A Phase 4 interventional study of Gammaplex in Primary Immune Deficiency Disorders, Common Variable Immunodeficiency and X-linked Agammaglobulinemia, sponsored by Bio Products Laboratory. Completed at 10 sites in 3 countries. Open to participants aged 2 Years to 16 Years. Per ClinicalTrials.gov, last updated 2014-12-23.
Sponsored by Bio Products Laboratory · Phase 4, Interventional, and Prevention
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
41 studies on the registry are indexed under Wiskott-Aldrich Syndrome; 4 are open to participants now.
This study's enrollment of 25 is above the median of 20 across 29 interventional studies indexed under Wiskott-Aldrich Syndrome.
Browse Wiskott-Aldrich Syndrome studies →Bio Products Laboratory is the lead sponsor of 27 studies on the registry; none are open to participants now.
Compared within Wiskott-Aldrich Syndrome, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Primary Immunodeficiency Diseases. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biological: Gammaplex
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.
Adverse Events
Number of subjects with serious, acute, bacterial infections as a measure of efficacy
Time frame: 12 months
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
Time frame: From week 15 onwards
Therapeutic Efficacy
Number of days off school
Time frame: 12 months
Therapeutic Efficacy
Number of days in hospital
Time frame: 12 months
Therapeutic Efficacy
Visits to physicians and/or emergency room
Time frame: 12 months
Therapeutic Efficacy
Number of days on therapeutic antibiotics
Time frame: 12 months
First enrollment: 06 April 2011; Last Subject completed 23 April 2014; Nine recruiting sites globally: United States (US) (seven sites), Chile (one site) and Israel (one site)
| Milestone | Gammaplex |
|---|---|
| Started | 25 |
| Completed | 24 |
| Not completed | 1 |
| Withdrew: Patient could not comply with visits | 1 |
Number of subjects with serious, acute, bacterial infections as a measure of efficacy
| participants | Gammaplex |
|---|---|
| Adverse Events | 2 |
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
| participants | Gammaplex |
|---|---|
| Therapeutic Efficacy | 7 |
Number of days off school
| days | Gammaplex |
|---|---|
| Therapeutic Efficacy | 7.8 ± 12.06 |
Number of days in hospital
| days | Gammaplex |
|---|---|
| Therapeutic Efficacy | 0.3 ± 0.87 |
Visits to physicians and/or emergency room
| visits | Gammaplex |
|---|---|
| Therapeutic Efficacy | 4.0 ± 4.67 |
Number of days on therapeutic antibiotics
| days | Gammaplex |
|---|---|
| Therapeutic Efficacy | 32.0 ± 28.28 |
Collected over AEs were documented from the date Informed Consent Form or Assent was signed until 30 days after the last dose of Gammaplex was infused.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Gammaplex | — | 3/25 (12%) | 25/25 (100%) |
| Event | Gammaplex |
|---|---|
| left lower lobe pneumoniaRespiratory, thoracic and mediastinal disorders | 1/25 |
| left lower lobe pneumoniaRespiratory, thoracic and mediastinal disorders | 1/25 |
| Acute Gastroenterirtis (AGE)Gastrointestinal disorders | 1/25 |
| Event | Gammaplex |
|---|---|
| HeadacheNervous system disorders | 13/25 |
| PyrexiaGeneral disorders | 9/25 |
| NasopharyngitisInfections and infestations | 8/25 |
| CoughRespiratory, thoracic and mediastinal disorders | 8/25 |
| Nasal congestionRespiratory, thoracic and mediastinal disorders | 8/25 |
| Acute sinusitisInfections and infestations | 7/25 |
| Upper respiratory tract infectionInfections and infestations | 6/25 |
| NauseaGastrointestinal disorders | 6/25 |
| Viral upper respiratory tract infectionInfections and infestations | 5/25 |
| FatigueGeneral disorders | 5/25 |
| Age, Customized(participants) | Gammaplex |
|---|---|
| 2 to 5 year age group | 3 |
| 6 to 11 year age group | 12 |
| 12 to 16 year age group | 10 |
| Sex: Female, Male(Participants) | Gammaplex |
|---|---|
| Female | 6 |
| Male | 19 |
| Region of Enrollment(participants) | Gammaplex |
|---|---|
| United States | 22 |
| Israel | 2 |
| Chile | 1 |
This study is completed, as verified in Dec 2014. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Immunoglobulins, Intravenous
Bio Products Laboratory