RecruitingNCT07461519

Gonadic Function and Pubertal Development in Female Patients With Classic Galactosemia


Sponsor

IRCCS Azienda Ospedaliero-Universitaria di Bologna

Enrollment

60 participants

Start Date

Feb 12, 2025

Study Type

OBSERVATIONAL

Conditions

Summary

Newborn screening and a galactose-free diet have improved early outcomes in galactosemia, but long-term issues such as primary ovarian insufficiency (POI) remain significant. This study aims to clarify clinical, hormonal, developmental, and fertility-related factors in affected girls through a large multicenter Italian cohort.


Eligibility

Sex: FEMALEMax Age: 45 Years

Inclusion Criteria2

  • Female patients followed at participating Pediatric Endocrinology and Inherited Metabolic Disease Centers, born between January 1st, 1980 and December 31st, 2024, with a diagnosis of classic galactosemia and confirmed by genetic testing.
  • Obtaining informed consent.

Exclusion Criteria2

  • Patients with known chromosomal abnormalities (e.g., trisomy 21, Turner syndrome, Fragile X syndrome, Kabuki syndrome, or other genetic conditions associated with pubertal disorders);
  • Patients with primary ovarian insufficiency due to causes other than galactosemia.

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Locations(19)

SSD Nutrizione Clinica e Metabolismo - IRCCS Azienda Ospedaliero-Universitaria di Bologna

Bologna, Italy, Italy

UOC Pediatria - IRCCS Azienda Ospedaliero-Universitaria di Bologna

Bologna, Italy, Italy

Ospedale Pediatrico "Giovanni XXIII"

Bari, Italy

Ospedale Pediatrico Microcitemico "A. Cao"

Cagliari, Italy

AOU Policlinico di Catania

Catania, Italy

AOU "Dulbecco", Policlinico Germaneto-Università di Catanzaro

Catanzaro, Italy

Azienda Ospedaliera Universitaria Meyer IRCCS

Florence, Italy

IRCCS Istituto G. Gaslini

Genova, Italy

AOU Policlinico G. Martino

Messina, Italy

ASST Santi Paolo e Carlo - Presidio San Paolo - Università degli studi di Milano

Milan, Italy

Ospedale dei Bambini "Vittore Buzzi"

Milan, Italy

Fondazione IRCCS San Gerardo dei Tintori

Monza, Italy

AORN Santobono Pausilipon

Naples, Italy

Azienda Ospedaliera Universitaria "Federico II"

Naples, Italy

Azienda Ospedale-Università di Padova

Padova, Italy

Ospedale dei Bambini di Palermo

Palermo, Italy

Ospedale Guglielmo da Saliceto

Piacenza, Italy

Ospedale Pediatrico Bambin Gesù IRCCS

Roma, Italy

Ospedale Infantile Regina Margherita, AOU Città della Salute e della Scienza di Torino

Torino, Italy

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NCT07461519


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