Hemophilia APASODOBLEDEMI2NCT05450640
Satisfaction Regarding the Dispensing of Emicizumab (HEMLIBRA®) for Patients With Haemophilia A in France
An observational study of Hemophilia A, run by Hospices Civils de Lyon at 1 site in France.
Trial summary
Haemophilia A (HA) is a rare constitutional haemorrhagic disease whose drug management is based on the use of chronic lifelong replacement therapy. The occurrence of an inhibitor is a dreaded complication that impacts conventional management, consisting in using factor VIII (FVIII)-based replacement therapy, most… Read more about this study
- Who is it for?
- Ages 0 years and older
- Conditions the study is for
- Hemophilia A
- What is being tested?
- satisfaction survey
- How many people
- 176 participants
- Where
- 1 siteFrance
- Study dates
- 9 Jan 2023to 9 Jan 2023
- 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 0 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: With approximately one in 6,000 men with haemophilia A, we estimated about 2,900 patients in France with 35% of cases with severe haemophilia, 15% of cases with moderate haemophilia and 50% of cases with minor severity. The eligible population for inclusion in the study considered all patients, regardless of age, treated with Emicizumab (HEMLIBRA®), whether they choose dispensing in community pharmacies, or retained dispensing in hospital pharmacy. The Laboratory Roche-Chugai which commercialize Emicizumab (HEMLIBRA®) estimated about 1'000 patients eligible to the inclusion criteria. We assumed that the uptake of the dispensing circuit, and therefore the number of patients choosing dispensing in community pharmacies, approximated the number of trained pharmacists.
Who may be able to join
- Patient with hemophilia type A with or without inhibitors
- Patients treated with Emicizumab (Hemlibra®)
- Age over 18 years old or legal representative of a minor haemophilia type A patient or his/her relative/caregiver for a non-autonomous adult patient
Who cannot join
- Patient under guardianship / curatorship
- Opposition expressed to use personal data
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Evaluation of the Evolution of the Patient Care Pathway in the Context of the Implementation of the DOuBLE EMIcizumab Dispensing Circuit in France
Haemophilia A (HA) is a rare constitutional haemorrhagic disease whose drug management is based on the use of chronic lifelong replacement therapy. The occurrence of an inhibitor is a dreaded complication that impacts conventional management, consisting in using factor VIII (FVIII)-based replacement therapy, most often for prophylaxis. Although effective, these treatments can only be administered intravenously, leading to accessibility constraints and significant mental burden for patients and their relatives.
Before June 15, 2021 in France, the emicizumab (HEMLIBRA®) was available only in hospital pharmacies for the prevention or reduction of bleedings. The introduction of the dual dispensing circuit in hospital or community pharmacies, left to patient's choice, is effective from this date. These changes have important organizational consequences for patients and health professionals alongside the pathway of care. Therefore, the effectiveness of this new organization requires to be evaluated with a national French study, called PASO DOBLE DEMI. The aims of this study are twofold :
Read more
- To evaluate the direct impact of the training programs provided to the new placeholders of the dispensing circuit ; the community pharmacists, II. To evaluate satisfaction of patients or their relatives regarding the emicizumab treatment whether they chose dispensing in the community pharmacy, or kept the dispensing at the hospital pharmacy.
The methodology was based on the 4-level of the Kirkpatrick's evaluation model; 1) the immediate reaction of community pharmacists following the trainings (Reaction), 2) their knowledge acquisition (Learning), 3) their professional practice (Behavior) and 4 ) the patients' satisfaction related to their treatment whether dispensing in hospital or in community pharmacies (Results).
The PASO DOBLE DEMI II study was based on the fourth level of the evaluation model and particularly to evaluate to what extent the dispensing of Emicizumab (HEMLIBRA ®) treatment in community pharmacies has contributed to the improvement of the satisfaction of patients with HA.
The availability of the treatment in community pharmacy assumes an improvement of the treatment accessibility for the patient at several levels :
- Global accessibility: what choice has the patient expressed to access his treatment from a practical point of view?
- Adaptation: Has the patient adapted to the new treatment delivery organization?
- Availability of resources: are the special needs of the patients taken into consideration?
