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

NCT03907241 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: SCGAM-03 -
• Study Type: interventional
• Target: 27
• Locations: 2 countries
• Sites: 7

Brief Summary

Summary for SCGAM-03: Clinical phase III study to monitor the safety, tolerability and efficacy of subcutaneous human immunoglobulin (Octanorm) in patients with primary immunodeficiency diseases who have completed the SCGAM-01 trial. Summary for SCGAM-03 in Canada: 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

Conditions

Primary Immunodeficiency

Outcome Measures

Primary Outcomes (25)

• Occurrence of All Treatment-emergent Adverse Events (TEAEs)

  Number of TEAEs

  From study start to end, up to 3.5 years

• Occurrence of Temporally Associated TEAEs

  From study start to end, up to 3.5 years

• Number of Temporally Associated TEAEs by Infusion Rate

  Number of temporally associated TEAEs by infusion rate. Only includes systemic TEAEs without infections and without infusion site reactions

  From study start to end, up to 3.5 years

• Local Injection-site Reactions

  From study start to end, up to 3.5 years

• Blood Pressure

  Systolic and diastolic.

  From study start to end, up to 3.5 years

• Body Temperature

  From study start to end, up to 3.5 years

• Respiratory Rate

  From study start to end, up to 3.5 years

• Sodium

  Changes in sodium levels from baseline to end of study

  From study start to end, up to 3.5 years

• Potassium

  Changes in potassium levels from baseline to end of study

  From study start to end, up to 3.5 years

• Blood Glucose

  Changes in blood glucose from baseline to end of study

  From study start to end, up to 3.5 years

• ALAT

  Changes in ALAT (alanine transaminase) from baseline to end of study

  From study start to end, up to 3.5 years

• ASAT

  Changes in ASAT (aspartate aminotransferase) from baseline to end of study

  From study start to end, up to 3.5 years

• LDH

  Changes in LDH (lactate dehydrogenase) from baseline to end of study

  From study start to end, up to 3.5 years

• Total Bilirubin

  Changes in total bilirubin from baseline to end of study

  From study start to end, up to 3.5 years

• Blood Urea Nitrogen

  Changes in blood urea nitrogen from baseline to end of study

  From study start to end, up to 3.5 years

• Creatinine

  Changes in creatinine from baseline to end of study

  From study start to end, up to 3.5 years

• Urine pH

  Changes in urine pH from baseline to end of study

  From study start to end, up to 3.5 years

• Number of Participants With a Change in Urine Glucose

  Number of Participants with a Change in Urine Glucose

  From study start to end, up to 3.5 years

• Number of Participants With a Change in Urine Ketones

  Number of Participants With a Change in Urine Ketones at baseline and end of study

  From study start to end, up to 3.5 years

• Number of Participants With a Change in Urine Leukocytes

  Number of participants with a change in urine leukocytes at baseline and end of study

  From study start to end, up to 3.5 years

• Number of Participants With a Change in Urine Hemoglobin

  Number of participants with a change in urine hemoglobin at baseline and end of study

  From study start to end, up to 3.5 years

• Complete Red Blood Cell Count

  Changes in complete red blood cell count from baseline to end of study

  From study start to end, up to 3.5 years

• Haematocrit

  Changes in haematocrit from baseline to end of study

  From study start to end, up to 3.5 years

• Haemoglobin

  Changes in haemoglobin from baseline to end of study

  From study start to end, up to 3.5 years

• Complete White Blood Cell Count

  Changes in complete white blood cell count from baseline to end of study

  From study start to end, up to 3.5 years

Secondary Outcomes (4)

• Measurement of Trough Total IgG Levels

  From study start to end, up to 3.5 years

• Number of Participants With Serious Bacterial Infections (SBIs).

  From study start to end, up to 3.5 years

• SF-36 Health Survey.

  From study start to end, up to 3.5 years

• CHQ-PF50 (Child Health Questionnaire-Parent Form)

  From study start to end, up to 3.5 years

Study Arms (1)

Octanorm 16.5%

EXPERIMENTAL

octanorm 16.5%, human normal immunoglobulin for subcutaneous (SC) administration.

Drug: Octanorm 16.5%

Interventions

Octanorm 16.5% DRUG

Human normal immunoglobulin

Octanorm 16.5%

Eligibility Criteria

• Age: 2 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.
• C-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.

You may not qualify if:

• 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.
• Either:
• SCGAM-01 patients (United States, Canada):
• 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):
• C-a Acute infection requiring intravenous antibiotic treatment within 2 weeks prior to and during the screening period.
• C-b Known history of adverse reactions to IgA in other products.
• C-c Patients with body mass index \>40 kg/m2.
• C-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).
• C-e Requirement of any routine premedication for IgG administration.
• C-f History of malignancies of lymphoid cells and immunodeficiency with lymphoma.
• C-g Severe liver function impairment (ALAT 3 times above upper limit of normal).

Sponsors & Collaborators

• Octapharma: lead

Study Sites (7)

Octapharma Research Site

Irvine, California, 92697, United States

Location

Octapharma Research Site

San Diego, California, 92123, United States

Location

Octapharma Research Site

Centennial, Colorado, 80112, United States

Location

Octapharma Research Site

Papillion, Nebraska, 68046, United States

Location

Octapharma Research Site

Toledo, Ohio, 43617, United States

Location

Octapharma Research Site

Frisco, Texas, 75034, United States

Location

Octapharma Research Site

Edmonton, Alberta, T6G2V2, Canada

Location

MeSH Terms

Conditions

Primary Immunodeficiency Diseases

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases

Results Point of Contact

• Title: Mikaela Raymond
• Organization: CRMG

ctgov@clinicalresearchmgt.com

866-337-1868

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 7, 2018
• First Posted: April 8, 2019
• Study Start: March 1, 2016
• Primary Completion: September 5, 2019
• Study Completion: September 5, 2019
• Last Updated: October 27, 2020
• Results First Posted: September 22, 2020

Record last verified: 2020-10

Locations

CA (1); US (6)