Haematological Disorders
Conditions
Interventions
LIVIG 10 percent Human Normal Immunoglobulin
Sponsors
National Bioproducts Institute NPC
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: a. Documented diagnosis (confirmed by the PI/treatment specialist prior to enrolment) of a form of PID involving defective or lack of antibody formation and requiring immunoglobulin replacement, as defined according to the International Union of Immunological Societies (IUIS) Phenotypic Classification of human inborn errors of immunity, 2024 (1). b. Medically stable for at least 6 weeks, with no acute illness or significant disease progression at the time of enrolment that may increase the risk of developing a serious bacterial infection, as confirmed by Investigator. c. Male or female, aged =2 years at time of enrolment. d. Signed and dated informed consent and where applicable, age-appropriate assent. e. Willing and able to adhere to the IP regimen. f. For females of reproductive potential: use of stable form of contraception (e.g. abstinence from heterosexual intercourse, intrauterine device, diaphragm or condom [for male partner] with spermicidal jelly or foam, or birth control pills/patches/injectables).
Exclusion criteria
Exclusion criteria: a. A known history of, or positive at screening for, one or more of the following: hepatitis B surface antigen , hepatitis C virus, human immunodeficiency virus Type 1/2. b. Abnormal laboratory values at screening that meet any one of the following criteria (abnormal tests could be repeated once to determine if they persisted): i. Persistent alanine aminotransferase and aspartate amino transferase >2.5 times the upper limit of normal for the testing laboratory, ii. Persistent severe neutropenia as defined as an absolute neutrophil count of 500 /mm3, iii. Creatinine clearance value <60% of normal for age and gender, either measured, or calculated according to the Cockcroft-Gault formula. c. Diagnosed with or had a malignancy (other than adequately treated basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix unless the disease-free period prior to screening exceeded 5 years. d. Receiving anti-coagulation therapy (although low dose aspirin, =325 mg/day, is permitted) or had a history of thrombotic episodes (including deep vein thrombosis, myocardial infarction, cerebrovascular accident, pulmonary embolism) or sickle cell disease with crisis within 12 months prior to screening or had a history of thrombophilia. e. Abnormal protein loss (protein losing enteropathy, nephrotic syndrome). f. Anaemia that would preclude phlebotomy for laboratory studies according to standard practice at the site. g. Opportunistic infections associated with severe non-humoral immune deficiency. h. Continuous systemic antibiotics at doses sufficient to treat or prevent bacterial infections and in the opinion of the PI, could not stop these for the duration of the study without putting the participant at risk of increased infections. i. A bleeding disorder or thrombocytopenia with a platelet count less than 20,000 /µL. j. Women of childbearing potential meeting any one of the following criteria: i. Positive pregnancy test. ii. Breast feeding. iii. Intend to begin nursing during the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary Endpoints a.Efficacy Endpoints i.The rate of serious bacterial infections per person-years during the 12-month IP efficacy evaluation period. Serious bacterial infections include: •bacteraemia or sepsis •bacterial meningitis •osteomyelitis or septic arthritis •bacterial pneumonia •visceral abscess b.Safety and Tolerability Endpoints Safety and tolerability include: i.Annual rate of treatment-emergent AEs (TEAEs) and adverse drug reactions (ADRs) defined as AEs assessed by the inves¬tigator as related to treatment. ii.Rate of Infusion-related AEs reported during or within 72 hours following administration of IVIG. iii.Rate of ADRs by participant. iv.Rate of ADRs per the number of infusions administered. v.Categorisation of ADRs by type, severity, duration, seriousness, causality and temporal association with IP. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary Endpoints a. Pharmacokinetic (PK) Endpoints i. IgG (Total) trough levels • Before each IVIG/IP infusion • Trends over a 28-week period, starting after 6 administrations of IP ii. PK Parameters Blood PK parameters in all adult participants (minimum of 20) after 5 or 6 administrations of the IP in Period 2, namely: • Plasma concentration-time curve • Half-life (T½) • Area under the cue (AUC) • Volume of distribution (Vd) • Cmax • Tmax • Elimination rate constant b. Efficacy Endpoints Secondary efficacy endpoints include: i. All other infections, regardless of site ii. Antibiotic treatment iii. Days lost from work/school iv. Hospitalisation, and v. Fever episodes of =2 days. | — |
Countries
South Africa
Contacts
Public ContactLize Schutte
ACRO Clinical Research Manager
Outcome results
None listed