Primary Immunodeficiency DiseasePhase 3NGAM-05NCT01313507
High Infusion Rate Study of Immunoglobulin Intravenous (Human) 10% (NewGam)
A Phase 3 study testing NewGam in people with Primary Immunodeficiency Disease, run by Octapharma at 6 sites in United States.
Trial summary
This was a prospective, open-label, non-controlled, non-randomized multicenter Phase III study of 2 multiple-dose intravenous NewGam regimens (every 3 weeks or every 4 weeks, continuing the patient's infusion interval in the main study NCT01012323 [NGAM-01]) for 3 months. Read more about this study
- Who is it for?
- Ages 2 to 75 years
- Conditions the study is for
- Primary Immunodeficiency Disease
- What is being tested?
- NewGam
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 6 sitesUnited States
- Study dates
- May 2011to Sep 2012
- Phase
- Phase 3
- 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 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Completion of the main study NGAM-01.
- At each of the last 3 infusions in the main study NGAM-01, administration of NewGam at the maximum infusion rate of 0.08 mL/kg/min and without the need for premedication.
Who cannot join
- Any condition or circumstance that would have led to the exclusion of the subject from the NGAM-01 study.
- Administration of any immunoglobulin infusion other than NewGam between conclusion of the NGAM-01 study and the beginning of the present study.
- A deviation of the subject's treatment interval of more than 7 days between the last infusion of NewGam in the NGAM-01 study and the first infusion of NewGam in the present study.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Immunoe Research Center
Centennial, Colorado
Rush Universtity Medical Center
Chicago, Illinois
University of California Irvine
Irvine, California
Midlands Pediatrics
Papillion, Nebraska
Seattle Children's Hospital
Seattle, Washington
Cardinal Glennon Children's Hospital
St Louis, Missouri
About this study
Official title: Clinical Study to Evaluate the Safety and Tolerability of Immunoglobulin Intravenous (Human) 10% (NewGam) Administered at High Infusion Rates to Patients With Primary Immunodeficiency Diseases (Extension of Study NGAM-01)
This was a prospective, open-label, non-controlled, non-randomized multicenter Phase III study of 2 multiple-dose intravenous NewGam regimens (every 3 weeks or every 4 weeks, continuing the patient's infusion interval in the main study NCT01012323 [NGAM-01]) for 3 months. The primary objective of the study was to assess the safety and tolerability of high infusion rates of NewGam.
Read the detailed description
Patients received NewGam via an infusion pump to control precise infusion rates. All NewGam infusions started at a rate of 0.01 mL/kg/min (60 mg/kg/h) for the first 30 minutes followed by 0.03 mL/kg/min (180 mg/kg/h) for the next 15 minutes. If tolerated, further increments were made at predefined patterns with the following maximum rates: 0.10 mL/kg/min (600 mg/kg/h) in the first infusion; if this was tolerated, 0.12 mL/kg/min (720 mg/kg/h) in the second infusion; if this was tolerated, 0.14 mL/kg/min (840 mg/kg/h) in all subsequent infusions.
If an adverse event occurred during an infusion, the rate was reduced to half the rate at which the event occurred or the infusion was interrupted until symptoms subsided. The infusion was then resumed at a rate tolerated by the patient.
- Conditions
- Primary Immunodeficiency Disease
- Being tested
- NewGam
- Keywords
- Primary Immunodeficiency Disease, PID
- Medical subject headings
- Primary Immunodeficiency Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NewGamBiological
The initial dose and dosing interval for each participant was the dose and dosing interval the participant received at the end of the NGAM-01 study. The dose of NewGam, solvent/detergent treated human normal immunoglobulin 10%, remained the same throughout the study, as long as the minimum trough level of serum immunoglobulin G (IgG) was above 5 g/L. If the serum IgG trough level dropped to 5 g/L or less, the dose was to be adjusted at the investigator's discretion. NewGam was supplied as a solution for infusion.
| Study group | Role | What they receive |
|---|---|---|
| NewGam | Experimental | Participants received NewGam 200-800 mg/kg intravenously every 3 or 4 weeks for 3 months (5 or 4 total infusions, respectively). Receives: Biological: NewGam |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Who Experienced at Least 1 Adverse Event Causally Related to the Administration of the Study Drug
An adverse event was considered to be causally related to the administration of the study drug if it judged to be probably or possibly related to the study drug, as assessed by the investigator.
Measured: Baseline (follow-up visit of study NGAM-01) to the end of the study (follow-up visit of study NGAM-05) (up to 4 months)
Percentage of Participants Who Experienced at Least 1 Adverse Event Temporally Related to the Study Drug
An adverse event was considered to be temporally related to the study drug if it started during an infusion or within 72 hours after the end of an infusion.
Measured: Baseline (follow-up visit of study NGAM-01) to the end of the study (follow-up visit of study NGAM-05) (up to 4 months)
Secondary outcomes (1)
Change From Baseline in the Quality of Life (QoL) at the End of the Study
QoL was assessed with the Child Health Questionnaire-Parent Form (CHQ-PF50), completed by a parent or guardian, in participants < 14 years of age at the start of the previous study NGAM-01 and with the Short Form-36 Health Survey (SF-36-HS) in participants ≥ 14 years of age. The CHQ-PF50 consists of 50 items organized into 15 subscales.The 15 subscales could be combined into 2 summary scores, physical and psychosocial. The calculated scores were transformed so that each scale had a range of 0-100. A higher score indicates better health. The SF-36-HS is composed of 36 items. Responses to the 36 items were combined to create 8 scales. The 8 scales could be further combined into 2 scores: Physical component summary and mental component summary. The item and scale scores were transformed to a range of 0-100 with a mean of 50 and a standard deviation of 10 in the general US population. A higher score indicates better health. For both instruments, a positive change indicates improvement.
