Health Services AccessibilityPHAREONCT04775888
Accessibility of Prophylaxis and On-demand Treatment for Persons With Haemophilia and Other Coagulation Deficiencies
An observational study of Health Services Accessibility and Blood Coagulation Factor Deficiencies, run by RESCUe - RESeau Cardiologie Urgence / RESUVal - RESeau des Urgences de la vallée du Rhône at 34 sites in France.
Trial summary
The current treatment of people with haemophilia and other bleeding deficiencies is largely based on clotting factor replacement therapy. The injections can be repeated several times a week according to a personalized schedule. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Health Services Accessibility, Blood Coagulation Factor Deficiencies
- How many people
- 500 participants
- Where
- 34 sitesFrance
- Study dates
- 21 Sep 2020to 21 May 2021
- 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 included subjects comprise people with haemophilia or other coagulation deficiencies, with no limitation of age, treated by prophylaxis or on-demand treatment, in one of the authorized hospital pharmacies in the Auvergne-Rhone-Alpes region (France).
Included:
- Living person on the study start date and presenting with haemophilia or other coagulation deficiencies;
- Treated with prophylaxis or on-demand anti-haemorrhagic treatments;
- Location of medication dispensing within the hospital pharmacies of the HEsora network (Hémophilie Soins Rhône Alpes): 27 eligible hospital pharmacies in Rhône Alpes + <10 eligible hospital pharmacies in Auvergne
Excluded:
- Home delivery (not included in the study via hospital pharmacy)
Trial locations and contacts
34 sites in 1 country.
34 sites in 1 country
France34 sites, 34 recruiting
- Recruiting
Centre Hospitalier Albertville-Moûtiers
Albertville, 73200
Thomas Gailland
- Recruiting
Centre Hospitalier Annecy Genevois
Annecy, 74370
Christelle Roch
- Recruiting
Centre Hospitalier d'Ardèche Nord
Annonay, 07103
Sophie Vernardet
- Recruiting
Centre Hospitalier d'Ardèche Méridionale
Aubenas, 07200
Alain Lenoir
- Recruiting
Centre Hospitalier Henri Mondor d'Aurillac
Aurillac, 15000
Valérie Millot
- Recruiting
Centre Hospitalier de Belley
Belley, 01300
Marie Geneste
- Recruiting
Centre Hospitalier Fleyriat
Bourg-en-Bresse, 01012
Stéphanie Claveranne
- Recruiting
Centre hospitalier de Bourg St Maurice
Bourg-Saint-Maurice, 73700
Valérie Chaminant
- Recruiting
Centre Hospitalier Pierre Oudot
Bourgoin, 38300
Clément Beyron
- Recruiting
Groupement Hospitalier Est, Hospices Civils de Lyon
Bron, 69500
Valérie Chamouard
- Recruiting
Centre Hospitalier de Chambéry - Hôpital Savoie
Chambéry, 73000
Elodie Bernard
- Recruiting
Centre Hospitalier Universitaire de Clermont-Ferrand
Clermont-Ferrand, 63100
Anne Boyer
- Recruiting
Centre Hospitalier Alpes Léman
Contamine-sur-Arve, 74130
Catherine Diakhate
- Recruiting
Hôpital Nord Ouest de Villefranche-sur-Saône
Gleizé, 69400
Magali Bourdelin
- Recruiting
Centre Hospitalier Universitaire Grenoble - Alpes
La Tronche, 38700
Sophie Cerena
- Recruiting
Hôpital Edouard herriot, Hospices Civils de Lyon
Lyon, 69003
Elodie Lanoi
- Recruiting
Hôpital de la Croix-Rousse, Hospices Civils de Lyon
Lyon, 69004
Anne-Claire Uhrès
- Recruiting
Centre Hospitalier du Forez
Montbrison, 42600
Hadyl Asfari
- Recruiting
Centre Hospitalier de Montélimar
Montélimar, 26200
Jérémy Rolain
- Recruiting
Hôpital de Montluçon
Montluçon, 03100
Carole Rimpici
- Recruiting
Centre Hospitalier de Moulins-Yzeure
Moulins, 03006
Emmanuel Deligeard
- Recruiting
Centre Hospitalier Du Haut Bugey
Oyonnax, 01100
Nathalie Dey
- Recruiting
Centre Hospitalier des Vals d'Ardeche
Privas, 07007
Dominique Quinard
- Recruiting
Centre Hospitalier de Roanne
Roanne, 42300
Maud Rossignol
- Recruiting
Hôpitaux Drôme Nord
Romans-sur-Isère, 26100
Xavier Cussoneau
- Recruiting
Pharmacie Centrale, Hospices Civils de Lyon
Saint-Genis-Laval, 69230
Isabelle Carpentier
- Recruiting
Centre Hospitalier Intercommunal Sud-Léman Valserine
Saint-Julien-en-Genevois, 74164
Alexandra Combes
- Recruiting
Centre Hospitalier Uuniversitaire Nord, Saint-Étienne
Saint-Priest-en-Jarez, 42270
Jihen Boussetta
- Recruiting
Hôpitaux du Pays du Mont-Blanc
Sallanches, 74700
Marion Filippi
- Recruiting
Hôpital Nord-Ouest Tarare-Grandris
Tarare, 69170
Christine Vray
- Recruiting
Hopitaux du Léman
Thonon-les-Bains, 74203
Denis Hardelin
- Recruiting
Centre Hospitalier de Valence
Valence, 26000
Stéphanie Bourget
- Recruiting
Centre Hospitalier de Vichy
Vichy, 03200
Jérôme Tavernier
- Recruiting
Centre Hospitalier Lucien Hussel
Vienne, 38200
Anne-Sophie Leromain
About this study
Official title: Prophylaxis and On-demand Treatment for Persons With Haemophilia and Other Coagulation Deficiencies: a Comparison of Perceived and Observed Accessibility in the Auvergne-Rhône-Alpes Region (France)
The current treatment of people with haemophilia and other bleeding deficiencies is largely based on clotting factor replacement therapy. The injections can be repeated several times a week according to a personalized schedule. To date, medications are exclusively dispensed in hospital pharmacies to ensure traceability and safety. This retrocession imposes accessibility constraints on patients and on their caregivers, increasing the burden of the disease, particularly in the organization of personal and professional daily life. The PHAREO study aims to investigate patients' perception of accessibility to anti-haemophilia drugs in relation to an evaluation of spatial accessibility in the Auvergne-Rhône-Alpes region (France) in order to consider, if necessary, ways of improving the pathway for patients and their caregivers.
Read the detailed description
Haemophilia is a rare constitutional hemorrhagic disease whose drug management is based on the use of chronic lifelong replacement therapy. For many years, the reference treatment, particularly in children, has been based on the use of anti-haemophilic drugs for prophylaxis requiring repeated injections several times a week according to a personalized schedule. In contrast, on-demand treatment is less and less used, particularly in patients with severe forms of haemophilia. Clinical and biological diagnosis, as well as the implementation and monitoring of treatments, are carried out within specialized hospital care structures affiliated with the French national reference center. Medications are dispensed as part of hospital retrocessions. This organization imposes constraints on patients and their caregivers due to their limited accessibility.
The burden related to this disease is probably due to the systematic use of specialized hospital teams such as doctors, nurses for self-injection training for example, and pharmacists. It now seems important to reflect on the evolution of patient pathways that were previously exclusively hospital-centred towards ambulatory care. To do this, various reflections need to be undertaken, including that relating to the accessibility of medication. Indeed, the monthly renewals of these chronic treatments force patients and their caregivers, as well as the parents of children, to go to the hospital, which frequently makes the organization of daily and professional life more cumbersome.
