Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12606000535572
Ethics application status
Approved
Date submitted
15/12/2006
Date registered
22/12/2006
Date last updated
14/06/2022
Date data sharing statement initially provided
14/06/2022
Type of registration
Retrospectively registered

Titles & IDs
Public title
Phase II Study of CUV1647 in Erythropoietic Protoporphyria (EPP)
Scientific title
A Multicentre, Phase II, Open Label Study to Evaluate the Safety of CUV1647 and to Evaluate the Effect of Subcutaneous Implants of CUV1647 on the Time to Artificially Provoked Symptoms in Patients with Erythropoietic Protoporphyria (EPP)
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Erythropoietic Protoporphyria (EPP) 1503 0
Condition category
Condition code
Human Genetics and Inherited Disorders 1601 1601 0 0
Other metabolic and endocrine disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
CUV1647 (20 mg implant) every two months for a total of 12 months. Patients will serve as their own contols for all tested parameters.
Intervention code [1] 1509 0
Prevention
Comparator / control treatment
No comparator.
Control group
Historical

Outcomes
Primary outcome [1] 2209 0
Time to appearance of artificially provoked symptoms when hands are exposed to light from a solar simulator
Timepoint [1] 2209 0
At baseline and then every two months.
Primary outcome [2] 2210 0
Amount of rescue medication used to treat phototoxic reactions.
Timepoint [2] 2210 0
Secondary outcome [1] 3852 0
Changes is skin melanin density
Timepoint [1] 3852 0
From baseline to 2, 4, 6, 8, 10 and 12 months.
Secondary outcome [2] 3853 0
Quality of life as measured by SF36v2
Timepoint [2] 3853 0
At baseline and again at 12 months.
Secondary outcome [3] 3854 0
Number and severity of phototoxic reactions
Timepoint [3] 3854 0
Recorded over the 12 month study period.

Eligibility
Key inclusion criteria
Patients with EPP (confirmed by elevated free protoporphyrin in peripheral erythrocytes and/or ferrochelatase mutation)- Fitzpatrick skin types I to IV.
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Any other photodermatosis such as polymorphic light eruption (PLE), discoid lupus erythematosus (DLE) or solar urticaria- females who are pregnant, lactating or of child bearing age not using adequate methods of contraception- Any evidence of clinically significant organ dysfunction or any clinically significant deviation from normal in the clinical or laboratory determinations.- Acute history of drug or alcohol abuse (in the last 12 months).- History of disorders of the gastrointestinal, hepatic, renal, cardiovascular, respiratory, endocrine, neurological, haematological or systemic disease judged to be clinically significant by the Investigator.

Study design
Purpose of the study
Prevention
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Not applicable
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Not applicable
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Data analysis is complete
Reason for early stopping/withdrawal
Other reasons/comments
Other reasons
it was planned that study participation for each subject would be for a period of approximately one year, with 6 implants at intervals of 60 days. However, following highly encouraging results for interim data to Day 120 (after 2 implants), the study was terminated prematurely, to progress the investigation of this product in a multi-centre study (CUV017).
Date of first participant enrolment
Anticipated
1/09/2006
Actual
1/09/2006
Date of last participant enrolment
Anticipated
Actual
10/10/2006
Date of last data collection
Anticipated
Actual
20/02/2007
Sample size
Target
10
Accrual to date
Final
5
Recruitment outside Australia
Country [1] 446 0
Switzerland
State/province [1] 446 0

Funding & Sponsors
Funding source category [1] 1745 0
Commercial sector/Industry
Name [1] 1745 0
Clinuvel Pharmaceuticals Limited
Country [1] 1745 0
Primary sponsor type
Commercial sector/Industry
Name
Clinuvel Pharmaceuticals
Address
Country
Australia
Secondary sponsor category [1] 1540 0
None
Name [1] 1540 0
Not applicable
Address [1] 1540 0
Country [1] 1540 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 3223 0
Triemli Hospital-Zurich Canton Ethics Committee
Ethics committee address [1] 3223 0
Ethics committee country [1] 3223 0
Swaziland
Date submitted for ethics approval [1] 3223 0
Approval date [1] 3223 0
03/07/2006
Ethics approval number [1] 3223 0
Ethics committee name [2] 3224 0
Royal Melbourne Hospital
Ethics committee address [2] 3224 0
Ethics committee country [2] 3224 0
Australia
Date submitted for ethics approval [2] 3224 0
Approval date [2] 3224 0
Ethics approval number [2] 3224 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 27443 0
Address 27443 0
Country 27443 0
Phone 27443 0
Fax 27443 0
Email 27443 0
Contact person for public queries
Name 10698 0
Dr Dennis Wright
Address 10698 0
Level 13
1 Collins Street
Melbourne VIC 3000
Country 10698 0
Australia
Phone 10698 0
+61 3 96604900
Fax 10698 0
+61 3 96604999
Email 10698 0
dennis.wright@clinuvel.com
Contact person for scientific queries
Name 1626 0
Professor Elisabeth Minder
Address 1626 0
Chefärztin Zentrallabor
Stadtspital Triemli
CH-8063 Zürich
Country 1626 0
Switzerland
Phone 1626 0
+41 44 4662320
Fax 1626 0
+41 44 4662744
Email 1626 0
Elisabeth.Minder@triemli.stzh.ch

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.