- Social acceptability: is the patient willing to follow the proposed care pathway? The issue of treatment cost is not considered in this study because the cost of antihemophilic treatments is totally covered by the French government for all patients regardless of their insurance coverage
- Conditions
- Hemophilia A
- Being tested
- satisfaction survey
- Keywords
- Hemophilia A, Accessibility, Emicizumab (HEMLIBRA®), Patient satisfaction
- Medical subject headings
- Hemophilia A, Patient Satisfaction
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
satisfaction surveyOther
1 eQuestionnaire per patient completed during study
| Study group | Role | What they receive |
|---|---|---|
| Patients with haemophilia A with dispensing Emicizumab (HEMLIBRA®) in community pharmacy | This group of patients have chosen the dispensing of Emicizumab (HEMLIBRA®) in community pharmacy. This choice involves the contribution of the community pharmacist in the dispensing circuit who does not replace the hospital pharmacist but interact together for prescriptions renewals, emergency treatment or sharing information if necessary. Receives: Other: satisfaction survey | |
| Patients with haemophilia A with dispensing Emicizumab (HEMLIBRA®) in hospital pharmacy | This group of patients have kept the dispensing of Emicizumab (HEMLIBRA®) in hospital pharmacy Receives: Other: satisfaction survey |
What is being measured
The primary outcome is the main question the study is designed to answer.
satisfaction levels reported through a specific questionnaire
This questionnaire is composed of 40 questions for an estimated time to fulfil not exceeding 8 minutes. The overall satisfaction levels consists in collecting the patient's perception based on the 4-item of the Likert-scale (strongly dissatisfied, rather not satisfied, rather satisfied, strongly satisfied) taking into account the 4 dimensions of accessibility (global accessibility, adaptation, availability of resources, social acceptability).
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jul 2022
- Study start9 Jan 2023
- Primary completion9 Jan 2023
- Study completion9 Jan 2023
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.
- More trials run by Hospices Civils de LyonThe sponsor's other registered studies.
- 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
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 176 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Valérie Chamouard, Principal investigator, Hospices Civils de Lyon
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05450640
- Sponsor's protocol ID
- PASODOBLEDEMI2
- Acronym
- PASODOBLEDEMI2
- Status last verified
- Mar 2023
- First submitted
- 5 Jul 2022
- Last updated
- 3 Mar 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Fraticelli L, Freyssenge J, Prome-Combel E, Agnellet E, Dargaud Y, Chamouard V. Evaluation of the Care Pathway in the Context of the Dispensing of Emicizumab (Hemlibra) in Community Pharmacies in France: Protocol for a Cross-sectional Study Based on the Kirkpatrick Model. JMIR Res Protoc. 2023 Mar 8;12:e43091. doi: 10.2196/43091.
PubMed 36884286
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Evolution of the Patient Care Pathway in the Context of the Implementation of the DOuBLE EMIcizumab Dispensing Circuit in France
- ClinicalTrials.gov ID
- NCT05450640
- Acronym / sponsor ID
- PASODOBLEDEMI2 / PASODOBLEDEMI2
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A, Accessibility, Emicizumab (HEMLIBRA®), Patient satisfaction
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 176 participants (actual)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- 9 Jan 2023 (actual)
- Primary completion
- 9 Jan 2023 (actual)
- Study completion
- 9 Jan 2023 (actual)
- First posted
- 11 Jul 2022 (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.
Haemophilia A (HA) is a rare constitutional haemorrhagic disease whose drug management is based on the use of chronic lifelong replacement therapy. The occurrence of an inhibitor is a dreaded complication that impacts conventional management, consisting in using factor VIII (FVIII)-based replacement therapy, most often for prophylaxis. Although effective, these treatments can only be administered intravenously, leading to accessibility constraints and significant mental burden for patients and their relatives.
Before June 15, 2021 in France, the emicizumab (HEMLIBRA®) was available only in hospital pharmacies for the prevention or reduction of bleedings. The introduction of the dual dispensing circuit in hospital or community pharmacies, left to patient's choice, is effective from this date. These changes have important organizational consequences for patients and health professionals alongside the pathway of care. Therefore, the effectiveness of this new organization requires to be evaluated with a national French study, called PASO DOBLE DEMI. The aims of this study are twofold :
- To evaluate the direct impact of the training programs provided to the new placeholders of the dispensing circuit ; the community pharmacists, II. To evaluate satisfaction of patients or their relatives regarding the emicizumab treatment whether they chose dispensing in the community pharmacy, or kept the dispensing at the hospital pharmacy.
