Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in PID Patients (CARES10)

Kedrion Biopharma

Status and phase: Completed; Phase 3

Conditions: Primary Immunodeficiency Disease

Treatments: Biological: Kedrion IVIG 10%

Study type: Interventional

Funder types: Industry

Identifiers

NCT03961009

KIG10_US3_PID01

Details and patient eligibility

About

The purpose of this study was to assess efficacy and safety of Kedrion Immunoglobulin 10% (KIg10) in participants with Primary Immunodeficiency (PID).

Enrollment: 47 patients

Sex: All

Ages: 2 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Written informed consent/assent obtained from participant/participant's parent(s) or legally acceptable representative indicating that they understand the purpose of and procedures required for the study and are willing to participate in it.
• Confirmed clinical diagnosis of a PID, requiring treatment with IVIg and have documented agammaglobulinemia (defined as the total absence of one or more classes of antibodies) or hypogammaglobulinemia (defined as low levels of one or more classes [that is at least 2 standard deviations under the mean level per age])
• Male or female, ages 2 to 70 years at screening
• Received 200 to 800 mg/kg of a commercially available IVIg therapy in the range of 21- or 28-day intervals (±3 days or ±4 days, respectively) for at least 3 infusion cycles prior to screening
• At least 2 documented IgG trough levels while receiving an IVIg, of greater than or equals to (>=) 6 gram per liter (g/L) obtained at 2 infusion cycles within 12 months (1 must be within 6 months) prior to Informed Consent Form (ICF) signature
• Participant is willing to comply all requirements of the protocol.
• Females of child-bearing potential with a negative urine pregnancy test and who agree to employ adequate birth control measures during the study
• Authorization to access personal health information
• Participant previously participating in a clinical trial with another experimental IVIg may be enrolled if they have received stable commercially available IVIg therapy for at least 3 infusion cycles (21 or 28 days) prior to screening
• Participant currently on treatment with any subcutaneous immunoglobulin (SCIG) can be enrolled if they are switched to stable commercially available IVIg therapy for at least 3 infusion cycles (21 or 28 days) prior to screening

Exclusion criteria

• Newly diagnosed PID and and naïve to IgG replacement therapy
• Dysgammaglobulinemia (defined as a deficiency in one or more classes of antibodies, but not severe enough to require substitutive therapy) or isolated IgG subclass deficiency or profound primary T-cell deficiency (defined as the absence or severe reduction of T lymphocytes [CD3+ <300 cells per cubic millimeter (cell/mm3)] and an absent or particularly low proliferative response [10% of the lower normal range] to phytohaemagglutinin P [PHA])
• Has history of severe or serious reactions or hypersensitivity to IVIg or other injectable forms of IgG
• Has history of thrombotic events (including deep vein thrombosis, cerebrovascular accident, pulmonary embolism, transient ischemic attacks, or myocardial infarction), as defined by at least 1 event in participant's lifetime
• Has IgA deficiency with documented antibodies to IgA
• Have received blood products that have not undergone viral inactivation measures within 12 months prior to Informed Consent Form (ICF) signature
• Has significant protein losing enteropathy, nephrotic syndrome, or lymphangiectasia
• Has an acute infection as documented by culture or diagnostic imaging and/or a body temperature >= 38 degree Celsius (°C) (>=100.4 degree Fahrenheit (°F) within 7 days prior to screening
• Has acquired immunodeficiency syndrome (AIDS) and/or hepatitis B/C active disease at ICF signature
• Has levels of Alanine aminotransferase (ALT) or aspartate aminotransferase (AST), 2.5 times of the upper limit of normal for the laboratory designated for the study
• Using an implanted venous access device
• Has profound anemia (haemoglobin less than10 gram per deciliter [g/dl]) or persistent severe neutropenia (<= 1000 neutrophils per millimeter cube (mm^3) or lymphopenia of less than 500 cells per microliter
• Have severe chronic condition such as renal failure (creatinine concentration > 2.0 times the upper limit of normal) with proteinuria, congestive heart failure (New York Heart Association III/IV), cardiomyopathy, cardiac arrhythmia associated with thromboembolic events (e.g., atrial fibrillation), unstable or advanced ischemic heart disease, hyper viscosity, or any other condition that the Investigator believes is likely to interfere with evaluation of the study drug or with satisfactory conduct of the trial. Normal values for serum creatinine are the following: a) Female (18+ years): 45 - 84 micromole per liter (mcmol/L) or 0.51 - 0.95 milligrams per deciliter (mg/dl); b) Male (18+ years): 59 - 103 mcmol/L or 0.67 - 1.17 mg/dl
• Has history of a malignant disease other than properly treated carcinoma in situ of the cervix or basal cell or squamous cell carcinoma of the skin within 24 months prior to ICF signature
• Has history of pharmacoresistant epilepsy or multiple episodes of migraine (defined as at least 1 episode within 6 months of ICF signature) not completely controlled by medication
• Participants must not be receiving steroids (oral or parenteral daily dose of >= 0.15 milligram per kilogram per day (mg/kg/day) of prednisone or equivalent) or other immunosuppressive drugs or chemotherapy
• Females who are pregnant, breast feeding or planning a pregnancy during the course of the study. Women who become pregnant during the study will be withdrawn from the study
• Has participated in another clinical study within 3 weeks prior to study ICF signature
• Has history of drug or alcohol abuse within the 6 months before screening
• Has Employed or a direct relative of an employee of the CRO, the study site, or the Sponsor
• Previously treated under this protocol
• Unable to provide informed consent or provide informed consent by a legally authorized representative

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

47 participants in 1 patient group

Kedrion IVIG 10%

Experimental group

Description:

Participants received intravenous infusion of Kedrion IVIG 10% at a dose of 200 to 800 milligrams per kilogram (mg/kg) body weight on every 21 or 28 days for period of 48 weeks.

Treatment:

Biological: Kedrion IVIG 10%