Hemophilia AAHEADNCT02078427
ADVATE/ ADYNOVI Hemophilia A Outcome Database (AHEAD)
An observational study of Hemophilia A, run by Baxalta now part of Shire at 110 sites in 22 countries.
Trial 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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia A
- What is being tested?
- ADVATE and ADYNOVI
- How many people
- 951 participants
- Where
- 110 sitesin 22 countries
- Study dates
- 28 Jun 2011to 16 Jan 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The study population will include male and female participants of any race and age who have a diagnosis of hemophilia A (Factor VIII (FVIII) =5%). Participants must have been prescribed rAHF-PFM or rAHF-PEG for the management of hemophilia A by the treating physician prior to the decision to enroll in the study.
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
110 sites in 22 countries.
110 sites in 22 countries
Italy16 sites
Azienda Ospedaliera Policlinico Consorziale Di Bari
Bari, 70124
Policlinico S Orsola Malpighi
Bologna, 40138
Azienda Ospedaliera Universitaria Vittorio Emanuele, Ferrarotto, S. Bambino
Catania, 95120
Ospedale Pugliese -Ciaccio
Catanzaro, 88100
Az. Osp. Univ. Careggi
Florence, 50134
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122
Azienda Ospedaliera Universitaria Federico II
Naples, 80131
Azienda Ospedaliera di Padova Clinica Medica II
Padova, 35100
AOUP P. Giaccone
Palermo, 90133
Ospedale di Macerata
Province of Macerata, 62100
Azienda Ospedaliera Bianchi Melacrino Morelli
Reggio Calabria, 89123
Università degli studi di Roma "La Sapienza"
Roma, 00161
Ospedale Pediatrico Bambino Gesù
Roma, 00165
Policlinico Universitario Gemelli
Roma, 00168
Centro Emofilia e Coagulopatie Rare
Scorrano, 73025
Ospedale Molinette
Torino, 10126
France13 sites
"Hôpital Morvan CHRU de Brest"
Brest, 29200
CHU Côte de Nacre - CRTH
Caen, 14033
Centre Hospitalier Générale - CTH
Chambéry, 73011
CTRH - CHU Bocage
Dijon, 21079
Hôpital André Mignot
Le Chesnay, 78157
Hôpital de la Mère et de l'Enfant - CHU de LIMOGES
Limoges, 87043
CHRU Hôtel Dieu - CRTH
Nantes, 44093
Hôpital Cochin
Paris, 75014
CHU de Reims Hôpital Maison Blanche - CRTH
Reims, 51092
Centre Régional de Traitement de l'Hémophilie et des Maladies hémorragiques. CHU de Rennes - Hôpital Pontchaillou
Rennes, 35033
CHRU Charles Nicolle
Rouen, 76031
CIC
Saint-Priest-en-Jarez, 42270
CHRU Purpan CRTH - Pavillon Sénac
Toulouse, 31059
Brazil9 sites
Fundação HEMOPA
Belém, Pará, 66033-000
Hemocentro da UNICAMP
Campinas, São Paulo, 13083-970
Hemepar - Pr
Curitiba, Paraná, 80045-145
Hemoce - Ce
Fortaleza, Ceará, 60431086
Hemorgs - Rs
Porto Alegre, Parthenon, 90650-000
Hemocentro Ribeirão Preto - SP
Ribeirão Preto, São Paulo, 14051140
Hemorio - Rj
Rio de Janeiro, 20211030
Faculdade de Medicina da Universidade de São Paulo
São Paulo, 5403000
Hemocentro do Espírito Santo
Vitória, Espírito Santo, 29040-090
China9 sites
Beijing Children's Hospital Affiliated to Capital University of Medical Sciences
Beijing, 100045
Nanfang Hospital Affiliated to Nanfang Medical University
Guangzhou, Guangdong
The Blood Center of Shandong Province
Jinan, Shandong
Nanjing Drum Tower Hospital The Affiliated Hospital of Nanjing University Medical School
Nanjing
Shenzhen Children's Hospital
Shenzhen, Futian
Shenzhen Second People's Hospital
Shenzhen, 518037
Institute of Hematology, Blood Disease Hospital, PUMC&CAMS
Tianjin, Heping
Tongji Hospital, Tongji Medical College of Huazhong University of Science & Technology
Wuhan, Hubei
The Affiliated Hospital of Xuzhou Medical University
Xuzhou, Jiangsu, 221006
Switzerland8 sites
Universitätsspital Basel
Basel, CH-4031
Inselspital Bern
Bern, 3010
Centre Hospitalier Universitaire Vaudois
Lausanne, 1011
Luzerner Kantonsspital
Lucerne, CH-6000
Kantonsspital St. Gallen
Sankt Gallen, 9007
FMH Kinder und Jugendmedizin, im Wabern-Zentrum
Wabern, CH-3084
Kinderspital Zürich
Zurich, CH-8032
Universitätsspital Zürich
Zurich, CH-8091
Canada6 sites
Stollery Children's Hospital, University of Alberta
Edmonton, Alberta, T6G 2B7
The Moncton City Hospital
Moncton, New Brunswick, E1C 6Z8
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
Royal University Hospital
Saskatoon, Saskatchewan, S7N 0W8
St-Michael's Hospital
Toronto, Ontario, M5B 1W8
Sick Kids Hospital
Toronto, Ontario, M5G 1X8
Hungary5 sites
Heim Pál Gyermekkórház
Budapest, H-1089
Debreceni Egyetem Klinikai Kozpont
Debrecen, H-4032
Mohács City Hospital
Mohács, H-7700
Jósa András County Hospital
Nyíregyháza, H-4400
Markusovszky Hospital
Szombathely, H-9700
Russia5 sites
SBHI Chelyabinsk Regional Children's Clinical Hospital
Chelyabinsk
Federal State Budget Institution "Hematology Research Center" of Ministry of Healthcare of Russian Federation
Moscow, 125167
SBHI of the Republic of Kareliya Republican Hospital n.a. V.A. Baranov
Petrozavodsk
State Budget Healthcare Institution of Saint-Petersburg "City polyclinic #37"
Saint Petersburg, 191186
Clinics of FSBEI High Education Samara SMU of MoH of Russia
Samara, 443079
Czechia4 sites
Fakultní nemocnice Brno
Brno, 613 00
Fakultní nemocnice Ostrava, Oddělení dětské hematologie a hematoonkologie
Ostrava, 70852
Fakultní nemocnice Ostrava
Ostrava, 70852
Fakultní nemocnice v Motole
Prague, 150 06
Poland4 sites
Szpital Uniwersytecki nr 1 im. dr Andrzeja Jurasza
Bydgoszcz, 85-094
Samodzielny Publiczny Szpital Kliniczny nr 1
Gdansk, 80-952
Samodzielny Publiczny Dzieciecy Szpital Kliniczny
Warsaw, 02-091
Uniwersytecki Szpital Kliniczny we Wrocławiu
Wroclaw, 50-556
Portugal4 sites
Centro Hospitalar e Universitade de Coimbra
Coimbra, 3000-602
Centro Hospitalar Lisboa Norte Hospital de Sta. Maria
Lisbon, 1649-035
Hospital do Divino Espírito Santo
Ponta Delgada, Azores, 9500-370
Centro Hospitalar de São João, E.P.E.
