Primary ImmunodeficiencyNCT03339778
The Benefit of 5% IVIG for Patients With Primary Immunodeficiency Disorders Who Experience Adverse Events on 10% IVIG Preparations
An observational study of Primary Immunodeficiency, run by IMMUNOe Research Centers.
Trial summary
Patients with primary immunodeficiency disorders (PID) on intravenous immunoglobulin (IVIG) treatment may experience adverse events (AEs). Patients who experience AEs on any 10% IVIG solution will be changed to octagam 5% for six infusions to evaluate the potential benefit for reduction of AEs on a lower concentration… Read more about this study
- Who is it for?
- Ages 10 to 75 years
- Conditions the study is for
- Primary Immunodeficiency
- How many people
- 15 participants
- Where
- No sites listed yet
- Study dates
- Jun 2015to Sep 2017
- Phase
- Observational
- Are there results?
- None posted in the registry
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 10 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Diagnosis of PID - specifically common variable immunodeficiency (CVID)
Who may be able to join
- Participants, or legal guardians with assent by underage children, will sign informed consent/assent and are willing to comply with all aspects of the study
- Diagnosis of CVID according IUIS Expert Committee
- Participants on a 10% product who experience AEs
- Ages between 10 and 75 years of age
- Participants on 10% IVIG therapy every 21±3 days or 28±3 days between 300 - 800 mg/Kg body weight
Who cannot join
- Acute infection requiring antibiotic therapy within 7 days prior to visit 1
- Presence of any condition that is likely to interfere with the evaluation of the study medication or satisfactory conduct of the trial
- History of anaphylactic or severe systemic reactions to human immunoglobulin
- IgA deficient patients with antibodies against IgA and a history of hypersensitivity
- Females who are pregnant or lactating
About this study
Official title: An Investigator Driven Observational Study to Determine the Benefit of Octagam 5% for Treatment of Patients Diagnosed With Primary Immunodeficiency Disorders (PID) on Intravenous Immunoglobulin (IVIG) Therapy That Experience Adverse Events (AEs) on Any 10% IVIG Preparation
Patients with primary immunodeficiency disorders (PID) on intravenous immunoglobulin (IVIG) treatment may experience adverse events (AEs). Patients who experience AEs on any 10% IVIG solution will be changed to octagam 5% for six infusions to evaluate the potential benefit for reduction of AEs on a lower concentration IVIG product.
Read the detailed description
Patients with PID require life long immunoglobulin (Ig) replacement therapy with IVIG being the most common form. As more 10% IVIG products are FDA approved, the older and well characterized 5% IVIG products are becoming less used. Currently, the standard of care for patients who experience AEs on IVIG is to move to a subcutaneous (SCIG) delivery and product. This study will evaluate the AEs on a 10% product and octagam 5%. The study will enroll 15 patients after an AE on any 10% product who will then be infused with octagam 5% for six infusions. AEs will be documented and compared to the 10% product along with changes in biomarkers. The study data may document another therapeutic option for patients who experience AEs - SCIG and octagam 5%.
- Conditions
- Primary Immunodeficiency
- Medical subject headings
- Primary Immunodeficiency Diseases
What is being measured
The primary outcome is the main question the study is designed to answer.
The change in the number of AEs post-infusion between any 10% IVIG product and octagam 5%
Measured: AEs will be documented at screening and up to 72 hours post-infusion for six infusions up to 24 weeks
Secondary outcomes (4)
The change in levels of inflammatory biomarkers associated with AEs between any 10% IVIG and octagam 5%
Measured: Levels will be documented at screening and up to 72 hours post-infusion for six infusions up to 24 weeks
Safety Evaluations (complete blood count [CBC])
CBC
Measured: Screening and prior to each infusion (six infusions total) up to 24 weeks
Safety evaluations (Complete Metabolic profile[CMP])
CMP
Measured: Screening and prior to each infusion (six infusions total) up to 24 weeks
Safety evaluations (IgG trough level)
IgG trough level
Measured: Screening and prior to last infusion up to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2017
- Study startJun 2015
- Primary completionNov 2016
- Study completionSep 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Primary ImmunodeficiencyStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- IMMUNOe Research Centers
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Isaac Melamed, MD, Principal investigator, IMMUNOe Research Centers
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03339778
- Sponsor's protocol ID
- IIS201401-PID
- Status last verified
- Nov 2017
- First submitted
- 31 Mar 2015
- Last updated
- 13 Nov 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
Geha RS, Notarangelo LD, Casanova JL, Chapel H, Conley ME, Fischer A, Hammarstrom L, Nonoyama S, Ochs HD, Puck JM, Roifman C, Seger R, Wedgwood J; International Union of Immunological Societies Primary Immunodeficiency Diseases Classification Committee. Primary immunodeficiency diseases: an update from the International Union of Immunological Societies Primary Immunodeficiency Diseases Classification Committee. J Allergy Clin Immunol. 2007 Oct;120(4):776-94. doi: 10.1016/j.jaci.2007.08.053.
PubMed 17952897Deane S, Selmi C, Naguwa SM, Teuber SS, Gershwin ME. Common variable immunodeficiency: etiological and treatment issues. Int Arch Allergy Immunol. 2009;150(4):311-24. doi: 10.1159/000226232. Epub 2009 Jul 1.
PubMed 19571563Kaveri SV, Maddur MS, Hegde P, Lacroix-Desmazes S, Bayry J. Intravenous immunoglobulins in immunodeficiencies: more than mere replacement therapy. Clin Exp Immunol. 2011 Jun;164 Suppl 2(Suppl 2):2-5. doi: 10.1111/j.1365-2249.2011.04387.x.
