A study to evaluate the efficacy and safety of GAMMAPLEX in patients with chronic Idiopathic Thrombocytopenic Purpura

Phase 3

Completed

• Conditions: Chronic Idiopathic Thrombocytopenic Purpura

• Registration Number: CTRI/2010/091/000016
• Lead Sponsor: Bio Products Laboratory

Brief Summary

This clinical trial is a phase III, multicenter, open-label study to evaluate the efficacy and safety of GAMMAPLEX® in Chronic Idiopathic Thrombocytopenic Purpura, wherein the subjects will receive infusions of GAMMAPLEX on Days 1 and 2 (first treatment course). If the platelet count is not maintained for the desired length of time after the first course of GAMMAPLEX, and at the discretion of the study investigator and subject, subjects may receive up to a further 2 courses ofGAMMAPLEX within the period Day 32 to Day 90 of the first treatment course. This study is being conducted in approximately 12 centres in India, 5 centres in USA and 9 centres in Argentina, with approximately 31 subjects to be competetively enrolled globally throughout the centres. The primary objective of the study is to determine the efficacy by determining if GAMMAPLEX raises the platelet count of subjects with chronic ITP to a threshold of 50 x 10\_9/L, similar to that of a historical control. The secondary objectives of the study are: 1) to determine the safety of GAMMAPLEX at the dosage used in this study 2) to determine if GAMMAPLEXmaintains platelet counts of ≥ 50 x 10\_9/L in subjects with chronic ITP for a period of time similar to that of a historical control.

Detailed Description

Not available

Study Timeline

• Study Start: 2010-03-03
• Last Update Posted: 2013-07-01

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 31

Inclusion Criteria

• 1. Males and females aged between 18 and 70 years.
• 2. Confirmed diagnosis of chronic ITP of at least 6 monthsduration.
• 3. Platelet count ≤20 x 109/L at enrollment.
• 4. Absence of other conditions that, in the opinion of theinvestigator, could cause thrombocytopenia.
• 5. If subjects are currently being treated with corticosteroids thetreatment regimen/dose must have been stable (for a minimum of 2 weeks before Day 1 infusion).
• However,subjects must remain on a stable treatment regimen.
• If there is any intent to alter the corticosteroid treatmentregimen (e.g., tapering of corticosteroids) before Day 10, subjects may not be included in the study.
• 6. Ifsubjects are currently being treated with cyclophosphamide, azathioprine or attenuated androgens, the treatmentregimen and dose must have been stable for a minimum of 2 months before infusion on Day 1.
• However, if thereis any intent to alter the treatment regimen before Day 10, subjects may not be included in the study.
• 7)Splenectomized subjects and both Rh(D)+ and Rh(D)- subjects may be included.
• 8. The subject has signed aninformed consent form (subjects must be at least 18 years old), and/or the subject?s legal guardian has signed theinformed consent form if indicated.
• 9. If a subject is a female of child-bearing potential, she must have a negativeresult on a urine-based HCG pregnancy test.
• 10. If a subject is a female who is or becomes sexually active, shemust practice contraception by using a method of proven reliability for the duration of the study.

