelocytic Leukemia, a rare type of blood cancer. The treatment being studied is QTX-2101, taken by mouth as a capsule, and it is given together with all-trans retinoic acid, a medicine that helps leukemia cells mature into normal blood cells. The study also compares this approach with arsenic trioxide given through a vein, which is a standard treatment for this disease. The purpose of the study is to understand how well this treatment works and how safe it is.

The study is designed for adults with newly diagnosed, low-risk disease. Treatment is given in phases over time, including an early treatment period and later treatment cycles called consolidation, which are used to help keep the disease under control after the first response. During the study, blood tests, heart checks, and other routine medical checks are done, and side effects are watched closely. The study also looks at how the body handles QTX-2101 and whether the leukemia goes away to a very deep level, meaning no clear signs of the cancer marker PML/RARA can be found in the bone marrow.

Who Can Join the Study?

Be at least 18 years old and younger than 71 years old when signing the informed consent form, which is the written permission to join the study.
Be able to give written informed consent, meaning the person understands the study and signs the form voluntarily.
Have a diagnosis of acute promyelocytic leukemia (APL) confirmed by one of these tests:
- t(15;17) translocation found by fluorescence in situ hybridization or cytogenetics. A translocation means parts of two chromosomes have swapped places.
- PML/RARA gene expression found by RT-qPCR, which is a test that measures specific gene activity.
Have low-risk or intermediate-risk APL, meaning the white blood cell count at diagnosis was 10 × 10⁹/L or lower.
For Part 1 only, have low-risk APL and have already completed:
- Induction treatment, which is the first phase of treatment.
- 3 cycles of consolidation treatment with IV ATO and ATRA.
  - IV means given into a vein.
  - ATO is arsenic trioxide.
  - ATRA is all-trans retinoic acid.
- Have a documented mCR at study entry, meaning a molecular complete remission, where tests show no detectable signs of the leukemia at that level.
For Part 2 only, have newly diagnosed low-risk APL, meaning the diagnosis is recent and treatment has not yet been completed.
Have enough organ function, shown by all of the following:
- Total bilirubin of 3.0 mg/dL or lower. Bilirubin is a substance measured in blood that helps show how well the liver is working.
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) at 3 times the upper limit of normal or lower. These are liver enzymes, which are proteins that can rise when the liver is stressed or injured.
- Creatinine clearance of 30 mL/min or higher. This is a measure of kidney function.
Have an ECOG performance status of 2 or less, meaning the person is able to care for themselves and is not too limited in daily activities. ECOG is a scale doctors use to measure how well a person can function.
For females who can become pregnant, have a negative pregnancy test at the screening visit and again right before the first study treatment.
Be willing and able to:
- Attend all scheduled study visits.
- Follow the treatment plan.
- Receive the study treatment at the study site through the end of the study period.
- Have the required blood tests and other study procedures.
- Follow the contraception guidance, meaning instructions to help prevent pregnancy during the study.
- Complete the full treatment and follow-up steps unless the study doctor says stopping is medically needed.

Who Cannot Join the Study?

Anyone who has had ATRA treatment for more than 7 days before the first study dose, in Part 2 only. ATRA is a medicine used in this study.
Anyone with a past cancer that is still being treated with radiation or chemotherapy, or who has not been free of that cancer for at least 2 years before screening, except for certain allowed cancers such as localized prostate cancer treated with hormone therapy alone, breast cancer treated with hormone therapy alone, basal cell carcinoma, non-melanoma skin cancer, or cervical carcinoma in situ.
Anyone with an active, serious, or important uncontrolled infection that needs hospital care. An uncontrolled infection means the infection is not being kept under control.
Anyone with a known malabsorption syndrome or another condition that may stop the body from absorbing the study medicine properly, such as after gastrectomy (surgery to remove part or all of the stomach), or anyone who cannot swallow oral medicine.
Anyone with another severe short-term or long-term medical problem, mental health problem, or abnormal lab result that could raise the risk of taking part, affect the study results, or make the person unsuitable for the study.
Anyone with immunocompromise, meaning a weakened immune system with a higher risk of unusual infections, including people with HIV if their CD4 count is 350 cells/mm3 or less, or if they had an opportunistic infection in the last 12 months. An opportunistic infection is an infection that can happen more easily when the immune system is weak. HIV-positive people must also have been on stable antiretroviral therapy for at least 4 weeks and have an HIV viral load below 400 copies/mL before screening.
Anyone with active or chronic hepatitis B or active hepatitis C infection. People with past hepatitis C may still qualify only if they finished curative treatment at least 12 weeks before screening and have no detectable virus at screening.
Anyone who must stay on anticoagulation without interruption, such as people with a mechanical heart valve, because of a higher bleeding risk during treatment. Anticoagulation means blood-thinning medicine.
Pregnant females, breastfeeding females, males who will not follow the required birth control rules, or females who can become pregnant and will not follow the required birth control rules.
Anyone likely to stop the study after treatment or during follow-up only to join another interventional study for APL or another experimental treatment, unless the investigator or sponsor recommends it.
Anyone with suspected leukemia involvement in the central nervous system, which includes the brain and spinal cord.
Anyone with Grade 2 or higher neuropathy. Neuropathy means nerve damage that can cause pain, numbness, or weakness.
Anyone with a history of torsade de pointes, a dangerous type of abnormal heart rhythm.
Anyone with certain ECG heart test problems, including:
- Congenital long QT syndrome, a condition present from birth that affects the heart’s electrical timing.
- A history of, or current, serious fast heart rhythms in the upper or lower chambers of the heart.
- Slow resting heart rate below 50 beats per minute.
- QTc longer than 450 milliseconds on the screening ECG. QTc is a measure of how long the heart takes to recharge between beats.
- Right bundle branch block plus left anterior hemiblock or bifascicular block, which are types of heart conduction problems.
Anyone with unresolved side effects from prior intravenous ATO treatment, in Part 1 only, unless those side effects are mild Grade 1 or have fully gone away before joining the study.
Anyone with current heart failure, or heart failure that happened within the 3 months before screening. Heart failure means the heart cannot pump blood as well as it should.
Anyone with a known reason they cannot take ATO or ATRA, or any ingredient in these medicines, including allergy to soy or peanut for ATRA.
Anyone who received another investigational agent within 30 days before screening, or within fewer than 5 half-lives after the previous investigational treatment ended, whichever is shorter. An investigational agent is a study medicine that is still being tested.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Policlinico “Tor Vergata”, Università degli Studi di Roma TOR VERGATA	Rome	Italy
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France
Universitaet Leipzig	Leipzig	Germany
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Hospital Universitario Y Politecnico La Fe	Valencia	Spain

