Clinical trials: Genetic Liver Disorder
Cytomegalovirus Infection
CMV Antiviral Prevention Strategies in D+R-Liver Transplants ("CAPSIL")
This is a trial of preemptive therapy vs. prophylaxis for prevention of Cytomegalovirus (CMV) disease in R-D+ liver transplant patients.
- Trial name
- NCT01552369
- Testing
- Valganciclovir
- Where
- 6 sites in United States
- Volunteers
- 205 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Obstructive Pulmonary Disease, Alpha1-antitrypsin Deficiency
Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency
The purpose of this study is to conduct a pilot study to evaluate the safety and efficacy of weekly administration of Alpha1-Proteinase Inhibitor (A1PI) augmentation therapy in subjects with A1PI deficiency and emphysema/ chronic…
- Trial name
- NCT02722304
- Testing
- ARALAST NP 60 mg/kg, ARALAST NP 120 mg/kg, GLASSIA 60 mg/kg
- Where
- 17 sites in United States, Australia and 1 more
- Volunteers
- 7 people
- Sponsor
- Baxalta now part of Shire
Acute Intermittent Porphyria, Hereditary Coproporphyria
Panhematin for Prevention of Acute Attacks of Porphyria
The purpose of this study is to determine if Panhematin is safe and effective for prevention of acute attacks of porphyria. The study aims to provide high quality evidence for the use on hemin for prevention of acute attacks of…
- Conditions
- Acute Intermittent Porphyria, Hereditary Coproporphyria, Variegate Porphyria
- Trial name
- NCT02922413
- Testing
- Hemin for injection
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- The University of Texas Medical Branch, Galveston
Wilson Disease
Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
The main objective of the study is to evaluate the change in liver copper (Cu) concentration following 48 weeks of treatment with ALXN1840 in adult participants with Wilson Disease (WD) who have been previously treated for at…
- Trial name
- NCT04422431
- Testing
- Bis-Choline Tetrathiomolybdate
- Where
- 12 sites in United States, Canada and 6 more
- Volunteers
- 31 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hepatocellular Carcinoma
A Study of LY2157299 in Participants With Advanced Hepatocellular Carcinoma
The main purpose of this study is to evaluate the safety and effectiveness of the study drug known as LY2157299 in participants with hepatocellular carcinoma.
- Trial name
- NCT02178358
- Testing
- LY2157299, Sorafenib
- Where
- 16 sites in China, Hong Kong and 3 more
- Volunteers
- 132 people
- Sponsor
- Eli Lilly and Company
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Dasatinib, Ifosfamide, Carboplatin, and Etoposide in Treating Young Patients With Metastatic or Recurrent Malignant Solid Tumors
RATIONALE: Dasatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs in chemotherapy, such as ifosfamide, carboplatin, and etoposide, work in different ways to stop the growth of…
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00788125
- Testing
- carboplatin, dasatinib, etoposide phosphate
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- City of Hope Medical Center
Wilson Disease
Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants.
This was a Phase 1, randomized, 2-period, 2-sequence, cross-over study designed to determine the effect of ALXN1840 on the metabolism of celecoxib, a sensitive cytochrome P450 2C9 (CYP2C9) substrate, in healthy male and female…
- Trial name
- NCT04526197
- Testing
- ALXN1840, Celecoxib
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Alpha-1 Antitrypsin Deficiency
Long-Term Follow-up Study of ADVM-043
ADVM-043-03 is a long-term follow-up (LTFU) study of subjects who participated in the ADVM-043-01 (NCT02168686) multi-center gene therapy clinical study (ADVANCE) that evaluated ADVM-043 for the treatment of Alpha-1 Antitrypsin…
- Trial name
- NCT03804021
- Testing
- No Intervention
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Adverum Biotechnologies, Inc.
Chronic Pancreatitis
Alpha-1 Antitrypsin (AAT) Enhances Islet Autograft Survival in Patients With Chronic Pancreatitis
Primary objective: To describe and compare the safety and efficacy of treatment with AAT in chronic pancreatitis patients who undergo total pancreatectomy and islet autotransplantation (TP-IAT).
- Trial name
- NCT02947087
- Testing
- Prolastin-C
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Medical University of South Carolina
Alagille Syndrome
Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome
The study is a randomized, double-blind, placebo-controlled study to evaluate the safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 versus placebo on the biochemical markers and…
- Trial name
- IMAGO (NCT01903460)
- Testing
- LUM001
- Where
- 3 sites in United Kingdom
- Volunteers
- 20 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
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.
Erythropoietic Protoporphyria
A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)
The main objectives of the study were to evaluate the safety and tolerability of afamelanotide by measuring treatment emergent adverse events (AEs) and to determine whether afamelanotide can improve the quality of life of EPP…
- Trial name
- NCT04578496
- Testing
- Afamelanotide
- Volunteers
- 16 people
- Sponsor
- Clinuvel Pharmaceuticals Limited
Alpha-1 Antitrypsin Deficiency, Emphysema
Alpha-1 Carrier Genomics Study
The goal of this study is to better understand why some Alpha-1 genotype MZ (PiMZ) individuals develop chronic obstructive pulmonary disease (COPD) while others do not.
- Conditions
- Alpha-1 Antitrypsin Deficiency, Emphysema, COPD, Smoking
- Trial name
- NCT02810327
- Testing
- Genetic Sequencing
- Where
- United States
- Volunteers
- 117 people
- Sponsor
- Medical University of South Carolina