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Active clinical trials for "Hepatolenticular Degeneration"

Results 21-30 of 52

Clinical Profile and Out Come of Children With Wilson's Disease

Clinical Outcome in WILSON Disease

Clinical profile and out come of Children with Wilson's disease :A single tertiary center study

Not yet recruiting7 enrollment criteria

Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants

Wilson Disease

This is a Phase 1, randomized, 2-period, 2-sequence, cross-over study designed to determine the effect of ALXN1840 on the metabolism of bupropion, a sensitive cytochrome P450 2B6 (CYP2B6) substrate, in healthy male and female participants. The safety and tolerability of ALXN1840 will be determined along with ALXN1840 pharmacokinetics (PK) in plasma as measured via total molybdenum with the coadministration of bupropion.

Completed12 enrollment criteria

Study of Zinc for Wilson Disease

Wilson Disease

OBJECTIVES: I. Establish the safety and efficacy of extended maintenance zinc therapy in 200 patients with Wilson disease. II. Establish further the role of zinc in the prophylactic treatment of presymptomatic patients by increasing the current cohort from 80 to at least 100 patients. III. Establish further the role of zinc therapy in pregnant patients with Wilson disease. IV. Establish further the role of zinc therapy in children with Wilson disease.

Completed1 enrollment criteria

Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants.

Wilson Disease

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 participants. The safety and tolerability of ALXN1840 were determined along with ALXN1840 pharmacokinetics (PK) in plasma as measured via total molybdenum with the coadministration of celecoxib.

Completed12 enrollment criteria

Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840

Wilson Disease

This exploratory study will investigate the effects of ALXN1840 on copper balance in participants with Wilson disease (WD).

Completed13 enrollment criteria

Early Detection of Cardiac Affection in Patients of Wilson's Disease

Cardiovascular DiseasesWilson's Disease

Wilson disease (WD) is a rare autosomal recessive disorder caused by a genetic defect in ATP7B resulting in limited excretion of excess copper into the bile Pathological copper accumulation occurs in the entire body, with the liver and the brain being primarily affected

Not yet recruiting4 enrollment criteria

Inhibitory rTMS in Dystonic Wilson Patients

Wilson DiseaseMovement Disorders1 more

Wilson disease is a genetic disorder resulting in copper accumulation in liver, brain and eye. The neurologic complications include dystonic syndrome, which is a prolonged and excessive muscle activation responsible for abnormal postures. Hand dystonia prevents daily life activities such as writing, which is particularly disabling, since writing is the only mean of communication in these patients with significant slurred speech. Treatment is limited and only partially effective. Low frequency (<or=1Hz) repetitive transcranial magnetic stimulation (rTMS) has shown inhibiting properties when applied over the cortex. Since dystonia has been correlated to hyperactivation of the neurons of the somatosensory cortex (SSC), we hypothesize that one single 20-minute session of 1 Hz rTMS applied on left SSC will improve writing of the right dystonic hand, assessed immediately at the end of the session.

Completed16 enrollment criteria

Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease...

Wilson Disease

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 least 1 year with standard of care (that is, trientine, penicillamine, or zinc). In the Treatment Period, efficacy and safety of ALXN1840 will be assessed at Week 48.

Completed17 enrollment criteria

Efficacy and Safety, Long-term Study of Zinc Acetate to Treat Wilson's Disease in Japan.

Wilson's Disease

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.

Completed3 enrollment criteria

A Phase 1 Study to Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride...

Wilson's Disease

To evaluate the effects of a single dose of trientine in children ≥6 years and adult patients with Wilson's disease.

Completed11 enrollment criteria
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