Clinical trial NCT01009931: Phase II Study of TPA Plus Dexamethasone & CMT in Hematologic Malignancies
Official title: A Phase II Study of 12-O-tetradecanoylphorbol-13-acetate (TPA) Plus Dexamethasone & Choline Magnesium Trisalicylate in the Treatment of Patients With Relapsed/Refractory Acute Myelogenous Leukemia
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Leukemia
Keywords: Acute Myelogenous Leukemia
Relapsed AML
Refractory AML
Lead sponsor: Rutgers, The State University of New Jersey
Sponsor class: OTHER
Collaborators: name: Rutgers Cancer Institute of New Jersey
name: National Cancer Institute (NCI)
name: Biosuccess Biotech Co., Ltd.
First posted: 2009-11-09
Enrollment: 1
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This phase II trial is studying the side effects and how well giving tetradecanoylphorbol acetate together with dexamethasone and choline magnesium trisalicylate works in treating patients with relapsed or refractory acute myeloid leukemia.


Eligibility criteria: Inclusion Criteria

* Must have a histologically documented relapsed/refractory AML for which there is no standard therapy that has been demonstrated to have curative or palliative potential.
* ECOG performance status of 0-2.
* Must be 18 years or older.
* Estimated life expectancy \> 1 month.
* Laboratory data:

  * total bilirubin ≤ 1.5 x upper limit of normal unless due to Gilbert's syndrome
  * serum creatinine ≤ 2.0 mg/dl
  * AST ≤ 3.0 x upper limit of normal
  * Cardiac ejection fraction \> 40%
  * FEV1.0 \> 50% predicted
* Prior therapy: \> 3 weeks since chemotherapy, biological therapy or radiation; anticipated maximum hematological improvement since last dose of chemotherapy. (Concurrent hydroxyurea administration will be allowed to control WBC count, platelet count, or symptoms).
* No active infections.
* Negative pregnancy test for women of childbearing potential.
* No uncontrolled psychiatric illness or medical illness that the principal investigator feels will compromise the patient's tolerance of the study medication.
* Must provide informed consent.

Exclusion Criteria

* Patients with an allergy to proton pump inhibitors, required for GI prophylaxis; or salicylates are excluded.
* Pregnant or lactating women
* Age \<18 years. Because no dosing or adverse event data are currently available on the use of TPA alone or in combination with dexamethasone in patients \< 18 years of age, children are excluded from this study but will be eligible for future pediatric Phase II combination trials.
* The effects of TPA on the developing human fetus are unknown. For this reason, women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation and for 10 weeks after. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. Should the female partner of a participant in this study become pregnant or suspect she is pregnant during this study, the PI of this study will be available to provide advice about further medical/obstetric care/referral for the female partner.
* Patients with active CNS involvement (documented by radiographic lesions and/or malignant cells in the CSF) will be excluded from this clinical trial because of their poor prognosis and because they often develop progressive neurologic dysfunction that would confound the evaluation of neurologic and other adverse events.
* Patients with treatment of any other investigational drug within the last 30 days prior to entering the study.

Primary outcomes:
measure: Response Rate > 20% for 12-O-tetradecanoylphorbol-13- Acetate (TPA)+ Dexamethasone + Choline Magnesium Trisalicylate(Trilisate)
timeFrame: 42 months
measure: Grade 3 and 4 Non-hematologic Treatment-related Toxicity Rates < 25%
timeFrame: 43 months
Secondary outcomes:
measure: Effects of Treatment on Immunophenotype, Signaling Profile, and Nuclear NF-kB Expression
timeFrame: 48 months
Other outcomes:
Locations:
city: New Brunswick
country: United States
facility: Rutgers Cancer Institute of New Jersey
state: New Jersey
Study officials:
affiliation: Rutgers Cancer Institute of New Jersey
name: Roger Strair, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: