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An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A

Primary Purpose

Hemophilia A

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
rFVIIIFc
Sponsored by
Bioverativ, a Sanofi company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A focused on measuring prophylaxis treatment, Hemophilia A, Hemophilia, episodic treatment

Eligibility Criteria

undefined - 5 Years (Child)MaleDoes not accept healthy volunteers

Key Inclusion Criteria:

  • Ability of the participant's legally authorized representative (e.g. their parent or legal guardian) to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local participant privacy regulations.
  • Weight >=3.5 kg at the time of screening.
  • Severe hemophilia A defined as less than (<) 1 IU/dL (<1%) endogenous FVIII documented in the medical record or as tested during the Screening Period.

Key Exclusion Criteria:

  • Any exposure to blood components, factor VIII replacement products, including commercially available rFVIIIFc at any time prior to or during screening.
  • History of positive inhibitor testing. A prior history of inhibitors was defined based on a patient's historical positive inhibitor test using the local laboratory Bethesda value for a positive inhibitor test (ie, equal to or above lower level of detection).
  • History of hypersensitivity reactions associated with any rFVIIIFc administration.
  • Other coagulation disorder(s) in addition to hemophilia A.
  • Any concurrent clinically significant major disease that, in the opinion of the Investigator, would make the participant unsuitable for enrollment.
  • Current systemic treatment with chemotherapy and/or other immunosuppressant drugs.

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

Sites / Locations

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Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

rFVIIIFc

Arm Description

Participants were to receive rFVIIIFc as follows- Prophylaxis regimen (PR): rFVIIIFc 25-80 international units per kilogram (IU/kg), at 3- to 5-day intervals until participant reached greater than or equal to (>=) 50 exposure days (ED: 24-hour period in which >=1 injection/dose of rFVIIIFc was given), or study withdrawal/end of study. Adjustments to dose/dosing interval was done as needed by investigator; Treatment with an optional ER (Episodic regimen) can be initiated before PR at investigators discretion; ITI: rFVIIIFc 200 IU/kg, daily for participants who, after exposure to rFVIIIFc, had positive high titer inhibitor (>=5.00 Bethesda Units per milliliter [BU/mL]) or positive low titer inhibitor (>=0.60 and <5.00 BU/mL) and had poorly controlled bleeding despite increased rFVIIIFc doses, or required bypassing agent to treat bleeding.

Outcomes

Primary Outcome Measures

Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
A positive/confirmed inhibitor result occurs when a participant has a value >=0.6 BU/mL by central laboratory testing using Nijmegen-modified Bethesda assay, that is confirmed on re-testing of a separate sample collected >=2 weeks after the initial sample. Confirmed inhibitor development was based on all participants who had reached >=10 EDs and had >=1 inhibitor test performed at or beyond this milestone or who had an inhibitor. Exposure day (ED) is a 24-hour period in which participant received >=1 dose of rFVIIIFc injections. Participants who did not develop an inhibitor but reached the milestone number of EDs were included in the denominator during calculation of percentage. Additionally, any participant who developed an inhibitor following the initial rFVIIIFc administration was included in the numerator and denominator during calculation of percentage.

