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

Primary Purpose

Idiopathic Thrombocytopenic Purpura

Status
Terminated
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
GMA161
GMA161
GMA161
GMA161
GMA161
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Idiopathic Thrombocytopenic Purpura focused on measuring idiopathic thrombocytopenia purpura

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Experimental

Experimental

Experimental

Experimental

Experimental

Arm Label

1

2

3

4

5

Arm Description

Cohort 1

Cohort 2

Cohort 3

Cohort 4

Cohort 5

Outcomes

Primary Outcome Measures

Evaluate safety, tolerability and pharmacokinetics (PK) of single intravenous (IV) infusions of GMA161 in patients with idiopathic thrombocytopenic purpura (ITP)

Secondary Outcome Measures

Full Information

First Posted
October 25, 2005
Last Updated
April 8, 2015
Sponsor
Genzyme, a Sanofi Company
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1. Study Identification

Unique Protocol Identification Number
NCT00244257
Brief Title
Safety Study of GMA161 in Patients With Idiopathic Thrombocytopenic Purpura (ITP)
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)
Study Type
Interventional

2. Study Status

Record Verification Date
April 2015
Overall Recruitment Status
Terminated
Why Stopped
Study terminated due to low enrollment.
Study Start Date
August 2005 (undefined)
Primary Completion Date
July 2008 (Actual)
Study Completion Date
July 2008 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genzyme, a Sanofi Company

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Idiopathic Thrombocytopenic Purpura
Keywords
idiopathic thrombocytopenia purpura

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
10 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Type
Experimental
Arm Description
Cohort 1
Arm Title
2
Arm Type
Experimental
Arm Description
Cohort 2
Arm Title
3
Arm Type
Experimental
Arm Description
Cohort 3
Arm Title
4
Arm Type
Experimental
Arm Description
Cohort 4
Arm Title
5
Arm Type
Experimental
Arm Description
Cohort 5
Intervention Type
Biological
Intervention Name(s)
GMA161
Intervention Description
0.1 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Intervention Type
Biological
Intervention Name(s)
GMA161
Intervention Description
0.3 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Intervention Type
Biological
Intervention Name(s)
GMA161
Intervention Description
0.6 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Intervention Type
Biological
Intervention Name(s)
GMA161
Intervention Description
1.0 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Intervention Type
Biological
Intervention Name(s)
GMA161
Intervention Description
3.0 mg/kg, IV infusion on Day 0 and monitored for 7 days with collection of blood samples
Primary Outcome Measure Information:
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Genzyme, a Sanofi Company
Official's Role
Study Director
Facility Information:
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
City
San Diego
State/Province
California
ZIP/Postal Code
92121
Country
United States
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States

12. IPD Sharing Statement

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

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