A Study to Find How Safe and Effective GAMMAPLEX® is in Subjects With Chronic Idiopathic Thrombocytopenic Purpura (ITP)

Condition(s) targeted: Chronic Idiopathic Thrombocytopenic Purpura

Intervention: Gammaplex, intravenous immunoglobulin (Biological)

Phase: Phase 3

Status: Completed

Sponsored by: Bio Products Laboratory

Official(s) and/or principal investigator(s):
Tim J Aldwinckle, MD, Principal Investigator, Affiliation: Bio Products Laboratory

To determine if GAMMAPLEX raises the platelet count of subjects with chronic ITP to a threshold of 50 x 109/L, similar to that of published response >60%. Also to assess the safety of GAMMAPLEX and determine if platelet counts are maintained at 50 x 109/L in subjects with chronic ITP for.

Official title: A Phase III, Multicenter, Open-Label Study To Evaluate the Efficacy and Safety of GAMMAPLEX® in Chronic Idiopathic Thrombocytopenic Purpura

Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: The Number of Subjects With Chronic ITP Treated With Gammaplex Whose Platelet Count Reached a Threshold of 50 x 10^9/L.

Secondary outcome:

The Safety of GAMMAPLEX at the Dosage Used in This Study.

Duration of Time That the Platelet Count of Subjects With Chronic ITP Treated With Gammaplex Remained ≥ 50 x 10^9/L.

Detailed description: The primary objective is to determine if BPL's GAMMAPLEX raises the platelet count of subjects with chronic ITP to a threshold of 50 x 109/L, similar to that of published response >60%. The secondary objectives are: 1) to determine the safety of GAMMAPLEX at the dosage used in this study. 2) to determine if GAMMAPLEX maintains platelet counts of ³ 50 x 109/L in subjects with chronic ITP for a period of time similar to that of a published data.

Minimum age: 18 Years. Maximum age: 70 Years. Gender(s): Both.

Criteria:

Inclusion Criteria: 1. Males and females aged between 18 and 70 years. 2. Confirmed diagnosis of chronic ITP of at least 6 months duration. 3. Platelet count of less than or equal to 20 x 10 9/L at enrollment. 4. Absence of other conditions that, in the opinion of the investigator, could cause thrombocytopenia. 5. If subjects were treated with corticosteroids the treatment regimen/dose must have been stable (for a minimum of 2 weeks before screening). The dose of corticosteriod or other immunosuppressant should have remained constant until Day 32. 6) If subjects were being treated with cyclophosphamide, azathioprine or attenuated androgens, the treatment regimen and dose must have been stable for a minimum of 2 months before Day 1. The dose of corticosteriod or other immunosuppressant should have remained constant until Day 32. 7) Splenectomized subjects and both Rh(D)+ and Rh(D)- subjects may be included. 8) The subject has signed an informed consent form (subjects must be at least 18 years old), and/or the subject's legal guardian has signed the informed consent form if indicated 9) If a subject is a female of child-bearing potential, she must have a negative result on a urine-based HCG pregnancy test. 10) If a subject is a female who is or becomes sexually active, she must practice contraception by using a method of proven reliability for the duration of the study. Exclusion Criteria: 1. A history of any severe or anaphylactic reaction to blood or any blood-derived product, or any severe reaction to IGIV or any other IgG preparation. 2. Intolerance to any component of the investigational product. 3. Received any live virus vaccine within the last 3 months prior to Day1. 4. Received an IGIV preparation within 1 month prior to Day 1. 5. Were currently receiving, or has received, any investigational agent within the 1 month prior to Day 1. 6. Received any blood, blood product, or blood derivative within the 1 month prior to Day 1. 7. Received Rituximab within the 3 months before Day 1. 8. Pregnant or nursing. 9. Tested positive for any of the following at screening: HBsAg, NAT for HCV, NAT for HIV, Antibodies to HCV or HIV 1 or 2. 10. Had levels greater than 2. 5 times the upper limit of normal at screening, as defined by the central laboratory, of alanine aminotransferase or aspartate aminotransferase. 11. Had severe renal impairment (defined as serum creatinine greater than 2 times the upper limit of normal or BUN greater than 2. 5 times the upper limit of normal for the range of the laboratory doing the analysis); on dialysis; a history of acute renal failure. 12. Known to have abused alcohol, opiates, psychotropic agents, or other chemicals or drugs within the past 12 months. 13. History of deep vein thrombosis (DVT) or thrombotic complications of IGIV therapy. 14. Any history or sign of hyperviscosity, transient ischemic attack (TIA), stroke, other thromboembolic event, or unstable angina. 15. Suffered from any acute or chronic medical conditions (e. g., renal disease or predisposing conditions for renal disease, coronary artery disease, or protein losing enteropathy) that, in the opinion of the investigator, may interfere with the conduct of the study. 16. An acquired medical condition, such as chronic lymphocytic leukemia, lymphoma, multiple myeloma, chronic or recurrent neutropenia (defined as an absolute neutrophil count (ANC) < 1 x 109/L). 17. Non-controlled arterial hypertension (systolic blood pressure >160 mmHg and/or diastolic blood pressure >90 mmHg). 18. Anemic (hemoglobin <10 g/dL) at screening. 19. Unlikely to adhere to the protocol requirements of the study or is likely to be uncooperative.

