Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

Safety Study of GMA161 in Patients With Idiopathic Thrombocytopenic Purpura (ITP)

This study has been terminated.
(Study terminated due to low enrollment.)
Information provided by:
Genzyme, a Sanofi Company Identifier:
First received: October 25, 2005
Last updated: July 29, 2009
Last verified: June 2008

This study is designed to investigate the safety of a single infusion of GMA161 in patients with idiopathic thrombocytopenic purpura, as well as, the way the drug enters and leaves the body. In addition, throughout the study, platelet counts and other blood cell numbers will be measured. NOTE: A decision was made to terminate this study in June 2008 due to low enrollment.

Condition Intervention Phase
Idiopathic Thrombocytopenic Purpura
Biological: GMA161
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase I, Open-Label, Multi-center, Single-Dose, Dose-Escalating, Safety, Tolerability, Immunogenicity, Pharmacokinetics and Pharmacodynamic Study of GMA161 in Patients With Idiopathic Thrombocytopenic Purpura (ITP)

Resource links provided by NLM:

Further study details as provided by Genzyme, a Sanofi Company:

Primary Outcome Measures:
  • Evaluate safety, tolerability and pharmacokinetics (PK) of single intravenous (IV) infusions of GMA161 in patients with idiopathic thrombocytopenic purpura (ITP) [ Time Frame: up to 3 years ] [ Designated as safety issue: No ]

Enrollment: 10
Study Start Date: August 2005
Estimated Study Completion Date: July 2008
Arms Assigned Interventions
Experimental: 1
Cohort 1
Biological: GMA161
0.1 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Experimental: 2
Cohort 2
Biological: GMA161
0.3 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Experimental: 3
Cohort 3
Biological: GMA161
0.6 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Experimental: 4
Cohort 4
Biological: GMA161
1.0 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Experimental: 5
Cohort 5
Biological: GMA161
3.0 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples


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

Inclusion Criteria:

  • Willing and able to provide written informed consent prior to any study-related procedures
  • Chronic idiopathic thrombocytopenic purpura diagnosed for at least 6 months
  • A platelet count of < 100,000/mm^3 on 2 determinations at least 6 weeks apart, including 1 determination within 7 days prior to initiating study treatment. Patients on a stable dose of corticosteroids for 2 weeks prior to study entry and with a platelet count of < 100,000/mm^3 may be enrolled
  • Eastern Cooperative Oncology Group (ECOG) Performance status of 0 or 1, with a life expectancy of at least 6 months

Exclusion Criteria:

  • Women who are pregnant or lactating
  • Women of childbearing potential unless using a medically acceptable contraceptive precaution with the use of spermicide or are sexually inactive
  • Women who plan to become pregnant within 6 months after the screening phase
  • Evidence of excessive bleeding requiring hospitalization within 6 weeks of study entry or a red cell transfusion within 6 weeks of study entry
  • Absolute neutrophil count < 2,000/mm^3
  • Total bilirubin > 2 mg/dL or alanine transaminase (ALT) or aspartate transaminase (AST) > 3 times the upper limit of normal ranges in the institutional laboratory
  • Creatinine > 2 mg/dL
  • History of drug-induced thrombocytopenia, marrow failure syndrome, such as aplastic anemia or myelodysplasia, or thrombocytopenia related to viral or bacterial infection
  • Elevated (≥ 1.5 times the upper limit of normal range) prothrombin time (PT) or partial thromboplastin time (PTT) (other than related to a lupus anticoagulant or contact factor defect)
  • Evidence of active bacterial infection or viral infection
  • Active hemolysis that requires red cell transfusion within 6 weeks of study entry (Patients with evidence of concurrent autoimmune hemolysis [Evan's Syndrome] will be allowed)
  • History of clinically significant cardiac or pulmonary disease
  • History of cancer, other than: basal cell skin cancer, squamous cell skin cancer with no previous chemotherapy treatment or disease-free carcinoma in situ of the cervix, for a minimum of 5 years from the time of Screening
  • HIV infection or acute or persistent hepatitis B and C viral infection (characterized by positive hepatitis B surface antigen (HBsAg), positive anti-hepatitis C virus [HCV] or polymerase chain reaction [PCR] assays for HCV)
  • History of concurrent autoimmune disorders requiring systemic treatment for involvement of organ systems other than cytopenias or thyroid disease
  • Treatment with cyclophosphamide, vincristine, rituximab, or any other monoclonal antibody within 12 weeks of study infusion
  • Treatment with intravenous immunoglobulin (IVIg) within 2 weeks of study drug infusion or Rh(D) immune globulin intravenous within 4 weeks of study drug infusion
  • Treatment with an agent, other than IVIg or corticosteroids, for ITP within 4 weeks of study entry. The dose level of corticosteroids may not be increased or decreased within 2 weeks of study entry
  • Use of any investigational drug within 12 weeks before Screening
  • Other pathology that might interfere with the assessment of the safety or efficacy of the test article or other clinically significant, uncontrolled medical condition that, in the Investigator's opinion, might interfere with the assessment or follow-up.
  • Patients who have been splenectomized within 2 months of study entry.
  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: NCT00244257

United States, California
Los Angeles, California, United States, 90033
San Diego, California, United States, 92121
United States, Maryland
Bethesda, Maryland, United States, 20892
United States, Massachusetts
Boston, Massachusetts, United States, 02115
United States, New York
New York, New York, United States, 10021
United States, Ohio
Cleveland, Ohio, United States, 44195
Sponsors and Collaborators
Genzyme, a Sanofi Company
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

No publications provided

Responsible Party: Medical Monitor, Genzyme Corporation Identifier: NCT00244257     History of Changes
Obsolete Identifiers: NCT00245999
Other Study ID Numbers: GMA16100104
Study First Received: October 25, 2005
Last Updated: July 29, 2009
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Autoimmune Diseases
Blood Coagulation Disorders
Blood Platelet Disorders
Hematologic Diseases
Hemorrhagic Disorders
Immune System Diseases
Pathologic Processes
Signs and Symptoms
Skin Manifestations
Thrombotic Microangiopathies processed this record on March 01, 2015