Phase 3
N=51
A Study of NewGam, Human Immunoglobulin 10%, in Patients With Primary Immunodeficiency Diseases
Primary Immunodeficiency Diseases
Bottom Line
View on ClinicalTrials.gov: NCT01012323 ↗Enrolled (actual)
51
Serious AEs
9.8%
Results posted
Mar 2017
Primary outcome: Primary: Number of Serious Bacterial Infections Per Person-year of Treatment — 0.080 Serious infections per person-year of tr
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- NewGam (Biological)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- Octapharma
- Primary completion
- Jun 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Serious Bacterial Infections Per Person-year of Treatment |
0.080 | — |
| SECONDARY IgG Trough Level Concentration |
9.91; 10.32 | — |
| SECONDARY Trough Level Concentration of Antibodies Against Haemophilus Influenzae |
1.96; 2.51 | — |
| SECONDARY Trough Level Concentration of Antibodies Against Measles |
854.08; 1257.76 | — |
| SECONDARY Trough Level Concentration of Antibodies Against Streptococcus Pneumoniae |
1.03; 1.10; 1.97; 2.10; 1.86; 1.95 | — |
| SECONDARY Trough Level Concentration of Antibodies Against Cytomegalovirus |
30.75; 30.16 | — |
| SECONDARY Trough Level Concentration of Antibodies Against Tetanus |
2.70; 2.37 | — |
| SECONDARY Trough Level Concentration of Antibodies Against Varicella-zoster Virus |
125.13; 119.02 | — |
| SECONDARY Total Number of Infections |
189 | — |
| SECONDARY Number of Non-serious Infections |
185; 13; 4; 28; 14; 90 | — |
| SECONDARY Time to Resolution of Serious and Other Infections |
14.3; 18.4 | — |
| SECONDARY Percentage of Participants Treated With Antibiotics |
82.4 | — |
| SECONDARY Number of Antibiotic Treatment Episodes Per Person-year of Treatment |
3.045 | — |
| SECONDARY Number of Antibiotic Treatment Days Per Person-year of Treatment |
87.301 | — |
| SECONDARY Number of Participants Hospitalized Due to an Infection |
1 | — |
| SECONDARY Percentage of Participants With at Least 1 Episode of Fever |
21.6 | — |
| SECONDARY Percentage of Participants That Missed School or Work Due to an Infection |
49.0 | — |
| SECONDARY Changes in the Physical and Psychosocial Child Health Questionnaire-Parent Form Scores From Baseline to the End of the Study |
2.77; 0.26 | — |
| SECONDARY Changes in the Physical and Mental Short Form-36 Health Survey Scores From Baseline to the End of the Study |
-2.23; 2.59 | — |
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.
Eligibility Criteria
Inclusion Criteria
- 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.
Exclusion Criteria
- 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).
- 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.
Data sourced from ClinicalTrials.gov (NCT01012323). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.