NCT01404949

Brief Summary

The purpose of this study is to find what effects, good and/or bad, treatment with two drugs has on leukemia. The first medicine is tretinoin (also called all-trans retinoic acid, ATRA, or Vesanoid). It is an approved medicine that causes the leukemia cells in APL to mature. It is related to vitamin A. The second is arsenic trioxide (Trisenox). It is an approved medicine for APL that comes back after earlier treatment. APL is most often treated with tretinoin and standard chemotherapy drugs. These chemotherapy drugs can cause infection and bleeding. They can also damage the heart and normal bone marrow cells. This can lead to a second leukemia years later. In this study, the investigators are using tretinoin and arsenic trioxide together. Both drugs work to treat APL. They have been used together in only a limited number of people. The investigators want to use these drugs together to reduce the amount of standard chemotherapy and decrease side effects. The patient will receive standard chemotherapy with a drug called idarubicin only if they have a higher chance of the leukemia coming back or a higher risk of side effects.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
17

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jul 2011

Longer than P75 for phase_2

Geographic Reach
2 countries

12 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2011

Completed
26 days until next milestone

First Submitted

Initial submission to the registry

July 27, 2011

Completed
1 day until next milestone

First Posted

Study publicly available on registry

July 28, 2011

Completed
9.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2021

Completed
10 months until next milestone

Results Posted

Study results publicly available

November 30, 2021

Completed
Last Updated

November 30, 2021

Status Verified

February 1, 2021

Enrollment Period

9.6 years

First QC Date

July 27, 2011

Results QC Date

November 2, 2021

Last Update Submit

November 2, 2021

Conditions

Keywords

Tretinoinall-trans retinoic acidATRAVesanoidArsenic TrioxideATOTrisenox11-040

Outcome Measures

Primary Outcomes (1)

  • To Determine the Rate of Molecular Remission

    after induction with combined tretinoin and ATO (along with idarubicin in patients with high-risk disease or who develop leukocytosis) in APL.

    4 years

Secondary Outcomes (6)

  • Participants Who Experienced a Complete Remission

    4 years

  • To Determine the Proportion of Patients in Molecular Remission

    4 years

  • To Determine the Disease-free and Event-free Survival of Patients

    4 years

  • To Determine the Toxicity of This Treatment Program

    4 years

  • To Characterize the Differentiation of APL Cells During Treatment

    4 years

  • +1 more secondary outcomes

Study Arms (1)

Tretinoin and Arsenic Trioxide

EXPERIMENTAL

This is a multicenter, phase II trial to study the efficacy of combined tretinoin and ATO in the treatment of newly diagnosed APL in an effort to reduce or eliminate the amount of standard chemotherapy required for long-term remission.

Drug: Tretinoin and Arsenic Trioxide

Interventions

See Detailed Description

Tretinoin and Arsenic Trioxide

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Previously untreated patients with a morphologic diagnosis of APL, confirmed by demonstration of t(15;17) using conventional cytogenetics OR florescence in situ hybridization (FISH), OR a positive RT-PCR assay for PML-RAR at the subject's local institution.
  • Age ≥18 years. Karnofsky performance status of ≥ 60%.
  • Adequate renal function as demonstrated by a serum creatinine ≤ 2.0 mg/dl or a creatinine clearance of \> 60 ml/min.
  • Adequate hepatic function as demonstrated by a bilirubin \< 2.0 mg/dl (unless attributable to Gilbert's disease) and an alkaline phosphatase, AST, and ALT ≤ 2.5 times the upper limit of normal.
  • Normal cardiac function as demonstrated by a left ventricular ejection fraction ≥ 50% on echocardiogram or MUGA scan.
  • QTc ≤ 500 msec on baseline ECG.
  • Negative serum pregnancy test in women of childbearing potential.
  • Ability to swallow oral medication.
  • Men and women of child-bearing potential must be willing to practice an effective method of birth control during treatment and at least 4 months after treatment is finished.
  • Patients with central nervous system involvement by APL are eligible and may receive concomitant treatment with radiation therapy and/or intrathecal chemotherapy in accordance with standard medical practice.

You may not qualify if:

  • Previous treatment for APL, except tretinoin, which may be given for up to 7 days prior to study entry.
  • Active serious infections not controlled by antibiotics.
  • Pregnant women or women who are breast-feeding.
  • Concurrent active malignancy requiring immediate therapy.
  • Clinically significant cardiac disease (NY Heart Association Class III or IV), including chronic arrhythmias, or pulmonary disease.
  • Other serious or life-threatening conditions deemed unacceptable by the principal investigator.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

University of Southern California

Los Angeles, California, 90033, United States

Location

Northwestern University

Evanston, Illinois, 60208, United States

Location

National Heart, Lung, and Blood Institute (NIH)

Bethesda, Maryland, 20824, United States

Location

Memorial Sloan Kettering at Basking Ridge

Basking Ridge, New Jersey, 07920, United States

Location

Memorial Sloan Kettering Monmouth

Middletown, New Jersey, 07748, United States

Location

Memorial Sloan Kettering Cancer Center @ Suffolk

Commack, New York, 11725, United States

Location

Memorial Sloan Kettering Westchester

Harrison, New York, 10604, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

New York Presbyterian Hospital-Weill Medical College of Cornell University

New York, New York, 10065, United States

Location

Memorial Sloan Kettering at Mercy Medical Center

Rockville Centre, New York, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Princess Margaret Hospital/Ontario Cancer Institute

Toronto, Ontario, Canada

Location

Related Links

MeSH Terms

Conditions

Leukemia, Promyelocytic, Acute

Interventions

TretinoinArsenic Trioxide

Condition Hierarchy (Ancestors)

Leukemia, Myeloid, AcuteLeukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Vitamin ARetinoidsCarotenoidsPolyenesAlkenesHydrocarbons, AcyclicHydrocarbonsOrganic ChemicalsCyclohexenesCyclohexanesCycloparaffinsHydrocarbons, AlicyclicHydrocarbons, CyclicTerpenesDiterpenesPigments, BiologicalBiological FactorsArsenicalsInorganic ChemicalsOxidesOxygen Compounds

Results Point of Contact

Title
Dr. Jae Park, MD
Organization
Memorial Sloan Kettering Cancer Center

Study Officials

  • Jae Park, MD

    Memorial Sloan Kettering Cancer Center

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 27, 2011

First Posted

July 28, 2011

Study Start

July 1, 2011

Primary Completion

February 1, 2021

Study Completion

February 1, 2021

Last Updated

November 30, 2021

Results First Posted

November 30, 2021

Record last verified: 2021-02

Locations