Main objective: determine whether afamelanotide implants can reduce the severity of the skin disease in patients with VPSecondary objectives: Evaluate the safety and tolerability of afamelanotide in patients with VP. Evaluate the impact of…
ID
Source
Brief title
Condition
- Other condition
- Metabolic and nutritional disorders congenital
Synonym
Health condition
inborn error of heme biosynthesis
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The change in severity of skin disease from baseline to Days 28, 56, 84, 112,
140 and 168 and then from Day 168 to Day 196, as measured by the 11-point VAS
IGA scale.
Secondary outcome
The change in severity from baseline to Days 28, 56, 84, 112, 140 and 168 and
then from Day 168 to Day 196, as measured by: The 5-point IGA; The Patient*s
Global Assessment using a VAS. - The change in the number of new skin lesions
formed during the preceding 28 days from baseline to Days 28, 56, 84, 112, 140
and 168 then from Day 168 to Day 196 as counted by the Investigator. - The
change in Quality of Life from baseline to Day 28, 56, 84, 112, 140 and 168
then from Day 168 to Day 196, as measured by the three instruments: WPAI:GH,
VP-derived QOLEB, VP-QoL. - Change in outdoors light exposure over time (Daily
Diary). Trauma events will be tabulated.
Background summary
Afamelanotide 16mg has been shown to be effective in the prevention of
phototoxicity in EPP and had obtained marketing authorisation in the European
Union for this indication in adult patients. The results of the clinical trials
conducted by CLINUVEL, as well as of one long term observational study and the
ongoing post-authorisation pharmacovigilance activities, confirmed the positive
safety profile of afamelanotide to date. As the photosensitising agent is the
same in VP as it is in EPP, it is believed that afamelanotide could be of
benefit to VP patients with cutaneous symptoms. In view of the chronic nature
of the disease, and the pharmacokinetic profile of afamelanotide 16mg, an
intensified dosage scheme to that used in EPP (once every 28 days rather than
once every 60 days), but similar to that applied previously in vitiligo
studies would be expected to be effective.
Study objective
Main objective: determine whether afamelanotide implants can reduce the
severity of the skin disease in patients with VP
Secondary objectives: Evaluate the safety and tolerability of afamelanotide in
patients with VP. Evaluate the impact of afamelanotide on the quality of life
of patients with VP.
Study design
Afamelanotide 16 mg every 28±2 days (up to six doses), as a controlled-release
implant.
This is an 8-month (including the screening and follow-up period) open label
study in adult patients with confirmed VP-related skin disease.
Intervention
Afamelanotide 16 mg every 28±2 days (up to six doses)
Study burden and risks
The results of the clinical trials conducted by CLINUVEL in other indications,
as well as of one long term observational study and the ongoing
post-authorisation pharmacovigilance activities in erythropoietic
protoporphyria (EPP) patients, confirmed the positive safety profile of
afamelanotide to date. To date, SCENESSE® has been administered to more than
903 patients - EPP and other indications - and healthy volunteers in total as
part of its clinical development. The most commonly reported adverse reactions
are nausea, experienced by approximately 19% of subjects who received treatment
with the drug during clinical studies, headache (20%), and implant site
reactions (21%; mainly discolouration, pain, haematoma, erythema). In most
cases these adverse reactions are reported to be mild in severity and transient
in nature. Most adverse events occur within 24-72 hours following the
administration of the implant. *Participation in the study may also require
time for additional hospital appointments and for matters that are necessary to
meet the research requirements. Each visit lasts on average about 3 hours.*
Wesley House, Bull Hill 1
Leatherhead, Surrey KT22 7AH
GB
Wesley House, Bull Hill 1
Leatherhead, Surrey KT22 7AH
GB
Listed location countries
Age
Inclusion criteria
Male or female patients with confirmed diagnosis of VP.
Patients with VP-related skin symptoms assessed with a score equal or above 7
on the 11-point VAS IGA.
Exclusion criteria
Known allergy to afamelanotide or the polymer or to lignocaine/lidocaine or
other local anaesthetic.
Had two or more acute attacks of hepatic porphyria lasting more than two days,
within 12 months prior to the Screening period.
History of certain malignant and premalignant skin lesions.
Individual or family history of melanoma.
Severe hepatic disease.
Renal impairment (eGFR (MDRD) < 30 ml/min*1.73m2).
Female who is pregnant or lactating.
Females of child-bearing potential not using adequate contraceptive measures.
Sexually active man with a partner of child-bearing potential who is not using
adequate contraceptive measures.
Not suitable for trial participation in the opinion of the Investigator.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2018004164-60-NL |
| CCMO | NL69926.078.19 |