Secondary Outcome Measures

Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR])
ABR was annualized number of bleeding episodes during efficacy period (EP) per participant annualized to a 1-year interval of time. Bleeding episodes were classified as spontaneous if parent/caregiver/participant records bleeding event when there is no known contributing factor such as definite trauma or antecedent "strenuous" activity and as traumatic when there is known reason for bleed. ABR=(Number of bleeding episodes during EP divided by total number of days during EP)*365.25. EP was sum of all intervals of time during which participants were treated with rFVIIIFc per treatment regimens of study excluding surgical/rehabilitation periods and large injection intervals (greater than [>]28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Annualized Number of Spontaneous Joint Bleeding Episodes
Bleeding episodes were classified as spontaneous if parent/caregiver/participant records a bleeding event when there was no known contributing factor such as a definite trauma or antecedent "strenuous" activity. Annualized spontaneous joint bleeding episodes = (Total number of spontaneous joint bleeding episodes during EP divided by total number of days during EP)*365.25. EP reflects sum of all intervals of time during which participants were treated with rFVIIIFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals (> 28 days). Bleeding episodes were summarized by treatment regimen. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Number of rFVIIIFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale
Using e-diary, each participant's parent/caregiver rated treatment response to any bleeding episode at approximately 8-12 hours from time of injection and prior to additional doses of rFVIIIFc given for same bleeding episodes, using 4-point scale: 1=Excellent: abrupt pain relief and/or improvement in signs of bleeding within approximately 8 hour after initial injection; 2=Good: definite pain relief and/or improvement in signs of bleeding within approximately 8 hour after injection, but possibly requiring more than 1 injection after 24-48 hour for complete resolution; 3=Moderate: Probable/slight beneficial effect within 8 hour after initial injection and requires more than 1 injection and 4=None: No improvement or condition worsens within approximately 8 hour after initial injection. Participants included in more than 1 treatment regimen if their regimen changed during study.
Total Number of Exposure Days (EDs)
An ED was defined as a 24-hour period in which a participant received one or more doses of rFVIIIFc injections, with the time of the first injection of rFVIIIFc defined as the start of the ED. Participants who did not have a particular injection type were counted as having zero injections for that type.
Total Annualized rFVIIIFc Consumption Per Participant for the Prevention and Treatment of Bleeding Episodes
Total annualized rFVIIIFc consumption (in IU/kg) was calculated for each participant as: Annualized consumption = (Total IU/kg of rFVIIIFc during EP divided by total number of days during EP)*365.25. EP reflects the sum of all intervals of time during which participants are treated with rFVIIIFc according to the treatment regimens of the study excluding surgical/rehabilitation periods and large injection intervals (>28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Number of Injections of rFVIIIFc Required to Resolve a Bleeding Episode
Number of Injections of rFVIIIFc required to resolve a bleeding episode during EP were reported. EP reflects the sum of all intervals of time during which participants were treated with rFVIIIFc according to the treatment regimens of the study excluding surgical/rehabilitation periods and large injection intervals (>28 days). All injections given from the initial sign of a bleed, until the last date/time within the bleed window were counted. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Average Dose Per Injection of rFVIIIFc Required to Resolve a Bleeding Episode
The average dose of rFVIIIFc per injection per bleeding episode was calculated as the average of all doses (IU/kg) administered to treat the bleeding episode during EP. EP begins with the first treatment regimen dose of rFVIIIFc and ends with the last dose (regardless of the reason for dosing). Surgery/rehabilitation periods were not included in the EP. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Change From Baseline in rFVIIIFc Incremental Recovery (IR)
Blood samples were taken at trough (predose) and Cmax (maximum concentration) for assessment of incremental recovery, measured by the one-stage clotting assay. IR (International Units per deciliter [IU/dL] per IU/kg) = (Cmax for FVIII activity - Pre-dose FVIII activity) (IU/dL) divided by actual dose (IU/kg), where Cmax (maximum concentration) is 30-minute FVIII activity post-dose and FVIII activity less than (<)0.5 IU/dL was set to 0 IU/dL for calculation of IR.
Number of Participants With Response to Immune Tolerance Induction (ITI)
Complete Success was defined as meeting all of the following criteria: Negative inhibitor titers in 2 consecutive determinations at least 4 weeks apart; IR >=66% of baseline in 2 consecutive determinations at least 4 weeks apart; Half life >=6 hours. Partial Success was defined as meeting the first criteria for complete success and one of the other 2 after 33 months of ITI.

