CLINICAL PHASE III STUDY TO MONITOR THE SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES, INCLUDING (BUT NOT LIMITED TO) THOSE WHO HAVE COMPLETED THE SCGAM-01 TRIAL
Primary Immunodeficiency
Bottom Line
View on ClinicalTrials.gov: NCT03907241 ↗Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Octanorm 16.5% (Drug)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- Octapharma
- Primary completion
- Sep 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Occurrence of All Treatment-emergent Adverse Events (TEAEs) |
8; 19; 22; 155; 8; 12 | — |
| PRIMARY Occurrence of Temporally Associated TEAEs |
2; 7; 13; 72; 5; 6 | — |
| PRIMARY Number of Temporally Associated TEAEs by Infusion Rate |
0; 0; 0; 1; 1; 0 | — |
| PRIMARY Local Injection-site Reactions |
0; 4; 3; 5 | — |
| PRIMARY Blood Pressure |
100.00; 105.00; 116.60; 126.21; 102.50; 103.00 | — |
| PRIMARY Body Temperature |
36.85; 36.98; 36.63; 36.56; 36.55; 36.58 | — |
| PRIMARY Respiratory Rate |
23.50; 19.00; 16.00; 16.07; 17.50; 15.50 | — |
| PRIMARY Sodium |
0.50; 0.25; 1.75; 0.41 | — |
| PRIMARY Potassium |
0.35; 0.25; 0.18; -0.05 | — |
| PRIMARY Blood Glucose |
-1.03; 3.49; 0.22; -0.69 | — |
| PRIMARY ALAT |
-2.50; -5.75; -1.50; 0.71 | — |
| PRIMARY ASAT |
-3.00; -4.00; -3.00; 0.59 | — |
| PRIMARY LDH |
23.50; -110.50; -52.50; -8.47 | — |
| PRIMARY Total Bilirubin |
0.86; 0.94; 1.58; 0.52 | — |
| PRIMARY Blood Urea Nitrogen |
0.36; -2.12; 0.09; -0.49 | — |
| PRIMARY Creatinine |
-4.86; -27.72; 4.20; -0.62 | — |
| PRIMARY Urine pH |
0.00; 0.13; -0.50; -0.34 | — |
| PRIMARY Number of Participants With a Change in Urine Glucose |
2; 4; 4; 15; 0; 0 | — |
| PRIMARY Number of Participants With a Change in Urine Ketones |
2; 4; 4; 13; 0; 0 | — |
| PRIMARY Number of Participants With a Change in Urine Leukocytes |
2; 3; 4; 14; 0; 0 | — |
| PRIMARY Number of Participants With a Change in Urine Hemoglobin |
2; 4; 4; 14; 0; 0 | — |
| PRIMARY Complete Red Blood Cell Count |
0.14; -0.06; 0.17; 0.01 | — |
| PRIMARY Haematocrit |
0.02; 0.00; 0.02; 0.00 | — |
| PRIMARY Haemoglobin |
8.00; -2.08; 4.25; 1.29 | — |
| PRIMARY Complete White Blood Cell Count |
1.45; -0.05; 0.40; -0.24 | — |
| SECONDARY Measurement of Trough Total IgG Levels |
10.99; 11.92; 12.17; 13.17; 12.50; 15.45 | — |
| SECONDARY Number of Participants With Serious Bacterial Infections (SBIs). |
0; 0; 0; 1 | — |
| SECONDARY SF-36 Health Survey. |
12.39; 0.42; -1.12; 0.38 | — |
| SECONDARY CHQ-PF50 (Child Health Questionnaire-Parent Form) |
-0.79; 0.94; 2.76; -9.07; 0.50; 9.92 | — |
Summary
Eligibility Criteria
Inclusion Criteria for SCGAM-03:
- Completion of the main study SCGAM-01, with good tolerance of Octanorm (as determined by the investigator).
- For adult patients: freely given written informed consent. For patients below the legal age of majority: freely given written informed consent from parents/legal guardians and written informed assent from the child/adolescent in accordance with local requirements.
- For female patients of child-bearing potential, a negative result in a urine pregnancy test conducted at the Screening visit.
- Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study.
Inclusion Criteria for SCGAM-03 in Canada:
Either:
SCGAM-01 patients (United States, Canada):
- Completion of the main study SCGAM-01, with good tolerance of octanorm (as determined by the investigator).
Or:
De novo patients (Canada only):
- C-a Age of ≥18 years and ≤75 years.
1C-b Confirmed diagnosis of PI as defined by ESID and PAGID and requiring immunoglobulin replacement therapy due to hypogammaglobulinaemia or agammaglobulinaemia. The exact type of PI should be recorded.
- C-c Availability of the IgG trough levels of 2 previous SCIG infusions before enrolment, and maintenance of ≥5.0 g/L in the trough levels of these 2 previous infusions.
And:
- For adult patients: freely given written informed consent. For patients below the legal age of majority: freely given written informed consent from parents/legal guardians and written informed assent from the child/adolescent in accordance with local requirements.
- For female patients of child-bearing potential, a negative result in a urine pregnancy test conducted at the Screening Visit.
- Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study.
Exclusion Criteria for SCGAM-03:
- Subject being without any IgG treatment for period greater than approximately 5 weeks between the last infusion of Octanorm in the SCGAM-01 study and the first infusion of Octanorm in the SCGAM-03 study.
- Exposure to blood or any blood product or derivative, other than IgG used for regular PID treatment, within the 3 months before the first infusion in this study.
- Planned pregnancy during the course of the study.
Exclusion Criteria for SCGAM-03 in Canada:
- Either:
SCGAM-01 patients (United States, Canada):
1 Subject being without any IgG treatment for period greater than 5 weeks between the last infusion of octanorm in the SCGAM-01 study and the first infusion of octanorm in the SCGAM-03 study.
Or:
De novo patients (Canada only):
1C-a Acute infection requiring intravenous antibiotic treatment within 2 weeks prior to and during the screening period.
1C-b Known history of adverse reactions to IgA in other products.
1C-c Patients with body mass index >40 kg/m2.
1C-d Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived products, or any component of the investigational product (such as Polysorbate 80).
1C-e Requirement of any routine premedication for IgG administration.
1C-f History of malignancies of lymphoid cells and immunodeficiency with lymphoma.
1C-g Severe liver function impairment (ALAT 3 times above upper limit of normal).
1C-h Known protein-losing enteropathies or proteinuria.
1C-i Presence of renal function impairment (creatinine >120 μM/L or creatinine >1.35 mg/dL), or predisposition for acute renal failure (e.g., any degree of pre-existing renal insufficiency or routine treatment with known nephritic drugs).
1C-j Treatment with oral or parenteral steroids for ≥30 days or when given intermittently or as bolus at daily doses ≥0.15 mg/kg.
1C-k Treatment with immunosuppressive or immunomodulatory drugs.
1C-l Live viral vaccination (such as measles, rubella, mumps and varicella) within the last 2 months prior to first infusion of octanorm.
And:
- Exposure to blood or any blood product or plasma derivatives, other tha
Data sourced from ClinicalTrials.gov (NCT03907241). 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.