Porto, 4200-319
Spain4 sites
Hospital Teresa Herrera-Materno Infantil
A Coruña, 15006
Hospital Hospital Sant Joan de Déu
Barcelona, 08035
Hospital Universitari Vall d'Hebron (HUVH)
Barcelona, 08035
Hospital Universitario Son Espases
Palma de Mallorca, 07120
United Kingdom4 sites
Hampshire Hospitals NHS Foundation Trust, Basingstoke and North Hampshire Hospital
Basingstoke, Hampshire, RG24 9NA
Cambridge University Hospital NHS Foundation Trust, Addenbrooke's Hospital
Cambridge, CB2 0QQ
East Kent Hospitals University Foundation Trust, Kent & Canterbury Hospital
Canterbury, CT1 3NG
Great Ormond Street Hospital for Children NHS Trust
London, WC1N 3JH
Austria3 sites
Landes Frauen und Kinderklinik Linz (LFKK Linz)
Linz, 4017
Kepler Universitätsklinikum Klinik für Kinder-und Jugendheilkunde
Linz, 4020
Medizinische Universitaet Wien
Vienna, 1090
Colombia3 sites
IPS FUSA SAS Centro Integral de Coagulacion
Atlántico
Integral Solutions SD SAS
Bogotá
Fundación Oftalmológica de Santander FOSCAL
Floridablanca, 681004
Greece3 sites
Aghia Sofia Children's Hospital
Goudi, Athens, 11527
Laikon General Hospital
Goudi, Athens, 11527
General Hospital of Thessaloniki "Ippokratio"
Thessaloniki, 546 42
Sweden3 sites
Sahlgrenska University Hospital
Gothenburg, SE 41345
Kliniska studier i Sverige - Forum Söder
Malmö, SE-205 02
Karolinska Universitetssjukhuset Solna
Stockholm, 171 76
Australia2 sites
Royal Prince Alfred Hospital
Camperdown, New South Wales, 2050
South Metropolitan Health Service trading as Fiona Stanley Hospital
Murdoch, Western Australia, 6050
Norway2 sites
Rikshospitalet
Oslo, N-0027
Rikshospitalet, Oslo University Hospital
Oslo, N-0424
About this study
Official title: ADVATE/ ADYNOVI Hemophilia A Outcome Database
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
- Conditions
- Hemophilia A
- Being tested
- ADVATE, ADYNOVI
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ADVATEBiological
Also called octocog alfa, rAHF-PFM
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
ADYNOVIBiological
Also called rurioctocog alfa pegol, rAHF-PEG
Antihemophilic Factor (Recombinant) Pegylated
| Study group | Role | What they receive |
|---|---|---|
| rAHF-PFM | Participants treated with rAHF-PFM alone Receives: Biological: ADVATE | |
| rAHF-PEG | Participants treated with rAHF-PEG alone Receives: Biological: ADYNOVI | |
| rAHF-PFM then rAHF-PEG | Participants treated with rAHF-PFM and subsequently switched to rAHF-PEG Receives: Biological: ADVATE; Biological: ADYNOVI |
What is being measured
The primary outcome is the main question the study is designed to answer.
Joint Health Outcomes - Assessed by Physical Exam Using Only the Pain, Bleeding, and Physical Exam Parameters of the Gilbert Scale
The World Federation of Hemophilia developed a musculoskeletal evaluation system, commonly referred to as the Gilbert test, to measure hemophilia joint health status.The Gilbert test needs to be performed in the absence of acute bleed, acute pain, and acute inflammation into the evaluated joint. Four parameters are used in each Gilbert test: pain (score: 0-3), bleeding (score: 0-3), physical exam (score: 0-12), and X-ray evaluation (score: 0-13) Scores of 0, represent no pain, no bleeding, no physical exam issues, and/or no x-ray issues. Higher scores for each of these categories represents worsening conditions.
Measured: Up to approximately 12 years
Secondary outcomes (32)
Annualized Bleed Rate, All Joints
The annualized bleed rate for all joints will be calculated per participant and summarized over the set of available participants with a minimum observation period of 90 days per treatment regimen.
Measured: 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
Annualized Bleed Rate, All Bleeds
The annualized bleed rate for all bleeds will be calculated per participant and summarized over the set of available participants.with a minimum observation period of 90 days per treatment regimen.