Full Information

First Posted
August 29, 2014
Last Updated
March 15, 2022
Sponsor
Bioverativ, a Sanofi company
Collaborators
Swedish Orphan Biovitrum
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1. Study Identification

Unique Protocol Identification Number
NCT02234323
Brief Title
An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A
Official Title
An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc; BIIB031) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia A
Study Type
Interventional

2. Study Status

Record Verification Date
March 2022
Overall Recruitment Status
Completed
Study Start Date
January 12, 2015 (Actual)
Primary Completion Date
September 23, 2019 (Actual)
Study Completion Date
September 23, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bioverativ, a Sanofi company
Collaborators
Swedish Orphan Biovitrum

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The primary objective of the study was to evaluate the safety of rFVIIIFc (BIIB031) in previously untreated participants (PUPs) with severe hemophilia A. The secondary objectives were to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in PUPs, to evaluate rFVIIIFc consumption for the prevention and treatment of bleeding episodes in PUPs, and to describe experience with the use of rFVIIIFc for immune tolerance induction (ITI) in participants with inhibitors.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A
Keywords
prophylaxis treatment, Hemophilia A, Hemophilia, episodic treatment

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
108 (Actual)

8. Arms, Groups, and Interventions

Arm Title
rFVIIIFc
Arm Type
Experimental
Arm Description
Participants were to receive rFVIIIFc as follows- Prophylaxis regimen (PR): rFVIIIFc 25-80 international units per kilogram (IU/kg), at 3- to 5-day intervals until participant reached greater than or equal to (>=) 50 exposure days (ED: 24-hour period in which >=1 injection/dose of rFVIIIFc was given), or study withdrawal/end of study. Adjustments to dose/dosing interval was done as needed by investigator; Treatment with an optional ER (Episodic regimen) can be initiated before PR at investigators discretion; ITI: rFVIIIFc 200 IU/kg, daily for participants who, after exposure to rFVIIIFc, had positive high titer inhibitor (>=5.00 Bethesda Units per milliliter [BU/mL]) or positive low titer inhibitor (>=0.60 and <5.00 BU/mL) and had poorly controlled bleeding despite increased rFVIIIFc doses, or required bypassing agent to treat bleeding.
Intervention Type
Biological
Intervention Name(s)
rFVIIIFc
Other Intervention Name(s)
Eloctate; BIIB031; efmoroctocog alfa; recombinant coagulation factor VIII Fc fusion protein; antihemophilic factor [recombinant], Fc fusion protein
Intervention Description
Administered as specified in arm description
Primary Outcome Measure Information:
Title
Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
Description
A positive/confirmed inhibitor result occurs when a participant has a value >=0.6 BU/mL by central laboratory testing using Nijmegen-modified Bethesda assay, that is confirmed on re-testing of a separate sample collected >=2 weeks after the initial sample. Confirmed inhibitor development was based on all participants who had reached >=10 EDs and had >=1 inhibitor test performed at or beyond this milestone or who had an inhibitor. Exposure day (ED) is a 24-hour period in which participant received >=1 dose of rFVIIIFc injections. Participants who did not develop an inhibitor but reached the milestone number of EDs were included in the denominator during calculation of percentage. Additionally, any participant who developed an inhibitor following the initial rFVIIIFc administration was included in the numerator and denominator during calculation of percentage.
Time Frame
Up to 3 years
Secondary Outcome Measure Information:
Title
Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR])
Description
ABR was annualized number of bleeding episodes during efficacy period (EP) per participant annualized to a 1-year interval of time. Bleeding episodes were classified as spontaneous if parent/caregiver/participant records bleeding event when there is no known contributing factor such as definite trauma or antecedent "strenuous" activity and as traumatic when there is known reason for bleed. ABR=(Number of bleeding episodes during EP divided by total number of days during EP)*365.25. EP was sum of all intervals of time during which participants were treated with rFVIIIFc per treatment regimens of study excluding surgical/rehabilitation periods and large injection intervals (greater than [>]28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Time Frame
Up to 3 years
Title
Annualized Number of Spontaneous Joint Bleeding Episodes
Description
