Clinical trials: Genetic Liver Disorder
- Condition Genetic Liver Disorder
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Alagille Syndrome (ALGS)
A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)
The main aim of the study is to check if TAK-625 improves symptoms of Alagille Syndrome (ALGS), side effect from the study treatment or TAK-625, and how much TAK-625 stays in their blood over time.
- Trial name
- NCT05543174
- Testing
- TAK-625
- Where
- 8 sites in Japan
- Volunteers
- 7 people
- Sponsor
- Takeda
Alagille Syndrome
A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
This study will collect information from patients with Alagille syndrome (ALGS) as they use odevixibat (Bylvay) in their daily lives.
- Trial name
- NCT06850038
- Where
- 10 sites in United States
- Volunteers
- 30 people
- Sponsor
- Ipsen
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Alagille Syndrome
A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
This study will collect information from patients with ALGS who are using odevixibat in their daily lives. Odevixibat is a medication that helps patients with ALGS, a rare disease that affects the liver and causes itching.
- Trial name
- NCT07585097
- Where
- 14 sites in France, Italy
- Volunteers
- 30 people
- Sponsor
- Ipsen
Wilson's Disease
Efficacy and Safety, Long-term Study of Zinc Acetate to Treat Wilson's Disease in Japan.
The purpose of this long-term study is to determine whether Zinc Acetate is effective and safe in the treatment of Wilson's disease among Japanese.
- Trial name
- NCT00212355
- Testing
- NPC-02
- Volunteers
- 37 people
- Sponsor
- Nobelpharma
Urea Cycle Disorders
Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders
Treatment with liver cell infusion for children with urea cycle disorders (UCD).
- Trial name
- NCT01195753
- Testing
- HHLivC
- Where
- 5 sites in United States, Canada
- Volunteers
- 10 people
- Sponsor
- Cytonet GmbH & Co. KG
Sickle Cell Disease
Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO).
The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis.
- Trial name
- NCT01090323
- Testing
- ICL670
- Where
- 39 sites in United States, Canada and 3 more
- Volunteers
- 185 people
- Sponsor
- Novartis Pharmaceuticals
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
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 sunlight exposure in adults and adolescents with EPP or XLP.
- Trial name
- INSPIRE (NCT06144840)
- Testing
- Dersimelagon
- Where
- 35 sites in United States, Australia and 9 more
- Volunteers
- 165 people
- Sponsor
- Tanabe Pharma America, Inc.
Alagille Syndrome
A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
An open-label, single-arm, multi-centre program providing treatment access to ALGS patients with cholestatic pruritus in the US.
- Trial name
- NCT04530994
- Testing
- maralixibat
- Where
- 14 sites in United States
- Sponsor
- Mirum Pharmaceuticals, Inc.
Alagille Syndrome
Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome
Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in Patients with Alagille Syndrome.
- Trial name
- ASSERT (NCT04674761)
- Testing
- Odevixibat
- Where
- 32 sites in United States, Belgium and 11 more
- Volunteers
- 52 people
- Sponsor
- Albireo
Urea Cycle Disorders, Carbamoylphosphate Synthetase I Deficiency
Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders
Urea cycle disorders are rare inherited diseases that generally have a poor outcome. In this study, neonates and infants with UCD will be included within the first 3 months of life and will be treated by repetitive application of…
- Conditions
- Urea Cycle Disorders, Carbamoylphosphate Synthetase I Deficiency, Ornithine Transcarbamylase Deficiency, Citrullinemia
- Trial name
- NCT00718627
- Testing
- Human Heterologous Liver Cells
- Where
- 2 sites in Germany
- Volunteers
- 12 people
- Sponsor
- Cytonet GmbH & Co. KG
Wilson's Disease
A Phase 1 Study to Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson's Disease Patients
To evaluate the effects of a single dose of trientine in children ≥6 years and adult patients with Wilson's disease.
- Trial name
- NCT01874028
- Testing
- trientine dihydrochloride
- Where
- Germany
- Volunteers
- 20 people
- Sponsor
- Univar BV
Alagille Syndrome
Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)
This is a long-term, open-label study with a double-blind, placebo-controlled, randomized drug withdrawal period in children with Alagille Syndrome (ALGS) designed to evaluate the safety and efficacy of LUM001 (Also known as…
- Trial name
- ICONIC (NCT02160782)
- Testing
- LUM001 (Maralixibat)
- Where
- 9 sites in Australia, Belgium and 4 more
- Volunteers
- 31 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Wilson Disease
Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months.
- Trial name
- NCT02426905
- Testing
- trientine dihydrochloride
- Where
- 4 sites in Germany, Greece and 2 more
- Volunteers
- 90 people
- Sponsor
- Univar BV
Erythropoietic Protoporphyria
HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.
- Trial name
- NCT05883748
- Testing
- DISC-1459
- Where
- 26 sites in United States, Australia and 9 more
- Volunteers
- 230 people
- Sponsor
- Disc Medicine, Inc
Wilson Disease
Early Access Program for ALXN1840 in Patients With Wilson Disease
This is an open-label, single-arm, multi-center EAP, designed to provide early access to ALXN1840 for eligible patients with WD.
- Trial name
- NCT05686564
- Testing
- ALXN1840
- Sponsor
- Alexion Pharmaceuticals, Inc.
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Expanded Access Program of Bitopertin For Participants With EPP or XLP
The goal of this expanded access program is to provide bitopertin to patients with EPP and XLP who have no satisfactory treatment options available in the US and to learn if bitopertin is safe to treat EPP and XLP.
- Trial name
- NCT07603401
- Testing
- DISC-1459
- Where
- 8 sites in United States
- Sponsor
- Disc Medicine, Inc
Acute Hepatic Porphyria
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).
- Trial name
- NCT04056481
- Testing
- Givosiran
- Sponsor
- Alnylam Pharmaceuticals
Bile Acid Synthesis Disorders
The REPLACE Registry for Cholbam® (Cholic Acid)
This is a prospective, observational, non-interventional patient registry study designed to document product safety and effectiveness outcomes for 10 years in patients treated with Cholbam, including those who have been using…
- Trial name
- NCT03115086
- Testing
- Cholbam
- Where
- 24 sites in United States
- Volunteers
- 55 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC)
A Database Study of Maralixibat (TAK-625) in Participants With Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)
This study is a database study in Japan for maralixibat (TAK-625) used to treat participants with Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC).
- Trial name
- NCT07293897
- Testing
- Maralixibat
- Where
- Japan
- Volunteers
- 50 people
- Sponsor
- Takeda