Measured: 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
Annualized bleed rate, pre-existing target joints at baseline
The annualized bleed rate for pre-existing target joints at baseline will be calculated per participant and summarized over the set of available participants with a minimum observation period of 90 days per treatment regimen.
Measured: 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
Incidence of New Target Joints
The incidence of new target joints will be calculated as the total number of new target joints in all participants divided by the total number of observation days.
Measured: 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
Status of joint health by X-ray by Pettersson scale
The status of joint health by X-ray by Pettersson score will be summarized for each observational year.
Measured: 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.
Status of Joint Health by Magnetic Resonance Imaging (MRI) Scoring System- Using The Lund Scoring System (LSS)
LSS score format= A(e:s:h). Sum of values for Subchondral Cyst (score: 1-6), irregularity/erosion of Subchondral Cortex (score: 1-4), and Chondral Destruction (score: 1-6) gives value for the A component of score. e, s, h components represent effusion/hemarthrosis, hypertrophic synovial, \& hemosiderin deposition (score: 0-4 for each). Max. score is 16(4:4:4).
Subchondral Cyst:
- ≥1 bone
- ≥2 bones
- >3 cysts in ≥1 bone
- >3 cysts ≥2 bones
- Largest size >4 mm: ≥1 bone
- Largest size >4 mm: ≥2 bones
Subchondral Cortex
- ≥1 bone
- ≥2 bones
- Involve > half joint surface: ≥1 bone
- Involve > half of joint surface: ≥2 bones
Chondral Destruction
- ≥1 bone
- ≥2 bones
- Full thickness defect (FTD): ≥1 bone
- FTD: ≥2 bones
- FTD involves >1/3 of joint surface: ≥1 bone
- FTD involves >1/3 of joint surface: ≥2 bones
Effusion/hemarthrosis (e): Hypertrophic synovial (s): Hemosiderin (h): (0-4 for each):
- 0 absent
- 1 equivocal
- 2 small
- 3 moderate
- 4 large
Measured: 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
Status of joint health using the Hemophilia Joint Health Score (HJHS)
The International Prophylaxis Study Group (IPSG) developed a scoring system for musculoskeletal evaluation, the HJHS, optimized for use in children with no or minimal joint disease.
The HJHS includes the following parameters: swelling, duration of swelling, muscle atrophy, joint pain, crepitus on motion, flexion loss, extension loss, strength and global gait.
Measured: 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
Overall effectiveness assessment for prophylaxis therapy
- Excellent: Same or lower breakthrough bleed rate (BBR) within last 12 months (M) compared with prior prophylaxis; if participant did not receive prior prophylaxis with rAHF-PFM, rAHF-PEG or other Factor VIII (FVIII), same or better than expected outcome according to investigator's expectation
- Good: Minor increase in BBR within last 12M compared with prior prophylaxis; if participant did not receive prophylaxis with rAHF-PFM, rAHF-PEG or other FVIII, slightly less than expected outcome according to investigator's expectation
- Fair: Moderate increase in BBR in last 12M compared with prior prophylaxis; if participant did not receive prophylaxis with rAHF-PFM, rAHF-PEG or other FVIII, somewhat less than expected outcome according to investigator's expectation
- Poor: Significant increase in BBR in the 12M compared with prior prophylaxis; if participant did not receive prophylaxis with rAHF-PFM, rAHF-PEG or other FVIII, little to no benefit according to investigator's expectation
Measured: 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
Compliance with the dosing prescribed and its relationship with effectiveness
Evaluation of patients´ compliance to prescribed prophylactic treatment will be performed by the treating physician. Compliance will be categorized according to a 4-point table (Highly compliant, Fairly compliant, Moderately compliant, Poorly compliant)
Measured: 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
Overall effectiveness assessment for on-demand treatment
- Excellent: Bleed episodes typically respond to same or fewer number of infusion and same or lower dose as compared with previous on-demand treatment or investigator's expectation
- Good: Most bleed episodes typically respond to same number of infusion and dose but some require more infusions or higher dose as compared with previous on-demand treatment or investigator's expectation
- Fair: Bleed episodes typically require more infusions and/or higher dose than expected as compared with previous on-demand treatment or investigator's expectation
- Poor: Bleed episodes routinely fail to respond to same number of infusion and dose and require additional or different factor concentrate for hemostatic control as compared with previous on-demand treatment or investigator's expectation
Measured: 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
Global effectiveness assessment for on-demand treatment
- Excellent: Full relief of pain and cessation of bleeding as evidenced by objective signs (e.g., swelling, tenderness, irritability, inconsolability, and decreased range of motion in the case of musculoskeletal hemorrhage) within approximately 8 hours of a single infusion. No additional infusion is required for the control of bleeding. Administration of further infusions to maintain hemostasis would not affect this scoring.
- Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hours after the infusion. Possibly requires more than 1 infusion for complete resolution.
- Fair: Probable or slight relief of pain and slight improvement in signs of bleeding within approximately 8 hours after the infusion. Requires more than 1 infusion for complete resolution.
- Poor: No improvement or condition worsens.
Measured: 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
Number of rAHF-PFM or rAHF-PEG units required for bleed cessation
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG)
Measured: 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
Number of rAHF-PFM or rAHF-PEG infusions required for bleed cessation
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM) Antihemophilic Factor (Recombinant) - Pegylated (rAHF-PEG)
Measured: 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
Incidence of target joint intervention, including surgery, radiosynovectomy, and chemosynovectomy
Measured: 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
Incidence of pseudo tumor development
Measured: 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
Quality of Life: HAL questionnaire - for adult patients
The the lHAL measures activities involving the upper extremities, basic activities involving ower extremities and complex activities involving the lower extremities as well as an overall physical activity score for adults.
Measured: 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
Quality of Life: SF-12v2 questionnaire - for adult patients
The SF-12v2 measures generic health-related quality of life for adults.
Measured: 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
Quality of Life: EQ-5D questionnaire - for adult patients
The EQ-5D measures health utility in adult participants.
Measured: 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
Quality of Life: PedHAL questionnaire - for pediatric patients
The PedHAL measures activities involving the upper extremities, basic activities involving the lower extremities and complex activities involving the lower extremities as well as an overall physical activity score for children.
For participants 4-13 years of age:
- PedHAL (parent version)
For participants 14-17 years of age:
- PedHAL (child version)
Measured: 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
Quality of Life: SF-10 questionnaire - for pediatric patients
The SF-10 measures generic health-related quality of life for children and is parent-completed.
Measured: 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
Quality of Life: EQ-5D (14 and up) questionnaire - for pediatric patients
The EQ-5D measures health utility in subjects aged 14 and up.
Measured: 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
Chronic pain associated with hemophilia, as measured over a period of 4 weeks on an annual basis, using the visual analog scale (VAS)
The VAS assesses the pain using a scale of 0 (no pain) to 10 (unbearable pain).
During screening visit and on an annual basis, the investigators shall ask participants to rate the average level of chronic pain associated with hemophilia over the period of 4 weeks prior to visit date using the VAS.
Measured: 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
Acute pain associated with hemophilia, as measured with individual bleeding episodes, using the visual analog scale (VAS)
The VAS assesses the pain using a scale of 0 (no pain) to 10 (unbearable pain).
Participants will be asked to provide ratings on level of acute pain associated with each bleeding episode using the VAS. The VAS scores will be recorded in the participant diary.
