Clinical Assessment of Pharmacokinetics, Efficacy, and Safety of 10% IVIg in Pediatric PID Patients (KIDCARES10)

NCT04944979 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: KB070
• Study Type: interventional
• Target: 30
• Locations: 6 countries
• Sites: 21

Brief Summary

The purpose of this study is to assess efficacy, safety and pharmacokinetics of Kedrion Immunoglobulin 10% (KIg10) in pediatric patients with Primary Immunodeficiency Disease (PID).

Conditions

Primary Immunodeficiency Disease

Keywords

Immunoglobulin; Kedrion IVIG 10%; IgG Antibodies; Agammaglobulinemia; Hypogammaglobulinemia; Pediatric

Outcome Measures

Primary Outcomes (1)

• Incidence Rate of Acute Serious Bacterial Infections

  Incidence rate (i.e., the mean number of acute serious bacterial infections per patientyear) of acute serious bacterial infections (bacterial pneumonia, bacteremia/sepsis, bacterial meningitis, visceral abscess, osteomyelitis/septic arthritis) according to pre-specified criteria).

  From Baseline (Day 1) up to week 51/52

Secondary Outcomes (45)

• Serum Immunoglobulin G (IgG) trough levels

  Before each infusion of KIg10 and at the study termination visit (Week 51/52)

• Immunoglobulin G (IgG) subclasses levels (IgG1, IgG2, IgG3, IgG4)

  Before infusions 1, 5, 9 and 13 for the 28-day infusion schedule, and before infusions 1, 7, 11 and 17 for the 21-day infusion schedule

• Frequency of patients with total Immunoglobulin G (IgG) below 6 g/L

  Day 1 up to week 51/52

• Anti-tetanus toxoid antibody level

  Before infusions 1, 5, 9 and 13 for the 28-day infusion schedule and before infusions 1, 7, 11 and 17 for the 21-day infusion schedule

• Anti-pneumococcal capsular polysaccharide antibody level

  Before infusions 1, 5, 9 and 13 for the 28-day infusion schedule and before infusions 1, 7, 11 and 17 for the 21-day infusion schedule

Study Arms (1)

Experimental: Kedrion IVIG 10%

EXPERIMENTAL

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

Biological: Kedrion IVIG 10%

Interventions

Kedrion IVIG 10% BIOLOGICAL

Kedrion intravenous immunoglobulin (IVIg) 10%

Experimental: Kedrion IVIG 10%

Eligibility Criteria

• Age: 2 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Written informed consent/assent obtained from the patient and his/her 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 as defined by 2017 International Union of Immunological Societies (IUIS) Phenotypic Classification for Primary Immunodeficiencies (Bousfiha A, 2018 - and subsequent revisions) and The European Society for Immunodeficiencies (ESID) Registry Working Definitions for the Clinical Diagnosis of Inborn Errors of Immunity (Seidel MG et al., 2019 - and subsequent revisions) and requiring treatment with IVIg. 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 \[i.e., at least 2 standard deviations under the mean level per age\]).
• (NOTE: IVIg treatment is generally requested in the absence of IgG independently from whether other antibodies are absent).
• Male or female, age from 2 up to \< 16 years, at the time of screening.
• Received 200 to 800 mg/kg of a commercially available IVIg therapy in the range of 21- or 28-day intervals (±3 or ±4 days, respectively) for at least 3 infusions prior to screening.
• (NOTE: Other IVIgs will be prohibited after ICF signature and until study end, week 51/52).
• At least 2 documented IgG trough levels while receiving an IVIg, of ≥ 6 g/L obtained at 2 infusions within 12 months (1 must be within 6 months) prior to ICF signature.
• Patient and his/her parent(s)/legal guardian(s) are willing to comply with all requirements of the protocol.
• Females of child-bearing potential with a negative pregnancy test (serum or urine) and who agree to employ adequate birth control measures during the study, such as:
• sexual abstinence, to be evaluated in relation to the preferred and usual lifestyle of the subject;
• male or female condom with or without spermicide;
• cap, diaphragm or sponge with spermicide;
• progestogen-only oral hormonal contraception, if already used in the past on medical prescription.
• Adequate birth control measures should be maintained throughout the study under parental control.
• Authorization to access personal health information.

