Clinical trial NCT02732184: A Multiple Dose, Dose Escalation Trial of AEB1102 in Patients With AML or MDS
Official title: A Phase 2 Dose-Escalation Study of AEB1102 (Co-ArgI-PEG) in Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Refractory to Hypomethylating Agents
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Acute Myeloid Leukemia
Myelodysplastic Syndrome
Keywords: AML
MDS
Lead sponsor: Aeglea Biotherapeutics
Sponsor class: INDUSTRY
First posted: 2016-04-08
Enrollment: 29
Sex: ALL
Minimum age (years): 18
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is the first study of the safety of increasing dose levels of AEB1102 in patients with Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome. The study will also evaluate the amounts of AEB1102 in blood, the effects of AEB1102 on blood amino acid levels and the antitumor effects of AEB1102.


Eligibility criteria: Inclusion Criteria:

* Willing and able to provide informed consent
* Age 18 and older
* Diagnosis of AML or MDS according to the WHO criteria
* AML relapsed or refractory to at least one attempt at induction or subjects not candidates for aggressive induction regimens
* MDS refractory to treatment with HMA therapy or with recurrence or progression of MDS following a response to an HMA
* Has adequate organ function: AST and ALT \< 3X the ULN, serum bilirubin \< 2X the ULN, serum creatinine of \< 2 mg/dL, or a calculated creatinine clearance of \> 50 mL/minute
* ECOG Performance Score of 0 -2
* Recovered from the effects of any prior systemic therapy, radiotherapy or surgery
* Willing to use physician approved birth control method

Exclusion Criteria:

* Current CNS Leukemia
* Acute promyelocytic leukemia or AML with a t(15;17) (q22;q12) cytogenetic abnormality or Bcr/Abl positive leukemia
* \< 60 days from ASCT; has chronic graft-versus host disease (GVHD) or requires continued treatment with systemic immunosuppressive agents
* Uncontrolled infection
* Known HIV, hepatitis B or hepatitis C.
* Other active malignancy that requires therapy
* If female, is lactating or breast feeding
* Hypersensitivity to PEG or other component of AEB1102 (Co-ArgI-PEG)

Primary outcomes:
measure: Maximum Tolerated Dose and Recommended Phase 2 Dose
timeFrame: 4 weeks
Secondary outcomes:
measure: Safety profile (changes in physical exam, laboratory measures, reported adverse events)
timeFrame: 4 Weeks
Other outcomes:
Locations:
city: Ann Arbor
country: United States
facility: Comprehensive Cancer Center at University of Michigan
state: Michigan
city: Saint Louis
country: United States
facility: Washington University Medical School
state: Missouri
city: Cleveland
country: United States
facility: University Hospitals Case Medical Center
state: Ohio
city: Cleveland
country: United States
facility: The Cleveland Clinic
state: Ohio
city: Nashville
country: United States
facility: Vanderbilt-Ingram Cancer Center
state: Tennessee
city: Dallas
country: United States
facility: Baylor Scott & White
state: Texas
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Toronto
country: Canada
facility: Princess Margaret Cancer Centre
state: Ontario
city: Montreal
country: Canada
facility: Jewish General Hospital
state: Quebec
Study officials:
affiliation: Aeglea BioTherapeutics, Inc.
name: Jim Joffrion
role: STUDY_DIRECTOR
References: