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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Gammaplex (intravenous immunoglobulin) | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Gammaplex, intravenous immunoglobulin | Biological | Dosage form: Gammaplex® is a sterile liquid of 5 % w/v normal immunoglobulin. Gammaplex® contains 5 g/100 mL of human normal immunoglobulin (i.e. 50 g/L, of which virtually 100% is IgG). The first course of GAMMAPLEX will be administered as an intravenous infusion of 1 g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen may be administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX. |
| Measure | Description | Time Frame |
|---|---|---|
| The Number of Subjects With Chronic ITP Treated With Gammaplex Whose Platelet Count Reached a Threshold of 50 x 10^9/L. | The number of subjects with chronic ITP treated following treatment with Gammaplex who attained a platelet count of ≥ 50 x 10^9/L by Day 9. | 9 days |
| Measure | Description | Time Frame |
|---|---|---|
| The Safety of GAMMAPLEX at the Dosage Used in This Study. | The safety variables used to assess safety were the following:
|
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Inclusion Criteria:
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:
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| Name | Affiliation | Role |
|---|---|---|
| Tim J Aldwinckle, MD | Bio Products Laboratory | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mid Florida Hematology & Oncology | Orange City | Florida | 32763 | United States | ||
| Rush University Medical Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 24892422 | Derived | 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. |
| Label | URL |
|---|---|
| Sponsor website | View source |
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This was an open label study. All enrolled subjects received study medication.
First enrollment: 04 September 2007. Last Subject completed: 05 August 2011. Thirty-five sites in the United States, India and Argentina, all hospital clinics.
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| ID | Title | Description |
|---|---|---|
| FG000 | GAMMAPLEX | The first course of GAMMAPLEX was administered as an intravenous infusion of 1g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen was administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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|
|
| AEs were documented from the date the informed consent form was signed until the End of Study visit on Day 90. |
| Duration of Time That the Platelet Count of Subjects With Chronic ITP Treated With Gammaplex Remained ≥ 50 x 10^9/L. | Blood samples were collected to measure platelet counts and the duration of time for which the platelet count remained ≥50 x 10^9/L was measured. | Days 1, 2, 3, 5, 9, 14, 21, 32. |
| Chicago |
| Illinois |
| 60612 |
| United States |
| 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 |
| Cancer Care Centers of South Texas | San Antonio | Texas | 78229 | United States |
| Hospital Churruca | Capital Federal | Buenos Aires | C1437JCP | Argentina |
| Hospital Italiano de La Plata | La Plata | Buenos Aires | B1900AXU | Argentina |
| CER San Juan | San Juan | San Juan Province | 5400 | Argentina |
| Instituto de Diagnóstico Hematológico Ambulatorio (IDHEA) | Rosario | Sante Fe | S2000JKR | Argentina |
| I. A. D. T. (Instituto Argentino de Diagnóstico y Tratamiento) | Buenos Aires | C1122AAL | Argentina |
| Hospital Britanico | Buenos Aires | C1280AEB | Argentina |
| Centro de Educación Médica e Investigaciones Clinicas Dr. Norberto Quirno (CEMIC) | Buenos Aires | C1431FWO | Argentina |
| J.R. Vidal Hospital | Corrientes | Argentina |
| Hospital Privado de Cordoba | Córdoba | X5016KEH | Argentina |
| 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 |
| Vedanta | Ahmedabad | Gujarat | 380009 | India |
| Gurukrupa Hospital | Ahmedabad | Gujarat | 380061 | India |
| M S Patel Cancer Centre, Shree Krishna Hospital | Gokal Nagar, Karamsad | Gujarat | 388325 | India |
| Manipal Hospital | Bangalore | Karnataka | 560017 | India |
| Vinaya Hospital & research Centre | Mangalore | Karnataka | 5750003 | India |
| Kasturba Medical College, Manipal Acunova KMC Research Centre | Mangalore | Karnataka | 575001 | India |
| Father Muller Medical College Hospital | Mangalore | Karnataka | 575002 | India |
| Deenanath Mangeshkar Hospital | Pune | Maharashtra | 411004 | India |
| S.K. Soni Hospital Sect 5 | Jaipur | Rajasthan | 300201 | India |
