Status:

NOT_YET_RECRUITING

Identifying Biomarkers & Dysregulated Biological Pathways in Blood and Urine of Congenital Central Hypoventilation Syndrome (CCHS) Patients

Lead Sponsor:

Assistance Publique - Hôpitaux de Paris

Conditions:

Congenital Central Hypoventilation Syndrome (CCHS)

Eligibility:

All Genders

18+ years

Phase:

NA

Brief Summary

The CCHS study is a prospective, open-label, monocentric, interventional study with diagnostic and prognostic objectives, conducted in two phases. The first phase aims to identify biomarkers and dysre...

Eligibility Criteria

Inclusion

  • CCHS patients :
  • Age 18 years old or older;
  • Carry a polyA expansion mutation in PHOX2B;
  • Receive nocturnal mechanical ventilation;
  • Patients that are under the care and treatment in the CCHS center: Hôpital Universitaire Pitié-Salpêtrière
  • Written informed consent from the patient
  • Affiliated to The French social security except patient on AME (state medical aid)
  • Control group :
  • Age 18 years old or older.
  • Healthy with no major medical illnesses in the past year (such as diabetes, cancer, pregnancy, lungs disease).
  • Matched for sex, age (+/- 3 years), origin and BMI category with a CCHS patient
  • Written informed consent of the control
  • Affiliated to The French social security except patient on AME (state medical aid)

Exclusion

  • CCHS patients :
  • Age lower than 18 years old;
  • Pregnancy or breastfeeding
  • Patients with diaphragmatic (phrenic nerve) pacing;
  • Patients with late onset CCHS;
  • Patients that were diagnosed with a major medical illnesses/condition other than CCHS in the past year (such as diabetes, cancer, lungs disease, a sleep disorder, or pregnancy)
  • Patients that suffer from a sleep disorder such as insomnia, restless legs syndrome, nightmares
  • Patients who use medications that are likely to impair sleep structure
  • Individuals under guardianship, or permanently legally incompetent adults, under judicial protection, deprived of liberty, patients unable to express their consent.
  • Control group :
  • Age lower than 18 years old;
  • Pregnancy or breastfeeding
  • Controls that were diagnosed with a major medical illnesses/condition in the past year (such as diabetes, cancer, lungs disease, a sleep disorder, or pregnancy)
  • Controls that suffer from a sleep disorder such as insomnia, restless legs syndrome, nightmares
  • Controls who use medications that are likely to impair sleep structure.
  • Individuals under guardianship, or permanently legally incompetent adults, under judicial protection, deprived of liberty, patients unable to express their consent.

Key Trial Info

Start Date :

November 1 2025

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

December 2 2028

Estimated Enrollment :

40 Patients enrolled

Trial Details

Trial ID

NCT06997146

Start Date

November 1 2025

End Date

December 2 2028

Last Update

October 6 2025

Active Locations (1)

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Page 1 of 1 (1 locations)

1

Pitié-Salpêtrière Hospital

Paris, France, 75013