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

April 8, 2015 updated by: Genzyme, a Sanofi Company

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)

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

10

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90033
      • San Diego, California, United States, 92121
    • Maryland
      • Bethesda, Maryland, United States, 20892
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
    • New York
      • New York, New York, United States, 10021
    • Ohio
      • Cleveland, Ohio, United States, 44195

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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
Biological: GMA161
0.3 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
0.6 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
1.0 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
3.0 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Experimental: 2
Cohort 2
Biological: GMA161
0.1 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
0.3 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
0.6 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
1.0 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
3.0 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Experimental: 4
Cohort 4
Biological: GMA161
0.1 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
0.3 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
0.6 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
1.0 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
3.0 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Experimental: 3
Cohort 3
Biological: GMA161
0.1 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
0.3 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
0.6 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
1.0 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
3.0 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Experimental: 5
Cohort 5
Biological: GMA161
0.1 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
0.3 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
0.6 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
1.0 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Biological: GMA161
3.0 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
up to 3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Genzyme, a Sanofi Company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2005

Primary Completion (Actual)

July 1, 2008

Study Completion (Actual)

July 1, 2008

Study Registration Dates

First Submitted

October 25, 2005

First Submitted That Met QC Criteria

October 25, 2005

First Posted (Estimate)

October 26, 2005

Study Record Updates

Last Update Posted (Estimate)

April 9, 2015

Last Update Submitted That Met QC Criteria

April 8, 2015

Last Verified

April 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GMA16100104

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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