Wilson's Disease MedDRA version: 20.0 Level: LLT Classification code 10047988 Term: Wilson's disease System Organ Class: 100000004850
Conditions
Interventions
Product Name: ALXN1840
Pharmaceutical Form: Tablet
INN or Proposed INN: ALXN1840
Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE
Concentration unit: mg milligram(s)
Concentration type: equal
Co
Sponsors
Aarhus University Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. A confirmed WD diagnosis based on the Leipzig criteria 2. Current trientine or penicillamine treatment (at least for 1 year) 3. Age above 18 4. Fertile women must accept to apply contraception at the time of the first PET-scan until 1 month after the last PET-scan: Spiral or hormonal contraception (contraceptive pill, implantation, transdermal patch, vaginal ring or depot injection) 5. At the day of the tracer administration, a negative pregnancy test for fertile women must be available. Pregnancy test will be performed as a urine test. 6. At the day of the tracer administration, a signed informed consent must be available Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 5 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Prior cholecystectomy or known biliary tract pathology 2. Participation in a medical trial including a PET scan during the past year 3. Assessed ineligibility by medical doctor on basis of blood parameters measured at inclusion, if these are abnormal. 4. Known hypersensitivity for 64-Cu, copper in general or one of the other ingredients in the tracer formula 5. Claustrophobia 6. Pregnancy, breastfeeding or the desire to become pregnant before the trial is terminated
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To investigate whether ALXN1840 increases biliary copper excretion in Wilson's Disease patients.;Secondary Objective: 1. To compare biliary copper excretion in WD-patients treated with ALXN1840 to healthy (from earlier study). 2. To investigate the effect of ALXN1840 on copper uptake and excretion in gallbladder, pancreas, kidney, heart, brain, small- and large intestine in WD compared to healthy (from earlier study). 3. To investigate 64-Cu kinetics in subjects.;Primary end point(s): A comparison of mean gallbladder and intestinal standard uptake value (SUV) between baseline and follow-up scans.;Timepoint(s) of evaluation of this end point: Second half of 2021. Patient health will be monitored. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Comparison of mean SUV's in other organs of interest (liver, kidney, pancreas, heart, brain) in baseline and follow-up scan. 2. 64-Cu measurements from all subjects for kinetics evaluation.;Timepoint(s) of evaluation of this end point: second half of 2021. | — |
Countries
Denmark
Contacts
Public ContactPublic information about the trial.
Aarhus University Hospital
Outcome results
None listed