HaemophiliaTRANSHEMO2NCT07418827
Determinants and Consequences of the Transition to Adulthood for Adolescents With Severe Haemophilia: TRANSHEMO 2, an Ancillary Study to the TRANSHEMO Project
An observational study of Haemophilia, run by Assistance Publique Hopitaux De Marseille at 25 sites in France.
Trial summary
Haemophilia is a rare genetic disorder which, in its severe form and in the absence of treatment, can be life-threatening. Since the 1960s and the introduction of coagulation factor concentrates, the life expectancy of people with haemophilia has increased rapidly. Read more about this study
- Who is it for?
- Ages 20 to 29 years
- Conditions the study is for
- Haemophilia
- What is being tested?
- questionnaires
- How many people
- 75 participants
- Where
- 25 sitesFrance
- Study dates
- 28 May 2026to Jun 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 20 to 29 years
- All sexes
- No healthy volunteers
- Adult
Study population: Young adults with severe haemophilia (haemophilia A or B)
Who may be able to join
- Young adults who participated in the TRANSHEMO project during adolescence and who are currently aged 20-29 years;
- Young adults with severe haemophilia (haemophilia A or B);
- Young adults registered in the FranceCoag registry;
- Young adults who have received the participant information sheet for the TRANSHEMO 2 project;
- Young adults who did not object to participation in the present study.
Who cannot join
- patients with comprehension difficulties;
- patients who are unable to read and/or write;
- patients who express opposition to participation in this study.
Trial locations and contacts
25 sites in 1 country. Contact the study team or a site near you to ask about taking part.
25 sites in 1 country
France25 sites, 25 recruiting
- Recruiting
CH Annecy - St Julien
Annecy
Claire REYNES, 0033450636608, creynes@ch-annecygenevois.fr
- Not yet recruiting
Chu de Bordeaux
Bordeaux
Sabine-Marie CASTET, 0033556794879, sabine-marie.castet@chu-bordeaux.fr
- Not yet recruiting
Chu de Caen
Caen
Yohann REPESSE, 0033231064849, repesse-y@chu-caen.fr
- Not yet recruiting
Centre hospitalier Métropole Savoie
Chambéry
Romain JAILLER, 0033479966183, romain.jailler@ch-metropole-savoie.fr
- Not yet recruiting
CHU Clermont-Ferrand
Clermont-Ferrand
Aurelien LEBRETON, 0033800800065, alebreton@chu-clermontferrand.fr
- Not yet recruiting
Chu de Dijon
Dijon
Fabienne GENRE VOLOT, 0033380293257, fabienne.volot@chu-dijon.fr
- Not yet recruiting
Chu de Grenoble
Grenoble
Claire BARRO, 0033476765487, CBarro@chu-grenoble.fr
- Not yet recruiting
CHRU de Lille
Lille
Mélanie DANIEL, 0033320444845, melanie.daniel@chu-lille.fr
- Not yet recruiting
CHU Limoges
Limoges
Thomas LAUVRAY, 0033555056801, thomas.lauvray@chu-limoges.fr
- Not yet recruiting
Hospices Civils de Lyon
Lyon
Sandrine MEUNIER, 0033472118810, sandrine.meunier@chu-lyon.fr
- Not yet recruiting
Assistance publique - Hôpitaux de Marseille
Marseille
Hervé CHAMBOST, 033491386776, herve.chambost@ap-hm.fr
- Not yet recruiting
CH Montmorency
Montmorency
Abel HASSOUN, 0033134066954, abel.hassoun@ch-simoneveil.fr
- Not yet recruiting
CHU Montpellier
Montpellier
Christine BIRON-ANDREANI, 0033467337771, c-biron@chu-montpellier.fr
- Not yet recruiting
Chu de Nancy
Nancy
Birgit FROTSCHER, 0033383153784, b.frotscher@chu-nancy.fr
- Not yet recruiting
CHU Nantes
Nantes
Marc TROSSAËRT, 0033240087468, marc.trossaert@chu-nantes.fr
- Not yet recruiting
AP-HP (Hôpital Kremlin Bicetre)
Paris
Roseline D'OIRON, 033145212862, roseline.doiron@aphp.fr
- Not yet recruiting
AP-HP (Hôpital NECKER)
Paris
Laurent FRENZEL, 0033144495273, laurent.frenzel@aphp.fr
- Not yet recruiting
CHU Reims
Reims
Valérie POULLE, 0033326784652, vproulle@chu-reims.fr
- Not yet recruiting
CHU de Rennes
Rennes
Benoît GUILLET, 0033299282410, benoit.guillet@chu-rennes.fr
- Not yet recruiting
CHU de Rouen
Rouen
Pierre CHAMOUNI, 0033232880249, pierre.chamouni@chu-rouen.fr
- Not yet recruiting
CHU La Reunion
Saint-Denis, La Réunion
