Rare DiseasesRaroDentAXE3NCT05070988
Oral Health Related Quality of Life of Patients With Rare Diseases: a Qualitative Approach
An observational study of Rare Diseases, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
The main objective of the study is to assess the oral health-related quality of life of patients with rare diseases and followed in the rare disease centers of expertise at Necker Hospital in Paris by semi-structured interviews. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Rare Diseases
- What is being tested?
- Semi-structured interview
- How many people
- 28 participants
- Where
- 1 siteFrance
- Study dates
- 20 Oct 2021to 29 Nov 2021
- 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The study population is that of the centers of expertise for rare diseases at the Necker Hospital in Paris, France.
Who may be able to join
- Minor and adult patients with rare diseases aged of at least 6 years old and followed at the Necker Enfant Malades Hospital, Paris
- Patients with the 3 most frequent rare diseases of each rare disease Necker reference center participating in the study (10 centers : Chronic And Malformative Disorders Of The Esophagus / Inflammatory diseases of the bile ducts and autoimmune hepatitis / Constitutional Bone Diseases / Inherited Metabolic Diseases / Biliary Atresia And Genetic Cholestases / Vascular Diseases Of The Liver / Developmental Anomalies and Malformative Syndromes of Ile-De-France / Rare Causes Intellectual Disabilities / Complex congenital heart defects / Childhood cerebrovascular pathologies)
- Patients who consulted between 1.1.2017 and 1.1.2020 the relevant rare disease reference center at Necker hospital
- Patients seen at least once in the medical genetics department of Necker Hospital
- Information and non-opposition of adult patients / holders of parental authority of minor patients and minor patients to participate in the study
Who cannot join
- Patients who do not speak French
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Oral Health Related Quality of Life of Patients With Rare Diseases Followed at Necker Hospital: a Qualitative Approach
The main objective of the study is to assess the oral health-related quality of life of patients with rare diseases and followed in the rare disease centers of expertise at Necker Hospital in Paris by semi-structured interviews.
Read the detailed description
To date, few studies have used the qualitative method to analyze the oral health-related quality of life and the oral health care pathways of patients with rare diseases, all health sectors combined. The quantitative studies carried out using standardized questionnaires offer only a partial view of how patients feel, both children and adults.
The main objective of the study is to assess the oral health-related quality of life of patients with rare diseases and followed in the rare disease centers of expertise at Necker Hospital in Paris by means of a semi-structured interview.
- Conditions
- Rare Diseases
- Being tested
- Semi-structured interview
- Keywords
- Rare diseases, Oral health-related quality of life, Qualitative method
- Medical subject headings
- Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Semi-structured interviewOther
Semi-structured interview with the investigating doctor of the study lasting 30 minutes to an hour
| Study group | Role | What they receive |
|---|---|---|
| Rare diseases | Children and adult patients with rare diseases and followed at the Necker Enfant Malades Hospital, Paris Receives: Other: Semi-structured interview |
What is being measured
The primary outcome is the main question the study is designed to answer.
Oral health-related quality of life assessment
Qualitative method from a semi-structured interview.
Measured: Day 0
Secondary outcomes (2)
Description of oral health care pathways
Description from the semi-structured interviews.
Measured: Day 0
Inequalities in access to oral health care
Description of inequalities in access to oral health care. Description from the semi-structured interviews.
Measured: Day 0
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2021
- Study start20 Oct 2021
- Primary completion29 Nov 2021
- Study completion29 Nov 2021
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 Rare DiseasesStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Responsible party
- Sponsor
- Investigators
- Lisa Friedlander, DDS, PHD, associate professor, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05070988
- Sponsor's protocol ID
- APHP210890
- Other IDs
- 2021-A00429-32
- Acronym
- RaroDentAXE3
- Status last verified
- Sep 2025
- First submitted
- 27 Sep 2021
- Last updated
- 12 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Friedlander L, Berdal A, Cormier-Daire V, Lyonnet S, Garcelon N. Determinants of dental care use in patients with rare diseases: a qualitative exploration. BMC Oral Health. 2023 Jun 22;23(1):413. doi: 10.1186/s12903-023-03048-1.
