Genetic/Metabolic Liver Diseases

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Genetic/Metabolic Liver Diseases trials you may qualify for

100

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 39 trials

RecruitingPhase 2

NCT05519475

A Precision Medicine Approach Using Gene Silencing to Treat a Chronic Liver Disease Called Metabolic Dysfunction-Associa…

This study is researching an investigational drug, ALN-HSD called "study drug". This study is focused on participants who are known to have Metabolic dysfunctio…

United States120 participantsStarted 2023-02-09

Treatment: ALN-HSD, Placebo

RecruitingNot Applicable

NCT07567131

Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)

The purpose of this study is to collect information about the effects of EPP/XLP in adults and adolescents. This is an observational study in which participants…

United States50 participantsStarted 2026-04-30

RecruitingNot Applicable

NCT02012699

Integrated Cancer Repository for Cancer Research

The iCaRe2 is a multi-institutional resource created and maintained by the Fred \& Pamela Buffett Cancer Center to collect and manage standardized, multi-dimens…

United States999,999 participantsStarted 2013-11-01

RecruitingPhase 2

NCT07123155

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an…

United States45 participantsStarted 2025-10-30

Treatment: S-606001, Placebo

RecruitingNot Applicable

NCT04883905

ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

This global patient registry is being conducted to characterize the natural history and real-world clinical management of patients with AHP, and to further char…

United States150 participantsStarted 2021-04-26

RecruitingPhase 1/2

NCT06255782

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzym…

United States20 participantsStarted 2024-04-08

Treatment: ECUR-506

All recent trials (100 shown)

Active — Not RecruitingPhase 3

NCT06144840

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP

To investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunli…

United States165 participantsStarted 2023-12-11

Treatment: Dersimelagon, Placebo

Active — Not RecruitingPhase 3

NCT06910358

Study of Bitopertin in Participants With EPP or XLP (APOLLO)

The goal of this clinical trial is to learn if bitopertin works and is safe to treat EPP or XLP in participants 12 years or older. The main questions it aims to…

United States183 participantsStarted 2025-04-04

Treatment: Placebo, DISC-1459

Genetic/Metabolic Liver Diseases

Trial phase breakdown

Trial status

Actively recruiting — 39 trials

A Precision Medicine Approach Using Gene Silencing to Treat a Chronic Liver Disease Called Metabolic Dysfunction-Associa…

Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)

Integrated Cancer Repository for Cancer Research

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

All recent trials (100 shown)

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP

Study of Bitopertin in Participants With EPP or XLP (APOLLO)

Trial phase breakdown

Trial status

Actively recruiting — 39 trials

A Precision Medicine Approach Using Gene Silencing to Treat a Chronic Liver Disease Called Metabolic Dysfunction-Associa…

Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)

Integrated Cancer Repository for Cancer Research

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

All recent trials (100 shown)

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP

Study of Bitopertin in Participants With EPP or XLP (APOLLO)

Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.

Determine the Prevalence of Exocrine Pancreatic Insufficiency (EPI) in Pediatric and Adult Participants With Alagille Sy…

Liver Disease in Urea Cycle Disorders

Patient Preference Study: Standard of Care Versus Once-daily Trientine Tetrahydrochloride

Real World Evidence Study in Subjects With Wilson's Disease

Atorvastatin Therapy on Xanthoma in Alagille Syndrome

Pharmacokinetics of Afamelanotide in Erythropoietic Protoporphyria Patients

Structured Physical Activity Intervention in Patients With MASLD Receiving a Mediterranean Diet

A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

Exchangeable and Relative Exchangeable Copper as an Alternative to 24-Hour Urinary Copper in Wilson's Disease Monitoring

Quantitative Susceptibility Mapping (QSM) to Guide Iron Chelating Therapy

Diagnosis and Treatment of Patients With Cystic Fibrosis

Panhematin for Prevention of Acute Attacks of Porphyria

A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease

Effect of Bitopertin on the Liver and on Levels of Protoporphyrin IX in Bile, Blood, Liver, and Stool in Patients With E…

Prevalence of Acute Hepatic Porphyria

Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome

Effect of Oral Cimetidine in the Protoporphyrias