- Solid Tumors
- Pipeline Molecules
- Alliance Partners
Our mission is to provide healthcare professionals with unbiased clinical research information, easily.
Currently, you can access the following clinical trials being conducted worldwide:
Clinicaltrials.gov identifier NCT03515512
Recruitment Status Recruiting
First Posted May 3, 2018
Last update posted October 13, 2020
This research study is studying a targeted therapy drug as a possible treatment for IDH2 mutant acute myeloid leukemia or chronic myelomonocytic leukemia while undergoing hematopoietic stem cell transplantation. The drug involved in this study is: -Enasidenib.
This research study is a Phase I clinical trial, which tests the safety of an investigational drug and also tries to define the appropriate dose of the investigational drug to use for further studies. "Investigational" means that the drug is being studied. The FDA (the U.S. Food and Drug Administration) has approved enasidenib as a treatment option for some cancers, but not for the specific indication under study with this protocol. Enasidenib is currently used to treat AML with an IDH2 mutation that has come back or has not improved after previous AML treatment. This study is examining whether or not enasidenib may be beneficial and well-tolerated as an agent to prevent the relapse of IDH2-mutated AML or other myeloid neoplasms after participants have undergone hematopoietic stem cell transplantation (HSCT). IDH2 is an enzyme that, when mutated, can overproduce metabolites and compounds that contribute to the growth of tumors and cancerous cells. Enasidenib may help block the over production of these substances. There is an FDA-approved test available to detect IDH2 mutations in patients with AML, but for the purposes of participation in this clinical trial, an investigational test may be used to determine the presence of an IDH2 mutation. In this research study, the investigators are: - Looking for the maximum dose of enasidenib that individuals can take without experiencing severe side effects following HSCT. - Looking at how often Graft-Versus-Host-Disease (GVHD) occurs in participants taking enasidenib. GVHD is a complication of transplant. - Assessing the rates of relapse for participants taking enasidenib after HSCT.
Enasidenib will be administered orally once daily in 28-day cycles
Enasidenib may help block the over production of IDH2, which when mutated, can overproduce metabolites and compounds that contribute to the growth of tumors and cancerous cells
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, , Learn About Clinical Studies.-->
- Pathologically confirmed diagnosis of IDH2-mutant acute myeloid leukemia (AML),
myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML).
- IDH2 mutations will include any IDH2 R140 or R172 alterations
- Eligibility and enrollment will be based on local IDH2 mutational testing performed at
any center. The presence of an IDH2 mutation at the time of initial diagnosis or any
other time thereafter is necessary and sufficient. The presence of an IDH2 mutation at
time of enrollment is not necessary for the purposes of eligibility.
- Between the ages of 18 and 75 years
- Will undergo first allogeneic hematopoietic stem cell transplantation (HSCT) for their
malignancy. Conditioning may have been either conventional myeloablative (MAC) or
reduced intensity conditioning (RIC).
- HSCT Donor will be one of the following:
- 5/6 or 6/6 (HLA-A, B, DR) matched related donor
- 7/8 or 8/8 (HLA-A, B, DR, C) matched unrelated donor. Matching in the unrelated
setting must be at the allele level.
- Haploidentical related donor, defined as ≥ 3/6 (HLA-A, B, DR) matched --≥ 4/6
(HLA-A, B, DR) umbilical cord blood (UCB). Matching in the UCB setting is at the
antigen level. Recipients may receive either one or two UCB units. In the case of
2 UCB units, both units must have been at least 4/6 matched with the recipient.
- ECOG performance status ≤ 2
- Participants must have normal organ and marrow function as defined below:
- Absolute neutrophil count ≥ 1000/µL without growth factor support (e.g. GCSF) in
the previous 7 days
- Platelet count ≥ 50,000/µL without transfusional support in the previous 7 days
- AST (SGOT), ALT (SGPT) and Alkaline phosphatase < 3x institutional upper limit of
- Direct bilirubin < 2.0 mg/dL
- Calculated creatinine clearance ≥ 40 mL/min (Cockcroft-Gault formula)
- LVEF must be equal to or greater than 50%, as measured by MUGA scan or echocardiogram
- Female patients of childbearing potential must have a negative pregnancy test, as
measured by serum or urine testing
- The effects of enasidenib on the developing human fetus are unknown. For this reason
women of child-bearing potential and men must agree to use adequate contraception
(hormonal or barrier method of birth control; abstinence) during the entire study
treatment period and through 6 months after the last dose of treatment
- Ability to understand and the willingness to sign a written informed consent document
- Prior allogeneic hematopoietic stem cell transplants.
- Evidence of relapsed/recurrent/residual disease as assessed by bone marrow aspirate
and biopsy performed within 42 days prior to study entry.
- History of other malignancy(ies) unless
- the participant has been disease-free for at least 5 years and is deemed by the
investigator to be at low risk of recurrence of that malignancy, or
- the only prior malignancy was cervical cancer in situ and/or basal cell or
squamous cell carcinoma of the skin
- Known diagnosis of active hepatitis B or hepatitis C
- Current or history of congestive heart failure New York Heart Association (NHYA) class
3 or 4, or any history of documented diastolic or systolic dysfunction (LVEF 180 mmHg or diastolic BP >
- QTc interval (i.e., Friderica's correction [QTcF]) ≥ 450 ms or other factors that
increase the risk of QT prolongation or arrhythmic events (e.g., heart failure,
hypokalemia, family history of long QT interval syndrome) at screening
- Concomitant receipt of the following sensitive CYP substrate medications that have a
narrow therapeutic range (unless the participant can be transferred to other
medications at least 5 half-lives prior to the start of study treatment): paclitaxel
and docetaxel (CYP2C8), phenytoin (CYP2C9), S-mephenytoin (CYP2C19), thioridazine
(CYP2D6), theophylline, and tizanidine (CYP1A2)
- Concomitant receipt of the breast cancer resistance protein (BCRP)
transporter-sensitive substrate rosuvastain (unless the participant can be transferred
to another medication at least 5 half-lives prior to the start of study treatment)
- Uncontrolled intercurrent illness that would limit compliance with study requirements.
- HIV-positive participants on combination antiretroviral therapy are ineligible because
of the potential for pharmacokinetic interactions with study drug. In addition, these
participants are at increased risk of lethal infections when treated with
Contact: Amir T Fathi, MD 617-724-1124 email@example.com
United States, Maryland
Johns Hopkins Cancer Center
United States, Massachusetts
Massachusetts General Hospital
United States, Massachusetts
Dana Farber Cancer Institute
United States, Ohio
Ohio State University Comprehensive Cancer Center
Massachusetts General Hospital
Principal Investigator: Amir t Fathi, MD Massachusetts General Hospital