PubMed 21466545Maarschalk-Ellerbroek LJ, Hoepelman IM, Ellerbroek PM. Immunoglobulin treatment in primary antibody deficiency. Int J Antimicrob Agents. 2011 May;37(5):396-404. doi: 10.1016/j.ijantimicag.2010.11.027. Epub 2011 Jan 26.
PubMed 21276714
Study at a glance
From the registry record, last updated by the sponsor on 13 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Investigator Driven Observational Study to Determine the Benefit of Octagam 5% for Treatment of Patients Diagnosed With Primary Immunodeficiency Disorders (PID) on Intravenous Immunoglobulin (IVIG) Therapy That Experience Adverse Events (AEs) on Any 10% IVIG Preparation
- ClinicalTrials.gov ID
- NCT03339778
- Acronym / sponsor ID
- IIS201401-PID
- Conditions
- Primary Immunodeficiency
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- IMMUNOe Research CentersIndustry (pharma, biotech, devices)
- Study start
- Jun 2015 (actual)
- Primary completion
- Nov 2016 (actual)
- Study completion
- Sep 2017 (actual)
- First posted
- 13 Nov 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Patients with primary immunodeficiency disorders (PID) on intravenous immunoglobulin (IVIG) treatment may experience adverse events (AEs). Patients who experience AEs on any 10% IVIG solution will be changed to octagam 5% for six infusions to evaluate the potential benefit for reduction of AEs on a lower concentration IVIG product.
Detailed description
Patients with PID require life long immunoglobulin (Ig) replacement therapy with IVIG being the most common form. As more 10% IVIG products are FDA approved, the older and well characterized 5% IVIG products are becoming less used. Currently, the standard of care for patients who experience AEs on IVIG is to move to a subcutaneous (SCIG) delivery and product. This study will evaluate the AEs on a 10% product and octagam 5%. The study will enroll 15 patients after an AE on any 10% product who will then be infused with octagam 5% for six infusions. AEs will be documented and compared to the 10% product along with changes in biomarkers. The study data may document another therapeutic option for patients who experience AEs - SCIG and octagam 5%.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 10 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Diagnosis of PID - specifically common variable immunodeficiency (CVID)
Key inclusion criteria (5 listed)
- Participants, or legal guardians with assent by underage children, will sign informed consent/assent and are willing to comply with all aspects of the study
- Diagnosis of CVID according IUIS Expert Committee
- Participants on a 10% product who experience AEs
- Ages between 10 and 75 years of age
- Participants on 10% IVIG therapy every 21±3 days or 28±3 days between 300 - 800 mg/Kg body weight
Key exclusion criteria (5 listed)
- Acute infection requiring antibiotic therapy within 7 days prior to visit 1
- Presence of any condition that is likely to interfere with the evaluation of the study medication or satisfactory conduct of the trial
- History of anaphylactic or severe systemic reactions to human immunoglobulin
- IgA deficient patients with antibodies against IgA and a history of hypersensitivity
- Females who are pregnant or lactating
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
The change in the number of AEs post-infusion between any 10% IVIG product and octagam 5%
Time frame: AEs will be documented at screening and up to 72 hours post-infusion for six infusions up to 24 weeks
4 secondary outcomes
The change in levels of inflammatory biomarkers associated with AEs between any 10% IVIG and octagam 5%
Time frame: Levels will be documented at screening and up to 72 hours post-infusion for six infusions up to 24 weeks
Safety Evaluations (complete blood count [CBC])
Time frame: Screening and prior to each infusion (six infusions total) up to 24 weeks
Safety evaluations (Complete Metabolic profile[CMP])
Time frame: Screening and prior to each infusion (six infusions total) up to 24 weeks
Safety evaluations (IgG trough level)
Time frame: Screening and prior to last infusion up to 24 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Nov 2017.
- Background: PubMed 17952897Geha RS, Notarangelo LD, Casanova JL, Chapel H, Conley ME, Fischer A, Hammarstrom L, Nonoyama S, Ochs HD, Puck JM, Roifman C, Seger R, Wedgwood J; International Union of Immunological Societies Primary Immunodeficiency Diseases Classification Committee. Primary immunodeficiency diseases: an update from the International Union of Immunological Societies Primary Immunodeficiency Diseases Classification Committee. J Allergy Clin Immunol. 2007 Oct;120(4):776-94. doi: 10.1016/j.jaci.2007.08.053.
- Background: PubMed 19571563Deane S, Selmi C, Naguwa SM, Teuber SS, Gershwin ME. Common variable immunodeficiency: etiological and treatment issues. Int Arch Allergy Immunol. 2009;150(4):311-24. doi: 10.1159/000226232. Epub 2009 Jul 1.
- Background: PubMed 21466545Kaveri SV, Maddur MS, Hegde P, Lacroix-Desmazes S, Bayry J. Intravenous immunoglobulins in immunodeficiencies: more than mere replacement therapy. Clin Exp Immunol. 2011 Jun;164 Suppl 2(Suppl 2):2-5. doi: 10.1111/j.1365-2249.2011.04387.x.
- Background: PubMed 21276714Maarschalk-Ellerbroek LJ, Hoepelman IM, Ellerbroek PM. Immunoglobulin treatment in primary antibody deficiency. Int J Antimicrob Agents. 2011 May;37(5):396-404. doi: 10.1016/j.ijantimicag.2010.11.027. Epub 2011 Jan 26.
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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.
Isaac Melamed, MD principal investigator
IMMUNOe Research Centers
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Immunodeficiency, run by IMMUNOe Research Centers.
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 5 inclusion and 5 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 13 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The change in the number of AEs post-infusion between any 10% IVIG product and octagam 5% (time frame: AEs will be documented at screening and up to 72 hours post-infusion for six infusions up to 24 weeks). 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 NCT03339778, which also lists 4 linked publications.
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 13 Nov 2017.