Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12607000261415
Ethics application status
Approved
Date submitted
19/04/2007
Date registered
15/05/2007
Date last updated
8/05/2009
Type of registration
Prospectively registered

Titles & IDs
Public title
A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP)
Scientific title
A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants 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 1800 0
Condition category
Condition code
Human Genetics and Inherited Disorders 1887 1887 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
CUV1647 (16 mg/implant) contained in a poly(D,L-lactide-co-glycolide) implant core. The implant is fully bioresorbable and implanted subcutaneously. Drug is released from the active implant over 10 to 15 days. The study design consists of two parallel study arms with crossover between treatments every 60 days for 360 days. There is no wash out period in this study.
Intervention code [1] 1711 0
Treatment: Drugs
Comparator / control treatment
Placebo poly(D,L-lactide-co-glycolide) implant (without drug).
Control group
Placebo

Outcomes
Primary outcome [1] 2683 0
Determine whether CUV1647 can reduce the number of phototoxic reactions in patients with EPP
Timepoint [1] 2683 0
Patients record the number of reactions in a diary card completed every day and assessed every 30 days.
Primary outcome [2] 2684 0
Determine whether CUV1647 can reduce the severity of phototoxic reactions in patients with EPP
Timepoint [2] 2684 0
Patients record the severity of reactions in a diary card completed every day and assessed every 30 days.
Secondary outcome [1] 4539 0
Determine whether CUV1647 can increase the duration of sunlight tolerated by EPP patients.
Timepoint [1] 4539 0
Patients record the amount of time spent in sunlight in the diary card which is completed every day and reviewed every 30 days.
Secondary outcome [2] 4540 0
Determine whether CUV1647 increases melanin density in the skin at specified body sites.
Timepoint [2] 4540 0
Melanin density is measured every 30 days.
Secondary outcome [3] 4541 0
Evaluate the safety and tolerability of CUV1647 by measuring treatment-emergent adverse events (TEAEs).
Timepoint [3] 4541 0
TEAEs will be recorded daily in the patient diary card and assesed every 30 days.
Secondary outcome [4] 4542 0
Determine whether CUV1647 can improve the quality of life (QoL) of EPP patients.
Timepoint [4] 4542 0
QoL is measured with the SF36 v2 questionnaire every 60 days.

Eligibility
Key inclusion criteria
Subjects with a positive diagnosis of EPP
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
EPP patients with significant hepatic involvement
Any other photodermatosis

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Central randomisation by computer
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Stratified by centre, simple randomisation by using a randomisation table created by a computer software (i.e., computerised sequence generation).
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
The study is blinded to both treating doctor and patient. The study design consists of two parallel study arms with crossover between treatments every 60 days
Phase
Phase 3
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Date of first participant enrolment
Anticipated
1/06/2007
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
70
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment postcode(s) [1] 209 0
3000
Recruitment outside Australia
Country [1] 516 0
Germany
State/province [1] 516 0
Duesseldorf
Country [2] 517 0
Sweden
State/province [2] 517 0
Stockholm
Country [3] 518 0
France
State/province [3] 518 0
Paris
Country [4] 519 0
Italy
State/province [4] 519 0
Rome

