15N ammonium chloride ureagenesis validation clinical trial

Phase 1

• Conditions: Subject has a genetically confirmed diagnosis of any of the followingurea cycle disorders: ASS, CPS1, ASL, OTCSubjects without UCD can have other stable illness that not interferewith 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-IT
• Lead Sponsor: nicyte AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-08-16
• Last Update Posted: 12 March 2024

Recruitment & Eligibility

• Status: ot Recruiting
• 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 subj
Are the trial subjects under 18? yes
Number of subjects for this age range:
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