Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT02078427




Registration number
NCT02078427
Ethics application status
Date submitted
3/03/2014
Date registered
5/03/2014
Date last updated
25/01/2024

Titles & IDs
Public title
ADVATE/ ADYNOVI Hemophilia A Outcome Database (AHEAD)
Scientific title
ADVATE/ ADYNOVI Hemophilia A Outcome Database
Secondary ID [1] 0 0
061001
Universal Trial Number (UTN)
Trial acronym
AHEAD
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia A 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
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - ADVATE
Other interventions - ADYNOVI

rAHF-PFM - Participants treated with rAHF-PFM alone

rAHF-PEG - Participants treated with rAHF-PEG alone

rAHF-PFM then rAHF-PEG - Participants treated with rAHF-PFM and subsequently switched to rAHF-PEG


Other interventions: ADVATE
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method

Other interventions: ADYNOVI
Antihemophilic Factor (Recombinant) Pegylated
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Joint Health Outcomes - Assessed by Physical Exam Using Only the Pain, Bleeding, and Physical Exam Parameters of the Gilbert Scale
Timepoint [1] 0 0
Up to approximately 12 years
Secondary outcome [1] 0 0
Annualized Bleed Rate, All Joints
Timepoint [1] 0 0
Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit
Secondary outcome [2] 0 0
Annualized Bleed Rate, All Bleeds
Timepoint [2] 0 0
Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit
Secondary outcome [3] 0 0
Annualized bleed rate, pre-existing target joints at baseline
Timepoint [3] 0 0
Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit
Secondary outcome [4] 0 0
Incidence of New Target Joints
Timepoint [4] 0 0
Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit
Secondary outcome [5] 0 0
Status of joint health by X-ray by Pettersson scale
Timepoint [5] 0 0
Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit.
Secondary outcome [6] 0 0
Status of Joint Health by Magnetic Resonance Imaging (MRI) Scoring System- Using The Lund Scoring System (LSS)
Timepoint [6] 0 0
Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit
Secondary outcome [7] 0 0
Status of joint health using the Hemophilia Joint Health Score (HJHS)
Timepoint [7] 0 0
Screening visit; Annual/Interval visits:- Up to 8 years if rAHF-PFM alone- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit
Secondary outcome [8] 0 0
Overall effectiveness assessment for prophylaxis therapy
Timepoint [8] 0 0
Annual/Interval visits:- Up to 8 years if rAHF-PFM alone;- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years;- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit
Secondary outcome [9] 0 0
Compliance with the dosing prescribed and its relationship with effectiveness
Timepoint [9] 0 0
Annual/Interval visits:- Up to 8 years if rAHF-PFM alone;- Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years;- Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit
Secondary outcome [10] 0 0
Overall effectiveness assessment for on-demand treatment
Timepoint [10] 0 0
Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit
Secondary outcome [11] 0 0
Global effectiveness assessment for on-demand treatment
Timepoint [11] 0 0
Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit
Secondary outcome [12] 0 0
Number of rAHF-PFM or rAHF-PEG units required for bleed cessation
Timepoint [12] 0 0
Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit
Secondary outcome [13] 0 0
Number of rAHF-PFM or rAHF-PEG infusions required for bleed cessation
Timepoint [13] 0 0
Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit
Secondary outcome [14] 0 0
