A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency

NCT01289847 | Completed Phase 4 Results

• 2 other identifiers: GMX04IND 12569
• Study Type: interventional
• Target: 25
• Locations: 3 countries
• Sites: 10

Brief Summary

The main objective is to determine the efficacy of Gammaplex by measuring the number of serious acute bacterial infections during treatment with Gammaplex over a 12 month period. The secondary objectives are to assess the safety and tolerability of Gammaplex and to compare the data collected from adult subjects with PID from the GMX01 study

Conditions

Primary Immune Deficiency Disorders; Common Variable Immunodeficiency; X-linked Agammaglobulinemia; Hyper-IgM Syndrome; Wiskott-Aldrich Syndrome

Keywords

Primary Immune Deficiency Disorders; Common Variable Immunodeficiency; X-linked agammaglobulinemia; Hyper-IgM syndrome; Wiskott-Aldrich Syndrome; Immunoglobulins; Bacterial Infections

Outcome Measures

Primary Outcomes (1)

• Adverse Events

  Number of subjects with serious, acute, bacterial infections as a measure of efficacy

  12 months

Secondary Outcomes (5)

• Therapeutic Efficacy

  From week 15 onwards

• Therapeutic Efficacy

  12 months

• Therapeutic Efficacy

  12 months

• Therapeutic Efficacy

  12 months

• Therapeutic Efficacy

  12 months

Study Arms (1)

Gammaplex

EXPERIMENTAL

Biological: Gammaplex

Interventions

Gammaplex BIOLOGICAL

GAMMAPLEX 5g/100 mL, dose is 300-800 mg/kg/infusion every 21 or 28 days, intravenously. The total duration of treatment with GAMMAPLEX will be 12 months with a 3 month follow-up.

Gammaplex

Eligibility Criteria

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

You may qualify if:

• The subject is between the ages of or is equal to 2 and 16 years of age, of either sex, belonging to any ethnic group, and above a minimum weight of 10 kg. This weight is based on the amount of blood required for testing.
• Subjects already receiving IGIV replacement therapy require the following before their first infusion of Gammaplex:
• Documented IGIV dose(s) and treatment intervals for the last 2 consecutive routine IGIV treatments (one of which can be the screening visit result). The previous doses should also meet the following conditions before study entry: Have not changed by ± 50% of the mean dose for at least 3 months; be between 300 and 800 mg/kg/infusion; be given every 21-28 days, inclusive; be a licensed or investigational product (Phase III or IIIb).
• Documented previous IgG trough levels for the last 2 consecutive routine IGIV treatments for the last 2 consecutive routine IGIV treatments: Maintained at least 300 mg/dL above baseline serum IgG levels (defined as before initiation of any gamma globulin treatment for that subject); must be more than/equal to 600 mg/dL.
• If a subject is a female of child-bearing potential, she must have a negative result on an HCG-based pregnancy test.
• If a subject is a female who is or becomes sexually active, she must practice contraception by using a method of proven reliability for the duration of the study.
• The subject is willing to comply with all aspects of the protocol, including blood sampling, for the duration of the study.
• The subject, if old enough (generally 6 years to 16), has signed a Child Assent Form and the subject's parent or legal guardian has signed the Informed Consent Form, both approved by the IEC/IRB.

You may not qualify if:

• Has not been treated with IGIV (treatment naive subject)
• The subject has a history of any severe anaphylactic reaction to blood or any blood-derived product.
• The subject is known to be intolerant to any component of Gammaplex, such as sorbitol (i.e. intolerance to fructose).
• The subject has selective IgA deficiency, history of reaction to products containing IgA, or has a history of antibodies to IgA.
• Subjects who have completed the study and subjects who have withdrawn cannot participate in the study for a second time.
• The subject is currently receiving, or has received, any investigational agent, other than an immune serum globulin (ISG) preparation that is being evaluated in a Phase III or IIIb study, within the prior 3 months.
• The subject has been exposed to blood or any blood product or derivative within the last 6 months, other than a commercially available IGIV or other forms of commercially available and licensed ISG. If an unlicensed ISG product that is in Phase III or IIIb has been given, the subject cannot be infused with Gammaplex until 20 days after the last dose was given.
• The subject is pregnant or is nursing.
• The subject, at screening, has levels greater than 2.5 times the upper limit of normal as defined at the central laboratory of any of the following: (Alanine transaminase (ALT); Aspartate transaminase (AST) Lactate dehydrogenase (LDH)).
• The subject has a severe renal impairment (defined as serum creatinine greater than 2 times the upper limit of normal or BUN greater than 2.5 times the upper limit of normal for the range of the laboratory doing the analysis); the subject is on dialysis; the subject has a history of acute renal failure.
• The subject is known to abuse alcohol, opiates, psychotropic agents, or other chemicals or drugs, or has done so within the past 12 months.
• The subject has a history of DVT, or thrombotic complications of IGIV therapy.
• The subject suffers from any acute or chronic medical condition (e.g. renal disease or predisposing conditions for renal disease, or protein losing state) that, in the opinion of the investigator, may interfere with the conduct of the study.
• The subject has an acquired medical condition, such as, chronic or recurrent neutropenia (ANC \< 1000 x 109/L) or AIDS known to cause secondary immune deficiency, or is post or recovering from hematopoietic stem cell transplantation.
• The subject is receiving the following medication: Systemic long-term corticosteroids (i.e. not intermittent or burst, daily, \>1 mg of prednisone equivalent/kg/day).

Sponsors & Collaborators

• Bio Products Laboratory: lead

Study Sites (10)

Department of Medicine, University of California

Irvine, California, 92697., United States

Location

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

IMMUNOe International Reseach Centers

Centennial, Colorado, 80112, United States

Location

Family Allergy & Asthma Center, PC

Atlanta, Georgia, 30342, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

Allergy, Asthma & Immunology Clinic, P.A

Irving, Texas, 75063, United States

Location

Children's Hospital of Richmond, VA

Richmond, Virginia, 23219, United States

Location

Hospital de Niňos Roberto del Río

Santiago, 8380418, Chile

Location

Safra Children's Hospital, Sheba Medical Center

Tel Litwinsky, 52621, Israel

Location

Related Publications (1)

• Geng B, Clark K, Evangelista M, Wolford E. Low rates of headache and migraine associated with intravenous immunoglobulin infusion using a 15-minute rate escalation protocol in 123 patients with primary immunodeficiency. Front Immunol. 2023 Feb 2;13:1075527. doi: 10.3389/fimmu.2022.1075527. eCollection 2022.

  PMID: 36818468 DERIVED

MeSH Terms

Conditions

Primary Immunodeficiency Diseases; Common Variable Immunodeficiency; Bruton type agammaglobulinemia; Hyper-IgM Immunodeficiency Syndrome; Wiskott-Aldrich Syndrome; Bacterial Infections

Interventions

gamma-Globulins

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases; Dysgammaglobulinemia; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Lymphopenia; Leukopenia; Cytopenia; Hemorrhagic Disorders; Leukocyte Disorders; Genetic Diseases, X-Linked; Bacterial Infections and Mycoses; Infections

Intervention Hierarchy (Ancestors)

Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Results Point of Contact

• Title: Head of Medical Affairs
• Organization: Bio Products Laboratory

medinfo@bpl.co.uk

+44 20 8957 2200

Study Officials

• Tim J. Aldwinckle, MD

  Medical Director

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 27, 2011
• First Posted: February 4, 2011
• Study Start: March 1, 2011
• Primary Completion: April 1, 2014
• Study Completion: April 1, 2014
• Last Updated: December 23, 2014
• Results First Posted: December 23, 2014

Record last verified: 2014-12

Locations

US (8); IL (1); CL (1)