Measured: Baseline (follow-up visit of study NGAM-01) to the end of the study (follow-up visit of study NGAM-05) (up to 4 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Mar 2011 (estimated)
- Study startMay 2011
- Primary completionSep 2012
- Study completionSep 2012
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.
- Other trials for Primary Immunodeficiency DiseaseStudies of the same condition, open and under way.
- More trials run by OctapharmaThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- James N Moy, MD, Principal investigator, Rush Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01313507
- Sponsor's protocol ID
- NGAM-05
- Acronym
- NGAM-05
- Status last verified
- Feb 2017
- First submitted
- 9 Mar 2011
- Last updated
- 29 Mar 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 29 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study to Evaluate the Safety and Tolerability of Immunoglobulin Intravenous (Human) 10% (NewGam) Administered at High Infusion Rates to Patients With Primary Immunodeficiency Diseases (Extension of Study NGAM-01)
- ClinicalTrials.gov ID
- NCT01313507
- Acronym / sponsor ID
- NGAM-05 / NGAM-05
- Conditions
- Primary Immunodeficiency Disease
- Keywords
- Primary Immunodeficiency Disease, PID
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- May 2011
- Primary completion
- Sep 2012 (actual)
- Study completion
- Sep 2012 (actual)
- First posted
- 11 Mar 2011 (estimated)
- Results
- Posted 29 Mar 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This was a prospective, open-label, non-controlled, non-randomized multicenter Phase III study of 2 multiple-dose intravenous NewGam regimens (every 3 weeks or every 4 weeks, continuing the patient's infusion interval in the main study NCT01012323 [NGAM-01]) for 3 months. The primary objective of the study was to assess the safety and tolerability of high infusion rates of NewGam.
Detailed description
Patients received NewGam via an infusion pump to control precise infusion rates. All NewGam infusions started at a rate of 0.01 mL/kg/min (60 mg/kg/h) for the first 30 minutes followed by 0.03 mL/kg/min (180 mg/kg/h) for the next 15 minutes. If tolerated, further increments were made at predefined patterns with the following maximum rates: 0.10 mL/kg/min (600 mg/kg/h) in the first infusion; if this was tolerated, 0.12 mL/kg/min (720 mg/kg/h) in the second infusion; if this was tolerated, 0.14 mL/kg/min (840 mg/kg/h) in all subsequent infusions.
If an adverse event occurred during an infusion, the rate was reduced to half the rate at which the event occurred or the infusion was interrupted until symptoms subsided. The infusion was then resumed at a rate tolerated by the patient.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NewGam | Experimental | Biological: NewGamParticipants received NewGam 200-800 mg/kg intravenously every 3 or 4 weeks for 3 months (5 or 4 total infusions, respectively). |
Who may qualify
- Ages
- 2 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Completion of the main study NGAM-01.
- At each of the last 3 infusions in the main study NGAM-01, administration of NewGam at the maximum infusion rate of 0.08 mL/kg/min and without the need for premedication.
Key exclusion criteria (3 listed)
- Any condition or circumstance that would have led to the exclusion of the subject from the NGAM-01 study.
- Administration of any immunoglobulin infusion other than NewGam between conclusion of the NGAM-01 study and the beginning of the present study.
- A deviation of the subject's treatment interval of more than 7 days between the last infusion of NewGam in the NGAM-01 study and the first infusion of NewGam in the present study.
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 |
|---|---|---|
| NewGamThe initial dose and dosing interval for each participant was the dose and dosing interval the participant received at the end of the NGAM-01 study. The dose of NewGam, solvent/detergent treated human normal immunoglobulin 10%, remained the same throughout the study, as long as the minimum trough level of serum immunoglobulin G (IgG) was above 5 g/L. If the serum IgG trough level dropped to 5 g/L or less, the dose was to be adjusted at the investigator's discretion. NewGam was supplied as a solution for infusion. | Biological | NewGam |
Primary outcome
Percentage of Participants Who Experienced at Least 1 Adverse Event Causally Related to the Administration of the Study Drug
Time frame: Baseline (follow-up visit of study NGAM-01) to the end of the study (follow-up visit of study NGAM-05) (up to 4 months)
An adverse event was considered to be causally related to the administration of the study drug if it judged to be probably or possibly related to the study drug, as assessed by the investigator.
Percentage of Participants Who Experienced at Least 1 Adverse Event Temporally Related to the Study Drug
Time frame: Baseline (follow-up visit of study NGAM-01) to the end of the study (follow-up visit of study NGAM-05) (up to 4 months)
An adverse event was considered to be temporally related to the study drug if it started during an infusion or within 72 hours after the end of an infusion.
1 secondary outcome
Change From Baseline in the Quality of Life (QoL) at the End of the Study
Time frame: Baseline (follow-up visit of study NGAM-01) to the end of the study (follow-up visit of study NGAM-05) (up to 4 months)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
James N Moy, MD principal investigator
Rush Medical Center
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing NewGam in people with Primary Immunodeficiency Disease, run by Octapharma at 6 sites in United States.
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 2 inclusion and 3 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 29 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 29 Mar 2017). 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?
Percentage of Participants Who Experienced at Least 1 Adverse Event Causally Related to the Administration of the Study Drug (time frame: Baseline (follow-up visit of study NGAM-01) to the end of the study (follow-up visit of study NGAM-05) (up to 4 months)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 29 Mar 2017.