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AzTrials

Acute Promyelocytic Leukemia With PML-RARAPhase 2NCT01409161

Tretinoin and Arsenic Trioxide With or Without Gemtuzumab Ozogamicin in Treating Patients With Previously Untreated Acute Promyelocytic Leukemia

A Phase 2 study testing Arsenic Trioxide, Gemtuzumab Ozogamicin and Laboratory Biomarker Analysis in people with Acute Promyelocytic Leukemia With PML-RARA, run by M.D. Anderson Cancer Center at 5 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

This phase II trial studies how well tretinoin and arsenic trioxide with or without gemtuzumab ozogamicin works in treating patients with previously untreated acute promyelocytic leukemia. Read more about this study

Who is it for?
Ages 10 years and older
What is being tested?
Arsenic Trioxide, Gemtuzumab Ozogamicin and Laboratory Biomarker Analysis
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
151 participants
Where
5 sitesUnited States
Study dates
5 Oct 2011to 18 Dec 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

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 10 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • A diagnosis of APL based on the presence of the PML-RAR-alpha fusion gene by cytogenetics, polymerase chain reaction (PCR), or POD test
  • Ability to understand and the willingness to sign a written informed consent document indicating that they are aware of the investigational nature of the study
  • Patients in whom therapy for APL was initiated on an emergent basis are eligible (patients may have already started treatment with ATRA, ATO, and/or one dose of idarubicin due to the urgency to start therapy early)
  • Women of child-bearing potential must have a negative serum pregnancy test at screening; in addition to having a negative pregnancy test confirmed at screening, all female participants of childbearing potential must have a negative pregnancy test confirmed within 48 hours prior to dosing with the study drug
  • All sexually active subjects (males and females of child-bearing potential) agree to use 2 effective methods of contraception for the duration of the study

Who cannot join

  • Fridericia corrected QT (QTcF) interval on the electrocardiogram (EKG) greater than 480 milliseconds
  • Patients with creatinine > 2.5 times upper limit of normal unless felt to be related the underlying leukemia by the treating physician or hemolysis or Gilbert's disease
  • Patients with total bilirubin >= 2.0 times upper limit of normal unless felt to be related the underlying leukemia by the treating physician or hemolysis or Gilbert's disease
  • Patients with alanine aminotransferase (ALT)/aspartate aminotransferase (AST) > 3 times upper limit of normal unless felt to be related the underlying leukemia by the treating physician or hemolysis or Gilbert's disease

Trial locations and contacts

5 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Farhad Ravandi-Kashani contact

713-745-0394

fravandi@mdanderson.org

5 sites in 1 country

United States5 sites, 5 recruiting

  • M D Anderson Cancer Center

    Houston, Texas, 77030

    Farhad Ravandi-Kashani, 713-745-0394

    Farhad Ravandi-Kashani

    Recruiting

  • MD Anderson Regional Care Center-Katy

    Houston, Texas, 77094

    Farhad Ravandi-Kashani, 713-745-0394

    Farhad Ravandi-Kashani

    Recruiting

  • MD Anderson Regional Care Center-Bay Area

    Nassau Bay, Texas, 77058

    Farhad Ravandi-Kashani, 713-745-0394

    Farhad Ravandi-Kashani

    Recruiting

  • MD Anderson Regional Care Center-Sugar Land

    Sugar Land, Texas, 77478

    Farhad Ravandi-Kashani, 713-745-0394

    Farhad Ravandi-Kashani

    Recruiting

  • MD Anderson Regional Care Center-The Woodlands

    The Woodlands, Texas, 77384

    Farhad Ravandi-Kashani, 713-745-0394

    Farhad Ravandi-Kashani

    Recruiting

Other trials in United States

About this study

Official title: Phase II Study of Treatment of Acute Promyelocytic Leukemia (APL) With ATRA, Arsenic Trioxide and Gemtuzumab Ozogamicin (GO)

This phase II trial studies how well tretinoin and arsenic trioxide with or without gemtuzumab ozogamicin works in treating patients with previously untreated acute promyelocytic leukemia. Drugs used in chemotherapy, such as tretinoin and arsenic trioxide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Immunotoxins, such as gemtuzumab ozogamicin, may find certain cancer cells and kill them without harming normal cells. Giving tretinoin and arsenic trioxide together with gemtuzumab ozogamicin may kill more cancer cells.

Read the detailed description

PRIMARY OBJECTIVES:

  1. Assess whether a combination of all-trans retinoic acid (ATRA [tretinoin]), and arsenic trioxide (ATO) can produce long-term event-free survival in patients with low-risk untreated acute promyelocytic leukemia (APL).

II. Assess whether administration of gemtuzumab ozogamicin (GO) at the diagnosis in patients with high-risk APL (white blood cell [WBC] > 10,000) and if the WBC rises to > 10,000 after start of treatment (in patients with low-risk disease) will improve complete response (CR) rate without increasing toxicity in high-risk untreated APL.

OUTLINE:

INDUCTION: Patients receive tretinoin orally (PO) twice daily (BID), arsenic trioxide intravenously (IV) over 1-2 hours daily, and gemtuzumab ozogamicin IV over 2 hours once at weeks 1-4.

CONSOLIDATION: Patients achieving CR receive arsenic trioxide IV 5 days per week during weeks 1-4, 9-12, 17-20, and 25-28 and tretinoin PO BID for 2 weeks on and 2 weeks off. Treatment repeats every 8 weeks for up to 4 courses in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 6-12 months.

Being tested
Arsenic Trioxide, Gemtuzumab Ozogamicin, Laboratory Biomarker Analysis, Tretinoin
Medical subject headings
Leukemia, Promyelocytic, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Arsenic TrioxideDrug

    Also called Arsenic (III) Oxide, Arsenic Sesquioxide, Arsenous Acid, Arsenous Acid Anhydride

    Given IV

  • Gemtuzumab OzogamicinDrug

    Also called Calicheamicin-Conjugated Humanized Anti-CD33 Monoclonal Antibody, CDP-771, CMA-676, gemtuzumab

    Given IV

  • Laboratory Biomarker AnalysisOther

    Correlative studies

  • TretinoinDrug

    Also called 2,4,6,8-Nonatetraenoic acid, 3, 7-dimethyl-9-(2,6,6-trimethyl-1-cyclohexen-1-yl)-, (all-E)-, Aberel, Airol, Aknoten

    Given PO

Study groups in NCT01409161
Study groupRoleWhat they receive
Treatment (tretinoin, arsenic trioxide, gemtuzumab ozogamicin)ExperimentalINDUCTION: Patients receive tretinoin PO BID, arsenic trioxide IV over 1-2 hours daily, and gemtuzumab ozogamicin IV over 2 hours once at weeks 1-4. CONSOLIDATION: Patients achieving CR receive arsenic trioxide IV 5 days per week during weeks 1-4, 9-12, 17-20, and 25-28 and tretinoin PO BID for 2 weeks on and 2 weeks off. Treatment repeats every 8 weeks for up to 4 courses in the absence of disease progression or unacceptable toxicity.
Receives: Drug: Arsenic Trioxide; Drug: Gemtuzumab Ozogamicin; Other: Laboratory Biomarker Analysis; Drug: Tretinoin

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Event free survival

    Monitored using a Bayesian time-to-event model.

    Measured: The time from the start of treatment to first documentation of disease relapse or death, assessed up to 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Aug 2011 (estimated)
  2. Study start5 Oct 2011
  3. Primary completion18 Dec 2027 (estimated)
  4. Study completion18 Dec 2027 (estimated)

Resources

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
151 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01409161
Sponsor's protocol ID
2010-0981
Other IDs
NCI-2011-02767, 2010-0981
Status last verified
May 2026
First submitted
2 Aug 2011
Last updated
20 May 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Jen WY, Marvin-Peek J, Kantarjian HM, Alvarado Y, Borthakur G, Jabbour E, Wierda W, Kadia TM, Daver NG, DiNardo CD, Short NJ, Jain N, Ferrajoli A, Kornblau S, Yilmaz M, Ohanian M, McCue D, Burger J, Hammond D, Patel K, Issa GC, Pemmaraju N, Sasaki K, Maiti A, Abbas HA, Chien K, Takahashi K, Haddad F, Bose P, Masarova L, Montalban-Bravo G, Swaminathan M, Brandt M, Pierce S, Garcia-Manero G, Ravandi F. Long-term follow-up of a phase 2 study of all-trans retinoic acid, arsenic trioxide, and gemtuzumab ozogamicin in acute promyelocytic leukemia. Cancer. 2025 Jan 1;131(1):e35662. doi: 10.1002/cncr.35662. Epub 2024 Nov 25.

    PubMed 39584789

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Arsenic Trioxide, Gemtuzumab Ozogamicin and Laboratory Biomarker Analysis in people with Acute Promyelocytic Leukemia With PML-RARA, run by M.D. Anderson Cancer Center at 5 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 20 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Farhad Ravandi-Kashani (713-745-0394, fravandi@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Event free survival (time frame: The time from the start of treatment to first documentation of disease relapse or death, assessed up to 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01409161, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 May 2026.

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