Measured: 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
Number of days lost from school or work due to bleeding episodes
Measured: 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
Modalities of switching from a standard FVIII product to rAHF-PEG - 1
Difference in number of weekly prophylactic infusions between previous regimen and rAHF-PEG
Measured: 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
Modalities of switching from a standard FVIII product to rAHF-PEG - 2
Difference in number of weekly doses between previous regimen and rAHFPEG
Measured: 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
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
Measured: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Incidence of Inhibitors in Previously Treated Patients (PTPs) with Factor VIII (FVIII) Levels <1%, <=2%, and <= 5% with history of inhibitor
Measured: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Incidence of Inhibitors in Previously Untreated Patient (PUPs) and Minimally Treated Patients (MTPs) with Factor VIII (FVIII) Levels <1%, <=2%, and <= 5%
Measured: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Incidence of therapy-related serious adverse events
Measured: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Incidence of therapy-related non-serious adverse events
Measured: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Incidence of inhibitors after switching to rAHF-PEG
Incidence of inhibitors after switching to rAHF-PEG in the same subgroups of patients
Measured: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2014 (estimated)
- Study start28 Jun 2011
- Primary completion16 Jan 2024
- Study completion16 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 951 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Baxalta Innovations GmbH, now part of Shire
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02078427
- Sponsor's protocol ID
- 061001
- Acronym
- AHEAD
- Status last verified
- Jun 2024
- First submitted
- 3 Mar 2014
- Last updated
- 17 Jun 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Publications
Ozelo MC, Hermans C, Carcao M, Guillet B, Gu J, Guerra R, Tang L, Khair K. The effectiveness and safety of octocog alfa in patients with hemophilia A: up to 7-year follow-up of the real-world AHEAD international study. Ther Adv Hematol. 2024 Feb 15;15:20406207231218624. doi: 10.1177/20406207231218624. eCollection 2024.
PubMed 38371314
Study at a glance
From the registry record, last updated by the sponsor on 17 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ADVATE/ ADYNOVI Hemophilia A Outcome Database
- ClinicalTrials.gov ID
- NCT02078427
- Acronym / sponsor ID
- AHEAD / 061001
- Conditions
- Hemophilia A
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 951 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Collaborators
- Baxalta Innovations GmbH, now part of Shire
- Study start
- 28 Jun 2011 (actual)
- Primary completion
- 16 Jan 2024 (actual)
- Study completion
- 16 Jan 2024 (actual)
- First posted
- 5 Mar 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| rAHF-PFM | – | Biological: ADVATEParticipants treated with rAHF-PFM alone |
| rAHF-PEG | – | Biological: ADYNOVIParticipants treated with rAHF-PEG alone |
| rAHF-PFM then rAHF-PEG | – | Biological: ADVATE, Biological: ADYNOVIParticipants treated with rAHF-PFM and subsequently switched to rAHF-PEG |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study population will include male and female participants of any race and age who have a diagnosis of hemophilia A (Factor VIII (FVIII) =5%). Participants must have been prescribed rAHF-PFM or rAHF-PEG for the management of hemophilia A by the treating physician prior to the decision to enroll in the study.
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ADVATEAlso: octocog alfa, rAHF-PFMAntihemophilic Factor (Recombinant) - Plasma/Albumin Free Method | Biological | rAHF-PFM, rAHF-PFM then rAHF-PEG |
| ADYNOVIAlso: rurioctocog alfa pegol, rAHF-PEGAntihemophilic Factor (Recombinant) Pegylated | Biological | rAHF-PEG, rAHF-PFM then rAHF-PEG |
Primary outcome
Joint Health Outcomes - Assessed by Physical Exam Using Only the Pain, Bleeding, and Physical Exam Parameters of the Gilbert Scale
Time frame: Up to approximately 12 years
The World Federation of Hemophilia developed a musculoskeletal evaluation system, commonly referred to as the Gilbert test, to measure hemophilia joint health status.The Gilbert test needs to be performed in the absence of acute bleed, acute pain, and acute inflammation into the evaluated joint. Four parameters are used in each Gilbert test: pain (score: 0-3), bleeding (score: 0-3), physical exam (score: 0-12), and X-ray evaluation (score: 0-13) Scores of 0, represent no pain, no bleeding, no physical exam issues, and/or no x-ray issues. Higher scores for each of these categories represents worsening conditions.
