Study to evaluate the efficacy and safety of human immune globulin in patients with primary immune thrombocytopenia

• Register: EUCTR
• Last refreshed on: 2 September 2013
• Main ID: EUCTR2012-000796-16-DE
• Date of registration: 20/03/2012
• Prospective Registration: Yes
• Primary sponsor: OCTAPHARMA AG
• Public title: Study to evaluate the efficacy and safety of human immune globulin in patients with primary immune thrombocytopenia
• Scientific title: PROSPECTIVE, OPEN-LABEL, NON-CONTROLLED, MULTICENTER, PHASE III CLINICAL STUDY TO EVALUATE THE EFFICACY AND SAFETY OF OCTAGAM 10% IN PRIMARY IMMUNE THROMBOCYTOPENIA
• Date of first enrolment: 09/07/2012
• Target sample size: 30
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2012-000796-16
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Bulgaria, Czech Republic, Germany, Poland

Contacts

• Name: Clinical Research Department
• Affiliation: Octapharma Pharmazeutika Produktionsgesellschaft m.b.H.

• Name: Clinical Research Department
• Affiliation: Octapharma Pharmazeutika Produktionsgesellschaft m.b.H.

Key inclusion & exclusion criteria

Inclusion criteria:
• Age of >=18 years and <=65 years
• Confirmed diagnosis of chronic primary ITP of at least 12 months duration and fulfilling the following criteria...
• Platelet count of <30x10^9/L with or without bleeding manifestations.
• Freely given written informed consent from patient.
• Women of childbearing potential must have a negative result on a pregnancy test (human chorionic gonadotropine [HCG]-based assay) and need to practice contraception using a method of proven reliability for the duration of the study

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion criteria:
• Thrombocytopenia secondary to other diseases or drug-related thrombocytopenia.
• Administration of intravenous immunoglobulin (IVIG), anti-D or thrombopoetin receptor agonists or other platelet enhancing drugs (incl. immunosuppressive or other immunomodulatory drugs) within 3 weeks before enrolment, except for:
a) long-term corticosteroid therapy
b) long-term azathioprine, cyclophosphamide or attenuated androgen therapy
• Unresponsive to previous treatment with IVIG or anti-D immunoglobulin.
• Experimental treatment (e.g. Rituximab) within 3 months before enrolment.
• Splenectomy in the previous 4 weeks or planned splenectomy throughout the study period.
• Patient with Evans syndrome
• Known or suspected human immunodeficiency virus (HIV) or hepatitis C virus (HCV) infection.
• Live viral vaccination within the last 2 months before study entry.
• Emergency operation.
• Severe liver or kidney disease
• Congestive heart failure New York Heart Association (NYHA) class III or IV.
• Non-controlled arterial hypertension
• History of hypersensitivity to blood or plasma derived products, or any component of the investigational product.
• Known immunoglobulin A (IgA) deficiency and antibodies against IgA.
• History of, or suspected alcohol or drug abuse.
• Pregnant or nursing women.
• Unable to consent, or not capable to understand the nature, significance and implications of the clinical study, or unable or unwilling to comply with the study procedures.
• Vulnerable patients (e.g. kept in detention or institutionalised).
• Participating in another interventional clinical study or planned participation in another trial for the duration of this study.
• Receiving any investigational medicinal product (IMP) within 3 months before study entry.
• Patients with risk factors for thromboembolic events in whom the risks outweigh the potential benefit of Octagam treatment.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Primary Immune Thrombocytopenia (ITP)
MedDRA version: 14.1 Level: LLT Classification code 10023095 Term: ITP System Organ Class: 10005329 - Blood and lymphatic system disorders

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Intervention(s)

Trade Name: Octagam 10%
Product Name: Octagam 10%
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human normal immunoglobulin
CAS Number: 308067-58-5
Current Sponsor code: Octagam 10%
Other descriptive name: IMMUNOGLOBULIN G
Concentration unit: % percent
Concentration type: equal
Concentration number: 10-

Primary Outcome(s)

Primary end point(s): The primary efficacy measure is the PC and the increase in platelets to – and the maintenance of – specific thresholds.

Secondary Objective: To evaluate the safety of Octagam 10%.

Timepoint(s) of evaluation of this end point: continuously, see protocol

Main Objective: To assess the efficacy of Octagam 10% in correcting the platelet count (PC).

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: continuously, see protocol

Secondary end point(s): Secondary Efficacy Endpoints:
• All platelet measurements will be listed and presented in standard summary statistics for each category of response and in total.
• Number and proportion of responders with platelets reaching normal levels
• Maximum PC.
• Regression of haemorrhages.
• Relationship of any new haemorrhages to PC.
Secondary Safety Endpoints:
• Vital signs.
• Physical examinations.
• AEs.
• Laboratory parameters

Secondary ID(s)

GAMr-30

Source(s) of Monetary Support

Octapharma AG