|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
19 February 2015 |
|
Main ID: |
NCT00979745 |
|
Date of registration:
|
17/09/2009 |
|
Prospective Registration:
|
No |
|
Primary sponsor: |
|
|
Public title:
|
Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)
|
|
Scientific title:
|
A Phase III, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients With Erythropoietic Protoporphyria (EPP) |
|
Date of first enrolment:
|
September 2009 |
|
Target sample size:
|
70 |
|
Recruitment status: |
Completed |
|
URL:
|
http://clinicaltrials.gov/show/NCT00979745 |
|
Study type:
|
Interventional |
|
Study design:
|
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Prevention
|
|
Phase:
|
Phase 3
|
|
|
Countries of recruitment
|
|
Finland
|
France
|
Germany
|
Ireland
|
Netherlands
|
United Kingdom
| | |
|
Contacts
|
|
Name:
|
Jean-Charles Deybach, MD. PhD |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Centre Francais des Porphyries, Hopital Louis Mourier, Colombes, France |
|
|
Name:
|
Jorge Frank, MD, PhD |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Academisch Ziekenhuis Maastricht |
|
|
Name:
|
Gillian M Murphy, MD PhD |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Beaumont Hospital, Dublin, Ireland |
|
|
Name:
|
Eric JG Sijbrands, MD, PhD |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Erasmus Medical Center, Rotterdam |
|
|
Name:
|
Alex Anstey, MBBS, FRCP |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
St Woolos Hospital, Newport |
|
|
Name:
|
Raili Kauppinen, MD, PhD |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
University Central Hospital of Helsinki |
|
|
Name:
|
Sandra Hanneken, MD |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Heinrich-Heine Universität, Düsseldorf, Germany |
|
|
Name:
|
Lesley E Rhodes, MD PhD |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Hope Hospital, University of Manchester, UK |
| |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
- Male or female subjects with a diagnosis of EPP (confirmed by elevated free
protoporphyrin in peripheral erythrocytes) of sufficient severity that they have
requested treatment to alleviate their symptoms.
- Aged 18 - 70 years (inclusive)
- Written informed consent prior to the performance of any study-specific procedures.
Exclusion Criteria:
- Any allergy to afamelanotide or the polymer contained in the implant or to lignocaine
or other local anaesthetic to be used during the administration of study medication.
- EPP patients with significant hepatic involvement.
- Personal history of melanoma or dysplastic nevus syndrome.
- Current Bowen's disease, basal cell carcinoma, squamous cell carcinoma, or other
malignant or premalignant skin lesions.
- Any other photodermatosis such as PLE, DLE or solar urticaria.
- 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).
- Patient assessed as not suitable for the study in the opinion of the Investigator
(e.g. noncompliance history, allergic to local anaesthetics, faints when given
injections or giving blood).
- Female who is pregnant (confirmed by positive serum ß-HCG pregnancy test prior to
baseline) or lactating.
- Females of child-bearing potential (pre-menopausal, not surgically sterile) not using
adequate contraceptive measures (i.e. oral contraceptives, diaphragm plus spermicide,
intrauterine device).
- Sexually active men with partners of child bearing potential not using barrier
contraception during the trial and for a period of three months thereafter.
- Participation in a clinical trial of an investigational agent within 30 days prior to
the screening visit.
- Prior and concomitant therapy with medications which may interfere with the
objectives of the study, including drugs that cause photosensitivity or skin
pigmentation.
Age minimum:
18 Years
Age maximum:
70 Years
Gender:
Both
|
|
Health Condition(s) or Problem(s) studied
|
|
Erythropoietic Protoporphyria
|
|
Intervention(s)
|
|
Drug: Afamelanotide
|
|
Drug: Placebo
|
|
Primary Outcome(s)
|
|
Severity of phototoxic reaction measured by visual analogue scale
[Time Frame: 9 months]
|
|
Secondary Outcome(s)
|
|
Free protoporphyrin IX level
[Time Frame: 9 months]
|
|
Treatment emergent adverse events
[Time Frame: 9 months]
|
|
Number of phototoxic reactions
[Time Frame: 9 months]
|
|
Quality of life measured by patient completed questionnaire
[Time Frame: 9 months]
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|