Stephane VANDERBECKEN, 0033262906246, stephane.vanderbecken@chu-reunion.fr
- Not yet recruiting
CHU St Etienne
Saint-Etienne
Brigitte TARDY, 0033477127863, brigitte.tardy@chu-st-etienne.fr
- Not yet recruiting
CHU Strasbourg
Strasbourg
Dominique DESPREZ FLOR, 0033388128371, dominique.desprez@chru-strasbourg.fr
- Not yet recruiting
CHU Toulouse
Toulouse
Caroline OUDOT-CHALLARD, 0033561776803, oudotchallard.c@chu-toulouse.fr
- Not yet recruiting
CH Versailles
Versailles
Emmanuelle DE RAUCOURT, 0033139638745, ederaucourt@ch-versailles.fr
About this study
Haemophilia is a rare genetic disorder which, in its severe form and in the absence of treatment, can be life-threatening. Since the 1960s and the introduction of coagulation factor concentrates, the life expectancy of people with haemophilia has increased rapidly. Today, for most affected individuals, the disease is experienced as a chronic condition.
The transition process enabling adolescents and young adults (AYA) with a chronic disease to move into adult life can be complex, as they must face all the changes experienced by AYA in general, combined with issues related to their chronic condition and its management.
Read more
A successful transition involves a transfer of responsibility from parents to AYA regarding the management of their health condition, as well as the acquisition by AYA of knowledge, skills and autonomy. A difficult transition may lead to decreased adherence to follow-up or treatment, deterioration of overall health status and/or quality of life, or difficulties in entering adult life.
In this context, the national cross-sectional study TRANSHEMO was initiated in 2017. Its objective was to compare adherence to healthcare management between two groups of AYA with severe haemophilia (adolescents [for whom the transition is ongoing] versus young adults [for whom the transition may have been completed]), and to identify the determinants of this adherence. The results showed that young adults had a lower adherence rate than adolescents (82.2% vs. 61.2%, p<0.001). Among the determinants studied, being a young adult, having repeated at least one school year, and presenting psychological and emotional difficulties were factors that had a negative effect on adherence to healthcare management. However, the cross-sectional nature of the study represents a limitation that restricts causal inference. Complementing this project with a longitudinal study would address this limitation.
The results obtained from this new study (TRANSHEMO 2) may contribute to the literature by providing insights into both the determinants of sustained adherence to healthcare management among adolescents with severe haemophilia who become young adults, and the associations between these determinants.
The longitudinal design of the project will allow the establishment of a higher level of causal inference between the maintenance of adherence during the transition to adult life and its determinants, which represents a major epidemiological strength. Moreover, results addressing the issue of transition in the context of chronic diseases and derived from longitudinal studies remain scarce, making the findings of this project particularly original. Finally, haemophilia, a relatively frequent condition among rare diseases, could represent an interesting model for understanding the impact of transition in this type of pathology.
Study hypothesis The extent of the reduction in adherence to healthcare during the transition process, and/or the determinants of the maintenance of adherence identified in the longitudinal TRANSHEMO 2 project, may differ from those highlighted in the original cross-sectional TRANSHEMO project.
Specific aims
Main objective:
To compare the rate of adherence to healthcare among adolescents who participated in the TRANSHEMO project, using data collected during the original TRANSHEMO study when they were adolescents (before transition) and data to be collected as part of the TRANSHEMO 2 study when they have become young adults (after transition).