Incidence of target joint intervention, including surgery, radiosynovectomy, and chemosynovectomy
Timepoint [14] 0 0
Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; and Termination visit
Secondary outcome [15] 0 0
Incidence of pseudo tumor development
Timepoint [15] 0 0
Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit
Secondary outcome [16] 0 0
Quality of Life: HAL questionnaire - for adult patients
Timepoint [16] 0 0
Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit
Secondary outcome [17] 0 0
Quality of Life: SF-12v2 questionnaire - for adult patients
Timepoint [17] 0 0
Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit
Secondary outcome [18] 0 0
Quality of Life: EQ-5D questionnaire - for adult patients
Timepoint [18] 0 0
Enrollment visit;Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit
Secondary outcome [19] 0 0
Quality of Life: PedHAL questionnaire - for pediatric patients
Timepoint [19] 0 0
Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit
Secondary outcome [20] 0 0
Quality of Life: SF-10 questionnaire - for pediatric patients
Timepoint [20] 0 0
Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit
Secondary outcome [21] 0 0
Quality of Life: EQ-5D (14 and up) questionnaire - for pediatric patients
Timepoint [21] 0 0
Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit
Secondary outcome [22] 0 0
Chronic pain associated with hemophilia, as measured over a period of 4 weeks on an annual basis, using the visual analog scale (VAS)
Timepoint [22] 0 0
Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit
Secondary outcome [23] 0 0
Acute pain associated with hemophilia, as measured with individual bleeding episodes, using the visual analog scale (VAS)
Timepoint [23] 0 0
Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit
Secondary outcome [24] 0 0
Number of days lost from school or work due to bleeding episodes
Timepoint [24] 0 0
Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit
Secondary outcome [25] 0 0
Modalities of switching from a standard FVIII product to rAHF-PEG - 1
Timepoint [25] 0 0
Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit
Secondary outcome [26] 0 0
Modalities of switching from a standard FVIII product to rAHF-PEG - 2
Timepoint [26] 0 0
Enrollment visit; Screening visit; Annual/Interval visits: - Up to 8 years if rAHF-PFM alone - Up to 8 years if rAHF-PEG alone with minimum follow up of 4 years - Up to approx. 12 years rAHF-PFM and switched to rAHF-PEG; Termination visit
Secondary outcome [27] 0 0
Incidence of Inhibitors in Previously Treated Patients (PTPs) with Factor VIII (FVIII) Levels Lesser than (<)1%, Lesser Than or Equal to (<=) 2%, and <= 5% without history of inhibitor
Timepoint [27] 0 0
Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Secondary outcome [28] 0 0
Incidence of Inhibitors in Previously Treated Patients (PTPs) with Factor VIII (FVIII) Levels <1%, <=2%, and <= 5% with history of inhibitor
Timepoint [28] 0 0
Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Secondary outcome [29] 0 0
Incidence of Inhibitors in Previously Untreated Patient (PUPs) and Minimally Treated Patients (MTPs) with Factor VIII (FVIII) Levels <1%, <=2%, and <= 5%
Timepoint [29] 0 0
Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Secondary outcome [30] 0 0
Incidence of therapy-related serious adverse events
Timepoint [30] 0 0
Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Secondary outcome [31] 0 0
Incidence of therapy-related non-serious adverse events
Timepoint [31] 0 0
Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Secondary outcome [32] 0 0
Incidence of inhibitors after switching to rAHF-PEG
Timepoint [32] 0 0
Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)

