Primary Immune Deficiency DisordersPhase 4NCT01289847
A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency
A Phase 4 study testing Gammaplex in people with Primary Immune Deficiency Disorders and Common Variable Immunodeficiency, run by Bio Products Laboratory at 10 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 16 years
- Conditions the study is for
- Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked Agammaglobulinemia
- What is being tested?
- Gammaplex
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 10 sitesin 3 countries
- Study dates
- Mar 2011to Apr 2014
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
10 sites in 3 countries.
10 sites in 3 countries
United States8 sites
Family Allergy & Asthma Center, PC
Atlanta, Georgia, 30342
IMMUNOe International Reseach Centers
Centennial, Colorado, 80112
Rush University Medical Center
Chicago, Illinois, 60612
Department of Medicine, University of California
Irvine, California, 92697.
Allergy, Asthma & Immunology Clinic, P.A
Irving, Texas, 75063
Children's Hospital Los Angeles
Los Angeles, California, 90027
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Children's Hospital of Richmond, VA
Richmond, Virginia, 23219
Israel1 site
Safra Children's Hospital, Sheba Medical Center
Tel HaShomer, 52621
About this study
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
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
- Being tested
- Gammaplex
- Keywords
- Primary Immune Deficiency Disorders, Common Variable Immunodeficiency, X-linked agammaglobulinemia, Hyper-IgM syndrome, Wiskott-Aldrich Syndrome, Immunoglobulins, Bacterial Infections
- Medical subject headings
- Primary Immunodeficiency Diseases, Common Variable Immunodeficiency, Bruton type agammaglobulinemia, Hyper-IgM Immunodeficiency Syndrome, Wiskott-Aldrich Syndrome, Bacterial Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GammaplexBiological
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.
| Study group | Role | What they receive |
|---|---|---|
| Gammaplex | Experimental | Not described Receives: Biological: Gammaplex |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Number of subjects with serious, acute, bacterial infections as a measure of efficacy
Measured: 12 months
Secondary outcomes (5)
Therapeutic Efficacy
Number and proportion of subjects who maintain trough IgG levels at least as high as the average of the 2 previous trough levels before the first Gammaplex infusion
Measured: From week 15 onwards
Therapeutic Efficacy
Number of days off school
Measured: 12 months
Therapeutic Efficacy
Number of days in hospital
Measured: 12 months
Therapeutic Efficacy
Visits to physicians and/or emergency room
Measured: 12 months
Therapeutic Efficacy
Number of days on therapeutic antibiotics
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Feb 2011 (estimated)
- Study startMar 2011
- Primary completionApr 2014
- Study completionApr 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Immune Deficiency DisordersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bio Products Laboratory
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Tim J. Aldwinckle, MD, Study director, Medical Director
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01289847
- Sponsor's protocol ID
- GMX04
- Other IDs
- IND 12569
- Status last verified
- Dec 2014
- First submitted
- 27 Jan 2011
- Last updated
- 23 Dec 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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.
PubMed 36818468
Study at a glance
From the registry record, last updated by the sponsor on 23 Dec 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01289847
- Acronym / sponsor ID
- GMX04
- 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
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Bio Products LaboratoryUniversity, hospital or other
- Study start
- Mar 2011
- Primary completion
- Apr 2014 (actual)
- Study completion
- Apr 2014 (actual)
- First posted
- 4 Feb 2011 (estimated)
- Results
- Posted 23 Dec 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gammaplex | Experimental | Biological: Gammaplex |
Who may qualify
- Ages
- 2 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (18 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GammaplexGAMMAPLEX 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. | Biological | Gammaplex |
Primary outcome
Adverse Events
Time frame: 12 months
Number of subjects with serious, acute, bacterial infections as a measure of efficacy
5 secondary outcomes
Therapeutic Efficacy
Time frame: From week 15 onwards
Therapeutic Efficacy
Time frame: 12 months
Therapeutic Efficacy
Time frame: 12 months
Therapeutic Efficacy
Time frame: 12 months
Therapeutic Efficacy
Time frame: 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Dec 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36818468Geng 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Tim J. Aldwinckle, MD study director
Medical Director
10 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Gammaplex in people with Primary Immune Deficiency Disorders and Common Variable Immunodeficiency, run by Bio Products Laboratory at 10 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Dec 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Chile, Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Dec 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01289847, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Dec 2014.