Bleeding episodes were classified as spontaneous if parent/caregiver/participant records a bleeding event when there was no known contributing factor such as a definite trauma or antecedent "strenuous" activity. Annualized spontaneous joint bleeding episodes = (Total number of spontaneous joint bleeding episodes during EP divided by total number of days during EP)*365.25. EP reflects sum of all intervals of time during which participants were treated with rFVIIIFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals (> 28 days). Bleeding episodes were summarized by treatment regimen. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Time Frame
Up to 3 years
Title
Number of rFVIIIFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale
Description
Using e-diary, each participant's parent/caregiver rated treatment response to any bleeding episode at approximately 8-12 hours from time of injection and prior to additional doses of rFVIIIFc given for same bleeding episodes, using 4-point scale: 1=Excellent: abrupt pain relief and/or improvement in signs of bleeding within approximately 8 hour after initial injection; 2=Good: definite pain relief and/or improvement in signs of bleeding within approximately 8 hour after injection, but possibly requiring more than 1 injection after 24-48 hour for complete resolution; 3=Moderate: Probable/slight beneficial effect within 8 hour after initial injection and requires more than 1 injection and 4=None: No improvement or condition worsens within approximately 8 hour after initial injection. Participants included in more than 1 treatment regimen if their regimen changed during study.
Time Frame
Up to 3 years
Title
Total Number of Exposure Days (EDs)
Description
An ED was defined as a 24-hour period in which a participant received one or more doses of rFVIIIFc injections, with the time of the first injection of rFVIIIFc defined as the start of the ED. Participants who did not have a particular injection type were counted as having zero injections for that type.
Time Frame
Up to 3 years
Title
Total Annualized rFVIIIFc Consumption Per Participant for the Prevention and Treatment of Bleeding Episodes
Description
Total annualized rFVIIIFc consumption (in IU/kg) was calculated for each participant as: Annualized consumption = (Total IU/kg of rFVIIIFc during EP divided by total number of days during EP)*365.25. EP reflects the sum of all intervals of time during which participants are treated with rFVIIIFc according to the treatment regimens of the study excluding surgical/rehabilitation periods and large injection intervals (>28 days). Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Time Frame
Up to 3 years
Title
Number of Injections of rFVIIIFc Required to Resolve a Bleeding Episode
Description
Number of Injections of rFVIIIFc required to resolve a bleeding episode during EP were reported. EP reflects the sum of all intervals of time during which participants were treated with rFVIIIFc according to the treatment regimens of the study excluding surgical/rehabilitation periods and large injection intervals (>28 days). All injections given from the initial sign of a bleed, until the last date/time within the bleed window were counted. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Time Frame
Up to 3 years
Title
Average Dose Per Injection of rFVIIIFc Required to Resolve a Bleeding Episode
Description
The average dose of rFVIIIFc per injection per bleeding episode was calculated as the average of all doses (IU/kg) administered to treat the bleeding episode during EP. EP begins with the first treatment regimen dose of rFVIIIFc and ends with the last dose (regardless of the reason for dosing). Surgery/rehabilitation periods were not included in the EP. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Time Frame
Up to 3 years
Title
Change From Baseline in rFVIIIFc Incremental Recovery (IR)
Description
Blood samples were taken at trough (predose) and Cmax (maximum concentration) for assessment of incremental recovery, measured by the one-stage clotting assay. IR (International Units per deciliter [IU/dL] per IU/kg) = (Cmax for FVIII activity - Pre-dose FVIII activity) (IU/dL) divided by actual dose (IU/kg), where Cmax (maximum concentration) is 30-minute FVIII activity post-dose and FVIII activity less than (<)0.5 IU/dL was set to 0 IU/dL for calculation of IR.
Time Frame
Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108 and 120
Title
Number of Participants With Response to Immune Tolerance Induction (ITI)
Description
Complete Success was defined as meeting all of the following criteria: Negative inhibitor titers in 2 consecutive determinations at least 4 weeks apart; IR >=66% of baseline in 2 consecutive determinations at least 4 weeks apart; Half life >=6 hours. Partial Success was defined as meeting the first criteria for complete success and one of the other 2 after 33 months of ITI.
Time Frame
Up to 3 years