The PHAREO study aims to investigate patients' perception of accessibility to anti-haemophilia drugs in relation to an evaluation of spatial accessibility in order to consider, if necessary, ways of improving the pathway for patients and their caregivers.
The expected benefits of the study are to have: 1) an exhaustive description of the spatial accessibility of the cohort of people living with haemophilia to anti-haemophilia medication within the Auvergne Rhône Alpes region (France) and 2) a better understanding of their needs and their perceptions regarding access to on-demand and prophylactic treatments.
- Keywords
- haemophilia, accessibility, on-demand treatment, prophylactic treatment
- Medical subject headings
- Coagulation Protein Disorders, Hemophilia A
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the perception of persons with haemophilia and other coagulation deficiencies regarding the accessibility to prophylactic and on-demand antihemorrhagic treatment
The perception is defined by the satisfaction level of the survey participants, measured with on item in the questionnaire (Very satisfied / Rather satisfied / Rather not satisfied / Not satisfied).
Measured: Through study completion, on average 6 months.
To measure the accessibility of persons with haemophilia and other coagulation deficiencies regarding the accessibility to prophylactic and on-demand antihemorrhagic treatment
The accessibility is measured by spatial analysis, representing the distance between the home and/or work zip codes and the hospital pharmacy zip code.
Measured: Through study completion, on average 6 months.
To compare the perception and the spatial accessibility of persons with haemophilia and other coagulation deficiencies regarding prophylactic and on-demand antihemorrhagic treatment
The mean distances between the 4-groups of satisfaction level are compared using an analysis of variance (ANOVA).
Measured: Through study completion, on average 6 months.
Secondary outcomes (5)
To determine if the severity of the disease would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
The satisfaction level of participants are stratified on the severity of the disease (minor, moderate, major).
Measured: Through study completion, on average 6 months.
To determine if the age of the patient would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
The satisfaction level of participants are stratified on the age of patient.
Measured: Through study completion, on average 6 months.
To determine if the condition of access to hospital would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
The satisfaction level of participants are stratified on the condition of access to hospital (relative to traffic conditions, the physical condition of the patient, the parking difficulties).
Measured: Through study completion, on average 6 months.
To determine if the level of autonomy of the participant would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
The satisfaction level of participants are stratified on the level of autonomy of the participant (relative to wheelchair, crutch/walking stick or other mobility aid).
Measured: Through study completion, on average 6 months.
To determine if the availability of the hospital pharmacy would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
The satisfaction level of participants are stratified on the availability of the hospital pharmacy (relative to the waiting times).
Measured: Through study completion, on average 6 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Mar 2021
- Study start21 Sep 2020
- Primary completion30 Apr 2021
- Study completion21 May 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Health Services AccessibilityStudies 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
- 500 participants (estimated)
Sponsor and investigators
- Lead sponsor
- RESCUe - RESeau Cardiologie Urgence / RESUVal - RESeau des Urgences de la vallée du Rhône
- Sponsor type
- University, hospital or other
- Collaborators
- Hospices Civils de Lyon
- Responsible party
- Sponsor
- Investigators
- Laurie Fraticelli, PhD, Study director, RESCUe-RESUValJulie Freyssenge, PhD, Study director, RESCUe-RESUVal
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04775888
- Sponsor's protocol ID
- 2020PHAREO
- Acronym
- PHAREO
- Status last verified
- Feb 2021
- First submitted
- 11 Feb 2021
- Last updated
- 14 May 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Patient information is held under legal and ethical obligations of confidentiality. As the disease is rare, information should not be used or disclosed in a form that might identify a patient without his or her consent.
Publications
Leroy V, Freyssenge J, Renard F, Tazarourte K, Negrier C, Chamouard V. Access to treatment among persons with hemophilia: A spatial analysis assessment in the Rhone-Alpes region, France. J Am Pharm Assoc (2003). 2019 Nov-Dec;59(6):797-803. doi: 10.1016/j.japh.2019.07.006. Epub 2019 Aug 9.