Eligibility
Key inclusion criteria
- Participant has hemophilia A {FVIII lesser than or equal to (<=)5%}

- Participant is prescribed Antihemophilic Factor (Recombinant) - Plasma/Albumin Free
Method (rAHF-PFM) or Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG) by the
treating physician

- Participant or participant's legally authorized representative provides informed
consent
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Participant has known hypersensitivity to the active substance or any of the
excipients

- Participant has known allergic reaction to mouse or hamster proteins

- Participant has participated in another clinical study involving an investigational
product (IP) or device within 30 days prior to study enrollment or is scheduled to
participate in another clinical study involving another FVIII concentrate or device
during the course of this study

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/06/2011
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
16/01/2024
Sample size
Target
Accrual to date
Final
955
Recruitment in Australia
Recruitment state(s)
NSW,WA
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
South Metropolitan Health Service trading as Fiona Stanley Hospital - Murdoch
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
6050 - Murdoch
Recruitment outside Australia
Country [1] 0 0
Austria
State/province [1] 0 0
Linz
Country [2] 0 0
Austria
State/province [2] 0 0
Wien
Country [3] 0 0
Belgium
State/province [3] 0 0
Bruxelles
Country [4] 0 0
Brazil
State/province [4] 0 0
Ceará
Country [5] 0 0
Brazil
State/province [5] 0 0
Espírito Santo
Country [6] 0 0
Brazil
State/province [6] 0 0
Paraná
Country [7] 0 0
Brazil
State/province [7] 0 0
Parthenon
Country [8] 0 0
Brazil
State/province [8] 0 0
Pará
Country [9] 0 0
Brazil
State/province [9] 0 0
São Paulo
Country [10] 0 0
Brazil
State/province [10] 0 0
Rio de Janeiro
Country [11] 0 0
Canada
State/province [11] 0 0
Alberta
Country [12] 0 0
Canada
State/province [12] 0 0
New Brunswick
Country [13] 0 0
Canada
State/province [13] 0 0
Ontario
Country [14] 0 0
Canada
State/province [14] 0 0
Saskatchewan
Country [15] 0 0
China
State/province [15] 0 0
Futian
Country [16] 0 0
China
State/province [16] 0 0
Guangdong
Country [17] 0 0
China
State/province [17] 0 0
Heping
Country [18] 0 0
China
State/province [18] 0 0
Hubei
Country [19] 0 0
China
State/province [19] 0 0
Jiangsu
Country [20] 0 0
China
State/province [20] 0 0
Shandong
Country [21] 0 0
China
State/province [21] 0 0
Beijing
Country [22] 0 0
China
State/province [22] 0 0
Nanjing
Country [23] 0 0
China
State/province [23] 0 0
Shenzhen
Country [24] 0 0
Colombia
State/province [24] 0 0
Atlantico
Country [25] 0 0
Colombia
State/province [25] 0 0
Bogotá
Country [26] 0 0
Colombia
State/province [26] 0 0
Floridablanca
Country [27] 0 0
Czechia
State/province [27] 0 0
Brno
Country [28] 0 0
Czechia
State/province [28] 0 0
Ostrava
Country [29] 0 0
Czechia
State/province [29] 0 0
Praha 5
Country [30] 0 0
Denmark
State/province [30] 0 0
Copenhagen
Country [31] 0 0
France
State/province [31] 0 0
Brest
Country [32] 0 0
France
State/province [32] 0 0
CAEN Cedex
Country [33] 0 0
France
State/province [33] 0 0
Chambery Cedex
Country [34] 0 0
France
State/province [34] 0 0
Dijon Cedex
Country [35] 0 0
France
State/province [35] 0 0
Le Chesnay Cedex
Country [36] 0 0
France
State/province [36] 0 0
LIMOGES cedex
Country [37] 0 0
France
State/province [37] 0 0
Nantes Cedex
Country [38] 0 0
France
State/province [38] 0 0
Paris
Country [39] 0 0
France
State/province [39] 0 0
Reims Cedex
Country [40] 0 0
France
State/province [40] 0 0
RENNES Cedex 09
Country [41] 0 0
France
State/province [41] 0 0
Rouen
Country [42] 0 0
France
State/province [42] 0 0
Saint Priest en Jarez
Country [43] 0 0
France
State/province [43] 0 0
Toulouse Cedex 9
Country [44] 0 0
Greece
