NCT04775888

Brief 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. 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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
500

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Sep 2020

Shorter than P25 for all trials

Geographic Reach
1 country

34 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 21, 2020

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

February 11, 2021

Completed
18 days until next milestone

First Posted

Study publicly available on registry

March 1, 2021

Completed
2 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 30, 2021

Completed
21 days until next milestone

Study Completion

Last participant's last visit for all outcomes

May 21, 2021

Completed
Last Updated

May 14, 2021

Status Verified

February 1, 2021

Enrollment Period

7 months

First QC Date

February 11, 2021

Last Update Submit

May 13, 2021

Conditions

Keywords

haemophiliaaccessibilityon-demand treatmentprophylactic treatment

Outcome Measures

Primary Outcomes (3)

  • 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).

    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.

    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).

    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

    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

    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

    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

    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

    Through study completion, on average 6 months.

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
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)

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (34)

Centre Hospitalier Albertville-Moûtiers

Albertville, 73200, France

RECRUITING

Centre Hospitalier Annecy Genevois

Annecy, 74370, France

RECRUITING

Centre Hospitalier d'Ardèche Nord

Annonay, 07103, France

RECRUITING

Centre Hospitalier d'Ardèche Méridionale

Aubenas, 07200, France

RECRUITING

Centre Hospitalier Henri Mondor d'Aurillac

Aurillac, 15000, France

RECRUITING

Centre Hospitalier de Belley

Belley, 01300, France

RECRUITING

Centre Hospitalier Fleyriat

Bourg-en-Bresse, 01012, France

RECRUITING

Centre hospitalier de Bourg St Maurice

Bourg-Saint-Maurice, 73700, France

RECRUITING

Centre Hospitalier Pierre Oudot

Bourgoin, 38300, France

RECRUITING

Groupement Hospitalier Est, Hospices Civils de Lyon

Bron, 69500, France

RECRUITING

Centre Hospitalier de Chambéry - Hôpital Savoie

Chambéry, 73000, France

RECRUITING

Centre Hospitalier Universitaire de Clermont-Ferrand

Clermont-Ferrand, 63100, France

RECRUITING

Centre Hospitalier Alpes Léman

Contamine-sur-Arve, 74130, France

RECRUITING

Hôpital Nord Ouest de Villefranche-sur-Saône

Gleizé, 69400, France

RECRUITING

Centre Hospitalier Universitaire Grenoble - Alpes

La Tronche, 38700, France

RECRUITING

Hôpital Edouard herriot, Hospices Civils de Lyon

Lyon, 69003, France

RECRUITING

Hôpital de la Croix-Rousse, Hospices Civils de Lyon

Lyon, 69004, France

RECRUITING

Centre Hospitalier du Forez

Montbrison, 42600, France

RECRUITING

Centre Hospitalier de Montélimar

Montélimar, 26200, France

RECRUITING

Hôpital de Montluçon

Montluçon, 03100, France

RECRUITING

Centre Hospitalier de Moulins-Yzeure

Moulins, 03006, France

RECRUITING

Centre Hospitalier Du Haut Bugey

Oyonnax, 01100, France

RECRUITING

Centre Hospitalier des Vals d'Ardeche

Privas, 07007, France

RECRUITING

Centre Hospitalier de Roanne

Roanne, 42300, France

RECRUITING

Hôpitaux Drôme Nord

Romans-sur-Isère, 26100, France

RECRUITING

Pharmacie Centrale, Hospices Civils de Lyon

Saint-Genis-Laval, 69230, France

RECRUITING

Centre Hospitalier Intercommunal Sud-Léman Valserine

Saint-Julien-en-Genevois, 74164, France

RECRUITING

Centre Hospitalier Uuniversitaire Nord, Saint-Étienne

Saint-Priest-en-Jarez, 42270, France

RECRUITING

Hôpitaux du Pays du Mont-Blanc

Sallanches, 74700, France

RECRUITING

Hôpital Nord-Ouest Tarare-Grandris

Tarare, 69170, France

RECRUITING

Hopitaux du Léman

Thonon-les-Bains, 74203, France

RECRUITING

Centre Hospitalier de Valence

Valence, 26000, France

RECRUITING

Centre Hospitalier de Vichy

Vichy, 03200, France

RECRUITING

Centre Hospitalier Lucien Hussel

Vienne, 38200, France

RECRUITING

Related Publications (2)

  • 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.

    PMID: 31405805BACKGROUND
  • 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.

    PMID: 31294904BACKGROUND

MeSH Terms

Conditions

Coagulation Protein DisordersHemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesBlood Coagulation Disorders, InheritedHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Laurie Fraticelli, PhD

    RESCUe-RESUVal

    STUDY DIRECTOR
  • Julie Freyssenge, PhD

    RESCUe-RESUVal

    STUDY DIRECTOR

Central Study Contacts

Valérie Chamouard, PharmD

CONTACT

Carlos El Khoury, MD, PhD

CONTACT

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 11, 2021

First Posted

March 1, 2021

Study Start

September 21, 2020

Primary Completion

April 30, 2021

Study Completion

May 21, 2021

Last Updated

May 14, 2021

Record last verified: 2021-02

Data Sharing

IPD Sharing
Will not share

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.

Locations