32 secondary outcomes
Annualized Bleed Rate, All Joints
Time frame: 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
Annualized Bleed Rate, All Bleeds
Time frame: 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
Annualized bleed rate, pre-existing target joints at baseline
Time frame: 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
Incidence of New Target Joints
Time frame: 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
Status of joint health by X-ray by Pettersson scale
Time frame: 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.
Status of Joint Health by Magnetic Resonance Imaging (MRI) Scoring System- Using The Lund Scoring System (LSS)
Time frame: 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
Status of joint health using the Hemophilia Joint Health Score (HJHS)
Time frame: 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
Overall effectiveness assessment for prophylaxis therapy
Time frame: 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
Compliance with the dosing prescribed and its relationship with effectiveness
Time frame: 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
Overall effectiveness assessment for on-demand treatment
Time frame: 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
Global effectiveness assessment for on-demand treatment
Time frame: 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
Number of rAHF-PFM or rAHF-PEG units required for bleed cessation
Time frame: 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
Number of rAHF-PFM or rAHF-PEG infusions required for bleed cessation
Time frame: 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
Incidence of target joint intervention, including surgery, radiosynovectomy, and chemosynovectomy
Time frame: 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
Incidence of pseudo tumor development
Time frame: 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
Quality of Life: HAL questionnaire - for adult patients
Time frame: 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
Quality of Life: SF-12v2 questionnaire - for adult patients
Time frame: 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
Quality of Life: EQ-5D questionnaire - for adult patients
Time frame: 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
Quality of Life: PedHAL questionnaire - for pediatric patients
Time frame: 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
Quality of Life: SF-10 questionnaire - for pediatric patients
Time frame: 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
Quality of Life: EQ-5D (14 and up) questionnaire - for pediatric patients
Time frame: 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
Chronic pain associated with hemophilia, as measured over a period of 4 weeks on an annual basis, using the visual analog scale (VAS)
Time frame: 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
Acute pain associated with hemophilia, as measured with individual bleeding episodes, using the visual analog scale (VAS)
Time frame: 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
Number of days lost from school or work due to bleeding episodes
Time frame: 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
Modalities of switching from a standard FVIII product to rAHF-PEG - 1
Time frame: 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
Modalities of switching from a standard FVIII product to rAHF-PEG - 2
Time frame: 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
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
Time frame: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Incidence of Inhibitors in Previously Treated Patients (PTPs) with Factor VIII (FVIII) Levels <1%, <=2%, and <= 5% with history of inhibitor
Time frame: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Incidence of Inhibitors in Previously Untreated Patient (PUPs) and Minimally Treated Patients (MTPs) with Factor VIII (FVIII) Levels <1%, <=2%, and <= 5%
Time frame: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Incidence of therapy-related serious adverse events
Time frame: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Incidence of therapy-related non-serious adverse events
Time frame: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Incidence of inhibitors after switching to rAHF-PEG
Time frame: Throughout the study period of up to approximately: 8 years (rAHF-PFM alone), or 12 years (rAHF-PEG alone or after receiving rAHF-PFM)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jun 2024.
- Derived from this study: PubMed 38371314Ozelo MC, Hermans C, Carcao M, Guillet B, Gu J, Guerra R, Tang L, Khair K. The effectiveness and safety of octocog alfa in patients with hemophilia A: up to 7-year follow-up of the real-world AHEAD international study. Ther Adv Hematol. 2024 Feb 15;15:20406207231218624. doi: 10.1177/20406207231218624. eCollection 2024.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
110 sites in 22 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Baxalta now part of Shire at 110 sites in 22 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 110 sites in Italy, France, Brazil, China, Switzerland and 17 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Joint Health Outcomes - Assessed by Physical Exam Using Only the Pain, Bleeding, and Physical Exam Parameters of the Gilbert Scale (time frame: Up to approximately 12 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02078427, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jun 2024.