Trial Outcomes & Findings for Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID) (NCT NCT01581593)

NCT ID: NCT01581593

Last Updated: 2021-02-16

Results Overview

The incidence of acute serious bacterial infections, e.g. bacterial pneumonia, bacteremia/sepsis, bacterial meningitis, visceral abscess, osteomyelitis/septic arthritis, meeting EMA and FDA criteria.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 45 participants
• Primary outcome timeframe: 13 months
• Results posted on: 2021-02-16

Participant Flow

Participant milestones

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Overall Study STARTED	45
Overall Study COMPLETED	44
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID)

Baseline characteristics by cohort

Measure	Kedrion IVIG 10% 21 and 28 Day Infusion Schedule n=45 Participants Kedrion IVIG 10% 21 and 28 Day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Treatment duration - 12 months
Age, Categorical <=18 years	9 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	27 Participants n=5 Participants
Age, Categorical >=65 years	9 Participants n=5 Participants
Age, Continuous	39.9 years STANDARD_DEVIATION 20.7 • n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants
Sex: Female, Male Male	32 Participants n=5 Participants

PRIMARY outcome

Timeframe: 13 months

The incidence of acute serious bacterial infections, e.g. bacterial pneumonia, bacteremia/sepsis, bacterial meningitis, visceral abscess, osteomyelitis/septic arthritis, meeting EMA and FDA criteria.

Outcome measures

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule n=45 Participants Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Incidence of Acute, Serious Bacterial Infections in the Total ITT Population.	0.04 ASBI's/person-year Interval 0.0 to 0.11

SECONDARY outcome

Timeframe: 13 months

Outcome measures

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule n=45 Participants Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Infections Other Than Acute, Serious Bacterial Infections (ASBIs) in the Total ITT Population. Upper respiratory tract infection	0.42 infect./subject's study duration (years)
Infections Other Than Acute, Serious Bacterial Infections (ASBIs) in the Total ITT Population. Sinusitis	0.48 infect./subject's study duration (years)
Infections Other Than Acute, Serious Bacterial Infections (ASBIs) in the Total ITT Population. Viral upper respiratory tract infection	0.15 infect./subject's study duration (years)
Infections Other Than Acute, Serious Bacterial Infections (ASBIs) in the Total ITT Population. Bronchitis	0.11 infect./subject's study duration (years)
Infections Other Than Acute, Serious Bacterial Infections (ASBIs) in the Total ITT Population. Nasopharyngitis	0.11 infect./subject's study duration (years)
Infections Other Than Acute, Serious Bacterial Infections (ASBIs) in the Total ITT Population. Rhinitis	0.11 infect./subject's study duration (years)
Infections Other Than Acute, Serious Bacterial Infections (ASBIs) in the Total ITT Population. Acute sinusitis	0.15 infect./subject's study duration (years)
Infections Other Than Acute, Serious Bacterial Infections (ASBIs) in the Total ITT Population. Gastroenteritis viral	0.09 infect./subject's study duration (years)
Infections Other Than Acute, Serious Bacterial Infections (ASBIs) in the Total ITT Population. Herpes zoster	0.11 infect./subject's study duration (years)
Infections Other Than Acute, Serious Bacterial Infections (ASBIs) in the Total ITT Population. Urinary tract infection	0.11 infect./subject's study duration (years)

SECONDARY outcome

Timeframe: 13 months

Outcome measures

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule n=45 Participants Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Days Out of Work/School/Daycare Due to Infection in the Total ITT Population.	2.8 days Standard Deviation 6.56

SECONDARY outcome

Timeframe: 13 months

Outcome measures

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule n=45 Participants Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Days Unable to Perform Normal Daily Activities Due to Infection in the Total ITT Population.	3.4 days Standard Deviation 6.53

SECONDARY outcome

Timeframe: 13 months

Outcome measures

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule n=45 Participants Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Days on Therapeutic Antibiotics in the Total ITT Population.	23.3 days Standard Deviation 33.77

SECONDARY outcome

Timeframe: 13 months

Outcome measures

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule n=45 Participants Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Days of Unscheduled Visits to Physicians in the Total ITT Population.	0.9 days Standard Deviation 1.14