10. Eligibility

Sex
Male
Maximum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Ability of the participant's legally authorized representative (e.g. their parent or legal guardian) to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local participant privacy regulations. Weight >=3.5 kg at the time of screening. Severe hemophilia A defined as less than (<) 1 IU/dL (<1%) endogenous FVIII documented in the medical record or as tested during the Screening Period. Key Exclusion Criteria: Any exposure to blood components, factor VIII replacement products, including commercially available rFVIIIFc at any time prior to or during screening. History of positive inhibitor testing. A prior history of inhibitors was defined based on a patient's historical positive inhibitor test using the local laboratory Bethesda value for a positive inhibitor test (ie, equal to or above lower level of detection). History of hypersensitivity reactions associated with any rFVIIIFc administration. Other coagulation disorder(s) in addition to hemophilia A. Any concurrent clinically significant major disease that, in the opinion of the Investigator, would make the participant unsuitable for enrollment. Current systemic treatment with chemotherapy and/or other immunosuppressant drugs. Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Facility Information:
Facility Name
Research Site
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
Research Site
City
Duarte
State/Province
California
ZIP/Postal Code
91010
Country
United States
Facility Name
Research Site
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Research Site
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Research Site
City
Torrance
State/Province
California
ZIP/Postal Code
90509
Country
United States
Facility Name
Research Site
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46260
Country
United States
Facility Name
Research Site
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Facility Name
Research Site
City
Brewer
State/Province
Maine
ZIP/Postal Code
04412
Country
United States
Facility Name
Research Site
City
East Lansing
State/Province
Michigan
ZIP/Postal Code
48823
Country
United States
Facility Name
Research Site
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55404
Country
United States
Facility Name
Research Site
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63104
Country
United States
Facility Name
Research Site
City
Buffalo
State/Province
New York
ZIP/Postal Code
14209
Country
United States
Facility Name
Research Site
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
Research Site
City
Rochester
State/Province
New York
ZIP/Postal Code
14621
Country
United States
Facility Name
Research Site
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
Research Site
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Research Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Research Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75230
Country
United States
Facility Name
Research Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Facility Name
Research Site
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
Research Site
City
Brisbane
ZIP/Postal Code
4029
Country
Australia
Facility Name
Research Site
City
Parkville
ZIP/Postal Code
3052
Country
Australia
Facility Name
Research Site
City
Perth
ZIP/Postal Code
6008
Country
Australia
Facility Name
Research Site
City
Westmead
ZIP/Postal Code
2145
Country
Australia
Facility Name
Research Site
City
Campinas
ZIP/Postal Code
13083-878
Country
Brazil
Facility Name
Research Site
City
Canoas
ZIP/Postal Code
92425-900
Country
Brazil
Facility Name
Research Site
City
Ribeirão Preto
ZIP/Postal Code
14048-900
Country
Brazil
Facility Name
Research Site
City
Rio de Janeiro
ZIP/Postal Code
20211-030
Country
Brazil
Facility Name
Research Site
City
São Paulo
ZIP/Postal Code
01227-200
Country
Brazil
Facility Name
Research Site
City
Hamilton
ZIP/Postal Code
L8S 4K1
Country
Canada
Facility Name
Research Site
City
London