Secondary objective:
To identify the determinants of the maintenance of this adherence and the associations between these determinants, within the framework of a longitudinal study.
- Conditions
- Haemophilia
- Being tested
- questionnaires
- Keywords
- haemophilia, young adult, cohort study, genetic disorder, healthcare management
- Medical subject headings
- Hemophilia A, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
questionnairesOther
Participants will have questionnaires
| Study group | Role | What they receive |
|---|---|---|
| adolescents with severe hemophilia who participated in the TRANSHEMO project | Not described Receives: Other: questionnaires |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of follow-up visits over the last 2 years (FranceCoag registry)
Quantitative variable. Dichotomized in agreement and not in agreement with the recommended number
Measured: 1 day
Rate of Physician-reported adherence to clinical follow-up
Ordinal variable in 5 modalities (Very high/High/Average/Low/Very low). Dichotomized in very high and high to very low.
Measured: 1 day
Rate of Patient-reported adherence to clinical follow-up
Ordinal variable in 2 modalities : visiting Haemophilia Treatment Centre (HTC) at least once a year and Not visiting HTC every year.
Measured: 1 day
If under prophylaxis, number of prophylactic injections over the last 3 months (FranceCoag registry)
Quantitative variable. Dichotomized in agreement and not in agreement with the recommended number.
Measured: 1 day
If under prophylaxis, rate of physician-reported adherence to prophylaxis
Ordinal variable in 5 modalities (Very high/High/Average/Low/Very low). Dichotomized in very high and high to very low.
Measured: 1 day
Rate of Patient-reported adherence to treatment
Nominal variable.
If under prophylaxis:
- Dichotomized in having no difficulties in adherence to prophylaxis or having difficulties but missing treatment less than once a week and having difficulties and missing treatment once a week or more
If under on-demand treatment:
- Dichotomized in having no difficulties in recognizing early signs of haemorrhage and having difficulties inrecognizing early signs of haemorrhage
Measured: 1 day
Physician-reported number of haemorrhagic events over the last two years
Quantitative variable. Dichotomized in none and at least one
Measured: 1 day
Rate of composite endpoint
A composite quantitative endpoint was created by adding the criteria described above (seven for people under prophylaxis, five for people under ondemandtreatment). Adherent peoplewere defined as those having 4 to 7 points if under prophylaxis, and as those having 3 to 5 points if underon-demand treatment. Dichotomized in adherent and non adherent
Measured: 1 day
Secondary outcomes (9)
Quality of life analysis
Generic quality of life (QoL) measured by Short form 12 Health Survey (SF-12). Two subscores: Physical component score (PCS) for physical health-related quality of life (HRQoL), mental component score (MCS) for mental HRQoL. Scores range from 0 to 100 with higher scores indicating better HRQoL.
Measured: 1 day
Coping strategies analysis
Coping strategies measured by Brief Coping Orientation to Problems Experienced Scale (Brief COPE). 28-item questionnaire evaluating 14 coping strategies, which are grouped into four main strategies: Social support, problem solving, avoidance, and positive thinking. Scores ranged from 0 to 100. Higher scores indicate a more frequent use of the assessed coping strategy
Measured: 1 day
Autonomy analysis
Autonomy measured by autonomy questionnaire Noom. 15-item questionnaire classified into three dimensions: Attitudinal autonomy, Functional autonomy, Emotional autonomy. Scores range from 5 to 25 with higher scores indicating higher level of autonomy
Measured: 1 day
Time perspective analysis
Time perspective measured by Zimbardo Time Perspective Inventory (ZTPI). Two subscales "Negative past" (nine items) and "Future" (12 items) were retained from the original 56-item questionnaire, measuring tendency to focus on different aspects of the past and future. Score range from 1 to 5. Higher scores indicate stronger projection in the assessed time perspective .
Measured: 1 day
Family functioning analysis
Family functioning measured by Family Assessment Device (FAD). 6-item questionnaire. Scores range from 1 to 4 with lower score indicating better general family functioning.