SECONDARY outcome

Timeframe: 13 months

Outcome measures

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule n=45 Participants Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Number of Hospitalizations Due to Infection in ITT Population. 0 hospitalizations	42 Participants
Number of Hospitalizations Due to Infection in ITT Population. 1 hospitalization	2 Participants
Number of Hospitalizations Due to Infection in ITT Population. 2 hospitalizations	1 Participants
Number of Hospitalizations Due to Infection in ITT Population. ≥ 3 hospitalizations	0 Participants

SECONDARY outcome

Timeframe: 13 months

Outcome measures

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule n=45 Participants Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Days of Hospitalization Due to Infection in the Total ITT Population.	0.6 days Standard Deviation 2.21

SECONDARY outcome

Timeframe: 13 months

Outcome measures

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule n=45 Participants Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Yearly Hospitalization Rate Due to Infection in the Total ITT Population.	0.11 hospitalizations per year Standard Deviation 0.436

SECONDARY outcome

Timeframe: 13 months

Outcome measures

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule n=45 Participants Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Yearly Hospitalization Duration Due to Infection in the Total ITT Population.	0.55 days Standard Deviation 2.196

SECONDARY outcome

Timeframe: 13 months

Outcome measures

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule n=45 Participants Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Distribution of All-cause Hospitalizations in the Total ITT Population. 0 hospitalizations	41 Participants
Distribution of All-cause Hospitalizations in the Total ITT Population. 1 hospitalization	1 Participants
Distribution of All-cause Hospitalizations in the Total ITT Population. 2 hospitalizations	2 Participants
Distribution of All-cause Hospitalizations in the Total ITT Population. 3 hospitalizations	1 Participants
Distribution of All-cause Hospitalizations in the Total ITT Population. ≥ 4 hospitalizations	0 Participants

SECONDARY outcome

Timeframe: 13 months

Population: The duration of the hospitalizations was measured for each of four subjects who were hospitalized and was calculated dividing by the subject's study duration in days.

Outcome measures

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule n=4 Participants Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Duration of All-cause Hospitalizations	17.8 days Standard Deviation 12.07

SECONDARY outcome

Timeframe: 13 months

Outcome measures

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule n=45 Participants Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Distribution of Fever Episodes in the Total ITT Population. 0 fever episodes	34 Participants
Distribution of Fever Episodes in the Total ITT Population. 1 fever episode	6 Participants
Distribution of Fever Episodes in the Total ITT Population. 2 fever episodes	2 Participants
Distribution of Fever Episodes in the Total ITT Population. 3 fever episodes	1 Participants
Distribution of Fever Episodes in the Total ITT Population. 4 fever episodes	1 Participants
Distribution of Fever Episodes in the Total ITT Population. 5 fever episodes	1 Participants

SECONDARY outcome

Timeframe: 13 months

Population: The duration of fever episodes was measured for each of eleven subjects who experienced this event.

Outcome measures

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule n=11 Participants Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
Duration of Fever Episodes	6.5 days Standard Deviation 4.87

SECONDARY outcome

Timeframe: 13 months

Outcome measures

Measure	Kedrion IVIG 10% 21 and 28 Days Infusion Schedule n=45 Participants Kedrion IVIG 10% treatment 21 and 28-day infusion schedule Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 or 28 days; Duration - 12 months
IgG Trough Levels at Steady State in the Total ITT Population.	888 mg/dL Standard Deviation 197

Adverse Events

Kedrion IVIG 10%

Serious events: 4 serious events

Other events: 44 other events

Deaths: 0 deaths

Serious adverse events

Measure	Kedrion IVIG 10% n=45 participants at risk Kedrion IVIG 10% treatment. Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 to 28 days; Duration - 12 months
Infections and infestations Bacterial pneumonia	2.2% 1/45 • Number of events 1 • 13 months
Infections and infestations Herpes zoster	2.2% 1/45 • Number of events 1 • 13 months
Infections and infestations Infective exacerbation of bronchiectasis	2.2% 1/45 • Number of events 1 • 13 months
Psychiatric disorders Bipolar disorder	2.2% 1/45 • Number of events 2 • 13 months
Gastrointestinal disorders Diarrhoea	2.2% 1/45 • Number of events 1 • 13 months
Injury, poisoning and procedural complications Humerus Fracture	2.2% 1/45 • Number of events 1 • 13 months
Infections and infestations Pneumonia	2.2% 1/45 • Number of events 1 • 13 months

