NCT03988426

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

Completed Phase 3 Results posted Last updated 10 March 2020

What this trial tests

Phase 3 trial testing Octanorm in Primary Immune Deficiency Disorder in 25 participants. Completed in 26 January 2018.

Timeline

7 March 2017

Primary endpoint
26 January 2018

26 January 2018

Quick facts

Lead sponsor	Octapharma
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	25
Start date	7 March 2017
Primary completion	26 January 2018
Estimated completion	26 January 2018
Sites	5 locations across Russia

Drugs / interventions tested

• Octanorm — full drug profile →

Conditions studied

• Primary Immune Deficiency Disorder — all drugs for Primary Immune Deficiency Disorder →

Sponsor

Octapharma — full company profile →

Who can join

Adults 18 to 70, any sex, with Primary Immune Deficiency Disorder. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Serious Bacterial Infections Per Person-Year on Treatment Primary · Primary Treatment Period (24 Weeks)

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

Group	Value	95% CI
Octanorm	0

Number of Patients With Other Infections Secondary · Primary Treatment Period (24 Weeks)

The number of patients with all infections of any kind or seriousness.

Number of Patients With Infections

Group	Value	95% CI
Octanorm	14

Number of Patients with 0 Infections

Group	Value	95% CI
Octanorm	10

Number of Patients with 1 Infection

Group	Value	95% CI
Octanorm	9

Number of Patients with 2 Infections

Group	Value	95% CI
Octanorm	2

Number of Patients with 3 Infections

Group	Value	95% CI
Octanorm	0

Number of Patients with 4 Infections

Group	Value	95% CI
Octanorm	2

Number of Patients with 5 Infections

Group	Value	95% CI
Octanorm	1

Number of Other Infections Secondary · Primary Treatment Period (24 Weeks)

For other infections, the Medical Dictionary for Regulatory Activities (MedDRA) preferred term was used to determine the type of infection. They were grouped into the following categories as determined by a medical expert: Ear infections, eye infections, infections of the gastrointestinal tract, infections of the genitourinary tract, upper respiratory tract infections, lower respiratory tract infections, infections of the skin, and infections not elsewhere classified.

Number of Mild Infections

Group	Value	95% CI
All Patients	17

Number of Moderate Infections

Group	Value	95% CI
All Patients	9

Number of Severe Infections

Group	Value	95% CI
All Patients	0

Time to Resolution of Infections Secondary · Primary Treatment Period (24 Weeks) and Whole Treatment Period (up to 36 Weeks)

Since infections were reported as adverse events, the time to resolution of an infection was the time from the start date of the infection adverse event to the end date of the infection adverse event.

Primary Treatment Period

Group	Value	95% CI
Octanorm	9.53

Whole Treatment Period

Group	Value	95% CI
Octanorm	10.32

Number of Participants Using Antibiotics From 0 to > 20 Days Secondary · Primary Treatment Period (24 Weeks)

Number of patients using antibiotics during the whole treatment period (36 weeks) grouped per number of days with antibiotic usage.

Patients with 0 Treatment Days

Group	Value	95% CI
Octanorm	14

Patients with 5 Treatment Days

Group	Value	95% CI
Octanorm	2

Patients with 7 Treatment Days

Group	Value	95% CI
Octanorm	1

Patients with 8 Treatment Days

Group	Value	95% CI
Octanorm	2

Patients with >20 Treatment Days

Group	Value	95% CI
Octanorm	5

Annual Rate of Antibiotic Use Secondary · Primary Treatment Period (24 Weeks)

The number of antibiotic treatment episodes per person-year of treatment was calculated by the following formula: Total number of antibiotic treatment episodes / patient-years of Octanorm treatment

Group	Value	95% CI
Octanorm	1.73

Hospitalizations Due to Infection Secondary · Primary Treatment Period (24 Weeks)

Number of days spent in hospital due to infection

Group	Value	95% CI
Octanorm	0	± 0

Rate of Hospitalizations Due to Infection Secondary · Primary Treatment Period (24 Weeks)

Annual Rate of Hospitalizations due to Infection

Group	Value	95% CI
Octanorm	0

Episodes of Fever Secondary · Primary Treatment Period (24 Weeks) and Whole Treatment Period (up to 36 Weeks)

Number of episodes of fever

Primary Treatment Period

Group	Value	95% CI
Octanorm	6

Whole Treatment Period

Group	Value	95% CI
Octanorm	8

Rate of Episodes of Fever Secondary · Primary Treatment Period (24 Weeks)

The number of episodes of fever per person-year of treatment was calculated by the following formula: Total number of episodes of fever / patient-years of Octanorm treatment

Group	Value	95% CI
Octanorm	0.55

Patients With Days Missed From Work/Study Due to Infections and Treatment Secondary · Primary Treatment Period (24 Weeks)

Total number of patients who missed days from work or study due to infections or treatment thereof.

Group	Value	95% CI
Octanorm	3

Changes in the Subscales of the Form-36 Health Survey Scores From Baseline to the End of the Study Secondary · Baseline to the end of study (up to 36 weeks)

The SF-36-HS consists of 36 items organized into 8 subscales. The 8 subscales could be combined into 2 summary scores, physical and mental. The calculated summary scores were transformed to a range of 0-100, where a higher score indicates better health. A positive change score indicates improvement.

Physical Functioning

Group	Value	95% CI
Full Analysis Set	2.5	± 12.25

Role Physical

Group	Value	95% CI
Full Analysis Set	8.07	± 25.83

Bodily Pain

Group	Value	95% CI
Full Analysis Set	21.38	± 28.12

General Health

Group	Value	95% CI
Full Analysis Set	-1.08	± 15.16

Vitality

Group	Value	95% CI
Full Analysis Set	5.73	± 17.67

Social Functioning

Group	Value	95% CI
Full Analysis Set	7.81	± 23.26

Role Emotional

Group	Value	95% CI
Full Analysis Set	10.76	± 22.18

Mental Health

Group	Value	95% CI
Full Analysis Set	5.83	± 20.62

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to 36 weeks. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Octanorm

Serious: 0/25 (0%)

Deaths: 0/25

Other adverse events (9 terms — click to expand)

Reaction	System	Octanorm
Respiratory Tract Infection	Infections and infestations	—
Condition Aggravated	General disorders	—
Bronchitis	Infections and infestations	—
Dizziness	Nervous system disorders	—
Food Poisoning	Gastrointestinal disorders	—
Rhinitis	Infections and infestations	—
Pharyngitis	Infections and infestations	—
Blood and Lymphatic Infection	Blood and lymphatic system disorders	—
Investigations	Investigations	—

Data from ClinicalTrials.gov NCT03988426 adverse events section.

Sponsor's own description

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

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Patient-reported preferences for subcutaneous or intravenous administration of parenteral drug treatments in adults with immune disorders: a systematic review and meta-analysis.
   Bril V, Lampe J, Cooper N, Kiessling P, et al · · 2024 · cited 6× · PMID 39115099 · DOI 10.57264/cer-2023-0171
2. Efficacy and safety of octanorm (cutaquig^®) in adults with primary immunodeficiencies with predominant antibody deficiency: a prospective, open-label study.
   Latysheva E, Rodina Y, Sizyakina L, Totolian A, et al · · 2020 · cited 4× · PMID 32212944 · DOI 10.2217/imt-2020-0012