Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT05783687 |
| Other study ID # |
ORPH-131-009 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
June 28, 2023 |
| Est. completion date |
December 2025 |
Study information
| Verified date |
April 2024 |
| Source |
Orphalan |
| Contact |
Carla Bennett, Bsc. Hons |
| Phone |
07918 380893 |
| Email |
carla.bennett[@]orphalan.com |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial Summary
This non-interventional Real-World Evidence (RWE) study aims to describe non-ceruloplasmin
copper values obtained using a new NCC Speciation assay by taking a small (up to 10mLs)
volume of additional blood from patients with Wilson's Disease, around the time when routine
blood sampling is expected to be scheduled by the treating physician. Data will be collected
over an approximate 12-month period.
Description:
This is a non-interventional RWE study to describe NCC-Sp in relation to standard of care
(SoC) copper measurements at each visit and longitudinally over an approximate 12-month study
period in WD patients.
Data will be collected during routinely scheduled WD clinic visits over an approximate
12-month period.
Data collected will include:
- Relevant medical history and WD medication history
- All copper measurements and SoC clinical laboratory results prior to changing
therapy/study enrolment, and if available, for up to 2 years prior to baseline
- Concomitant medications at the time of study enrollment and if available, for up to 2
years prior to baseline.
- Routine blood sample results from SoC assessments including biochemistry, hematology and
coagulation measures, copper assessments (including serum ceruloplasmin and total copper
alongside a locally calculated estimation of NCC).
- Around the same time as the routine blood samples, an additional study specific study
blood samples of up to 10 mL will be collected for analysis of the NCC-Sp, serum total
copper and serum ceruloplasmin, copper fraction associated with ceruloplasmin and serum
zinc levels. Samples will be sent for analysis at the nominated central laboratory.
Results will only be made available to investigators at the end of the study. Central
laboratory samples taken for the study may be kept for up to 5 years for additional
testing (metal analyses).
- Routine 24-hour urinary copper excretion (µmol/l) collection may form part of the SoC
assessments conducted during regularly planned visits. This sample will be analyzed as
per standard practice by the local laboratory.
- Calculated New Wilson's Disease Index and WD medications, and other testing as collected
during routinely scheduled WD clinic visits and analyzed as per local hospital
procedures and facilities.
After providing informed consent, patients meeting all inclusion and no exclusion criteria
will be enrolled into the study. Patient's routine WD clinic visits will be scheduled
according to the standard clinical practice at the study center and at the discretion of the
treating physician. Enrolled patients are to be followed for approximately 12 months; first
visit will be recorded as the baseline visit. If a switch of therapy is made, patients are
typically re-assessed within a window of approximately 1- 3 months post initiation of the new
treatment with biochemical testing and in person consultation for some. In the absence of
starting a new treatment, patients are reviewed at approximately 6-month intervals, i.e., SoC
visits at 6 months and 12 months. Routine chemistry, hematology, coagulation, copper
assessments, 24h urine sampling for urinary copper excretion and other laboratory testing
used to assess the patient as part of SoC will be performed as usual by the site local
laboratory as determined by the treating physician per their standard practice for managing
patients with WD. Routine safety testing and the required laboratory values produced by the
local laboratory will be used to calculate the New Wilson's Disease Index.
An additional small volume (up to 10 mL) of blood will be drawn for central laboratory
assessment of NCC-Sp, serum total copper and serum ceruloplasmin, copper fraction associated
with ceruloplasmin and serum zinc levels around the same time as SoC blood draw is expected
to minimize burden to the patients. These study specific samples will be drawn, processed,
and shipped to a central laboratory either from the WD clinic, by a local commercial
laboratory, or from the patient's home (or patient-specified local address) by a qualified
home health care professional with experience in obtaining and processing blood specimens for
clinical trials. Results from centrally processed samples will only be made available to
investigators at the end of the study. Central laboratory samples taken for the study may be
kept for up to 5 years for additional testing for metal analyses.
The following standardized disease assessment questionnaires will be conducted as per the
schedule of assessments over the study:
- UWDRS (Part II)
- SF-12 quality of life questionnaire
- Morisky scale assessment for compliance (MMAS-8)
- Patient's Global Impression of Change (PGIC)
- Clinical Global Impression of Change (CGIC)
Central laboratory results (blood) will be transferred by the central laboratory vendor(s)
for inclusion in the Clinical Data Set. The date of all sample collections as well as the
results, units and normal range values from local laboratories and all other clinical data
collected for the study (medical and medication history etc.) will be entered into a
web-based electronic data capture (EDC) system by the study physician or study
coordinator/designee.
Physicians are not obligated to actively seek information on adverse reactions/serious
adverse reactions. However, if the Physician or other responsible person learns of any ADR
/special reporting situation and he/she considers the event to be reasonably related to
current treatment, the Investigator should document it in the eCRF. Adverse Drug Reactions
attributable to a drug product, should be reported directly to the Marketing Authorisation
Holder (MAH) of that product following the usual process.