Centro de Educación Médica e Investigaciones Clinicas Dr. Norberto Quirno (CEMIC), Buenos Aires C1431FWO, Argentina

Hospital Britanico, Buenos Aires C1280AEB, Argentina

I. A. D. T. (Instituto Argentino de Diagnóstico y Tratamiento), Buenos Aires C1122AAL, Argentina

Hospital Privado de Cordoba, Cordoba X5016KEH, Argentina

J.R. Vidal Hospital, Corrientes, Argentina

CER San Juan, San Juan 5400, Argentina

Sir Ganga Ram Hospital, New Delhi 110060, India

Apollo Hospitals, Hyderabad, Andhra Pradesh 500003, India

Mahavir Hospital, Hyderabad, Andhra Pradesh 500054, India

City Cancer Centre,, Suryarao pet, Vijayawada, Andhra Pradesh 520002, India

M. S Ramaiah Hospital, Karnataka, Bangalore 560054, India

Hospital Churruca, Capital Federal, Buenos Aires C1437JCP, Argentina

Hospital Italiano de La Plata, La Plata, Buenos Aires B1900AXU, Argentina

Mid Florida Hematology & Oncology, Orange City, Florida 32763, United States

Gurukrupa Hospital, Ahmedabad, Gujarat 380061, India

Vedanta, Ahmedabad, Gujarat 380009, India

M S Patel Cancer Centre, Shree Krishna Hospital, Gokal Nagar, Karamsad, Gujarat 388325, India

Rush University Medical Center, Chicago, Illinois 60612, United States

Manipal Hospital, Bangalore, Karnataka 560017, India

Father Muller Medical College Hospital, Mangalore, Karnataka 575002, India

Kasturba Medical College, Manipal Acunova KMC Research Centre, Mangalore, Karnataka 575001, India

Vinaya Hospital & research Centre, Mangalore, Karnataka 5750003, India

Deenanath Mangeshkar Hospital, Pune, Maharashtra 411004, India

Center for Cancer & Blood Disorders, Bethesda, Maryland 20817, United States

New York Hospital / Cornell University, Division of Pediatrics, New York, New York 10021, United States

Department of Pediatrics, SUNY at Stony Brook, Stony Brook, New York 11794-8111, United States

Oregon Health & Science University, Portland, Oregon 97239, United States

S.K. Soni Hospital Sect 5, Jaipur, Rajasthan 300201, India

Instituto de Diagnóstico Hematológico Ambulatorio (IDHEA), Rosario, Sante Fe S2000JKR, Argentina

Cancer Care Centers of South Texas, San Antonio, Texas 78229, United States

Sponsor website

Starting date: September 2007
Last updated: February 26, 2013