| Sir Ganga Ram Hospital | New Delhi | 110060 | India |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | GAMMAPLEX | The first course of GAMMAPLEX was administered as an intravenous infusion of 1g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen was administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| |||||||||||||||||||||||
| Age Continuous | Mean | Standard Deviation | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Region of Enrollment | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | The Number of Subjects With Chronic ITP Treated With Gammaplex Whose Platelet Count Reached a Threshold of 50 x 10^9/L. | The number of subjects with chronic ITP treated following treatment with Gammaplex who attained a platelet count of ≥ 50 x 10^9/L by Day 9. | Any subject who started treatment with GAMMAPLEX and whose central laboratory results taken prior to infusion on Day 1 were within specified protocol parameters, was included in the intent-to-treat (ITT) population for analysis of efficacy and safety. | Posted | Number | participants | 9 days |
|
|
| ||||||||||||||||||||||||||
| Secondary | The Safety of GAMMAPLEX at the Dosage Used in This Study. | The safety variables used to assess safety were the following:
| Any subject who started treatment with GAMMAPLEX and whose central laboratory results taken prior to infusion on Day 1 were within specified protocol parameters, was included in the intent-to-treat (ITT) population for analysis of efficacy and safety. | Posted | Number | 95% Confidence Interval | % of subjects with product related SAEs | AEs were documented from the date the informed consent form was signed until the End of Study visit on Day 90. |
|
| ||||||||||||||||||||||||||
| Secondary | Duration of Time That the Platelet Count of Subjects With Chronic ITP Treated With Gammaplex Remained ≥ 50 x 10^9/L. | Blood samples were collected to measure platelet counts and the duration of time for which the platelet count remained ≥50 x 10^9/L was measured. | Any subject who started treatment with GAMMAPLEX and whose central laboratory results taken prior to infusion on Day 1 were within specified protocol parameters, was included in the intent-to-treat (ITT) population for analysis of efficacy and safety. | Posted | Median | 95% Confidence Interval | days | Days 1, 2, 3, 5, 9, 14, 21, 32. |
|
|
An event was only included in the analysis of Adverse Events (AEs) if it had an onset date between the first GAMMAPLEX infusion and 30 days after the last GAMMAPLEX infusion, inclusive.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | GAMMAPLEX | The first course of GAMMAPLEX was administered as an intravenous infusion of 1g/kg on each of 2 consecutive days. If required, a further 1 or 2 courses on the same dosage regimen was administered in the period Day 32 to Day 90 following the first course of GAMMAPLEX. | 4 | 35 | 21 | 35 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dehydration | Metabolism and nutrition disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (8.1) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (8.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (8.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (8.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Vaginal haemorrhage | Reproductive system and breast disorders | MedDRA (8.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
| ||
| Headache | Nervous system disorders | Systematic Assessment |
|
Publication of the Study results will be allowed only with prior written approval from Sponsor, said consent not to be unreasonably withheld. At the request of Sponsor, the Institution and/or Investigator shall delete any Confidential Information pertaining to Sponsor's Inventions from any proposed publications prior to submitting or presenting the materials. Any and all publications will give appropriate recognition of any support received from Sponsor.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Tim Aldwinckle, MD | Bio Products Laboratory Limited | +44 2089572565 | Tim.Aldwinckle@bpl.co.uk |
| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| D020141 | Hemostatic Disorders |
| D007154 | Immune System Diseases |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
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| ID | Term |
|---|---|
| D005719 | gamma-Globulins |
| D016756 | Immunoglobulins, Intravenous |
| ID | Term |
|---|---|
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D007074 | Immunoglobulin G |
| D007132 | Immunoglobulin Isotypes |
| D000906 | Antibodies |
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| India |
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