Clinical trial assessing urea formation capacity in babies up to 12 months old using 15N ammonium chloride tracer

Phase 1

• Conditions: Subject has a genetically confirmed diagnosis of any of the following urea cycle disorders: ASS, CPS1, ASL, OTC Subjects without UCD can have other stable illness that not interfere with the clinical trial according to the investigator judgement; MedDRA version: 20.1Level: PTClassification code 10080020Term: Urea cycle disorderSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2021-000824-36-AT
• Lead Sponsor: nicyte AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-07-29
• Last Update Posted: 16 May 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

A subject must meet ALL of the following criteria at Screening to be eligible for this trial, with the exception of criteria 1 and 2 which do not apply to subjects without UCD and criterium 3 that only applies for subjects without UCD:
Only for subjects with UCD
1.Subject has a genetically confirmed diagnosis of any of the following urea cycle disorders: ASS, CPS1, ASL, OTC. Note: All subjects should have genotyping information available, however if an exact genetic diagnosis is not available, diagnosis of the UCD sub-type may be confirmed by well accepted biochemical parameters
2.Subject has neonatal or infantile onset of UCD signs and symptoms within the first 12 months of life; or subjects who have a family history of UCD and are asymptomatic after birth due to a therapeutic regimen started directly after birth;
Only for subjects without UCD
3.Subjects without UCD can have other stable illness that does not interfere with the clinical trial according to the investigator judgement;
For all subjects (with and without UCD)
4.Male and female subjects aged up to 12 months, inclusive;
5.Subject has a body weight within the 5-95 percentile of the corresponding age according to the WHO Child Growth Standards 2006;
6.Subject has stable clinical conditions (any acute condition needs to be stabilised/treated before inclusion);
7.The parent(s) / legal representative(s) agrees that the subject will not participate in any interventional clinical trial with an investigational drug suspected of having an interaction with the urea cycle or 15NH4Cl diagnostic tracer for the duration of the trial until the final follow-up telephone call;
8.Ability and willingness of the parent(s) / legal representative(s) to comply with the protocol requirements, including ability to bring the subject to the scheduled trial visits;
9.Written informed consent by the parent(s) / legal representative(s) of the subject.

Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

A subject must meet NONE of the following criteria at Screening to be eligible for this trial, with the exception of criterion 1 which does not apply to subjects with UCD:
Only for subjects with UCD
1.Subject has any suspected UCD of any sub-type. Note: subjects suspected of having a UCD of any sub-type, but without either confirmatory genotyping information or a typical biochemical diagnostic pattern for any UCD gene defect, will not be enrolled in this trial.
For all subjects (with and without UCD)
2.Subject is a premature neonate (up to 37 gestation weeks not completed);
3.Subject is in a period of significant post-natal weight drop based on the judgement of the investigator ;
4.Subject has received any investigational compound within 30 days (or 5 half-lives, whichever is longer) prior to first dose of diagnostic tracer and according to the investigator judgement could interfere with the clinical trial;
5.Subject has any other acute severe / other genetic / life limiting disorder that would interfere with ethical and/or medical standards in the conduct or follow up of the trial.
6.Subject has acute liver failure, clinical or radiological evidence of liver fibrosis or cirrhosis, or presents a hepatic or extrahepatic malignancy.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified