Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

Phase 1 Study to Evaluate MEDI4736

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2014 by MedImmune LLC
Information provided by (Responsible Party):
MedImmune LLC Identifier:
First received: April 14, 2014
Last updated: July 21, 2014
Last verified: July 2014

This is a multicenter, open-label, Phase 1 study to assess the safety and antitumor activity of MEDI4736 in subjects with myelodysplastic syndrome.

Condition Intervention Phase
Myelodysplastic Syndrome
Biological: MEDI4736
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 1 Study to Evaluate the Safety and Tolerability of MEDI4736 in Subjects With Myelodysplastic Syndrome After Treatment With Hypomethylating Agents

Resource links provided by NLM:

Further study details as provided by MedImmune LLC:

Primary Outcome Measures:
  • Subject's safety where no more than one out of six subjects experience DLTs at a given dose. [ Time Frame: 455 days ] [ Designated as safety issue: Yes ]
    DLT assessment will be done by reviewing adverse events (AEs), serious adverse events (SAEs), laboratory evaluations, vital signs, physical examinations, and electrocardiogram (ECG) results.

Secondary Outcome Measures:
  • Clinical outcome in terms of response: duration of response [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    As defined by IWG 2006 MDS response criteria

  • Clinical outcome in terms of response: transfusion requirements [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    As defined by IWG 2006 MDS response criteria and incidence of transfusions.

  • Clinical outcome in terms of response: progression-free survival (PFS) [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    As defined by IWG 2006 MDS response criteria

  • Clinical outcome in terms of response: survival (OS) [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    As defined by IWG 2006 MDS response criteria and collection of survival data

  • Pharmacokinetics: MEDI4736 concentrations in serum: peak concentration [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    Peak concentration of MEDI4736 in serum

  • Pharmacokinetics: MEDI4736 concentrations in serum: area under the concentration-time curve [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    Analysis of area under the concentration-time curve

  • Pharmacokinetics: MEDI4736 concentration in serum: clearance [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    Rate of MEDI4736 clearance

  • Pharmacokinetics: MEDI4736 concentration in serum: terminal half-life [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    MEDI4736 concentration terminal half-life

  • Immunogenicity [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    Determine by number of subjects who develop ADA (anti-drug antibody).

  • AML (Acute Myeloid Leukemia) transformation [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Rate of AML transformation.

  • Health-related quality of life (QOL): disease- and treatment-related symptoms [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Analysis of reporting of disease- and treatment-related symptoms

  • Health-related quality of life (QOL): pain [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Analysis of incidence of pain reporting

  • Health-related quality of life (QOL): health status [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Analysis of reporting of health status.

Estimated Enrollment: 70
Study Start Date: May 2014
Estimated Study Completion Date: October 2015
Estimated Primary Completion Date: October 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: MEDI4736
Evaluate MEDI4736 in MDS
Biological: MEDI4736
MEDI4736 will be administered by IV infusion

Detailed Description:

A dose-escalation and dose-expansion study of MEDI4736 (a monoclonal antibody that targets programmed cell death-1 ligand 1 [PD-L1]) to evaluate the safety, tolerability, PK, IM, and antitumor activity of MEDI4736 in adult patients with myelodysplastic syndrome.


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

Adult male or female subjects with pathologically confirmed MDS who failed to respond, relapsed after an initial response, or were unable to tolerate hypomethylating agents, ECOG performance status of 0 - 2, and adequate organ and marrow function.

Exclusion Criteria:

Concurrent chemotherapy, immunotherapy, biologic, or hormonal therapy for cancer treatment, prior MAb against CTLA-4, PD-1, or PD-L1, alllogenic or haploidentical transplant, current immunosuppressive medication or autoimmune or inflammatory disease.

  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02117219

Contact: Heidi Kacprowicz 301-398-0000

United States, Illinois
Research Site Recruiting
Chicago, Illinois, United States
United States, Massachusetts
Research Site Recruiting
Boston, Massachusetts, United States
United States, Michigan
Research Site Recruiting
Detroit, Michigan, United States
United States, South Carolina
Research Site Recruiting
Greenville, South Carolina, United States
United States, Texas
Research Site Recruiting
Houston, Texas, United States
Sponsors and Collaborators
MedImmune LLC
Study Director: Joyson Karakunnel, MD MedImmune LLC
  More Information

No publications provided

Responsible Party: MedImmune LLC Identifier: NCT02117219     History of Changes
Other Study ID Numbers: D4190C00007
Study First Received: April 14, 2014
Last Updated: July 21, 2014
Health Authority: United States: Food and Drug Administration
European Union: European Medicines Agency

Keywords provided by MedImmune LLC:
Myelodysplastic syndrome

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Bone Marrow Diseases
Hematologic Diseases
Pathologic Processes
Precancerous Conditions processed this record on November 24, 2014