PubMed 37349716
Study at a glance
From the registry record, last updated by the sponsor on 12 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Oral Health Related Quality of Life of Patients With Rare Diseases Followed at Necker Hospital: a Qualitative Approach
- ClinicalTrials.gov ID
- NCT05070988
- Acronym / sponsor ID
- RaroDentAXE3 / APHP210890
- Conditions
- Rare Diseases
- Keywords
- Rare diseases, Oral health-related quality of life, Qualitative method
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Study start
- 20 Oct 2021 (actual)
- Primary completion
- 29 Nov 2021 (actual)
- Study completion
- 29 Nov 2021 (actual)
- First posted
- 7 Oct 2021 (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.
The main objective of the study is to assess the oral health-related quality of life of patients with rare diseases and followed in the rare disease centers of expertise at Necker Hospital in Paris by semi-structured interviews.
Detailed description
To date, few studies have used the qualitative method to analyze the oral health-related quality of life and the oral health care pathways of patients with rare diseases, all health sectors combined. The quantitative studies carried out using standardized questionnaires offer only a partial view of how patients feel, both children and adults.
The main objective of the study is to assess the oral health-related quality of life of patients with rare diseases and followed in the rare disease centers of expertise at Necker Hospital in Paris by means of a semi-structured interview.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Rare diseases | – | Other: Semi-structured interviewChildren and adult patients with rare diseases and followed at the Necker Enfant Malades Hospital, Paris |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study population is that of the centers of expertise for rare diseases at the Necker Hospital in Paris, France.
Key inclusion criteria (5 listed)
- Minor and adult patients with rare diseases aged of at least 6 years old and followed at the Necker Enfant Malades Hospital, Paris
- Patients with the 3 most frequent rare diseases of each rare disease Necker reference center participating in the study (10 centers : Chronic And Malformative Disorders Of The Esophagus / Inflammatory diseases of the bile ducts and autoimmune hepatitis / Constitutional Bone Diseases / Inherited Metabolic Diseases / Biliary Atresia And Genetic Cholestases / Vascular Diseases Of The Liver / Developmental Anomalies and Malformative Syndromes of Ile-De-France / Rare Causes Intellectual Disabilities / Complex congenital heart defects / Childhood cerebrovascular pathologies)
- Patients who consulted between 1.1.2017 and 1.1.2020 the relevant rare disease reference center at Necker hospital
- Patients seen at least once in the medical genetics department of Necker Hospital
- Information and non-opposition of adult patients / holders of parental authority of minor patients and minor patients to participate in the study
Key exclusion criteria (1 listed)
- Patients who do not speak French
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 |
|---|---|---|
| Semi-structured interviewSemi-structured interview with the investigating doctor of the study lasting 30 minutes to an hour | Other | Rare diseases |
Primary outcome
Oral health-related quality of life assessment
Time frame: Day 0
Qualitative method from a semi-structured interview.
2 secondary outcomes
Description of oral health care pathways
Time frame: Day 0
Inequalities in access to oral health care
Time frame: Day 0
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 12 Sep 2025.
- Results publication: PubMed 37349716Friedlander L, Berdal A, Cormier-Daire V, Lyonnet S, Garcelon N. Determinants of dental care use in patients with rare diseases: a qualitative exploration. BMC Oral Health. 2023 Jun 22;23(1):413. doi: 10.1186/s12903-023-03048-1.
- 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.
Lisa Friedlander, DDS, PHD, associate professor principal investigator
Assistance Publique - Hôpitaux de Paris
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 Rare Diseases, run by Assistance Publique - Hôpitaux de Paris 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 1 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 12 Sep 2025. 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?
Oral health-related quality of life assessment (time frame: Day 0). 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 NCT05070988, 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 12 Sep 2025.