Los requisitos de elegibilidad de los participantes incluyen la edad, el sexo, el tipo y el estadio de la enfermedad, y los problemas de salud o tratamientos previos. Las pautas difieren de un estudio a otro e identifican quiénes pueden o no pueden participar. No hay garantía de que cada persona elegible que desee participar en un ensayo se inscribirá. Comunícate con el equipo del estudio para analizar la elegibilidad del estudio y la posible participación.
Criteria for Inclusion
Subjects must meet all of the following criteria to be eligible for participation in this
study.
1. Male or female, of the following age groups at the time of obtaining informed
consent:
a. Cohort A: Age ≥ 12 years
b. Cohort B: Age 6 – 11.9 years
2. Diagnosis of PH1, confirmed by genotyping for mutations in the AGXT gene.
3. Urine oxalate excretion ≥0.7 mmol per 1.73 m2 body surface area (BSA) in
24 hours.
4. Estimated glomerular filtration rate (eGFR) ≥40 mL/min per 1.73 m2 BSA.
5. Adults must be able to understand and give written informed consent for
participation in this study, including all evaluations and procedures as
specified by this protocol. Minors (subjects <18 years of age, or younger
than the age of majority, according to local regulations) must have a parent or
guardian who is able to understand and give written informed consent for
participation in this study, including all evaluations and procedures as
specified by this protocol. Adolescents (12-18 years of age, or older than
12 years but younger than the age of majority, according to local regulations)
must be able to provide written assent for participation. For children
<12 years of age assent may be obtained from the patient based on local
regulations or institutional standards.
Criteria for Exclusion
Subjects meeting any of the following criteria will be excluded from this study.
1. Prior renal and/or hepatic transplantation, or patients undergoing dialysis.
2. Participation in any clinical study involving administration of any
investigational drug for the treatment of PH1 within 30 days before
screening. (NOTE: medications used in the routine care of PH1 patients such
as vitamin B-6, crystal inhibitors such as citrate or phosphate, or medications
used for the treatment of acute stone episodes such as tamsulosin are allowed)
3. Pregnancy or lactation at the time of screening or enrollment.
4. Any disorder or alteration in mental status that would preclude understanding
of the informed consent process and/or completion of the study-related
evaluations.
5. Any significant illness, organ system dysfunction, or other condition that, in
the opinion of the Investigator, would interfere with the subject’s ability to
comply with the protocol requirements, including the ability to attend all
visits and undergo all assessments.