Clinical trial NCT01349790: Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia
Official title: Prospective, Open-label, Non-controlled, Multicenter, Phase III Clinical Study to Evaluate the Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Primary Thrombocytopenia
Keywords: ITP
Primary immune thrombocytopenia
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2011-05-09
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: NewGam is a newly developed human normal immunoglobulin solution for intravenous administration (IGIV). This study will evaluate the safety and efficacy of NewGam 10% in patients with primary immune thrombocytopenia.

Detailed description: The primary objective of the study is to assess the efficacy of NewGam in correcting the platelet count. The secondary objective of the study is to evaluate the safety of NewGam. Safety will be assessed by monitoring vital signs, physical examination, evaluation of adverse events (AE) and laboratory parameters, and viral safety testing.

Eligibility criteria: Inclusion Criteria:

1. Age of ≥ 18 and ≤ 65 years.
2. Confirmed diagnosis of chronic primary immune thrombocytopenia (ITP) (diagnosed with a threshold platelet count \< 100x10\^9/L) of at least 12 months duration.
3. Platelet count of no more than 20x10\^9/L with or without bleeding manifestations.
4. Freely given written informed consent from patient.
5. Women of childbearing potential must have a negative result on a pregnancy test (human chorionic gonadotropin \[HCG\]-based assay) and need to practice contraception using a method of proven reliability for the duration of the study.

Exclusion Criteria:

1. Thrombocytopenia secondary to other diseases (such as acquired immunodeficiency syndrome \[AIDS\] or systemic lupus erythematosus \[SLE\]), or drug-related thrombocytopenia.
2. Administration of intravenous immunoglobulin (IGIV), anti-D immunoglobulin or thrombopoietin receptor agonists, or other platelet enhancing drugs (including immunosuppressive or other immunomodulatory drugs) within 3 weeks before enrollment, except for:

   * Long-term corticosteroid therapy when the dose has been stable during the preceding 3 weeks and no dosage change is planned until study Day 22.
   * Long-term azathioprine, cyclophosphamide, or attenuated androgen therapy when the dose has been stable during the preceding 3 months and no dosage change is planned until study Day 22.
3. Unresponsive to previous treatment with IGIV or anti-D immunoglobulin.

Primary outcomes:
measure: Percentage of Responders
timeFrame: Day 1 to Day 8
Secondary outcomes:
measure: Percentage of Alternative Responders
timeFrame: Day 1 to Day 22
measure: Percentage of Complete Responders
timeFrame: Day 1 to Day 22
measure: Percentage of Alternative Responders Who Lost the Response
timeFrame: Day 1 to Day 22
measure: Percentage of Complete Responders Who Lost the Response
timeFrame: Day 1 to Day 22
measure: Time to a Response
timeFrame: Day 1 to Day 8
measure: Time to an Alternative Response
timeFrame: Day 1 to Day 22
measure: Time to a Complete Response
timeFrame: Day 1 to Day 22
measure: Duration of a Response
timeFrame: Day 1 to Day 22
measure: Duration of an Alternative Response
timeFrame: Day 1 to Day 22
measure: Duration of a Complete Response
timeFrame: Day 1 to Day 22
measure: Platelet Count by Visit
timeFrame: Day 1 to Day 22
measure: Maximum Platelet Count
timeFrame: Day 1 to Day 22
measure: Percentage of Responders Who Achieved a Normal Platelet Count
timeFrame: Day 1 to Day 22
measure: Bleeding Intensity
timeFrame: Day 1 to Day 22
measure: Percentage of Participants Who Achieved a Platelet Count > 30x10^9/L
timeFrame: Day 1 to Day 2
Other outcomes:
Locations:
city: Berlin
country: Germany
facility: Abdulgabar Salama
Study officials:
affiliation: Universitätsklinikum Charite, Med. Fakultät der Humboldt-Universität Berlin
name: Abdulgabar Salama, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Octapharma AG
name: Wolfgang Frenzel, MD
role: STUDY_DIRECTOR
References:
citation: Arbach O, Taumberger AB, Wietek S, Cervinek L, Salama A. Efficacy and safety of a new intravenous immunoglobulin (Panzyga(R) ) in chronic immune thrombocytopenia. Transfus Med. 2019 Feb;29(1):48-54. doi: 10.1111/tme.12573. Epub 2019 Jan 27.