Primary Immunodeficiency DiseasesPhase 3NCT01012323
A Study of NewGam, Human Immunoglobulin 10%, in Patients With Primary Immunodeficiency Diseases
A Phase 3 study testing NewGam in people with Primary Immunodeficiency Diseases, run by Octapharma at 7 sites in United States.
Trial summary
The purpose of this study was to determine the efficacy of NewGam in preventing serious bacterial infections and to determine the pharmacokinetic profile of NewGam. The safety of NewGam and its effect on quality of life were also evaluated. Read more about this study
- Who is it for?
- Ages 2 to 75 years
- Conditions the study is for
- Primary Immunodeficiency Diseases
- 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
- 51 participants
- Where
- 7 sitesUnited States
- Study dates
- Jan 2010to Jun 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
- Age of ≥ 2 years and ≤ 75 years.
- Confirmed diagnosis of common variable immunodeficiency (CVID) or X-linked agammaglobulinemia (XLA).
- Previously treated with a commercial immune globulin intravenous (human) every 21-28 days for at least 6 infusion intervals at a constant dose between 200 and 800 mg/kg body weight.
Who cannot join
- Acute infection requiring intravenous antibiotic treatment within 2 weeks prior to and during the screening period.
- Exposure to blood or any blood product or derivative, other than commercially available intravenous immunoglobulin (IVIG), within the past 3 months prior to enrollment.
- Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived products, or any component of the investigational product.
- Requirement of any routine pre-medication for IVIG infusion.
- Severe liver function impairment (alanine aminotransferase [ALAT] 3x > upper limit of normal).
- Presence of renal function impairment (creatinine > 120 μmol/L), or predisposition for acute renal failure (eg, any degree of pre-existing renal insufficiency or routine treatment with known nephritic drugs).
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- History of autoimmune hemolytic anemia.
- History of diabetes mellitus.
- Congestive heart failure New York Heart Association (NYHA) class III or IV.
- Non-controlled arterial hypertension (systolic blood pressure > 160 mmHg or diastolic blood pressure > 90 mmHg).
- History of deep vein thrombosis or thrombotic complications of IVIG therapy.
- A positive result at screening on any of the following viral markers: human immunodeficiency virus (HIV), hepatitis C virus (HCV), hepatitis B virus (HBV).
- Treatment with steroids (oral or parenteral, long-term, ie, 30 days or more, not intermittent or burst, daily, ≥ 0.15 mg of prednisone or equivalent/kg/day), immunosuppressive or immunomodulatory drugs.
- Planned vaccination during the study period.
- Treatment with any investigational agent within 3 months prior to enrollment.
- Known or suspected to abuse alcohol, drugs, psychotropic agents or other chemicals within the past 12 months prior to enrollment.
- Pregnant or nursing women.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Isaac Melamed, MD
Centennial, Colorado
James Moy, MD
Chicago, Illinois
William Smits, MD
Fort Wayne, Indiana
Sudir Gupta, MD
Irvine, California
Ai Lan Kobayashi, MD
Papillion, Nebraska
Hans Ochs, MD
Seattle, Washington
Dr. Alan Knutsen
St Louis, Missouri, 63104
About this study
Official title: Clinical Study to Evaluate the Efficacy, Pharmacokinetics and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Patients With Primary Immunodeficiency Diseases
The purpose of this study was to determine the efficacy of NewGam in preventing serious bacterial infections and to determine the pharmacokinetic profile of NewGam. The safety of NewGam and its effect on quality of life were also evaluated.
Read the detailed description
NewGam is a new 10% human normal immunoglobulin (IVIG) solution developed by Octapharma for intravenous administration. It is supplied as a liquid formulation ready to use. The primary therapeutic use of immunoglobulins is to provide antibodies to prevent viral and bacterial diseases (replacement therapy). IVIG has proved to be useful in a variety of clinical conditions other than for replacement of immunoglobulins; IVIG exhibits an immunomodulatory effect. Children and adults with a Primary Immunodeficiency Disease (PID) have an increased risk of recurrent bacterial and viral infections that typically attack the respiratory tract (sinusitis, bronchitis, pneumonia) but can also affect the gastrointestinal tract (gastroenteritis). Theses diseases can be severe and can lead to substantial morbidity. Responses to antibacterial therapy are often poor. At present, most primary immune deficiencies are not curable, but IVIGs have been shown to decrease the total number of severe infections and the duration of hospitalization.
- Conditions
- Primary Immunodeficiency Diseases
- Being tested
- NewGam
- Keywords
- 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
Also called Human normal immunoglobulin
The dose of NewGam, solvent/detergent treated human normal immunoglobulin 10%, remained the same throughout the study, as long as minimum trough levels of serum immunoglobulin G (IgG) was above 5 g/L. If serum IgG trough levels 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 weeks (17 infusions) or 4 weeks (13 infusions) for 1 year. Receives: Biological: NewGam |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Serious Bacterial Infections Per Person-year of Treatment
The number of serious bacterial infections per person-year of treatment was calculated by the following formula: Total number of serious bacterial infections / patient-years on NewGam treatment. Serious bacterial infections were defined as bacteraemia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess.
Measured: Baseline to end of the study (up to 12 months)
Secondary outcomes (18)
IgG Trough Level Concentration
Total IgG trough concentrations were measured in serum samples taken before each infusion.
Measured: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Haemophilus Influenzae
Trough level concentrations of antibodies against Haemophilus influenzae were measured in serum blood samples collected before the 1st infusion in all participants, before the 9th and 10th infusions in participants receiving NewGam every 3 weeks, before the 7th and 8th infusions in participants receiving NewGam every 4 weeks, and at the termination visit for all participants.
Measured: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Measles
Trough level concentrations of antibodies against measles were measured in serum blood samples collected before the 1st infusion in all participants, before the 9th and 10th infusions in participants receiving NewGam every 3 weeks, before the 7th and 8th infusions in participants receiving NewGam every 4 weeks, and at the termination visit for all participants.
Measured: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Streptococcus Pneumoniae
Trough level concentrations of antibodies against Streptococcus pneumoniae (serotypes types 6B, 14, 9V, 18C, 19F, 4, and 23F) were measured in serum blood samples collected before the 1st infusion in all participants, before the 9th and 10th infusions in participants receiving NewGam every 3 weeks, before the 7th and 8th infusions in participants receiving NewGam every 4 weeks, and at the termination visit for all participants.
Measured: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Cytomegalovirus
Trough level concentrations of antibodies against cytomegalovirus were measured in serum blood samples collected before the 1st infusion in all participants, before the 9th and 10th infusions in participants receiving NewGam every 3 weeks, before the 7th and 8th infusions in participants receiving NewGam every 4 weeks, and at the termination visit for all participants.
Measured: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Tetanus
Trough level concentrations of antibodies against tetanus were measured in serum blood samples collected before the 1st infusion in all participants, before the 9th and 10th infusions in participants receiving NewGam every 3 weeks, before the 7th and 8th infusions in participants receiving NewGam every 4 weeks, and at the termination visit for all participants.
Measured: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Varicella-zoster Virus
Trough level concentrations of antibodies against varicella-zoster virus were measured in serum blood samples collected before the 1st infusion in all participants, before the 9th and 10th infusions in participants receiving NewGam every 3 weeks, before the 7th and 8th infusions in participants receiving NewGam every 4 weeks, and at the termination visit for all participants.
Measured: Baseline to end of the study (up to 12 months)
Total Number of Infections
The number of infections included serious bacterial infections (bacterial pneumonia, bacteraemia/sepsis, osteomyelitis/septic arthritis, visceral abscess, bacterial meningitis) and other infections. For other infections, the Medical Dictionary for Regulatory Activities (MedDRA) preferred term was used to determine the type of infection. They were grouped into the following categories as determined by a medical expert: Ear infections, eye infections, infections of the gastrointestinal tract, infections of the genitourinary tract, upper respiratory tract infections, lower respiratory tract infections, infections of the skin, and infections not elsewhere classified.
Measured: Baseline to end of the study (up to 12 months)
Number of Non-serious Infections
The MedDRA preferred term was used to determine the type of non-serious infection. They were grouped into the following categories as determined by a medical expert: Ear infections, eye infections, infections of the gastrointestinal tract, infections of the genitourinary tract, upper respiratory tract infections, lower respiratory tract infections, infections of the skin, and infections not elsewhere classified. The total number of infections and the number in each category are reported.
Measured: Baseline to end of the study (up to 12 months)
Time to Resolution of Serious and Other Infections
Since infections were reported as adverse events, the time to resolution of an infection was the time from the start date of the infection adverse event to the end date of the infection adverse event.
Measured: Baseline to end of the study (up to 12 months)
Percentage of Participants Treated With Antibiotics
The total percentage of participants treated with antibiotics, as well as, the percentage of participants treated with antibiotics therapeutically and prophylactically are reported.
Measured: Baseline to end of the study (up to 12 months)
Number of Antibiotic Treatment Episodes Per Person-year of Treatment
The number of antibiotic treatment episodes per person-year of treatment was calculated by the following formula: Total number of antibiotic treatment episodes / patient-years of NewGam treatment.
Measured: Baseline to end of the study (up to 12 months)
Number of Antibiotic Treatment Days Per Person-year of Treatment
The number of antibiotic treatment days per person-year of treatment was calculated by the following formula: Total number of antibiotic treatment days / patient-years of NewGam treatment.
Measured: Baseline to end of the study (up to 12 months)
Number of Participants Hospitalized Due to an Infection
Measured: Baseline to end of the study (up to 12 months)
Percentage of Participants With at Least 1 Episode of Fever
Measured: Baseline to end of the study (up to 12 months)
Percentage of Participants That Missed School or Work Due to an Infection
Measured: Baseline to end of the study (up to 12 months)
Changes in the Physical and Psychosocial Child Health Questionnaire-Parent Form Scores From Baseline to the End of the Study
The Quality of Life (QoL) questionnaire Child Health Questionnaire-Parent Form (CHQ-PF50) was completed by a parent or guardian in study 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 summary scores were transformed to a range of 0-100, where a higher score indicates more positive functioning or better health status. A positive change score indicates improvement.
Measured: Baseline to end of the study (up to 12 months)
Changes in the Physical and Mental Short Form-36 Health Survey Scores From Baseline to the End of the Study
The Quality of Life (QoL) questionnaire Short Form-36 Health Survey (SF-36-HS) was completed by participants ≥ 14 years of age. The SF-36-HS consists of 36 items organized into 8 subscales. The 8 subscales could be combined into 2 summary scores, physical and mental. The calculated summary scores were transformed to a range of 0-100, where a higher score indicates better health. A positive change score indicates improvement.
Measured: Baseline to end of the study (up to 12 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2009 (estimated)
- Study startJan 2010
- Primary completionJun 2012
- Study completionJun 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 DiseasesStudies 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
- 51 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Premier Research
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01012323
- Sponsor's protocol ID
- NGAM-01
- Other IDs
- 2009-011434-10, 2009
- Status last verified
- Feb 2017
- First submitted
- 11 Nov 2009
- Last updated
- 16 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 16 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study to Evaluate the Efficacy, Pharmacokinetics and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Patients With Primary Immunodeficiency Diseases
- ClinicalTrials.gov ID
- NCT01012323
- Acronym / sponsor ID
- NGAM-01
- Conditions
- Primary Immunodeficiency Diseases
- Keywords
- PID
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 51 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Collaborators
- Premier Research
- Study start
- Jan 2010
- Primary completion
- Jun 2012 (actual)
- Study completion
- Jun 2012 (actual)
- First posted
- 13 Nov 2009 (estimated)
- Results
- Posted 16 Mar 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study was to determine the efficacy of NewGam in preventing serious bacterial infections and to determine the pharmacokinetic profile of NewGam. The safety of NewGam and its effect on quality of life were also evaluated.