Funding & Sponsors
Funding source category [1] 2035 0
Commercial sector/Industry
Name [1] 2035 0
Clinuvel Pharmaceuticals Limited
Country [1] 2035 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Clinuvel Pharmaceuticals Limited
Address
11/330 Collins St Melbourne VIC 3000
Country
Australia
Secondary sponsor category [1] 1843 0
None
Name [1] 1843 0
nil
Address [1] 1843 0
Country [1] 1843 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 3767 0
Ethik-Kommission der beiden Stadtspitaler Triemli und Waid Zuerich
Ethics committee address [1] 3767 0
Ethics committee country [1] 3767 0
Switzerland
Date submitted for ethics approval [1] 3767 0
Approval date [1] 3767 0
Ethics approval number [1] 3767 0
STZ 07/07
Ethics committee name [2] 5112 0
Melbourne Health Human Research Ethics Committee
Ethics committee address [2] 5112 0
Research Directorate Level 6 East, Main Block Royal Melbourne Hospital City Campus Grattan Street PARKVILLE VIC 3050
Ethics committee country [2] 5112 0
Australia
Date submitted for ethics approval [2] 5112 0
Approval date [2] 5112 0
18/07/2007
Ethics approval number [2] 5112 0
EC00243
Ethics committee name [3] 5646 0
Comite de Protection des Personnes Ile de France VIII
Ethics committee address [3] 5646 0
Hospital Ambroise Pare
9, avenue Charles de Gaulle
92100 Boulogne-Billancourt
Ethics committee country [3] 5646 0
France
Date submitted for ethics approval [3] 5646 0
Approval date [3] 5646 0
24/04/2008
Ethics approval number [3] 5646 0
New ethics HREC. Please modify.
Ethics committee name [4] 5647 0
Comitato Etico I.F.O.
Ethics committee address [4] 5647 0
via Chianesi, 53
00128 Roma
Ethics committee country [4] 5647 0
Italy
Date submitted for ethics approval [4] 5647 0
Approval date [4] 5647 0
30/07/2007
Ethics approval number [4] 5647 0
New ethics HREC. Please modify.
Ethics committee name [5] 5648 0
Ethikkommission an der Med. Fakultat der Heinrich-Heine-Univ. Duesseldorf
Ethics committee address [5] 5648 0
Kinderklinik Geb. 13.41
Moorenstr. 5
40225 Duesseldorf
Ethics committee country [5] 5648 0
Germany
Date submitted for ethics approval [5] 5648 0
Approval date [5] 5648 0
02/06/2008
Ethics approval number [5] 5648 0
New ethics HREC. Please modify.
Ethics committee name [6] 5649 0
Regionala etikprovningsnamnden i Stockholm
Ethics committee address [6] 5649 0
FE 289
Stockholm
171 77
Ethics committee country [6] 5649 0
Sweden
Date submitted for ethics approval [6] 5649 0
Approval date [6] 5649 0
07/05/2008
Ethics approval number [6] 5649 0
New ethics HREC. Please modify.
Ethics committee name [7] 6459 0
Multi-centre Research Ethics Committee for Wales
Ethics committee address [7] 6459 0
4th Floor, Churchill House
Churchill Way
Cardiff CF10 2TW
Ethics committee country [7] 6459 0
United Kingdom
Date submitted for ethics approval [7] 6459 0
Approval date [7] 6459 0
17/06/2008
Ethics approval number [7] 6459 0
New ethics HREC. Please modify.
Ethics committee name [8] 6460 0
Medisch Ethische Toetsings Commissie Erasmus MC
Ethics committee address [8] 6460 0
Erasmus MC/faculteit,
Kamer Fd-209,
Postbus 2040,
3000 CA ROTTERDAM
Ethics committee country [8] 6460 0
Netherlands
Date submitted for ethics approval [8] 6460 0
Approval date [8] 6460 0
18/09/2008
Ethics approval number [8] 6460 0
New ethics HREC. Please modify.

Summary
Brief summary
The purpose of this study is to determine if there is a difference between the number and severity of phototoxic reactions that occurr in patients with Erythropoietic Protoporphyria (EPP) treated with CUV1647 and placebo.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 27814 0
Address 27814 0
Country 27814 0
Phone 27814 0
Fax 27814 0
Email 27814 0
Contact person for public queries
Name 10900 0
Dennis Wright
Address 10900 0
Clinuvel Pharmaceuticals Limited 11/330 Collins St Melbourne VIC 3000
Country 10900 0
Australia
Phone 10900 0
+61 3 96604900
Fax 10900 0
Email 10900 0
[email protected]
Contact person for scientific queries
Name 1828 0
Dennis Wright
Address 1828 0
Clinuvel Pharmaceuticals Limited 11/330 Collins St Melbourne VIC 3000
Country 1828 0
Australia
Phone 1828 0
+61 3 96604900
Fax 1828 0
Email 1828 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

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