PubMed 31405805Susen S, Gruel Y, Godier A, Harroche A, Chambost H, Lasne D, Rauch A, Roullet S, Fontana P, Goudemand J, de Maistre E, Chamouard V, Wibaut B, Albaladejo P, Negrier C. Management of bleeding and invasive procedures in haemophilia A patients with inhibitor treated with emicizumab (Hemlibra(R) ): Proposals from the French network on inherited bleeding disorders (MHEMO), the French Reference Centre on Haemophilia, in collaboration with the French Working Group on Perioperative Haemostasis (GIHP). Haemophilia. 2019 Sep;25(5):731-737. doi: 10.1111/hae.13817. Epub 2019 Jul 11.
PubMed 31294904
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prophylaxis and On-demand Treatment for Persons With Haemophilia and Other Coagulation Deficiencies: a Comparison of Perceived and Observed Accessibility in the Auvergne-Rhône-Alpes Region (France)
- ClinicalTrials.gov ID
- NCT04775888
- Acronym / sponsor ID
- PHAREO / 2020PHAREO
- Keywords
- haemophilia, accessibility, on-demand treatment, prophylactic treatment
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- RESCUe - RESeau Cardiologie Urgence / RESUVal - RESeau des Urgences de la vallée du RhôneUniversity, hospital or other
- Collaborators
- Hospices Civils de Lyon
- Study start
- 21 Sep 2020 (actual)
- Primary completion
- 30 Apr 2021 (actual)
- Study completion
- 21 May 2021 (estimated)
- First posted
- 1 Mar 2021 (actual)
- 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 current treatment of people with haemophilia and other bleeding deficiencies is largely based on clotting factor replacement therapy. The injections can be repeated several times a week according to a personalized schedule. To date, medications are exclusively dispensed in hospital pharmacies to ensure traceability and safety. This retrocession imposes accessibility constraints on patients and on their caregivers, increasing the burden of the disease, particularly in the organization of personal and professional daily life. The PHAREO study aims to investigate patients' perception of accessibility to anti-haemophilia drugs in relation to an evaluation of spatial accessibility in the Auvergne-Rhône-Alpes region (France) in order to consider, if necessary, ways of improving the pathway for patients and their caregivers.
Detailed description
Haemophilia is a rare constitutional hemorrhagic disease whose drug management is based on the use of chronic lifelong replacement therapy. For many years, the reference treatment, particularly in children, has been based on the use of anti-haemophilic drugs for prophylaxis requiring repeated injections several times a week according to a personalized schedule. In contrast, on-demand treatment is less and less used, particularly in patients with severe forms of haemophilia. Clinical and biological diagnosis, as well as the implementation and monitoring of treatments, are carried out within specialized hospital care structures affiliated with the French national reference center. Medications are dispensed as part of hospital retrocessions. This organization imposes constraints on patients and their caregivers due to their limited accessibility.
The burden related to this disease is probably due to the systematic use of specialized hospital teams such as doctors, nurses for self-injection training for example, and pharmacists. It now seems important to reflect on the evolution of patient pathways that were previously exclusively hospital-centred towards ambulatory care. To do this, various reflections need to be undertaken, including that relating to the accessibility of medication. Indeed, the monthly renewals of these chronic treatments force patients and their caregivers, as well as the parents of children, to go to the hospital, which frequently makes the organization of daily and professional life more cumbersome.
The PHAREO study aims to investigate patients' perception of accessibility to anti-haemophilia drugs in relation to an evaluation of spatial accessibility in order to consider, if necessary, ways of improving the pathway for patients and their caregivers.
