Study to Evaluate the Efficacy, Tolerability and Safety of Octanorm in Patients With Primary Immunodeficiency Diseases

NCT03988426 | Completed Phase 3 Results

• 1 other identifier: SCGAM-04
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 5

Brief Summary

Clinical phase 3 study to evaluate the efficacy, tolerability and safety of subcutaneous human immunoglobulin (octanorm) in patients with primary immunodeficiency diseases.

Conditions

Primary Immune Deficiency Disorder

Outcome Measures

Primary Outcomes (1)

• Number of Serious Bacterial Infections Per Person-Year on Treatment

  Serious Bacterial Infections defined as bacteraemia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess

  Primary Treatment Period (24 Weeks)

Secondary Outcomes (18)

• Number of Patients With Other Infections

  Primary Treatment Period (24 Weeks)

• Number of Other Infections

  Primary Treatment Period (24 Weeks)

• Time to Resolution of Infections

  Primary Treatment Period (24 Weeks) and Whole Treatment Period (up to 36 Weeks)

• Number of Participants Using Antibiotics From 0 to > 20 Days

  Primary Treatment Period (24 Weeks)

• Annual Rate of Antibiotic Use

  Primary Treatment Period (24 Weeks)

Study Arms (1)

Octanorm

EXPERIMENTAL

Human Normal Immunoglobulin for Subcutaneous Administration (Octanorm) is a liquid formulation of normal human IgG at a concentration of 16.5% administered as a SC infusion at weekly intervals (either done at the study center \[during first training sessions and then for every 4th administration\] or at home by the patient or caregiver). The initial weekly dose was determined based on subjects' previous IVIG treatment.

Biological: Octanorm

Interventions

Octanorm BIOLOGICAL

Octanorm

Octanorm

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age of ≥18 years and ≤70 years.
• Confirmed diagnosis of PI requiring immunoglobulin replacement therapy due to hypogammaglobulinaemia or agammaglobulinaemia. The type of PI should be recorded.
• Patients with at least 4 infusions on regular treatment with any Intravenous Immunoglobulin (IVIG) prior to entering the study. Constant IVIG dose between 200 and 800 mg/kg body weight (the individual doses of the last 4 infusions should not vary by more than ±25% of the mean dose for the last 4 infusions).
• Availability of at least 2 IgG trough levels with an IgG level of ≥5.0 g/L from the period of the last 4 IVIG infusions.
• Negative result on a pregnancy test (Human Chorionic Gonadotrophin \[HCG\]-based assay in urine) for women of childbearing potential and use of a reliable method of contraception for the duration of the study. Women of non-childbearing potential must be post-menopausal (amenorrhoeic for at least 12 months) or surgically sterile.
• Examples for medically acceptable methods of birth control for this study include:
• Oral, implantable, transdermal or injectable contraceptives
• Intrauterine device
• Condoms; diaphragm or vaginal ring with spermicidal jellies or cream
• Sexual abstinence
• Vasectomised partner
• Patient must freely give written informed consent.
• Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study.

You may not qualify if:

• Acute infection requiring intravenous (IV) antibiotic treatment within 2 weeks prior to and during the screening period.
• Known history of adverse reactions to Immunoglobulin A in other products.
• Patients with body mass index \>40 kg/m2
• Exposure to blood or any blood product or plasma derivatives, other than IVIG treatment of PI, within the past 3 months prior to first infusion of octanorm.
• Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived products, or any component of the investigational medicinal product (IMP) (such as Polysorbate 80).
• History of malignancies of lymphoid cells and immunodeficiency with lymphoma.
• Severe liver function impairment (ALAT 3 times above upper limit of normal).
• Known protein-losing enteropathies or proteinuria.
• 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).
• Treatment with enteral or parenteral steroids for ≥30 days or when given intermittently or as bolus, at daily doses ≥0.15 mg/kg. Inhaled corticosteroids are allowed.
• Patients with chronic obstructive pulmonary disease (COPD) stage Global Initiative for Chronic Obstructive Lung Disease (GOLD) III or IV.
• Treatment with immunosuppressive drugs.
• Live viral vaccination (such as measles, rubella, mumps and varicella) within the last 2 months prior to first infusion of octanorm.
• Treatment with any IMP within 3 months prior to first infusion of octanorm.
• Presence of any condition that is likely to interfere with the evaluation of study medication or satisfactory conduct of the trial.

Sponsors & Collaborators

• Octapharma: lead

Study Sites (5)

The State Research Center, Institute of Immunology of the Federal Medical-Biological Agency

Moscow, 115478, Russia

Location

Federal Research Center of Pediatric Hematology, Oncology and Immunology of the Ministry of Health and Social Development of the Russian Federation

Moscow, 117997, Russia

Location

State Medical University

Rostov, 344022, Russia

Location

Pasteur Institute

Saint Petersburg, 197101, Russia

Location

Institute of Immunology and Physiology of the Ural Branch of the Russian Academy of sciences

Yekaterinburg, 620219, Russia

Location

Related Publications (1)

• Latysheva E, Rodina Y, Sizyakina L, Totolian A, Tuzankina I. Efficacy and safety of octanorm (cutaquig(R)) in adults with primary immunodeficiencies with predominant antibody deficiency: a prospective, open-label study. Immunotherapy. 2020 Apr;12(5):299-309. doi: 10.2217/imt-2020-0012. Epub 2020 Mar 26.

  PMID: 32212944 DERIVED

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

Study Officials

• Wolfgang Toeglhofer, MD

  Octapharma

  STUDY DIRECTOR

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: June 13, 2019
• First Posted: June 17, 2019
• Study Start: March 7, 2017
• Primary Completion: January 26, 2018
• Study Completion: January 26, 2018
• Last Updated: March 10, 2020
• Results First Posted: March 10, 2020

Record last verified: 2020-02

Data Sharing

• IPD Sharing: Will not share

Locations

RU (5)