Severe Forms of HemophiliaACADHEMNCT02834130
Input of a Multidisciplinary Device to Accompany the Announce of Diagnosis for Young Children With Serious Constitutional Hemorrhagic Disease and Their Families
A study testing Evaluation of psychological and psychopathological in people with Severe Forms of Hemophilia and Other Constitutional Bleeding Disorders, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
Trial summary
Severe forms of hemophilia and other constitutional bleeding disorders represent a group of rare diseases. In last decades, news therapies have dramatically increased life expectancy and joint's protection. Read more about this study
- Who is it for?
- Ages 3 to 12 years
- Conditions the study is for
- Severe Forms of Hemophilia, Other Constitutional Bleeding Disorders
- What is being tested?
- Evaluation of psychological and psychopathological
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteFrance
- Study dates
- Apr 2014to Apr 2016
- Phase
- Interventional
- 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 3 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients aged between 3 and 12 years
- Patients diagnosed and treated at the Center until inclusion
- Patients with FVIII :C < 2% or with FIX :C <2% or with FVII:C <2% or with FXIII:C <2% or fibrinogen <0,2 g/l, or with type 3 von Willebrand diseases or Thrombasthenia of Glanzmann diseases.
- Patient who participate to multidisciplinary support and announcement of diagnosis device
- Voluntarily given fully informed written and signed consents obtained before any study-related procedures are conducted
Who cannot join
- Patient with other clotting disorder or disease.
- Patient diagnosed or treated in other centre
- Patient with psychiatric disorder or disease
- Patient with other severe chronic disease
Trial locations and contacts
1 site in 1 country.
France1 site
Hopital Enfants de la Timone Assistance Publique Hôpitaux de Marseille
Marseille, 13354
About this study
Official title: Input of a Multidisciplinary Device Including a Psychological Approach, to Accompany the Announce of Diagnosis for Young Children With Serious Constitutional Hemorrhagic Disease and Their Families
Severe forms of hemophilia and other constitutional bleeding disorders represent a group of rare diseases. In last decades, news therapies have dramatically increased life expectancy and joint's protection.
The investigators propose to set up a single-centre, descriptive, transversal pilot study, designed for the inclusion of around thirty children from 2 to 10 years, with haemophilia or allied hereditary bleeding disorders (HBD), who were referred to the Hemophilia Treatment Center (HTC) of Marseille at diagnosis. For those children who were able to benefit from the totality or part of the device accompanying the diagnostic announcement in the past 10 years.
Read the detailed description
Severe forms of hemophilia and other constitutional bleeding disorders represent a group of rare diseases. In last decades, news therapies have dramatically increased life expectancy and joint's protection. The recurrence of joint bleedings is responsive of multifocal arthropathy in the natural history, but nowadays, the orthopedic outcome is good thanks to prophylactic treatments.
However, the announcement of the diagnosis of such hereditary bleeding disorders at a very young age represents a real psychological trauma for parents. Such an announcement may induce some psycho-developmental impairment.
An innovative support has been implemented at the Hemophilia Treatment Center in Marseille, in conjunction with the French Hemophilia Society. This program is based on a multidisciplinary partnership that favors the psychological support and the reinsurance of the parents.
With Rare Diseases Foundation support, the investigators have initiated this pilot study that will include more than 20 patients with severe hemophilia or constitutional bleeding disorder, who benefit from the device. This descriptive, monocentric, interventional study will permit to describe the psychological status, the quality of life of patients and their parents, but also to evaluate the impact of the device on initiation and observance of treatments like Long Term Prophylaxis.
- Being tested
- Evaluation of psychological and psychopathological
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Evaluation of psychological and psychopathologicalBehavioural
Also called Evaluation of quality of life
| Study group | Role | What they receive |
|---|---|---|
| children from 2 to 10 years with haemophilia or allied HBD, wh | Other | Not described Receives: Behavioral: Evaluation of psychological and psychopathological |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Treatment Exposure Days compared to the Number of Treatment Exposure days prescribed
Treatment Observance
Measured: 2 years
Secondary outcomes (4)
Assessment of patient's behavioral condition using the Child Behavior Check-list (CBCL) 1,5-5
Measured: 2 Years
Assessment of the patient's body consciousness through the "CORP-R" test (french test of body mapping)
Measured: 2 years
Assessment of patient's quality of life using the "Kidscreen" questionnaire for children
Measured: 2 years
Assessment of the patient's parents' Qaulity of life through the Short Form Health Survey (SF-36)
Measured: 2 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jul 2016 (estimated)
- Study startApr 2014
- Primary completionApr 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Forms of HemophiliaStudies 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique Hopitaux De Marseille
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Urielle DESALBRES, Study director, Assistance Publique Hôpitaux de Marseille
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02834130
- Sponsor's protocol ID
- 2014-05
- Other IDs
- 2014-A00367-40
- Acronym
- ACADHEM
- Status last verified
- Jul 2016
- First submitted
- 21 Oct 2015
- Last updated
- 19 Jul 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Input of a Multidisciplinary Device Including a Psychological Approach, to Accompany the Announce of Diagnosis for Young Children With Serious Constitutional Hemorrhagic Disease and Their Families
- ClinicalTrials.gov ID
- NCT02834130
- Acronym / sponsor ID
- ACADHEM / 2014-05
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Assistance Publique Hopitaux De MarseilleUniversity, hospital or other
- Study start
- Apr 2014
- Primary completion
- Apr 2016 (actual)
- First posted
- 15 Jul 2016 (estimated)
- 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.
Severe forms of hemophilia and other constitutional bleeding disorders represent a group of rare diseases. In last decades, news therapies have dramatically increased life expectancy and joint's protection.
The investigators propose to set up a single-centre, descriptive, transversal pilot study, designed for the inclusion of around thirty children from 2 to 10 years, with haemophilia or allied hereditary bleeding disorders (HBD), who were referred to the Hemophilia Treatment Center (HTC) of Marseille at diagnosis. For those children who were able to benefit from the totality or part of the device accompanying the diagnostic announcement in the past 10 years.
Detailed description
Severe forms of hemophilia and other constitutional bleeding disorders represent a group of rare diseases. In last decades, news therapies have dramatically increased life expectancy and joint's protection. The recurrence of joint bleedings is responsive of multifocal arthropathy in the natural history, but nowadays, the orthopedic outcome is good thanks to prophylactic treatments.
However, the announcement of the diagnosis of such hereditary bleeding disorders at a very young age represents a real psychological trauma for parents. Such an announcement may induce some psycho-developmental impairment.
An innovative support has been implemented at the Hemophilia Treatment Center in Marseille, in conjunction with the French Hemophilia Society. This program is based on a multidisciplinary partnership that favors the psychological support and the reinsurance of the parents.
With Rare Diseases Foundation support, the investigators have initiated this pilot study that will include more than 20 patients with severe hemophilia or constitutional bleeding disorder, who benefit from the device. This descriptive, monocentric, interventional study will permit to describe the psychological status, the quality of life of patients and their parents, but also to evaluate the impact of the device on initiation and observance of treatments like Long Term Prophylaxis.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| children from 2 to 10 years with haemophilia or allied HBD, wh | Other | Behavioral: Evaluation of psychological and psychopathological |
Who may qualify
- Ages
- 3 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Patients aged between 3 and 12 years
- Patients diagnosed and treated at the Center until inclusion
- Patients with FVIII :C < 2% or with FIX :C <2% or with FVII:C <2% or with FXIII:C <2% or fibrinogen <0,2 g/l, or with type 3 von Willebrand diseases or Thrombasthenia of Glanzmann diseases.
- Patient who participate to multidisciplinary support and announcement of diagnosis device
- Voluntarily given fully informed written and signed consents obtained before any study-related procedures are conducted
Key exclusion criteria (4 listed)
- Patient with other clotting disorder or disease.
- Patient diagnosed or treated in other centre
- Patient with psychiatric disorder or disease
- Patient with other severe chronic disease
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 |
|---|---|---|
| Evaluation of psychological and psychopathologicalAlso: Evaluation of quality of life | Behavioural | children from 2 to 10 years with haemophilia or allied HBD, wh |
Primary outcome
Number of Treatment Exposure Days compared to the Number of Treatment Exposure days prescribed
Time frame: 2 years
Treatment Observance
4 secondary outcomes
Assessment of patient's behavioral condition using the Child Behavior Check-list (CBCL) 1,5-5
Time frame: 2 Years
Assessment of the patient's body consciousness through the "CORP-R" test (french test of body mapping)
Time frame: 2 years
Assessment of patient's quality of life using the "Kidscreen" questionnaire for children
Time frame: 2 years
Assessment of the patient's parents' Qaulity of life through the Short Form Health Survey (SF-36)
Time frame: 2 Years
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 19 Jul 2016.
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.
Urielle DESALBRES study director
Assistance Publique Hôpitaux de Marseille
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Evaluation of psychological and psychopathological in people with Severe Forms of Hemophilia and Other Constitutional Bleeding Disorders, run by Assistance Publique Hopitaux De Marseille 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 5 inclusion and 4 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 19 Jul 2016. 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?
Number of Treatment Exposure Days compared to the Number of Treatment Exposure days prescribed (time frame: 2 years). 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 19 Jul 2016.