State/province [44] 0 0
Athens
Country [45] 0 0
Greece
State/province [45] 0 0
Thessaloniki
Country [46] 0 0
Hungary
State/province [46] 0 0
Budapest
Country [47] 0 0
Hungary
State/province [47] 0 0
Debrecen
Country [48] 0 0
Hungary
State/province [48] 0 0
Mohács
Country [49] 0 0
Hungary
State/province [49] 0 0
Nyíregyháza
Country [50] 0 0
Hungary
State/province [50] 0 0
Szombathely
Country [51] 0 0
Italy
State/province [51] 0 0
Bari
Country [52] 0 0
Italy
State/province [52] 0 0
Bologna
Country [53] 0 0
Italy
State/province [53] 0 0
Catania
Country [54] 0 0
Italy
State/province [54] 0 0
Catanzaro
Country [55] 0 0
Italy
State/province [55] 0 0
Firenze
Country [56] 0 0
Italy
State/province [56] 0 0
Macerata
Country [57] 0 0
Italy
State/province [57] 0 0
Milano
Country [58] 0 0
Italy
State/province [58] 0 0
Napoli
Country [59] 0 0
Italy
State/province [59] 0 0
Padova
Country [60] 0 0
Italy
State/province [60] 0 0
Palermo
Country [61] 0 0
Italy
State/province [61] 0 0
Reggio Calabria
Country [62] 0 0
Italy
State/province [62] 0 0
Roma
Country [63] 0 0
Italy
State/province [63] 0 0
Scorrano
Country [64] 0 0
Italy
State/province [64] 0 0
Torino
Country [65] 0 0
Norway
State/province [65] 0 0
Oslo
Country [66] 0 0
Poland
State/province [66] 0 0
Bydgoszcz
Country [67] 0 0
Poland
State/province [67] 0 0
Gdansk
Country [68] 0 0
Poland
State/province [68] 0 0
Warszawa
Country [69] 0 0
Poland
State/province [69] 0 0
Wroclaw
Country [70] 0 0
Portugal
State/province [70] 0 0
Açores
Country [71] 0 0
Portugal
State/province [71] 0 0
Coimbra
Country [72] 0 0
Portugal
State/province [72] 0 0
Lisboa
Country [73] 0 0
Portugal
State/province [73] 0 0
Porto
Country [74] 0 0
Russian Federation
State/province [74] 0 0
Chelyabinsk
Country [75] 0 0
Russian Federation
State/province [75] 0 0
Moscow
Country [76] 0 0
Russian Federation
State/province [76] 0 0
Petrozavodsk
Country [77] 0 0
Russian Federation
State/province [77] 0 0
Saint Petersburg
Country [78] 0 0
Russian Federation
State/province [78] 0 0
Samara
Country [79] 0 0
Slovenia
State/province [79] 0 0
Ljubljana
Country [80] 0 0
Spain
State/province [80] 0 0
A Coruña
Country [81] 0 0
Spain
State/province [81] 0 0
Barcelona
Country [82] 0 0
Spain
State/province [82] 0 0
Palma de Mallorca
Country [83] 0 0
Sweden
State/province [83] 0 0
Gothenburg
Country [84] 0 0
Sweden
State/province [84] 0 0
Malmö
Country [85] 0 0
Sweden
State/province [85] 0 0
Stockholm
Country [86] 0 0
Switzerland
State/province [86] 0 0
Basel
Country [87] 0 0
Switzerland
State/province [87] 0 0
Bern
Country [88] 0 0
Switzerland
State/province [88] 0 0
Lausanne
Country [89] 0 0
Switzerland
State/province [89] 0 0
Luzern 17
Country [90] 0 0
Switzerland
State/province [90] 0 0
St. Gallen
Country [91] 0 0
Switzerland
State/province [91] 0 0
Wabern
Country [92] 0 0
Switzerland
State/province [92] 0 0
Zürich
Country [93] 0 0
United Kingdom
State/province [93] 0 0
Hampshire
Country [94] 0 0
United Kingdom
State/province [94] 0 0
Cambridge
Country [95] 0 0
United Kingdom
State/province [95] 0 0
Canterbury
Country [96] 0 0
United Kingdom
State/province [96] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Baxalta now part of Shire
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
Baxalta Innovations GmbH, now part of Shire
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of the study is to document the natural history of hemophilia A disease and
long-term outcomes in terms of effectiveness, safety and quality of life in participants
receiving Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) or
Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG) in routine clinical practice
Trial website
https://clinicaltrials.gov/ct2/show/NCT02078427
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Director
Address 0 0
Takeda
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/ct2/show/NCT02078427