Exclusion Criteria

• 1. The subject has a history of any severe or anaphylactic reaction to blood or any bloodderivedproduct, or any severe reaction to IGIV or any other IgG preparation.
• 2. The subject is known to beintolerant to any component of the investigational product.
• 3. The subject has received any live virus vaccinewithin the last 3 months prior to Day 1.
• 4. The subject has received an IGIV preparation within 1 month prior toDay 1.
• 5. The subject is currently receiving, or has received, any investigational agent within the 1 month prior toDay 1.
• 6. The subject has received any blood, blood product, or blood derivative within the 1 month prior to Day1.
• 7. The subject has received Rituximab within the 3 months before Day 1.
• 8. The subject is pregnant or isnursing.
• 9. The subject is positive for any of the following at screening: HBsAg, NAT for HCV, NAT for HIV,Antibodies to HCV or HIV 1 or 2.
• 10. The subject, at screening, has levels greater than 2.5 times the upper limit ofnormal, as defined by the central laboratory, of alanine aminotransferase or aspartate aminotransferase.
• 11. Thesubject has a severe renal impairment (defined as serum creatinine greater than 2 times the upper limit of normalor BUN greater than 2.5 times the upper limit of normal for the range of the laboratory doing the analysis); thesubject is on dialysis; the subject has a history of acute renal failure.
• 12. The subject is known to have abusedalcohol, opiates, psychotropic agents, or other chemicals or drugs within the past 12 months.
• 13. The subject has ahistory of deep vein thrombosis (DVT) or thrombotic complications of IGIV therapy.
• 14 The subject has anyhistory or sign of hyperviscosity, transient ischemic attack (TIA), stroke, other thromboembolic event, or unstableangina.
• 15. The subject suffers from any acute or chronic medical conditions (e.g., renal disease or predisposingconditions for renal disease, coronary artery disease, or protein losing enteropathy) that, in the opinion of theinvestigator, may interfere with the conduct of the study.
• 16. The subject has an acquired medical condition, suchas chronic lymphocytic leukemia, lymphoma, multiple myeloma, chronic or recurrent neutropenia (defined as anabsolute neutrophil count (ANC) < 1 x 109/L).
• 17. The subject has non-controlled arterial hypertension (systolicblood pressure >160 mmHg and/or diastolic blood pressure >90 mmHg).
• 18. The subject is anemic (hemoglobin<10 g/dL) at screening.
• 19. The subject is unlikely to adhere to the protocol requirements of the study or is likelyto be uncooperative.

Study & Design

• Study Type: Not specified
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Efficacy: To determine if GAMMAPLEX raises the platelet count of subjectswith chronic ITP to a threshold of 50 x 10_9/L, similar to that of a historical control.	The primary efficacy variable will be analyzed using the intent?to-treat population ITT). The percent of subjects attaining a platelet count of ≥50 x 10_9/L by Day 9 will be compared with the historical response rate of >60%. A 1-sided 95% confidence interval will be constructed for the percent of subjects attaining a platelet count of ≥50 x 10_9/L by Day 9 for GAMMAPLEX. If the lower bound of this confidence interval is >0.60, GAMMAPLEX will be considered effective for the treatment of chronic ITP. Therfore, the primary efficacy variable will be the percent of subjects attaining a platelet count of ≥50 x 10_9/L by Day 9 (the 7th day after completing the second infusion).

Secondary Outcome Measures

Name	Time	Method
The secondary objectives are:1) to determine the safety of GAMMAPLEX at the dosage used in this study. 2) to determine if GAMMAPLEXmaintains platelet counts of ≥ 50 x 10_9/L in subjects with chronic ITP for a period of time similar to that of a historical control.	The variables used to assess safety will be the following: adverse events; vital signs; clinical laboratorytests and Direct Coombs? Test; any transmission of viruses; physical examination.The two secondary efficacy variables: Duration of time for which the platelet counts remain ≥50 x 10_9/L after the first course of GAMMAPLEX and changes in the signs of any bleeding/hemorrhage up to Day 32.The secondary efficacy variables will be analyzed by: 1) using the method of Kaplan and Meier to produce plots ofthe distribution of the time for which the platelet counts remain ≥50 x 10_9/L; 2) a 95% confidence interval for the median time will be constructed.

Trial Locations

Locations (12)

Site No. 202; 🇮🇳 Delhi, DELHI, India

Site No. 203; 🇮🇳 Bangalore, KARNATAKA, India

Site No. 205; 🇮🇳 Bangalore, KARNATAKA, India

Site No. 206; 🇮🇳 Hyderabad, ANDHRA PRADESH, India

Site No. 207; 🇮🇳 Bangalore, KARNATAKA, India

Site No. 209; 🇮🇳 Ahmadabad, GUJARAT, India

Site No. 211; 🇮🇳 Pune, MAHARASHTRA, India

Site No. 212; 🇮🇳 Hyderabad, ANDHRA PRADESH, India

Site No. 216; 🇮🇳 India

Site No. 217; 🇮🇳 Ahmadabad, GUJARAT, India

Scroll for more (2 remaining)

Site No. 202

🇮🇳Delhi, DELHI, India

Ajeet Nanda

Principal investigator

9910104842