You may not qualify if:

• Newly diagnosed PID 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 \[CD3+ \< 300 cell/mm3\] and an absent or particularly low proliferative response \[10% of the lower normal range\] to phytohaemagglutinin P \[PHA\]).
• History of severe or serious reactions or hypersensitivity to IVIg or other injectable forms of IgG.
• 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 patient's lifetime.
• IgA deficiency with documented antibodies to IgA.
• Received blood products that have not undergone viral inactivation measures within 12 months prior to ICF signature.
• Significant protein losing enteropathy, nephrotic syndrome, or lymphangiectasia.
• An acute infection as documented by culture or diagnostic imaging and/or a body temperature ≥38.5 °C (≥101.3 °F) within 7 days prior to screening.
• Acquired immunodeficiency syndrome (AIDS) and/or hepatitis B/C active disease at ICF signature.
• 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.
• Moderate or severe anemia, defined according to patient's age as shown in the following table (World Health Organization, 2011) or persistent severe neutropenia (≤ 500 neutrophils per mm3) or persistent lymphopenia of less than 500 cells per microliter.
• A severe chronic condition such as renal failure \[defined as abnormalities in kidney structure or function that are present for more than 3 months and have health implications. The disease is classified on the basis of cause and category of glomerular filtration rate (GFR) (G1 to G5) and albuminuria (A1 to A3) (KIDIGO, 2017). See the following table\], 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, hyperviscosity, 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.
• 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.
• History of pharmacoresistant epilepsy or multiple episodes of migraine (defined as at least 1 episode within 6 months of ICF signature) not controlled by medication.

Sponsors & Collaborators

• Kedrion S.p.A.: lead

Study Sites (21)

Benioff Children&#39;s Hospital - Mission Bay

San Francisco, California, 94158, United States

Location

IMMUNOe Health and Research Centers

Centennial, Colorado, 80112, United States

Location

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States

Location

Velocity Clinical Research - MedPharmics - Lafayette

Lafayette, Louisiana, 70508, United States

Location

Louisiana State University Shreveport

Shreveport, Louisiana, 71103, United States

Location

Duke Children's Hospital & Health Center

Durham, North Carolina, 27706, United States

Location

Asthma and Allergy Center - Toledo

Toledo, Ohio, 43617, United States

Location

Vital Prospects Clinical Research Institute PC

Tulsa, Oklahoma, 74136, United States

Location

Dél-Pesti Centrumkórház - Országos Hematológiai És Infektológiai Intézet

Budapest, Hungary

Location

SST Spedali Civili di Brescia

Brescia, Italy

Location

Azienda Ospedaliero-Universitaria - Ospedale Pediatrico Meyer

Florence, Italy

Location

I.R.C.C.S. Istituto Giannina Gaslini

Genova, Italy

Location

Fondazione IRCCS Ca&#39; Granda Ospedale Maggiore Policlinic

Milan, Italy

Location

Azienda Ospedaliera Universitaria &#34;Federico II&#34;

Naples, Italy

Location

Fondazione Policlinico Tor Vergata

Roma, Italy

Location

IRCCS Ospedale Pediatrico Bambino Gesù

Roma, Italy

Location

Centro Hospitalar Lisboa Central - Hospital Dona Estefânia

Lisbon, Portugal

Location

Centro Hospitalar Universitário do Porto - Hospital Santo António

Porto, Portugal

Location

Children&#39;s City Clinical Hospital No. 9 named after G.N. Speransky, Moscow City Health Department

Moscow, Russia

Location

Dmitry Rogachev National Medical Research Center for Pediatric Hematology, Oncology and Immunology

Moscow, Russia

Location

Národný ústav detských chorôb (National Institute of Pediatric Diseases)

Bratislava, Slovakia

Location

MeSH Terms

Conditions

Primary Immunodeficiency Diseases; Agammaglobulinemia

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases

Study Officials

• Chiara Azzari

  Azienda Ospedaliero-Universitaria Ospedale Pediatrico Meyer - Italy

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 11, 2021
• First Posted: June 30, 2021
• Study Start: March 31, 2021
• Primary Completion: April 30, 2026
• Study Completion (Estimated): October 30, 2026
• Last Updated: January 15, 2026

Record last verified: 2026-01

Locations

PT (2); US (8); HU (1); SL (1); IT (7); RU (2)