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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01440946




Registration number
NCT01440946
Ethics application status
Date submitted
16/09/2011
Date registered
27/09/2011
Date last updated
19/12/2020

Titles & IDs
Public title
Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B
Scientific title
An Open-label, Multicenter Evaluation of Safety, Pharmacokinetics and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in the Prevention and Treatment of Bleeding Episodes in Pediatric Subjects With Hemophilia B
Secondary ID [1] 0 0
2011-003076-36
Secondary ID [2] 0 0
9HB02PED
Universal Trial Number (UTN)
Trial acronym
Kids B-LONG
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia B 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - rFIXFc
Treatment: Drugs - FIX

Experimental: rFIXFc Prophylaxis - At Baseline and at Day 1, participants receive a single intravenous (IV) injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg will be administered in clinic as an IV injection.

Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.


Treatment: Drugs: rFIXFc
Vials of rFIXFc were combined as needed, based on the actual labeled potency to achieve the participant's calculated dose. Partial vial use was allowed, in order to achieve the calculated dose.

Treatment: Drugs: FIX
Vials of prestudy FIX (provided by the participants) were combined as needed, based on the nominal labeled potency (e.g., 250 IU, 500 IU, and 1000 IU), to achieve the participant's calculated dose.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Occurence of Factor IX (FIX) Inhibitor Development
Timepoint [1] 0 0
Up to 50 weeks +/- 7 days, or up to 50 EDs if reached prior to Week 50
Secondary outcome [1] 0 0
Annualized Bleeding Rate
Timepoint [1] 0 0
Up to 50 weeks +/- 7 days (efficacy period as defined in description)
Secondary outcome [2] 0 0
Annualized Joint Bleeding Rate (Spontaneous)
Timepoint [2] 0 0
Up to 50 weeks +/- 7 days (efficacy period as defined in description)
Secondary outcome [3] 0 0
Participant Assessment of Response to Injections to Treat a Bleeding Episode
Timepoint [3] 0 0
Up to 50 weeks +/- 7 days
Secondary outcome [4] 0 0
Physician's Global Assessment of the Participant's Response to His rFIXFc Regimen
Timepoint [4] 0 0
Up to 50 weeks +/- 7 days
Secondary outcome [5] 0 0
Annualized rFIXFc Consumption by Type of Injection
Timepoint [5] 0 0
Up to 50 weeks +/- 7 days (efficacy period as defined in description)
Secondary outcome [6] 0 0
Number of Days From the Last Prophylaxis Injection to a Spontaneous Bleeding Episode
Timepoint [6] 0 0
Up to 50 weeks +/- 7 days (efficacy period as defined in description)
Secondary outcome [7] 0 0
Number of Injections Required for Resolution of a Bleeding Episode
Timepoint [7] 0 0
Up to 50 weeks +/- 7 days (efficacy period as defined in description)
Secondary outcome [8] 0 0
Total Dose Required for Resolution of a Bleeding Episode
Timepoint [8] 0 0
Up to 50 weeks +/- 7 days (efficacy period as defined in description)
Secondary outcome [9] 0 0
Maximum Plasma Activity (Cmax; One-stage Activated Partial Thromboplastin Time [aPTT] Clotting Assay)
Timepoint [9] 0 0
Baseline (28±7 days before Day 1) Prestudy FIX Dosing: predose; 30±5 min, 3 hrs±30 min, 10±2 hrs, 24±3 hrs, 48±4 hrs postdose. Day 1 rFIXFc Dosing: predose; 30±5 min, 3 hrs ±30 min, 10±2 hrs, 24±3 hrs, 72±7 hrs, 120±12 hrs, 168±16 hrs postdose.
Secondary outcome [10] 0 0
Terminal Half Life (t1/2; One-stage aPTT Clotting Assay)
Timepoint [10] 0 0
Baseline (28±7 days before Day 1) Prestudy FIX Dosing: predose; 30±5 min, 3 hrs±30 min, 10±2 hrs, 24±3 hrs, 48±4 hrs postdose. Day 1 rFIXFc Dosing: predose; 30±5 min, 3 hrs ±30 min, 10±2 hrs, 24±3 hrs, 72±7 hrs, 120±12 hrs, 168±16 hrs postdose.
Secondary outcome [11] 0 0
Clearance (CL; One-stage aPTT Clotting Assay)
Timepoint [11] 0 0
Baseline (28±7 days before Day 1) Prestudy FIX Dosing: predose; 30±5 min, 3 hrs±30 min, 10±2 hrs, 24±3 hrs, 48±4 hrs postdose. Day 1 rFIXFc Dosing: predose; 30±5 min, 3 hrs ±30 min, 10±2 hrs, 24±3 hrs, 72±7 hrs, 120±12 hrs, 168±16 hrs postdose.
Secondary outcome [12] 0 0
Volume of Distribution at Steady State (Vss; One-stage aPTT Clotting Assay)
Timepoint [12] 0 0
Baseline (28±7 days before Day 1) Prestudy FIX Dosing: predose; 30±5 min, 3 hrs±30 min, 10±2 hrs, 24±3 hrs, 48±4 hrs postdose. Day 1 rFIXFc Dosing: predose; 30±5 min, 3 hrs ±30 min, 10±2 hrs, 24±3 hrs, 72±7 hrs, 120±12 hrs, 168±16 hrs postdose.
Secondary outcome [13] 0 0
Dose Normalized Area Under the Curve (DNAUC; One-stage aPTT Clotting Assay)
Timepoint [13] 0 0
Baseline (28±7 days before Day 1) Prestudy FIX Dosing: predose; 30±5 min, 3 hrs±30 min, 10±2 hrs, 24±3 hrs, 48±4 hrs postdose. Day 1 rFIXFc Dosing: predose; 30±5 min, 3 hrs ±30 min, 10±2 hrs, 24±3 hrs, 72±7 hrs, 120±12 hrs, 168±16 hrs postdose.
Secondary outcome [14] 0 0
Mean Residence Time (MRT; One-stage aPTT Clotting Assay)
Timepoint [14] 0 0
Baseline (28±7 days before Day 1) Prestudy FIX Dosing: predose; 30±5 min, 3 hrs±30 min, 10±2 hrs, 24±3 hrs, 48±4 hrs postdose. Day 1 rFIXFc Dosing: predose; 30±5 min, 3 hrs ±30 min, 10±2 hrs, 24±3 hrs, 72±7 hrs, 120±12 hrs, 168±16 hrs postdose.
Secondary outcome [15] 0 0
Incremental Recovery (IR; One-stage aPTT Clotting Assay)
Timepoint [15] 0 0
Baseline (28±7 days before Day 1) Prestudy FIX Dosing: predose; 30±5 min, 3 hrs±30 min, 10±2 hrs, 24±3 hrs, 48±4 hrs postdose. Day 1 rFIXFc Dosing: predose; 30±5 min, 3 hrs ±30 min, 10±2 hrs, 24±3 hrs, 72±7 hrs, 120±12 hrs, 168±16 hrs postdose.

Eligibility
Key inclusion criteria
Key

* Severe hemophilia B defined as = 2 IU/dl (= 2%) endogenous FIX
* Male < 12 years and weight = 13 kg
* History of at least 50 documented prior exposure days to FIX
* No history of, or currently detectable, inhibitor

Key
Minimum age
No limit
Maximum age
11 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* Other coagulation disorders in addition to Hemophilia B
* History of anaphylaxis associated with any FIX or IV immunoglobulin administration

NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/06/2012
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/11/2014
Sample size
Target
Accrual to date
Final
30
Recruitment in Australia
Recruitment state(s)
VIC,WA
Recruitment hospital [1] 0 0
Research Site - Parkville
Recruitment hospital [2] 0 0
Research Site - Subiaco
Recruitment postcode(s) [1] 0 0
- Parkville
Recruitment postcode(s) [2] 0 0
- Subiaco
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Hawaii
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
Ireland
State/province [8] 0 0
Dublin
Country [9] 0 0
Netherlands
State/province [9] 0 0
Utrecht
Country [10] 0 0
South Africa
State/province [10] 0 0
Johannesburg
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Basingstoke
Country [12] 0 0
United Kingdom
State/province [12] 0 0
Cambridge
Country [13] 0 0
United Kingdom
State/province [13] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Bioverativ Therapeutics Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Swedish Orphan Biovitrum
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
The primary objective of the study is to evaluate the safety of Recombinant Human Coagulation Factor IX Fc Fusion Protein (rFIXFc) in previously treated pediatric subjects with hemophilia B. Secondary objectives of this study in this study population are as follows: to evaluate the efficacy of rFIXFc for prevention and treatment of bleeding episodes; to evaluate and assess the pharmacokinetics (PK) of rFIXFc; to evaluate rFIXFc consumption for prevention and treatment of bleeding episodes
Trial website
https://clinicaltrials.gov/study/NCT01440946
Trial related presentations / publications
Fischer K, Kulkarni R, Nolan B, Mahlangu J, Rangarajan S, Gambino G, Diao L, Ramirez-Santiago A, Pierce GF, Allen G. Recombinant factor IX Fc fusion protein in children with haemophilia B (Kids B-LONG): results from a multicentre, non-randomised phase 3 study. Lancet Haematol. 2017 Feb;4(2):e75-e82. doi: 10.1016/S2352-3026(16)30193-4.
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Bioverativ Therapeutics Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01440946