Trial Outcomes & Findings for Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B (NCT NCT01271868)

NCT ID: NCT01271868

Last Updated: 2021-03-15

Results Overview

• Recruitment status: TERMINATED
• Study phase: PHASE3
• Target enrollment: 9 participants
• Primary outcome timeframe: Treatment Study: at least 50 ED (approximately 6 months); actual mean subject duration: 39.7 ± 12.4 months
• Results posted on: 2021-03-15

Participant Flow

Participant milestones

Measure	IB1001
Pharmacokinetic Study STARTED	9
Pharmacokinetic Study COMPLETED	9
Pharmacokinetic Study NOT COMPLETED	0
Treatment Study STARTED	9
Treatment Study COMPLETED	9
Treatment Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B

Baseline characteristics by cohort

Measure	IB1001 n=9 Participants PK study and Treatment Study
Age, Customized 0 to 5 years	3 years n=5 Participants
Age, Customized 6 to 11 years	6 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	7 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	2 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Treatment Study: at least 50 ED (approximately 6 months); actual mean subject duration: 39.7 ± 12.4 months

Outcome measures

Measure	IB1001 n=33 Number of Infusions Treatment phase: Prophylaxis treatment (n=8) and on demand treatment (n=1)
Number of Infusions Required for Bleed Control 0 infusions	4 Number of Infusions
Number of Infusions Required for Bleed Control 1 infusion	25 Number of Infusions
Number of Infusions Required for Bleed Control 2 infusions	1 Number of Infusions
Number of Infusions Required for Bleed Control 3 infusions	1 Number of Infusions
Number of Infusions Required for Bleed Control 4 infusions	1 Number of Infusions
Number of Infusions Required for Bleed Control 5 infusions	1 Number of Infusions

SECONDARY outcome

Timeframe: Pre-infusion to 72 hours following infusion

Population: 9 subjects completed the PK phase. Only 6 subjects had adequate data for formal PK analysis.

Factor IX levels were assessed at the following time points:

Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours

Outcome measures

Measure	IB1001 n=6 Participants Treatment phase: Prophylaxis treatment (n=8) and on demand treatment (n=1)
Area Under the Curve (0-inf)	1062 IU*hr/dL Standard Deviation 322

SECONDARY outcome

Timeframe: Pre-infusion to 72 hours following infusion

Population: 9 subjects completed the PK phase. Only 6 subjects had adequate data for formal PK analysis.

Factor IX levels were assessed at the following time points:

Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours

Outcome measures

Measure	IB1001 n=6 Participants Treatment phase: Prophylaxis treatment (n=8) and on demand treatment (n=1)
Terminal Half-life	22.6 hours Standard Deviation 7

SECONDARY outcome

Timeframe: Pre-infusion to 72 hours following infusion

Population: 9 subjects completed the PK phase. Only 6 subjects had adequate data for formal PK analysis.

Factor IX levels were assessed at the following time points:

Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours

Outcome measures

Measure	IB1001 n=6 Participants Treatment phase: Prophylaxis treatment (n=8) and on demand treatment (n=1)
Concentration (Max)	51.7 IU/dL Standard Deviation 15.7

SECONDARY outcome

Timeframe: Pre-infusion to 72 hours following infusion

Population: 9 subjects completed the PK phase. Only 6 subjects had adequate data for formal PK analysis.

Factor IX levels were assessed at the following time points:

Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours

Outcome measures

Measure	IB1001 n=6 Participants Treatment phase: Prophylaxis treatment (n=8) and on demand treatment (n=1)
Incremental Recovery	0.84 IU/dL per IU/kg Standard Deviation 0.50

SECONDARY outcome

Timeframe: Pre-infusion to 72 hours following infusion

Population: 9 subjects completed the PK phase. Only 6 subjects had adequate data for formal PK analysis.

Factor IX levels were assessed at the following time points:

Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours

Outcome measures

Measure	IB1001 n=6 Participants Treatment phase: Prophylaxis treatment (n=8) and on demand treatment (n=1)
Clearance	7.3 mL/(kg*hr) Standard Deviation 3.1

SECONDARY outcome

Timeframe: Pre-infusion to 72 hours following infusion

Population: 9 subjects completed the PK phase. Only 6 subjects had adequate data for formal PK analysis.

Factor IX levels were assessed at the following time points:

Pre-infusion, post-infusion at 15-30 min, 4-6 hours, 24-26 hours, 68-72 hours

Outcome measures

Measure	IB1001 n=6 Participants Treatment phase: Prophylaxis treatment (n=8) and on demand treatment (n=1)
Volume of Distribution (Steady State)	244 mL/kg Standard Deviation 166

SECONDARY outcome

Timeframe: Treatment Study: at least 50 ED (approximately 6 months); actual mean subject duration: 41.7+/- 11.7 Months

Measure was assessed during the Treatment Study

Outcome measures

Measure	IB1001 n=8 Participants Treatment phase: Prophylaxis treatment (n=8) and on demand treatment (n=1)
Annualized Bleed Rate	0.26 bleeds/year Interval 0.0 to 1.6

Adverse Events

IB1001

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	IB1001 n=9 participants at risk IB1001 Safety population includes all subjects who received at least one dose of IB1001
Gastrointestinal disorders Vomiting	11.1% 1/9
Gastrointestinal disorders Toothache	11.1% 1/9
General disorders Pyrexia	44.4% 4/9
General disorders Swelling	11.1% 1/9
Infections and infestations Nasopharyngitis	22.2% 2/9
Infections and infestations Otitis Media	11.1% 1/9
Infections and infestations Pneumonia	11.1% 1/9
Injury, poisoning and procedural complications Contusion	11.1% 1/9
Injury, poisoning and procedural complications Radius fracture	11.1% 1/9
Injury, poisoning and procedural complications Upper limb fracture	11.1% 1/9
Injury, poisoning and procedural complications Limb injury	11.1% 1/9
Injury, poisoning and procedural complications Laceration	11.1% 1/9
Metabolism and nutrition disorders Iron deficiency	11.1% 1/9
Musculoskeletal and connective tissue disorders Muscular weakness	11.1% 1/9
Musculoskeletal and connective tissue disorders Joint swelling	11.1% 1/9
Musculoskeletal and connective tissue disorders Joint range of motion decreased	11.1% 1/9
Respiratory, thoracic and mediastinal disorders Cough	22.2% 2/9
Respiratory, thoracic and mediastinal disorders Epistaxis	11.1% 1/9
Skin and subcutaneous tissue disorders Rash	22.2% 2/9

Additional Information

Dr. David Schaaf

Aptevo Therapeutics

Phone: 206-859-6655

Email: schaafd@apvo.com

Results disclosure agreements

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