The methodology was based on the 4-level of the Kirkpatrick's evaluation model; 1) the immediate reaction of community pharmacists following the trainings (Reaction), 2) their knowledge acquisition (Learning), 3) their professional practice (Behavior) and 4 ) the patients' satisfaction related to their treatment whether dispensing in hospital or in community pharmacies (Results).
The PASO DOBLE DEMI II study was based on the fourth level of the evaluation model and particularly to evaluate to what extent the dispensing of Emicizumab (HEMLIBRA ®) treatment in community pharmacies has contributed to the improvement of the satisfaction of patients with HA.
The availability of the treatment in community pharmacy assumes an improvement of the treatment accessibility for the patient at several levels :
- Global accessibility: what choice has the patient expressed to access his treatment from a practical point of view?
- Adaptation: Has the patient adapted to the new treatment delivery organization?
- Availability of resources: are the special needs of the patients taken into consideration?
- Social acceptability: is the patient willing to follow the proposed care pathway? The issue of treatment cost is not considered in this study because the cost of antihemophilic treatments is totally covered by the French government for all patients regardless of their insurance coverage
Study design
- Observational model
- Other
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients with haemophilia A with dispensing Emicizumab (HEMLIBRA®) in community pharmacy | – | Other: satisfaction surveyThis group of patients have chosen the dispensing of Emicizumab (HEMLIBRA®) in community pharmacy. This choice involves the contribution of the community pharmacist in the dispensing circuit who does not replace the hospital pharmacist but interact together for prescriptions renewals, emergency treatment or sharing information if necessary. |
| Patients with haemophilia A with dispensing Emicizumab (HEMLIBRA®) in hospital pharmacy | – | Other: satisfaction surveyThis group of patients have kept the dispensing of Emicizumab (HEMLIBRA®) in hospital pharmacy |
Who may qualify
- Ages
- 0 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- With approximately one in 6,000 men with haemophilia A, we estimated about 2,900 patients in France with 35% of cases with severe haemophilia, 15% of cases with moderate haemophilia and 50% of cases with minor severity. The eligible population for inclusion in the study considered all patients, regardless of age, treated with Emicizumab (HEMLIBRA®), whether they choose dispensing in community pharmacies, or retained dispensing in hospital pharmacy. The Laboratory Roche-Chugai which commercialize Emicizumab (HEMLIBRA®) estimated about 1'000 patients eligible to the inclusion criteria. We assumed that the uptake of the dispensing circuit, and therefore the number of patients choosing dispensing in community pharmacies, approximated the number of trained pharmacists.
Key inclusion criteria (3 listed)
- Patient with hemophilia type A with or without inhibitors
- Patients treated with Emicizumab (Hemlibra®)
- Age over 18 years old or legal representative of a minor haemophilia type A patient or his/her relative/caregiver for a non-autonomous adult patient
Key exclusion criteria (2 listed)
- Patient under guardianship / curatorship
- Opposition expressed to use personal data
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 |
|---|---|---|
| satisfaction survey1 eQuestionnaire per patient completed during study | Other | Patients with haemophilia A with dispensing Emicizumab (HEMLIBRA®) in community pharmacy, Patients with haemophilia A with dispensing Emicizumab (HEMLIBRA®) in hospital pharmacy |
Primary outcome
satisfaction levels reported through a specific questionnaire
Time frame: 6 months
This questionnaire is composed of 40 questions for an estimated time to fulfil not exceeding 8 minutes. The overall satisfaction levels consists in collecting the patient's perception based on the 4-item of the Likert-scale (strongly dissatisfied, rather not satisfied, rather satisfied, strongly satisfied) taking into account the 4 dimensions of accessibility (global accessibility, adaptation, availability of resources, social acceptability).
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 3 Mar 2023.
- Derived from this study: PubMed 36884286Fraticelli L, Freyssenge J, Prome-Combel E, Agnellet E, Dargaud Y, Chamouard V. Evaluation of the Care Pathway in the Context of the Dispensing of Emicizumab (Hemlibra) in Community Pharmacies in France: Protocol for a Cross-sectional Study Based on the Kirkpatrick Model. JMIR Res Protoc. 2023 Mar 8;12:e43091. doi: 10.2196/43091.
- Individual participant dataSharing planned: No
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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.
Valérie Chamouard principal investigator
Hospices Civils de Lyon
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Hospices Civils de Lyon at 1 site in France.
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 2 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 3 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
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?
satisfaction levels reported through a specific questionnaire (time frame: 6 months). 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 NCT05450640, 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 3 Mar 2023.