Measured: 1 day
Rate of academic difficulty
Nominal variable with two modalities : at least one grade repetition at school and none
Measured: 1 day
Rate of type of healthcare transition in the HTC
Nominal variable with two modalities : same hospital and service for adolescents and adults and same hospital, two different services (paediatric/adult)
Measured: 1 day
Rate of paediatrician in the medical team of the HTC
Nominal variable with two modalities : yes and no
Measured: 1 day
Rate of setup of therapeutic education activities for transition in the HTC
Nominal variable with two modalities : yes and no
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Feb 2026
- Study start28 May 2026
- Primary completionDec 2026 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HaemophiliaStudies of the same condition, open and under way.
- More trials run by Assistance Publique Hopitaux De MarseilleThe 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
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 75 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique Hopitaux De Marseille
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- François Cremieux, Study director, AP-HM
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07418827
- Sponsor's protocol ID
- RCAPHM25_0354
- Other IDs
- 2025-A01711-48
- Acronym
- TRANSHEMO2
- Status last verified
- Oct 2026
- First submitted
- 26 Jan 2026
- Last updated
- 8 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Determinants and Consequences of the Transition to Adulthood for Adolescents With Severe Haemophilia: TRANSHEMO 2, an Ancillary Study to the TRANSHEMO Project
- ClinicalTrials.gov ID
- NCT07418827
- Acronym / sponsor ID
- TRANSHEMO2 / RCAPHM25_0354
- Conditions
- Haemophilia
- Keywords
- haemophilia, young adult, cohort study, genetic disorder, healthcare management
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 75 participants (estimated)
- Lead sponsor
- Assistance Publique Hopitaux De MarseilleUniversity, hospital or other
- Study start
- 28 May 2026 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 18 Feb 2026 (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 is a rare genetic disorder which, in its severe form and in the absence of treatment, can be life-threatening. Since the 1960s and the introduction of coagulation factor concentrates, the life expectancy of people with haemophilia has increased rapidly. Today, for most affected individuals, the disease is experienced as a chronic condition.
The transition process enabling adolescents and young adults (AYA) with a chronic disease to move into adult life can be complex, as they must face all the changes experienced by AYA in general, combined with issues related to their chronic condition and its management.
A successful transition involves a transfer of responsibility from parents to AYA regarding the management of their health condition, as well as the acquisition by AYA of knowledge, skills and autonomy. A difficult transition may lead to decreased adherence to follow-up or treatment, deterioration of overall health status and/or quality of life, or difficulties in entering adult life.
In this context, the national cross-sectional study TRANSHEMO was initiated in 2017. Its objective was to compare adherence to healthcare management between two groups of AYA with severe haemophilia (adolescents [for whom the transition is ongoing] versus young adults [for whom the transition may have been completed]), and to identify the determinants of this adherence. The results showed that young adults had a lower adherence rate than adolescents (82.2% vs. 61.2%, p<0.001). Among the determinants studied, being a young adult, having repeated at least one school year, and presenting psychological and emotional difficulties were factors that had a negative effect on adherence to healthcare management. However, the cross-sectional nature of the study represents a limitation that restricts causal inference. Complementing this project with a longitudinal study would address this limitation.
The results obtained from this new study (TRANSHEMO 2) may contribute to the literature by providing insights into both the determinants of sustained adherence to healthcare management among adolescents with severe haemophilia who become young adults, and the associations between these determinants.
The longitudinal design of the project will allow the establishment of a higher level of causal inference between the maintenance of adherence during the transition to adult life and its determinants, which represents a major epidemiological strength. Moreover, results addressing the issue of transition in the context of chronic diseases and derived from longitudinal studies remain scarce, making the findings of this project particularly original. Finally, haemophilia, a relatively frequent condition among rare diseases, could represent an interesting model for understanding the impact of transition in this type of pathology.
Study hypothesis The extent of the reduction in adherence to healthcare during the transition process, and/or the determinants of the maintenance of adherence identified in the longitudinal TRANSHEMO 2 project, may differ from those highlighted in the original cross-sectional TRANSHEMO project.
