Extension Study of Subcutaneous Immunoglobulin Human in Patients With Primary Immunodeficiency (PID)

• Register: EUCTR
• Last refreshed on: 2 March 2015
• Main ID: EUCTR2014-003605-15-Outside-EU/EEA
• Date of registration: 26/02/2015
• Prospective Registration: Yes
• Primary sponsor: CSL Behring AG
• Public title: Extension Study of Subcutaneous Immunoglobulin Human in Patients With Primary Immunodeficiency (PID)
• Scientific title: A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency (PID)
• Target sample size: 21
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-003605-15
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

United States

Contacts

• Name: Trial Registration Co-ordinator
• Affiliation: CSL Behring

• Name: Trial Registration Co-ordinator
• Affiliation: CSL Behring

Key inclusion & exclusion criteria

Inclusion criteria:
- Subjects with primary humoral immunodeficiency who have participated in the study ZLB04_009CR (NCT00419341), namely with a diagnosis of Common Variable Immunodeficiency (CVID) as defined by PAGID (Pan-American Group for Immunodeficiency) and ESID (European Society for Immunodeficiencies) or X-linked Agammaglobulinemia (XLA) as defined by PAGID and ESID
- Women of childbearing potential must be using and agree to continue using medically approved contraception and must have a negative pregnancy test at screening
- Written informed consent

Are the trial subjects under 18? yes
Number of subjects for this age range: 2
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 16
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 3

Exclusion criteria:
- Ongoing serious bacterial infection at the time of screening
- Malignancies of lymphoid cells such as lymphocytic leukemia, Non-Hodgkin's lymphoma, and immunodeficiency with thymoma
- Hypoalbuminemia, protein-losing enteropathies, and any proteinuria (defined by total urine protein concentration > 0.2 g/L)
- Other significant medical conditions that could increase the risk to the patient
- Females who are pregnant, breast-feeding or planning a pregnancy during the course of the study
- A positive result at screening on any of the following viral markers: Human immunodeficiency virus (HIV), Hepatitis C virus (HCV) or Hepatitis B virus (HBV)
- Aspartate aminotransferase (ASAT) or Alanine aminotransferase (ALAT) concentration > 2.5 times Upper Normal Limit (UNL) at Completion Visit of study ZLB04_009CR (NCT00419341)
- Creatinine concentration > 1.5 times UNL at Completion Visit of study ZLB04_009CR (NCT00419341)
- Participation in a study with an investigational product other than IgPro20 within 3 months prior to enrollment
- Evidence of uncooperative attitude
- Any condition that is likely to interfere with evaluation of the study drug or satisfactory conduct of the trial
- Subjects who are employees at the investigational site, relatives or spouse of the investigator


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Primary Immune Deficiency (PID)
MedDRA version: 17.1 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Intervention(s)

Trade Name: Hizentra®
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Human Normal Immunoglobulin
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN
Concentration unit: % (W/V) percent weight/volume
Concentration type: equal
Concentration number: 20-

Primary Outcome(s)

Main Objective: The purpose of this study is to determine whether a long-term use of a new human immunoglobulin G with proline (IgPro) is safe and effective in the treatment of primary immunodeficiency.

Primary end point(s): Annualized Rate of Serious Bacterial Infection

Acute serious bacterial infections included pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess.

Timepoint(s) of evaluation of this end point: For the duration of the study, up to approximately 104 weeks

Secondary Objective: Not applicable

Secondary Outcome(s)

Secondary end point(s): 1. Annualized Rate of Any Infection
2. Trough Levels of Total Immunoglobulin G (IgG) Serum Concentrations
3. Number of Days Out of Work / School / Kindergarten / Day Care or Inability to Perform Normal Activities Due to Infection
4. Annualized Rate of Days Out of Work / School / Kindergarten / Day Care or Inability to Perform Normal Activities Due to Infection
5. Number of Days of Hospitalization Due to Infection
6. Annualized Rate of Hospitalization Due to Infection
7. Use of Antibiotics for Infection Prophylaxis and Treatment
8. Rate of All AEs by Relatedness and Severity
9. Relatedness and Severity of All AEs (Percentage of Total AEs)
10. Number of Subjects With Any Temporally Associated Adverse Event (AE) Within 24 or 72 Hours After an Infusion
11. Rate of Temporally Associated AEs Within 24 or 72 Hours of an Infusion
12. Number of Subjects Reporting Mild, Moderate, or Severe Local AEs
13. Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Vital Signs
14. Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Routine Laboratory Parameters
15. Number of Subjects With Clinically Significant Changes From Baseline to the Completion Visit in Viral Safety Markers

Timepoint(s) of evaluation of this end point: 1, 3 - 9, 12: For the duration of the study, up to approximately 104 weeks
2: Before infusion at Weeks 1, 24, 48, 72, and 96
10, 11: Within 24 or 72 hours after each infusion
13: At weeks 1, 12, 24, 36, 48, 60, 72, 84, and 96
14, 15: At Week 1, and study completion (approximately 104 weeks)

Secondary ID(s)

IgPro20_3001

NCT00719680

Source(s) of Monetary Support

CSL Behring AG