Detailed description
NewGam is a new 10% human normal immunoglobulin (IVIG) solution developed by Octapharma for intravenous administration. It is supplied as a liquid formulation ready to use. The primary therapeutic use of immunoglobulins is to provide antibodies to prevent viral and bacterial diseases (replacement therapy). IVIG has proved to be useful in a variety of clinical conditions other than for replacement of immunoglobulins; IVIG exhibits an immunomodulatory effect. Children and adults with a Primary Immunodeficiency Disease (PID) have an increased risk of recurrent bacterial and viral infections that typically attack the respiratory tract (sinusitis, bronchitis, pneumonia) but can also affect the gastrointestinal tract (gastroenteritis). Theses diseases can be severe and can lead to substantial morbidity. Responses to antibacterial therapy are often poor. At present, most primary immune deficiencies are not curable, but IVIGs have been shown to decrease the total number of severe infections and the duration of hospitalization.
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 weeks (17 infusions) or 4 weeks (13 infusions) for 1 year. |
Who may qualify
- Ages
- 2 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Age of ≥ 2 years and ≤ 75 years.
- Confirmed diagnosis of common variable immunodeficiency (CVID) or X-linked agammaglobulinemia (XLA).
- Previously treated with a commercial immune globulin intravenous (human) every 21-28 days for at least 6 infusion intervals at a constant dose between 200 and 800 mg/kg body weight.
Key exclusion criteria (17 listed)
- Acute infection requiring intravenous antibiotic treatment within 2 weeks prior to and during the screening period.
- Exposure to blood or any blood product or derivative, other than commercially available intravenous immunoglobulin (IVIG), within the past 3 months prior to enrollment.
- Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived products, or any component of the investigational product.
- Requirement of any routine pre-medication for IVIG infusion.
- Severe liver function impairment (alanine aminotransferase [ALAT] 3x > upper limit of normal).
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 |
|---|---|---|
| NewGamAlso: Human normal immunoglobulinThe dose of NewGam, solvent/detergent treated human normal immunoglobulin 10%, remained the same throughout the study, as long as minimum trough levels of serum immunoglobulin G (IgG) was above 5 g/L. If serum IgG trough levels 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
Number of Serious Bacterial Infections Per Person-year of Treatment
Time frame: Baseline to end of the study (up to 12 months)
The number of serious bacterial infections per person-year of treatment was calculated by the following formula: Total number of serious bacterial infections / patient-years on NewGam treatment. Serious bacterial infections were defined as bacteraemia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess.
18 secondary outcomes
IgG Trough Level Concentration
Time frame: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Haemophilus Influenzae
Time frame: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Measles
Time frame: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Streptococcus Pneumoniae
Time frame: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Cytomegalovirus
Time frame: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Tetanus
Time frame: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Varicella-zoster Virus
Time frame: Baseline to end of the study (up to 12 months)
Total Number of Infections
Time frame: Baseline to end of the study (up to 12 months)
Number of Non-serious Infections
Time frame: Baseline to end of the study (up to 12 months)
Time to Resolution of Serious and Other Infections
Time frame: Baseline to end of the study (up to 12 months)
Percentage of Participants Treated With Antibiotics
Time frame: Baseline to end of the study (up to 12 months)
Number of Antibiotic Treatment Episodes Per Person-year of Treatment
Time frame: Baseline to end of the study (up to 12 months)
Number of Antibiotic Treatment Days Per Person-year of Treatment
Time frame: Baseline to end of the study (up to 12 months)
Number of Participants Hospitalized Due to an Infection
Time frame: Baseline to end of the study (up to 12 months)
Percentage of Participants With at Least 1 Episode of Fever
Time frame: Baseline to end of the study (up to 12 months)
Percentage of Participants That Missed School or Work Due to an Infection
Time frame: Baseline to end of the study (up to 12 months)
Changes in the Physical and Psychosocial Child Health Questionnaire-Parent Form Scores From Baseline to the End of the Study
Time frame: Baseline to end of the study (up to 12 months)
Changes in the Physical and Mental Short Form-36 Health Survey Scores From Baseline to the End of the Study
Time frame: Baseline to end of the study (up to 12 months)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 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.
The record lists no contact. Use the official record to reach the sponsor.
7 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 Diseases, run by Octapharma at 7 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 3 inclusion and 17 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 16 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 7 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 16 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?
Number of Serious Bacterial Infections Per Person-year of Treatment (time frame: Baseline to end of the study (up to 12 months)). 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 16 Mar 2017.