Specific aims
Main objective:
To compare the rate of adherence to healthcare among adolescents who participated in the TRANSHEMO project, using data collected during the original TRANSHEMO study when they were adolescents (before transition) and data to be collected as part of the TRANSHEMO 2 study when they have become young adults (after transition).
Secondary objective:
To identify the determinants of the maintenance of this adherence and the associations between these determinants, within the framework of a longitudinal study.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| adolescents with severe hemophilia who participated in the TRANSHEMO project | – | Other: questionnaires |
Who may qualify
- Ages
- 20 to 29 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- Young adults with severe haemophilia (haemophilia A or B)
Key inclusion criteria (5 listed)
- Young adults who participated in the TRANSHEMO project during adolescence and who are currently aged 20-29 years;
- Young adults with severe haemophilia (haemophilia A or B);
- Young adults registered in the FranceCoag registry;
- Young adults who have received the participant information sheet for the TRANSHEMO 2 project;
- Young adults who did not object to participation in the present study.
Key exclusion criteria (3 listed)
- patients with comprehension difficulties;
- patients who are unable to read and/or write;
- patients who express opposition to participation in 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 |
|---|---|---|
| questionnairesParticipants will have questionnaires | Other | adolescents with severe hemophilia who participated in the TRANSHEMO project |
Primary outcome
Number of follow-up visits over the last 2 years (FranceCoag registry)
Time frame: 1 day
Quantitative variable. Dichotomized in agreement and not in agreement with the recommended number
Rate of Physician-reported adherence to clinical follow-up
Time frame: 1 day
Ordinal variable in 5 modalities (Very high/High/Average/Low/Very low). Dichotomized in very high and high to very low.
Rate of Patient-reported adherence to clinical follow-up
Time frame: 1 day
Ordinal variable in 2 modalities : visiting Haemophilia Treatment Centre (HTC) at least once a year and Not visiting HTC every year.
If under prophylaxis, number of prophylactic injections over the last 3 months (FranceCoag registry)
Time frame: 1 day
Quantitative variable. Dichotomized in agreement and not in agreement with the recommended number.
If under prophylaxis, rate of physician-reported adherence to prophylaxis
Time frame: 1 day
Ordinal variable in 5 modalities (Very high/High/Average/Low/Very low). Dichotomized in very high and high to very low.
Rate of Patient-reported adherence to treatment
Time frame: 1 day
Nominal variable. If under prophylaxis: - Dichotomized in having no difficulties in adherence to prophylaxis or having difficulties but missing treatment less than once a week and having difficulties and missing treatment once a week or more If under on-demand treatment: - Dichotomized in having no difficulties in recognizing early signs of haemorrhage and having difficulties inrecognizing early signs of haemorrhage
Physician-reported number of haemorrhagic events over the last two years
Time frame: 1 day
Quantitative variable. Dichotomized in none and at least one
Rate of composite endpoint
Time frame: 1 day
A composite quantitative endpoint was created by adding the criteria described above (seven for people under prophylaxis, five for people under ondemandtreatment). Adherent peoplewere defined as those having 4 to 7 points if under prophylaxis, and as those having 3 to 5 points if underon-demand treatment. Dichotomized in adherent and non adherent
9 secondary outcomes
Quality of life analysis
Time frame: 1 day
Coping strategies analysis
Time frame: 1 day
Autonomy analysis
Time frame: 1 day
Time perspective analysis
Time frame: 1 day
Family functioning analysis
Time frame: 1 day
Rate of academic difficulty
Time frame: 1 day
Rate of type of healthcare transition in the HTC
Time frame: 1 day
Rate of paediatrician in the medical team of the HTC
Time frame: 1 day
Rate of setup of therapeutic education activities for transition in the HTC
Time frame: 1 day
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2026.
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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.
François Cremieux study director
AP-HM
25 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Haemophilia, run by Assistance Publique Hopitaux De Marseille at 25 sites 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 5 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: looking for participants now, as of the sponsor's last update on 8 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Noémie Resseguier, MD (0033491324272, noemie.resseguier@univ-amu.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of follow-up visits over the last 2 years (FranceCoag registry) (time frame: 1 day). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 8 Oct 2026.