Clinical trial NCT00504075: A Study to Find How Safe and Effective GAMMAPLEX® is in Subjects With Chronic Idiopathic Thrombocytopenic Purpura (ITP)
Official title: A Phase III, Multicenter, Open-Label Study To Evaluate the Efficacy and Safety of GAMMAPLEX® in Chronic Idiopathic Thrombocytopenic Purpura
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Chronic Idiopathic Thrombocytopenic Purpura
Keywords: Idiopathic Thrombocytopenic Purpura
Bleeding disorders
Immune System and Disorders
Lead sponsor: Bio Products Laboratory
Sponsor class: OTHER
First posted: 2007-07-19
Enrollment: 35
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

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.

Eligibility 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.

Primary outcomes:
measure: The Number of Subjects With Chronic ITP Treated With Gammaplex Whose Platelet Count Reached a Threshold of 50 x 10^9/L.
timeFrame: 9 days
Secondary outcomes:
measure: The Safety of GAMMAPLEX at the Dosage Used in This Study.
timeFrame: AEs were documented from the date the informed consent form was signed until the End of Study visit on Day 90.
measure: Duration of Time That the Platelet Count of Subjects With Chronic ITP Treated With Gammaplex Remained ≥ 50 x 10^9/L.
timeFrame: Days 1, 2, 3, 5, 9, 14, 21, 32.
Other outcomes:
Locations:
city: Orange City
country: United States
facility: Mid Florida Hematology & Oncology
state: Florida
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Bethesda
country: United States
facility: Center for Cancer & Blood Disorders
state: Maryland
city: New York
country: United States
facility: New York Hospital / Cornell University, Division of Pediatrics
state: New York
city: Stony Brook
country: United States
facility: Department of Pediatrics, SUNY at Stony Brook
state: New York
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: San Antonio
country: United States
facility: Cancer Care Centers of South Texas
state: Texas
city: Capital Federal
country: Argentina
facility: Hospital Churruca
state: Buenos Aires
city: La Plata
country: Argentina
facility: Hospital Italiano de La Plata
state: Buenos Aires
city: Rosario
country: Argentina
facility: Instituto de Diagnóstico Hematológico Ambulatorio (IDHEA)
state: Sante Fe
city: Buenos Aires
country: Argentina
facility: I. A. D. T. (Instituto Argentino de Diagnóstico y Tratamiento)
city: Buenos Aires
country: Argentina
facility: Hospital Britanico
city: Buenos Aires
country: Argentina
facility: Centro de Educación Médica e Investigaciones Clinicas Dr. Norberto Quirno (CEMIC)
city: Cordoba
country: Argentina
facility: Hospital Privado de Cordoba
city: Corrientes
country: Argentina
facility: J.R. Vidal Hospital
city: San Juan
country: Argentina
facility: CER San Juan
city: Hyderabad
country: India
facility: Apollo Hospitals
state: Andhra Pradesh
city: Hyderabad
country: India
facility: Mahavir Hospital
state: Andhra Pradesh
city: Suryarao pet, Vijayawada
country: India
facility: City Cancer Centre,
state: Andhra Pradesh
city: Karnataka
country: India
facility: M. S Ramaiah Hospital
state: Bangalore
city: Ahmedabad
country: India
facility: Vedanta
state: Gujarat
city: Ahmedabad
country: India
facility: Gurukrupa Hospital
state: Gujarat
city: Gokal Nagar, Karamsad
country: India
facility: M S Patel Cancer Centre, Shree Krishna Hospital
state: Gujarat
city: Bangalore
country: India
facility: Manipal Hospital
state: Karnataka
city: Mangalore
country: India
facility: Vinaya Hospital & research Centre
state: Karnataka
city: Mangalore
country: India
facility: Kasturba Medical College, Manipal Acunova KMC Research Centre
state: Karnataka
city: Mangalore
country: India
facility: Father Muller Medical College Hospital
state: Karnataka
city: Pune
country: India
facility: Deenanath Mangeshkar Hospital
state: Maharashtra
city: Jaipur
country: India
facility: S.K. Soni Hospital Sect 5
state: Rajasthan
city: New Delhi
country: India
facility: Sir Ganga Ram Hospital
Study officials:
affiliation: Bio Products Laboratory
name: Tim J Aldwinckle, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Dash CH, Gillanders KR, Stratford Bobbitt ME, Gascoigne EW, Leach SJ. Safety and efficacy of Gammaplex(R) in idiopathic thrombocytopenic purpura (ClinicalTrials.gov--NCT00504075). PLoS One. 2014 Jun 3;9(6):e96600. doi: 10.1371/journal.pone.0096600. eCollection 2014.