ZIP/Postal Code
N6A 4G5
Country
Canada
Facility Name
Research Site
City
Toronto
ZIP/Postal Code
M5G 1X8
Country
Canada
Facility Name
Research Site
City
Caen cedex 9
ZIP/Postal Code
14033
Country
France
Facility Name
Research Site
City
Le Kremlin Bicêtre cedex
ZIP/Postal Code
94270
Country
France
Facility Name
Research Site
City
Lille Cedex
ZIP/Postal Code
59037
Country
France
Facility Name
Research Site
City
Nantes Cedex 1
ZIP/Postal Code
44093
Country
France
Facility Name
Research Site
City
Strasbourg
ZIP/Postal Code
67200
Country
France
Facility Name
Research Site
City
Toulouse cedex
ZIP/Postal Code
31059
Country
France
Facility Name
Research Site
City
Tours cedex 9
ZIP/Postal Code
37044
Country
France
Facility Name
Research Site
City
Berlin
ZIP/Postal Code
13353
Country
Germany
Facility Name
Research Site
City
Bonn
ZIP/Postal Code
53127
Country
Germany
Facility Name
Research Site
City
Duesseldorf
ZIP/Postal Code
40225
Country
Germany
Facility Name
Research Site
City
Frankfurt
ZIP/Postal Code
60590
Country
Germany
Facility Name
Research Site
City
Hannöver
ZIP/Postal Code
30625
Country
Germany
Facility Name
Research Site
City
Dublin
ZIP/Postal Code
D12 N512
Country
Ireland
Facility Name
Research Site
City
Vicenza
State/Province
VI
ZIP/Postal Code
36100
Country
Italy
Facility Name
Research Site
City
Bari
ZIP/Postal Code
70123
Country
Italy
Facility Name
Research Site
City
Florence
ZIP/Postal Code
50134
Country
Italy
Facility Name
Research Site
City
Genova
ZIP/Postal Code
16148
Country
Italy
Facility Name
Research Site
City
Milan
ZIP/Postal Code
20122
Country
Italy
Facility Name
Research Site
City
Napoli
ZIP/Postal Code
80123
Country
Italy
Facility Name
Research Site
City
Padova
ZIP/Postal Code
35128
Country
Italy
Facility Name
Research Site
City
Rome
ZIP/Postal Code
161
Country
Italy
Facility Name
Research Site
City
Rome
ZIP/Postal Code
165
Country
Italy
Facility Name
Research Site
City
Leiden
ZIP/Postal Code
2333 ZA
Country
Netherlands
Facility Name
Research Site
City
Utrecht
ZIP/Postal Code
3584 CX
Country
Netherlands
Facility Name
Research Site
City
Auckland
ZIP/Postal Code
1023
Country
New Zealand
Facility Name
Research Site
City
Christchurch
ZIP/Postal Code
8011
Country
New Zealand
Facility Name
Research Site
City
Gdansk
ZIP/Postal Code
80-952
Country
Poland
Facility Name
Research Site
City
Kraków
ZIP/Postal Code
30-663
Country
Poland
Facility Name
Research Site
City
Lublin
ZIP/Postal Code
20-093
Country
Poland
Facility Name
Research Site
City
Warszawa
ZIP/Postal Code
02-091
Country
Poland
Facility Name
Research Site
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Research Site
City
Madrid
ZIP/Postal Code
28046
Country
Spain
Facility Name
Research Site
City
Stockholm
ZIP/Postal Code
171 76
Country
Sweden
Facility Name
Research Site
City
Cambridge
State/Province
Cambridgeshire
ZIP/Postal Code
CB2 0QQ
Country
United Kingdom
Facility Name
Research Site
City
Basingstoke
State/Province
Hampshire
ZIP/Postal Code
RG24 9NA
Country
United Kingdom
Facility Name
Research Site
City
Whitechapel
State/Province
London
ZIP/Postal Code
E1 1BB
Country
United Kingdom
Facility Name
Research Site
City
Cardiff
ZIP/Postal Code
CF14 4XW
Country
United Kingdom
Facility Name
Research Site
City
London
ZIP/Postal Code
WC1N3JH
Country
United Kingdom
Facility Name
Research Site
City
Sheffield
ZIP/Postal Code
S10 2TH
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Citations:
PubMed Identifier
35421219
Citation
Konigs C, Ozelo MC, Dunn A, Kulkarni R, Nolan B, Brown SA, Schiavulli M, Gunawardena S, Mukhopadhyay S, Jayawardene D, Winding B, Carcao M. First study of extended half-life rFVIIIFc in previously untreated patients with hemophilia A: PUPs A-LONG final results. Blood. 2022 Jun 30;139(26):3699-3707. doi: 10.1182/blood.2021013563. Erratum In: Blood. 2023 Mar 23;141(12):1495.
Results Reference
derived

Learn more about this trial

An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A

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