Clinical trial NCT03238079: Study of PlasmaCap IG in Adults and Children With PIDD
Official title: A Prospective, Open-Label, Multicenter Study of the Efficacy, Safety, Tolerability, and Pharmacokinetics of Therapure PlasmaCap IG in Adults and Children With Primary Immune Deficiency Diseases
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Primary Immune Deficiency Diseases (PIDD)
Lead sponsor: Therapure Biopharma Inc
Sponsor class: INDUSTRY
First posted: 2017-08-03
Enrollment: 74
Sex: ALL
Minimum age (years): 2
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to investigate the efficacy, safety, tolerability, and pharmacokinetic profile of the investigational medicinal product (IMP) and to determine, on the basis of historical control data, how it compares with other 10% intravenous immunoglobulin (IGIV) products currently licensed in North America for the treatment of subjects with primary immune deficiency diseases (PIDD).


Eligibility criteria: Inclusion Criteria:

* Subject has a confirmed clinical diagnosis of a PIDD, which requires treatment with IGIV:
* Subject/guardian has provided written informed consent (and assent, as applicable).
* Subject is between the ages of 2 and 70 years.
* Subject has received regular IGIV therapy at 21- or 28-day (±4 days) intervals for at least three consecutive months at a dose between 300-900 mg/kg/month prior to Screening or;
* Subject has received commercial SCIG at a dose of 300-900 mg/kg/month on any dosing schedule for at least 12 consecutive weeks prior to Screening. Subjects on SCIG must have received and tolerated IGIV treatment prior to SCIG treatment.
* Subject has a documented trough of ≥500 mg/dL in the 6 months prior to screening.
* Females of childbearing potential must be willing to use an effective form of birth control (eg, oral contraceptives) for the duration of the study, per IRB/REB guidelines.
* Subject agrees to comply with the requirements of the protocol.

Exclusion Criteria:

* Subject has secondary immunodeficiency.
* Subject has history of thrombotic events, such as deep vein thrombosis, myocardial infarction, cerebrovascular accident, pulmonary embolism, etc within the year prior to screening.
* Subject has had an immune globulin associated arterial or venous thrombotic/thromboembolic event (TEE) within 7 days of infusion or a TEE that is not associated with an immune globulin within one year of screening.
* Subject has received blood products (except for IGIV, SCIG, or albumin) within 6 months of screening.
* Subject has anemia (≤8.5 g/dL).
* Subject has levels of alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>3.0 times the upper limit of normal (ULN).
* Subject has severe neutropenia (≤1000 neutrophils per mm3).
* Subject is receiving other immunosuppressive or immunomodulatory drugs or chemotherapy.
* Subject is taking or has taken within the four weeks prior to screening prednisone at ≥0.15 mg/kg/day for more than 10 days.
* Subject has ever had a severe anaphylactic reaction to a blood or IgG product.
* Subject has lymphoid malignancy, leukemia, or any other history of malignancy within the past five years, except squamous cell or basal cell carcinoma of the skin (not melanoma).
* Subject has hypoalbuminemia, protein-losing enteropathy, or proteinuria greater than 300 mg/24 hours except for subjects with documented orthostatic proteinuria.
* Subject has immunoglobulin A (IgA) deficiency with known antibodies to IgA.
* Female who is pregnant, breastfeeding, or planning a pregnancy during the course of the study (women who become pregnant during the study will be withdrawn from the study).
* Any condition that is likely to interfere with evaluation of IMP or satisfactory conduct of the trial in the PI's opinion.
* Subjects who may not be compliant or have a history of non-compliance in the opinion of the PI.

Primary outcomes:
measure: Mean acute Serious Bacterial Infection (SBI) rate
timeFrame: 1 year
Secondary outcomes:
measure: Immunoglobulin G (IgG) trough concentration
timeFrame: up to 12 months per subject
measure: Days unable to perform daily activities
timeFrame: up to 12 months per subject
measure: Therapeutic IgG levels
timeFrame: up to 12 months per subject
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of California
state: California
city: Centennial
country: United States
facility: IMMUNOe Health & Research Centers
state: Colorado
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: West Palm Beach
country: United States
facility: Allergy Associates of the Palm Beaches, P.A.
state: Florida
city: Chevy Chase
country: United States
facility: Institute for Asthma and Allergy, PC
state: Maryland
city: Little Silver
country: United States
facility: Optimed Research Ltd.
state: New Jersey
city: Columbus
country: United States
facility: Optimed Research LTD
state: Ohio
city: Dallas
country: United States
facility: Allergy Partners of North Texas
state: Texas
city: Dallas
country: United States
facility: AARA Research Center
state: Texas
city: Irving
country: United States
facility: AAICPA
state: Texas
city: Milwaukee
country: United States
facility: The Medical College of Wisconsin, Inc.
state: Wisconsin
city: Montréal
country: Canada
facility: CHU Ste-Justine
state: Quebec
Study officials:
affiliation: Therapure Biopharma
name: Mark Krause
role: STUDY_DIRECTOR
References: