Health Trials

Metabolism, Inborn Errors

2

Review clinical trials related to Metabolism, Inborn Errors. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

A Phase 3 Study of NTLA-2001 in ATTRv-PN

This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.

Participants needed: 60
Trial details
Phase: Phase 3Age: 18-85Biological sex: AllType: InterventionalSponsor: Intellia TherapeuticsUpdated: Apr 16, 2026Locations: 14
Eligibility criteria

Diagnosis of ATTRv-PN [+1]

Other causes of amyloidosis (amyloidosis caused by non-TTR protein) [+9]

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Status: Recruiting

Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria

The overall aim of this study is to evaluate LNAA treatment as a potential alternative to conventional dietary treatment for PKU. This study investigates the effects of LNAA treatment compared to the classic dietary treatment on cerebral dopamine synthesis in patients with classic PKU. We will assess LNAAs effectiveness on neurotransmitter synthesis, cognitive function, mental health, and safety, compared to the standard diet.

Participants needed: 30
Trial details
Age: 18-50Biological sex: AllType: InterventionalSponsor: Rigshospitalet, DenmarkUpdated: Apr 6, 2025Locations: 2
Eligibility criteria

Treatment initiation within the first month of life [+4]

Unable or unwilling to adhere to the requirements of the study [+7]

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