Oral Health Related Quality of Life of Patients With Rare... | NCT05070988

Studies
Oral Health Related Quality of Life of Patients With Rare Diseases: a Qualitative Approach

NCT05070988

Sponsor

Assistance Publique - Hôpitaux de Paris

Status

Completed

Last Update Posted

Sep 12, 2025Actual

Enrollment

28Actual

Phase

Not provided

Conditions

Rare Diseases

Interventions

Semi-structured interview

Countries

France

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
2021-A00429-32	Other Identifier	ID RCB number

Status Module

Record Verification Date

Sep 2025

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Oct 20, 2021Actual

Primary Completion Date

Nov 29, 2021Actual

Completion Date

Nov 29, 2021Actual

First Submitted Date

Sep 27, 2021

First Submission Date that Met QC Criteria

Sep 27, 2021

First Posted Date

Oct 7, 2021Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Sep 5, 2025

Last Update Posted Date

Sep 12, 2025Actual

Name	Class
URC-CIC Paris Descartes Necker Cochin	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief 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.

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

28Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Rare diseases		Children and adult patients with rare diseases and followed at the Necker Enfant Malades Hospital, Paris	Other: Semi-structured interview

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Semi-structured interview	Other	Semi-structured interview with the investigating doctor of the study lasting 30 minutes to an hour	Rare diseases

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Oral health-related quality of life assessment	Qualitative method from a semi-structured interview.	Day 0

Secondary Outcomes

Measure	Description	Time Frame
Description of oral health care pathways	Description from the semi-structured interviews.	Day 0
Inequalities in access to oral health care	Description of inequalities in access to oral health care. Description from the semi-structured interviews.

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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

Exclusion Criteria:

Patients who do not speak French

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

The study population is that of the centers of expertise for rare diseases at the Necker Hospital in Paris, France.

Sampling Method

Non-Probability Sample

Name	Affiliation	Role
Lisa Friedlander, DDS, PHD, associate professor	Assistance Publique - Hôpitaux de Paris	Principal Investigator

Facility	Status	City	State	ZIP	Country	Contacts
Hôpital Necker-Enfants Malades		Paris		75015	France

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
37349716	Result	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.

ID	Term
D020969	Disease Attributes
D010335	Pathologic Processes
D013568	Pathological Conditions, Signs and Symptoms