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    The EU Clinical Trials Register currently displays   43206   clinical trials with a EudraCT protocol, of which   7151   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2021-003642-20
    Sponsor's Protocol Code Number:CUV156
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-07-09
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2021-003642-20
    A.3Full title of the trial
    A Proof of Concept, Phase IIa, Open Label Study to Evaluate the Safety and
    Efficacy of Subcutaneous Implants of Afamelanotide in Patients with
    Xeroderma Pigmentosum (XP)
    Eine „Proof of Concept“, Phase IIa, offene Studie zur Bewertung der Sicherheit und Wirksamkeit subkutaner Implantate von Afamelanotid bei Patienten mit Xeroderma Pigmentosum (XP)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients with
    Xeroderma Pigmentosum (XP)
    A.3.2Name or abbreviated title of the trial where available
    Phase IIa XP Study
    A.4.1Sponsor's protocol code numberCUV156
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCLINUVEL EUROPE LTD
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCLINIVEL EUROPE LIMITED
    B.4.2CountryIreland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCLINUVEL EUROPE LIMITED
    B.5.2Functional name of contact pointSara Dattoli
    B.5.3 Address:
    B.5.3.1Street AddressWesley House, Bull Hill
    B.5.3.2Town/ cityLeatherhead
    B.5.3.3Post codeKT22 7AH
    B.5.3.4CountryUnited Kingdom
    B.5.5Fax number7795107460
    B.5.6E-mailsara.dattoli@clinuvel.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name SCENESSE
    D.2.1.1.2Name of the Marketing Authorisation holderCLINUVEL EUROPE LIMITED
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/08/541
    D.3 Description of the IMP
    D.3.1Product nameSCENESSE
    D.3.4Pharmaceutical form Implant
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAfamelanotide
    D.3.9.1CAS number 75921-69-6
    D.3.9.4EV Substance CodeSUB33758
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number16
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    xeroderma pigmentosum
    E.1.1.1Medical condition in easily understood language
    xeroderma pigmentosum
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • Evaluate the impact of afamelanotide on minimal erythema dose (MED) in patients with XP
    E.2.2Secondary objectives of the trial
    • Evaluate the impact of afamelanotide on UV-induced DNA damage and repair capacity in patients with XP.
    • Evaluate the safety and tolerability of afamelanotide in patients with XP.
    • Evaluate the impact of afamelanotide on the skin disease severity of patients with XP.
    • Evaluate the impact of afamelanotide on the skin melanin density of patients with XP.
    • Evaluate the impact of afamelanotide on the quality of life of patients with XP.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male or female patient with a molecular-genetically confirmed diagnosis of XP-C.
    - Aged 18-75 years.
    - Providing written Informed Consent prior to the performance of any study-specific procedure.
    - Willing and able to comply with the conditions specified in the protocol and study procedures, in the opinion of the Investigator.

    E.4Principal exclusion criteria
    - Known allergy to afamelanotide or the polymer contained in the implant
    - Presence of severe hepatic disease or hepatic impairment.
    - Renal impairment.
    - Any other medical condition which may interfere with the study protocol.
    - Existing melanoma
    - Female who is pregnant (confirmed by positive urine β-HCG pregnancy test) 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) or a life-style excluding pregnancy, for up to three months after the last implant administration.
    - Sexually active man with a partner of child-bearing potential (pre-menopausal, not surgically sterile) who is not using adequate contraceptive measures, as described above.
    - Use of any other prior and concomitant therapy which may interfere with the objective of the study, within 30 days prior to the Screening visit.
    - Participation in a clinical trial for an investigational agent within 30 days prior to the Screening visit.
    - Not suitable for trial participation in the opinion of the Investigator.
    E.5 End points
    E.5.1Primary end point(s)
    • The change in MED (analysis will compare the individual fold increase in MED from Day 0 to the post-treatment assessment at Day 76).

    E.5.1.1Timepoint(s) of evaluation of this end point
    from Day 0 to the post-treatment assessment at Day 76
    E.5.2Secondary end point(s)
    • The changes in UV-induced DNA damage and repair capacity from baseline Day 0 to Day 76 or Premature Termination Visit (if applicable), as measured through analysis of UV photoproducts and DNA repair mechanisms on irradiated skin in comparison with non-irradiated skin (refer to the biopsy protocol for details).
    • The change in skin disease severity from baseline Day 0 to Day 70, Day 75 or 76 or Premature Termination Visit (if applicable) and Day 238(±14) as measured by:
    o 5-point IGA scale;
    o 11-point Likert-type scale;
    • The change in skin disease severity from Screening/Day -1/Day 0 to Day 75 or 76 or Premature Termination Visit (if applicable) and Day 238(±14) as measured by the European XP severity score;
    • The change in melanin density from baseline Day -1 or 0 to Days, 14 28, 42, 56, 75 or 76 or Premature Termination Visit (if applicable) and 238(±14) as measured by spectrophotometry.
    • The change in Quality of Life from Screening/Day 0/Day 1 to Day 70, 75 or 76 or Premature Termination Visit (if applicable) and 238(±14), as measured by the two instruments:
    o WPAI:GH
    o XP-derived QOLEB
    E.5.2.1Timepoint(s) of evaluation of this end point
    Day 0 to Day 76 or Premature Termination Visit (if applicable);
    From baseline Day 0 to Day 70, Day 75 or 76 or Premature Termination Visit (if applicable) and Day 238(±14);
    From Screening/Day -1/Day 0 to Day 75 or 76 or Premature Termination Visit (if applicable) and Day 238(±14)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    Twelve-week open label study in adult patients with confirmed XP plus six month follow up.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months9
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 6
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 6
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Provision of afamelanotide to patients will be discussed at the end of
    the study.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-09-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-07-28
    P. End of Trial
    P.End of Trial StatusOngoing
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