Accessibility of Prophylaxis and On-demand Treatment for Persons With Haemophilia and Other Coagulation Deficiencies

NCT ID: NCT04775888

Last Updated: 2021-05-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

500 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-09-21

Study Completion Date

2021-05-21

Brief Summary

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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

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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.

Conditions

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Health Services Accessibility Blood Coagulation Factor Deficiencies

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

Excluded:

\- Home delivery (not included in the study via hospital pharmacy)
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hospices Civils de Lyon

OTHER

Sponsor Role collaborator

RESCUe - RESeau Cardiologie Urgence / RESUVal - RESeau des Urgences de la vallée du Rhône

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Laurie Fraticelli, PhD

Role: STUDY_DIRECTOR

RESCUe-RESUVal

Julie Freyssenge, PhD

Role: STUDY_DIRECTOR

RESCUe-RESUVal

Locations

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Centre Hospitalier Albertville-Moûtiers

Albertville, , France

Site Status RECRUITING

Centre Hospitalier Annecy Genevois

Annecy, , France

Site Status RECRUITING

Centre Hospitalier d'Ardèche Nord

Annonay, , France

Site Status RECRUITING

Centre Hospitalier d'Ardèche Méridionale

Aubenas, , France

Site Status RECRUITING

Centre Hospitalier Henri Mondor d'Aurillac

Aurillac, , France

Site Status RECRUITING

Centre Hospitalier de Belley

Belley, , France

Site Status RECRUITING

Centre Hospitalier Fleyriat

Bourg-en-Bresse, , France

Site Status RECRUITING

Centre hospitalier de Bourg St Maurice

Bourg-Saint-Maurice, , France

Site Status RECRUITING

Centre Hospitalier Pierre Oudot

Bourgoin, , France

Site Status RECRUITING

Groupement Hospitalier Est, Hospices Civils de Lyon

Bron, , France

Site Status RECRUITING

Centre Hospitalier de Chambéry - Hôpital Savoie

Chambéry, , France

Site Status RECRUITING

Centre Hospitalier Universitaire de Clermont-Ferrand

Clermont-Ferrand, , France

Site Status RECRUITING

Centre Hospitalier Alpes Léman

Contamine-sur-Arve, , France

Site Status RECRUITING

Hôpital Nord Ouest de Villefranche-sur-Saône

Gleizé, , France

Site Status RECRUITING

Centre Hospitalier Universitaire Grenoble - Alpes

La Tronche, , France

Site Status RECRUITING

Hôpital Edouard herriot, Hospices Civils de Lyon

Lyon, , France

Site Status RECRUITING

Hôpital de la Croix-Rousse, Hospices Civils de Lyon

Lyon, , France

Site Status RECRUITING

Centre Hospitalier du Forez

Montbrison, , France

Site Status RECRUITING

Centre Hospitalier de Montélimar

Montélimar, , France

Site Status RECRUITING

Hôpital de Montluçon

Montluçon, , France

Site Status RECRUITING

Centre Hospitalier de Moulins-Yzeure

Moulins, , France

Site Status RECRUITING

Centre Hospitalier Du Haut Bugey

Oyonnax, , France

Site Status RECRUITING

Centre Hospitalier des Vals d'Ardeche

Privas, , France

Site Status RECRUITING

Centre Hospitalier de Roanne

Roanne, , France

Site Status RECRUITING

Hôpitaux Drôme Nord

Romans-sur-Isère, , France

Site Status RECRUITING

Pharmacie Centrale, Hospices Civils de Lyon

Saint-Genis-Laval, , France

Site Status RECRUITING

Centre Hospitalier Intercommunal Sud-Léman Valserine

Saint-Julien-en-Genevois, , France

Site Status RECRUITING

Centre Hospitalier Uuniversitaire Nord, Saint-Étienne

Saint-Priest-en-Jarez, , France

Site Status RECRUITING

Hôpitaux du Pays du Mont-Blanc

Sallanches, , France

Site Status RECRUITING

Hôpital Nord-Ouest Tarare-Grandris

Tarare, , France

Site Status RECRUITING

Hopitaux du Léman

Thonon-les-Bains, , France

Site Status RECRUITING

Centre Hospitalier de Valence

Valence, , France

Site Status RECRUITING

Centre Hospitalier de Vichy

Vichy, , France

Site Status RECRUITING

Centre Hospitalier Lucien Hussel

Vienne, , France

Site Status RECRUITING

Countries

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France

Central Contacts

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Valérie Chamouard, PharmD

Role: CONTACT

+33(0)472118829

Carlos El Khoury, MD, PhD

Role: CONTACT

+33(0)437021059

Facility Contacts

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Thomas Gailland

Role: primary

Christelle Roch

Role: primary

Sophie Vernardet

Role: primary

Alain Lenoir

Role: primary

Valérie Millot

Role: primary

Marie Geneste

Role: primary

Stéphanie Claveranne

Role: primary

Valérie Chaminant

Role: primary

Clément Beyron

Role: primary

Valérie Chamouard

Role: primary

Elodie Bernard

Role: primary

Anne Boyer

Role: primary

Catherine Diakhate

Role: primary

Magali Bourdelin

Role: primary

Sophie Cerena

Role: primary

Elodie Lanoi

Role: primary

Anne-Claire Uhrès

Role: primary

Hadyl Asfari

Role: primary

Jérémy Rolain

Role: primary

Carole Rimpici

Role: primary

Emmanuel Deligeard

Role: primary

Nathalie Dey

Role: primary

Dominique Quinard

Role: primary

Maud Rossignol

Role: primary

Xavier Cussoneau

Role: primary

Isabelle Carpentier

Role: primary

Alexandra Combes

Role: primary

Jihen Boussetta

Role: primary

Marion Filippi

Role: primary

Christine Vray

Role: primary

Denis Hardelin

Role: primary

Stéphanie Bourget

Role: primary

Jérôme Tavernier

Role: primary

Anne-Sophie Leromain

Role: primary

References

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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.

Reference Type BACKGROUND
PMID: 31405805 (View on PubMed)

Susen 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.

Reference Type BACKGROUND
PMID: 31294904 (View on PubMed)

Other Identifiers

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2020PHAREO

Identifier Type: -

Identifier Source: org_study_id

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