Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients with Xeroderma Pigmentosum (XP)

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) - Phase IIa XP Study

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2021-003642-20-DE

Enrollment

Registered

2021-07-09

Start date

2021-09-27

Completion date

Unknown

Last updated

2022-03-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

xeroderma pigmentosum

Interventions

Trade Name: SCENESSE Product Name: SCENESSE Pharmaceutical Form: Implant INN or Proposed INN: Afamelanotide CAS Number: 75921-69-6 Concentration unit: mg milligram(s) Concentration type: equal Concent

Eligibility

Sex/Gender

All

Inclusion criteria

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. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 6 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 6

Exclusion criteria

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 ß-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.

Design outcomes

Primary

Measure	Time frame
Main Objective: • Evaluate the impact of afamelanotide on minimal erythema dose (MED) in patients with XP;Secondary Objective: • 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.;Primary 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). ;Timepoint(s) of evaluation of this end point: from Day 0 to the post-treatment assessment at Day 76	—

Measure

Time frame

Main Objective: • Evaluate the impact of afamelanotide on minimal erythema dose (MED) in patients with XP;Secondary Objective: • 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.;Primary 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). ;Timepoint(s) of evaluation of this end point: from Day 0 to the post-treatment assessment at Day 76

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Secondary

Measure	Time frame
Secondary 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 ;Timepoint(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)	—

Measure

Time frame

Secondary 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 ;Timepoint(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)

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Countries

Germany

Contacts

Public ContactSara Dattoli

CLINUVEL EUROPE LIMITED

sara.dattoli@clinuvel.com7795107460

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026