Ovarian Function and Gynecological Profile of Patients Carrying a Pathogenic Variant of the HNF1B Gene (GYN-HNF1)

NCT ID: NCT04746053

Last Updated: 2024-02-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

26 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-04-12

Study Completion Date

2023-10-12

Brief Summary

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The main objective of this study is to study the ovarian function of patients with hepatocyte nuclear factor-1beta (HNF1B) abnormality, followed in the reference centers of Toulouse and Paris University Hospitals. The secondary objectives are to know the gynecological profile of these patients.

A dosage of anti-mullerian hormone (AMH) will be added to the usual balance, whatever the phase of the cycle and a pelvic ultrasound will be performed by the same operator by center. Patients will be received in a dedicated consultation to complete a questionnaire on gynecological and obstetric history, as well as their personal and family history. There will be no gynecological examination during this consultation.

Detailed Description

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HNF1B mutations can be responsible for genital malformations associated with renal development abnormalities. HNF1b is also a candidate gene involved in the development of the renal tract and the Mullerian system but there is no data in the literature regarding the ovarian function and clinical gynecological profile of these patients.

The main objective of this study is to study the ovarian function of patients with HNF1B abnormality, followed in the reference centers of Toulouse and Paris University Hospitals. The secondary objectives are to know the gynecological profile of these patients.

A dosage of AMH will be added to the usual balance, whatever the phase of the cycle and a pelvic ultrasound will be performed. Patients will be received in a dedicated consultation to complete a questionnaire on gynecological and obstetric history, as well as their personal and family history. There will be no gynecological examination during this consultation.

Conditions

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Genital Diseases, Female

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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patient with a mutation in the HNF1B gene

Patient with a mutation in the HNF1B gene and which are followed in the reference centers

Group Type OTHER

Pelvic ultrasound

Intervention Type OTHER

Pelvic or vaginal ultrasound

blood sample

Intervention Type BIOLOGICAL

blood sample (5ml)

Interventions

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Pelvic ultrasound

Pelvic or vaginal ultrasound

Intervention Type OTHER

blood sample

blood sample (5ml)

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Girls from 12 years of age carrying a pathogenic variant of the HNF1 B gene and monitored in the reference center for rare renal diseases
* Person affiliated or beneficiary of a social security scheme.
* Free, informed and written consent signed by the participant or her legal representative if a minor patient and the investigator (at the latest on the day of inclusion and before any examination required by the research).

Exclusion Criteria

* Woman unable to answer
* Pregnant or breastfeeding woman
* Refusal of participation by the patient or her legal representative
* Person under a protection system for adults (such as safeguard of justice, guardianship or curatorship)
* History of chemotherapy, radiotherapy, brachytherapy
* History of ovarian endometrium
Minimum Eligible Age

12 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No

Sponsors

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University Hospital, Toulouse

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Catherine PIENKOWSKI, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Toulouse

Locations

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Pitié Salpêtrière Hospital -

Paris, , France

Site Status

Necker Hospital

Paris, , France

Site Status

Toulouse University Hospital

Toulouse, , France

Site Status

Countries

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France

References

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Edghill EL, Bingham C, Ellard S, Hattersley AT. Mutations in hepatocyte nuclear factor-1beta and their related phenotypes. J Med Genet. 2006 Jan;43(1):84-90. doi: 10.1136/jmg.2005.032854. Epub 2005 Jun 1.

Reference Type BACKGROUND
PMID: 15930087 (View on PubMed)

Lindner TH, Njolstad PR, Horikawa Y, Bostad L, Bell GI, Sovik O. A novel syndrome of diabetes mellitus, renal dysfunction and genital malformation associated with a partial deletion of the pseudo-POU domain of hepatocyte nuclear factor-1beta. Hum Mol Genet. 1999 Oct;8(11):2001-8. doi: 10.1093/hmg/8.11.2001.

Reference Type BACKGROUND
PMID: 10484768 (View on PubMed)

Oram RA, Edghill EL, Blackman J, Taylor MJ, Kay T, Flanagan SE, Ismail-Pratt I, Creighton SM, Ellard S, Hattersley AT, Bingham C. Mutations in the hepatocyte nuclear factor-1beta (HNF1B) gene are common with combined uterine and renal malformations but are not found with isolated uterine malformations. Am J Obstet Gynecol. 2010 Oct;203(4):364.e1-5. doi: 10.1016/j.ajog.2010.05.022. Epub 2010 Jul 15.

Reference Type BACKGROUND
PMID: 20633866 (View on PubMed)

Other Identifiers

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2020-A02800-39

Identifier Type: OTHER

Identifier Source: secondary_id

RC31/20/0124

Identifier Type: -

Identifier Source: org_study_id

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