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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03988426
• Date of registration: 13/06/2019
• Prospective Registration: No
• Primary sponsor: Octapharma
• Public title: Study to Evaluate the Efficacy, Tolerability and Safety of Octanorm in Patients With Primary Immunodeficiency Diseases
• Scientific title: Clinical Phase 3 Study to Evaluate the Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases.
• Date of first enrolment: March 7, 2017
• Target sample size: 25
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03988426
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Russian Federation

Contacts

• Name: Wolfgang Toeglhofer, MD
• Affiliation: Octapharma

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Age of =18 years and =70 years.

2. Confirmed diagnosis of PI requiring immunoglobulin replacement therapy due to
hypogammaglobulinaemia or agammaglobulinaemia. The type of PI should be recorded.

3. 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).

4. 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.

5. 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

6. Patient must freely give written informed consent.

7. Willingness to comply with all aspects of the protocol, including blood sampling, for
the duration of the study.

Exclusion Criteria:

1. Acute infection requiring intravenous (IV) antibiotic treatment within 2 weeks prior
to and during the screening period.

2. Known history of adverse reactions to Immunoglobulin A in other products.

3. Patients with body mass index >40 kg/m2

4. 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.

5. 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).

6. History of malignancies of lymphoid cells and immunodeficiency with lymphoma.

7. Severe liver function impairment (ALAT 3 times above upper limit of normal).

8. Known protein-losing enteropathies or proteinuria.

9. 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).

10. 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.

11. Patients with chronic obstructive pulmonary disease (COPD) stage Global Initiative for
Chronic Obstructive Lung Disease (GOLD) III or IV.

12. Treatment with immunosuppressive drugs.

13. Live viral vaccination (such as measles, rubella, mumps and varicella) within the last
2 months prior to first infusion of octanorm.

14. Treatment with any IMP within 3 months prior to first infusion of octanorm.

15. Presence of any condition that is likely to interfere with the evaluation of study
medication or satisfactory conduct of the trial.

16. Known or suspected to abuse alcohol, drugs, psychotropic agents or other chemicals
within the past 12 months prior to first infusion of octanorm.

17. Known or suspected human immunodeficiency virus (HIV), hepatitis C virus (HCV), or
hepatitis B virus (HBV) infection.

18. Pregnant or nursing women; planned pregnancy during course of the study



Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Primary Immune Deficiency Disorder

Intervention(s)

Biological: Octanorm

Primary Outcome(s)

Number of Serious Bacterial Infections Per Person-Year on Treatment [Time Frame: Primary Treatment Period (24 Weeks)]

Secondary Outcome(s)

Rate of Episodes of Fever [Time Frame: Primary Treatment Period (24 Weeks)]

Number of Participants Using Antibiotics From 0 to > 20 Days [Time Frame: Primary Treatment Period (24 Weeks)]

Patients With Days Missed From Work/Study Due to Infections and Treatment [Time Frame: Primary Treatment Period (24 Weeks)]

Annual Rate of Infections [Time Frame: Up to 36 weeks]

Rate of Hospitalizations Due to Infection [Time Frame: Primary Treatment Period (24 Weeks)]

Number of Other Infections [Time Frame: Primary Treatment Period (24 Weeks)]

Number of Patients With Other Infections [Time Frame: Primary Treatment Period (24 Weeks)]

Proportion of Infusions With at Least 1 Temporally Associated AE [Time Frame: Up to 36 weeks]

Total Number of Adverse Events Regardless of Causality [Time Frame: Up to 36 weeks]

Episodes of Fever [Time Frame: Primary Treatment Period (24 Weeks) and Whole Treatment Period (up to 36 Weeks)]

Hospitalizations Due to Infection [Time Frame: Primary Treatment Period (24 Weeks)]

Number of Infusions With Infusion Site Reaction [Time Frame: Up to 36 weeks]

Number of Participants Experiencing Treatment-Emergent AEs [Time Frame: Up to 36 weeks]

Time to Resolution of Infections [Time Frame: Primary Treatment Period (24 Weeks) and Whole Treatment Period (up to 36 Weeks)]

Annual Rate of Antibiotic Use [Time Frame: Primary Treatment Period (24 Weeks)]

Changes in the Subscales of the Form-36 Health Survey Scores From Baseline to the End of the Study [Time Frame: Baseline to the end of study (up to 36 weeks)]

Number of Related Adverse Events [Time Frame: Up to 36 weeks]

Trough Levels of Serum Total IgG [Time Frame: At baseline and at last infusion (week 33)]

Secondary ID(s)

SCGAM-04

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 10/03/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03988426