Other adverse events

Measure	Kedrion IVIG 10% n=45 participants at risk Kedrion IVIG 10% treatment. Kedrion IVIG 10%: Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 to 28 days; Duration - 12 months
Infections and infestations Upper Respiratory Tract Infection	28.9% 13/45 • Number of events 18 • 13 months
Infections and infestations Sinusitis	24.4% 11/45 • Number of events 22 • 13 months
Infections and infestations Viral Upper Respiratory Infection	11.1% 5/45 • Number of events 7 • 13 months
Infections and infestations Rhinitis	8.9% 4/45 • Number of events 5 • 13 months
Infections and infestations Acute Sinusitis	6.7% 3/45 • Number of events 6 • 13 months
Infections and infestations Bronchitis	6.7% 3/45 • Number of events 3 • 13 months
Infections and infestations Gastroenteritis Viral	6.7% 3/45 • Number of events 4 • 13 months
Infections and infestations Nasopharyngitis	6.7% 3/45 • Number of events 4 • 13 months
Infections and infestations Urinary Tract Infection	6.7% 3/45 • Number of events 5 • 13 months
General disorders Pyrexia	24.4% 11/45 • Number of events 22 • 13 months
General disorders Fatigue	17.8% 8/45 • Number of events 11 • 13 months
Respiratory, thoracic and mediastinal disorders Cough	22.2% 10/45 • Number of events 19 • 13 months
Respiratory, thoracic and mediastinal disorders Oropharyngeal Pain	11.1% 5/45 • Number of events 9 • 13 months
Respiratory, thoracic and mediastinal disorders Asthma	11.1% 5/45 • Number of events 5 • 13 months
Respiratory, thoracic and mediastinal disorders Nasal Congestion	8.9% 4/45 • Number of events 6 • 13 months
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	6.7% 3/45 • Number of events 7 • 13 months
Respiratory, thoracic and mediastinal disorders Sneezing	6.7% 3/45 • Number of events 3 • 13 months
General disorders Adverse drug reaction	6.7% 3/45 • Number of events 3 • 13 months
Nervous system disorders Headache	26.7% 12/45 • Number of events 21 • 13 months
Nervous system disorders Sinus Headache	8.9% 4/45 • Number of events 5 • 13 months
Gastrointestinal disorders Diarrhoea	17.8% 8/45 • Number of events 12 • 13 months
Gastrointestinal disorders Nausea	8.9% 4/45 • Number of events 7 • 13 months
Gastrointestinal disorders Vomiting	8.9% 4/45 • Number of events 5 • 13 months
Musculoskeletal and connective tissue disorders Back Pain	15.6% 7/45 • Number of events 8 • 13 months
Musculoskeletal and connective tissue disorders Myalgia	6.7% 3/45 • Number of events 6 • 13 months
Musculoskeletal and connective tissue disorders Arthralgia	6.7% 3/45 • Number of events 4 • 13 months
Musculoskeletal and connective tissue disorders Pain in extremity	6.7% 3/45 • Number of events 3 • 13 months
Injury, poisoning and procedural complications Contusion	6.7% 3/45 • Number of events 3 • 13 months
Investigations Investigations	26.7% 12/45 • Number of events 21 • 13 months
Psychiatric disorders Insomnia	8.9% 4/45 • Number of events 4 • 13 months
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders	13.3% 6/45 • Number of events 18 • 13 months
Blood and lymphatic system disorders Lymphadenopathy	6.7% 3/45 • Number of events 3 • 13 months
Eye disorders Eye Disorders	8.9% 4/45 • Number of events 6 • 13 months
Metabolism and nutrition disorders Metabolism and nutrition disorders	8.9% 4/45 • Number of events 5 • 13 months
Immune system disorders Immune System Disorders	6.7% 3/45 • Number of events 4 • 13 months
Vascular disorders Vascular Disorders	6.7% 3/45 • Number of events 5 • 13 months

Additional Information

Dr. Mirella Calcinai, MD, Clinical Research Director

Kedrion S.p.A

Phone: +393486559761

Email: m.calcinai@kedrion.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place