The expected benefits of the study are to have: 1) an exhaustive description of the spatial accessibility of the cohort of people living with haemophilia to anti-haemophilia medication within the Auvergne Rhône Alpes region (France) and 2) a better understanding of their needs and their perceptions regarding access to on-demand and prophylactic treatments.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The included subjects comprise people with haemophilia or other coagulation deficiencies, with no limitation of age, treated by prophylaxis or on-demand treatment, in one of the authorized hospital pharmacies in the Auvergne-Rhone-Alpes region (France).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To assess the perception of persons with haemophilia and other coagulation deficiencies regarding the accessibility to prophylactic and on-demand antihemorrhagic treatment
Time frame: Through study completion, on average 6 months.
The perception is defined by the satisfaction level of the survey participants, measured with on item in the questionnaire (Very satisfied / Rather satisfied / Rather not satisfied / Not satisfied).
To measure the accessibility of persons with haemophilia and other coagulation deficiencies regarding the accessibility to prophylactic and on-demand antihemorrhagic treatment
Time frame: Through study completion, on average 6 months.
The accessibility is measured by spatial analysis, representing the distance between the home and/or work zip codes and the hospital pharmacy zip code.
To compare the perception and the spatial accessibility of persons with haemophilia and other coagulation deficiencies regarding prophylactic and on-demand antihemorrhagic treatment
Time frame: Through study completion, on average 6 months.
The mean distances between the 4-groups of satisfaction level are compared using an analysis of variance (ANOVA).
5 secondary outcomes
To determine if the severity of the disease would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
Time frame: Through study completion, on average 6 months.
To determine if the age of the patient would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
Time frame: Through study completion, on average 6 months.
To determine if the condition of access to hospital would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
Time frame: Through study completion, on average 6 months.
To determine if the level of autonomy of the participant would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
Time frame: Through study completion, on average 6 months.
To determine if the availability of the hospital pharmacy would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
Time frame: Through study completion, on average 6 months.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 2021.
- Background: PubMed 31405805Leroy V, Freyssenge J, Renard F, Tazarourte K, Negrier C, Chamouard V. Access to treatment among persons with hemophilia: A spatial analysis assessment in the Rhone-Alpes region, France. J Am Pharm Assoc (2003). 2019 Nov-Dec;59(6):797-803. doi: 10.1016/j.japh.2019.07.006. Epub 2019 Aug 9.
- Background: PubMed 31294904Susen S, Gruel Y, Godier A, Harroche A, Chambost H, Lasne D, Rauch A, Roullet S, Fontana P, Goudemand J, de Maistre E, Chamouard V, Wibaut B, Albaladejo P, Negrier C. Management of bleeding and invasive procedures in haemophilia A patients with inhibitor treated with emicizumab (Hemlibra(R) ): Proposals from the French network on inherited bleeding disorders (MHEMO), the French Reference Centre on Haemophilia, in collaboration with the French Working Group on Perioperative Haemostasis (GIHP). Haemophilia. 2019 Sep;25(5):731-737. doi: 10.1111/hae.13817. Epub 2019 Jul 11.
- Individual participant dataSharing planned: No. Patient information is held under legal and ethical obligations of confidentiality. As the disease is rare, information should not be used or disclosed in a form that might identify a patient without his or her consent.
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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.
Laurie Fraticelli, PhD study director
RESCUe-RESUVal
Julie Freyssenge, PhD study director
RESCUe-RESUVal
34 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Health Services Accessibility and Blood Coagulation Factor Deficiencies, run by RESCUe - RESeau Cardiologie Urgence / RESUVal - RESeau des Urgences de la vallée du Rhône at 34 sites in France.
The full registered description is in the About this study section and the official record.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 14 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 34 sites in France. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Valérie Chamouard, PharmD (+33(0)472118829, valerie.chamouard@chu-lyon.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To assess the perception of persons with haemophilia and other coagulation deficiencies regarding the accessibility to prophylactic and on-demand antihemorrhagic treatment (time frame: Through study completion, on average 6 months.). The study lists 3 primary outcomes in all. 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 NCT04775888, which also lists 2 linked publications.
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 14 May 2021.