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

Brief Title

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

Official Title

A Phase IV, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases (PID) in Children and Adolescents

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
    


Study Phase

Phase 4

Study Type

Interventional


Primary Outcome

Adverse Events

Secondary Outcome

 Therapeutic Efficacy

Condition

Primary Immune Deficiency Disorders

Intervention

Gammaplex

Study Arms / Comparison Groups

 Gammaplex
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

25

Start Date

March 2011

Completion Date

April 2014

Primary Completion Date

April 2014

Eligibility Criteria

        Inclusion Criteria:

          -  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.

          -  The subject has a primary immunodeficiency disease, which has as a significant
             component of hypogammaglobulinemia and/or antibody deficiency (e.g. common variable
             immunodeficiency, X-linked and autosomal forms of agammaglobulinemia, hyper-IgM
             syndrome, Wiskott-Aldrich Syndrome). NB Isolated deficiency of a single IgG subclass,
             or of specific antibodies without hypogammaglobulinemia per se, does not qualify for
             inclusion.

          -  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.

        Exclusion Criteria:

          -  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).

          -  The subject is receiving Immunosuppressive or Immunomodulatory drugs.

          -  The subject has non-controlled arterial hypertension.

          -  The subject has anemia (hemoglobin <10 g/dL) at screening.
      

Gender

All

Ages

2 Years - 16 Years

Accepts Healthy Volunteers

No

Contacts

Tim J. Aldwinckle, MD, , 

Location Countries

Chile

Location Countries

Chile

Administrative Informations


NCT ID

NCT01289847

Organization ID

GMX04

Secondary IDs

IND 12569

Responsible Party

Sponsor

Study Sponsor

Bio Products Laboratory


Study Sponsor

Tim J. Aldwinckle, MD, Study Director, Medical Director


Verification Date

December 2014