Other Sites

Site Name	City	Country
Szpital Uniwersytecki Nr 2 Im Dr Jana Biziela W Bydgoszczy	Bydgoszcz	Poland
Hospital Clinic De Barcelona	Barcelona	Spain
University Clinical Hospital Virgen De La Arrixaca	Murcia	Spain
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie	Cracow	Poland
Centre Hospitalier Universitaire De Nice	Nice	France
Centre Hospitalier Lyon Sud	Pierre Benite	France
Hopital Beaujon	Clichy	France
Servei De Salut De Les Illes Balears	Palma	Spain
Hospital San Pedro De Alcantara	Caceres	Spain
Spitalul Clinic Coltea	Bucharest	Romania
Spitalul Clinic Colentina Bucuresti	Bucharest	Romania
Spitalul Clinic Municipal De Urgenta Timisoara	Timisoara	Romania
Institutul Regional De Oncologie Iasi	Iasi	Romania
Institute Of Oncology Prof. Dr. Ion Chiricuta Cluj-Napoca	Cluj Napoca	Romania
Fundeni Clinical Institute	Bucharest	Romania
Hsuszzyj Vmub drdhtqvy	Barcelona	Spain
Cgem Di Naien	Vandoeuvre Les Nancy	France
Haslmfpe Do Ly Slmel Czra I Siuq Pgx	Barcelona	Spain
Hahvgxyh Unhbwnnfwbfao Hhrlirce Tjryd y Psixnb Igcyfjxn Cynvmk dcpjicflkyeqaztaq (ldko	Badalona	Spain

Trial status

Country	Status	Recruitment Start
France	Not yet recruiting	23.02.2026
Germany	Not yet recruiting	23.02.2026
Italy	Not yet recruiting	23.02.2026
Poland	Not yet recruiting	23.02.2026
Romania	Not yet recruiting	23.02.2026
Spain	Not yet recruiting	23.02.2026

Trial locations

Investigated drugs:

Arsenic Trioxide

QTX-2101 is the main study treatment. It is a capsule taken by mouth and contains arsenic trioxide. In this trial, it is being tested as part of the treatment plan for newly diagnosed low-risk acute promyelocytic leukemia, to see how well it works and how safe it is.

Arsenic trioxide is the comparison treatment in the study. It is given directly into a vein as an infusion. It is used to help treat the leukemia and serves as the standard treatment the study is comparing against the oral study medicine.

All-trans retinoic acid is used together with the study treatment. It is part of the treatment combination being tested in the trial and is given to help treat the leukemia by helping the abnormal blood cells mature in a more normal way.

Investigated diseases:

Acute promyelocytic leukaemia

Acute Promyelocytic Leukemia – A type of acute myeloid leukemia in which very immature white blood cells called promyelocytes build up in the bone marrow and blood. It develops when the cells stop maturing normally and begin to multiply in an uncontrolled way. The disease often progresses quickly, with the abnormal cells increasingly replacing normal blood-forming cells.

Trial ID:

2025-524810-28-00

Protocol code:

QTX-2101-301

Trial Phase:

Therapeutic confirmatory (Phase III)

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QTX-2101 and Arsenic Trioxide in Adult Patients With Acute Promyelocytic Leukemia

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?