NCT01746121

Brief Summary

Amelogenesis Imperfecta (AI) are a heterogeneous group of rare genetic diseases transmitted according to various mode of inheritance (X-linked, autosomal dominant, autosomal recessive) affecting the formation/mineralization of tooth enamel. These diseases exist in isolation with clinical manifestations limited to the oral cavity or may be associated to other symptoms in syndromes. Many different genes (AMELX, ENAM, ENAMELYSIN or MMP20, KLK4, DLX3, FAM83H, FAM20A WDR72…) coding for enamel matrix proteins, enamel matrix degrading proteins, proteins involved in hydroxyapatite formation and growth and mineralization processes have been discovered responsible for the clinical phenotypes (hypoplastic, hypomineralized, hypomature) encountered in AI. Genes involved in enamel formation but not yet identified in association with any form of AI include: AMELY, AMELOBLASTIN, TUFTELIN, AMELOTIN, A Pin protein, ODAM (Odontogenic ameloblast associated). In this research protocol the investigators explore the phenotype including the enamel ultrastructure and the genotype of a cohort of patients presenting AI.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
600

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Nov 2009

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2009

Completed
3 years until next milestone

First Submitted

Initial submission to the registry

October 22, 2012

Completed
2 months until next milestone

First Posted

Study publicly available on registry

December 10, 2012

Completed
3 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2013

Completed
2.8 years until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2016

Completed
Last Updated

July 11, 2018

Status Verified

February 1, 2013

Enrollment Period

3.3 years

First QC Date

October 22, 2012

Last Update Submit

July 10, 2018

Conditions

Outcome Measures

Primary Outcomes (1)

  • Natural history of Amelogenesis Imperfecta

    Familial, medical, dental history

    at day of enrollment

Secondary Outcomes (1)

  • Phenotype of Amelogenesis Imperfecta

    at day of enrollment

Other Outcomes (2)

  • Genetic Bases of Amelogenesis Imperfecta

    within 3 years after enrollment

  • Ultrastructure of teeth hard tissues

    within 3 years after enrollment

Study Arms (2)

Amelogenesis Imperfecta

Salivary and blood sampling, as part of routine care. Collection of exfoliated teeth

Genetic: Salivary and blood sampling, as part of routine care. Collection of exfoliated teeth.

healthy family members

Salivary and blood sampling, as part of routine care. Collection of exfoliated teeth

Genetic: Salivary and blood sampling, as part of routine care. Collection of exfoliated teeth.

Interventions

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

The recruitment of patients comes from several sources. A first cohort of 40 families is already followed by the reference center of Strasbourg. The reference center for rare diseases dental manifestations of Strasbourg recruiting patients at regional, interregional, national and international scale.

You may qualify if:

  • Patient presenting with AI
  • New patient or patient already known in the center
  • Child (in his primary dentition) or adult
  • Man or woman
  • Having signed a consent form or accepted to participate to the study
  • Patient affiliated to social security

You may not qualify if:

  • Patient with acquired enamel defects
  • Patient whose clinical diagnostic is not possible
  • Patient whose clinical file does not contain teeth photos
  • Patient who has not signed a consent form and accepted to participate to the study
  • Patient who is not affiliated to social security.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hôpitaux Universitaires de Strasbourg

Strasbourg, Alsace, 67091, France

Location

Related Publications (2)

  • Bloch-Zupan A, Rey T, Jimenez-Armijo A, Kawczynski M, Kharouf N; O-Rare consortium; Dure-Molla M, Noirrit E, Hernandez M, Joseph-Beaudin C, Lopez S, Tardieu C, Thivichon-Prince B; ERN Cranio Consortium; Dostalova T, Macek M Jr; International Consortium; Alloussi ME, Qebibo L, Morkmued S, Pungchanchaikul P, Orellana BU, Maniere MC, Gerard B, Bugueno IM, Laugel-Haushalter V. Amelogenesis imperfecta: Next-generation sequencing sheds light on Witkop's classification. Front Physiol. 2023 May 9;14:1130175. doi: 10.3389/fphys.2023.1130175. eCollection 2023.

  • Prasad MK, Geoffroy V, Vicaire S, Jost B, Dumas M, Le Gras S, Switala M, Gasse B, Laugel-Haushalter V, Paschaki M, Leheup B, Droz D, Dalstein A, Loing A, Grollemund B, Muller-Bolla M, Lopez-Cazaux S, Minoux M, Jung S, Obry F, Vogt V, Davideau JL, Davit-Beal T, Kaiser AS, Moog U, Richard B, Morrier JJ, Duprez JP, Odent S, Bailleul-Forestier I, Rousset MM, Merametdijan L, Toutain A, Joseph C, Giuliano F, Dahlet JC, Courval A, El Alloussi M, Laouina S, Soskin S, Guffon N, Dieux A, Doray B, Feierabend S, Ginglinger E, Fournier B, de la Dure Molla M, Alembik Y, Tardieu C, Clauss F, Berdal A, Stoetzel C, Maniere MC, Dollfus H, Bloch-Zupan A. A targeted next-generation sequencing assay for the molecular diagnosis of genetic disorders with orodental involvement. J Med Genet. 2016 Feb;53(2):98-110. doi: 10.1136/jmedgenet-2015-103302. Epub 2015 Oct 26.

Biospecimen

Retention: SAMPLES WITH DNA

saliva

MeSH Terms

Conditions

Amelogenesis Imperfecta

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

Developmental Defects of EnamelTooth AbnormalitiesStomatognathic System AbnormalitiesStomatognathic DiseasesTooth DiseasesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Specimen HandlingClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisPuncturesSurgical Procedures, OperativeInvestigative Techniques

Study Officials

  • Bloch-Zupan Agnes, DChD, PhD, HDR, PU-PH

    University Hospital of Strasbourg

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
FAMILY BASED
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 22, 2012

First Posted

December 10, 2012

Study Start

November 1, 2009

Primary Completion

March 1, 2013

Study Completion

January 1, 2016

Last Updated

July 11, 2018

Record last verified: 2013-02

Locations