Xeroderma PigmentosumPhase 2NCT05159752
A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum (XP)
A Phase 2 study testing Afamelanotide in people with Xeroderma Pigmentosum, run by Clinuvel Europe Limited at 1 site in Germany.
Trial summary
The CUV156 study will evaluate the safety of afamelanotide in XP-C patients, as well as the drug's ability to assist reparative processes following ultraviolet (UV) provoked DNA damage of the skin. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Xeroderma Pigmentosum
- What is being tested?
- Afamelanotide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteGermany
- Study dates
- 19 Oct 2021to Oct 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female patient with a molecular-genetically confirmed diagnosis of XP-C;
- Aged 18-75 years.
Who cannot join
- 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;
- Female who is pregnant (confirmed by positive urine beta-Human chorionic gonadotropin pregnancy test) or lactating;
- Females of child-bearing potential (pre-menopausal, not surgically sterile) not using highly effective contraceptive measures with a failure rate of less than 1% per year when used consistently and correctly (i.e. oral contraceptives, intrauterine device) or a life-style excluding pregnancy, for up to three months after the last implant administration;
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- Sexually active man with a partner of child-bearing potential (pre-menopausal, not surgically sterile) who is not using highly effective 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.
Trial locations and contacts
1 site in 1 country.
Germany1 site, 1 recruiting
- Recruiting
CLINUVEL investigative site
Regensburg
Head of Clinical Operations, mail@clinuvel.com
About this study
Official title: 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)
The CUV156 study will evaluate the safety of afamelanotide in XP-C patients, as well as the drug's ability to assist reparative processes following ultraviolet (UV) provoked DNA damage of the skin. It will assess whether SCENESSE® increases the amount of UV light needed to cause DNA damage of skin cells, as well as the extent of skin repair before and after treatment.
- Conditions
- Xeroderma Pigmentosum
- Being tested
- Afamelanotide
- Medical subject headings
- Xeroderma Pigmentosum
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AfamelanotideDrug
Patients will receive afamelanotide every two weeks for twelve weeks and undergo a follow up visit 6 months later.
| Study group | Role | What they receive |
|---|---|---|
| Afamelanotide | Experimental | Not described Receives: Drug: Afamelanotide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in minimal erythema dose (MED).
MED is the lowest dose of UV light that causes reddening of the skin.
Measured: From Baseline to Day 76.
Secondary outcomes (7)
Change in UV-induced DNA damage and repair capacity.
Analysis of UV photoproducts and DNA repair mechanisms.
Measured: From Baseline to Day 76.
Change in skin disease severity (A).
The higher the score, the more severe the disease.
Measured: From Baseline to Day 238.
Change in skin disease severity (B).
The higher the score, the more severe the disease.
Measured: From Baseline to Day 238.
Change in skin disease severity (C).
The higher the score, the more severe the disease.
Measured: From Baseline to Day 238.
Change in dermal melanin density.
Non-invasive quantitative skin reflectance measurement.
Measured: From Baseline to Day 238.
Change in quality of life assessed by a disease specific tool (A)
Higher scores represent worse health-related quality of life.
Measured: From Baseline to Day 238.
Change in quality of life assessed by a validated global quality of life tool (B)
Score calculated in impairment percentages, with higher numbers indicating greater impaired quality of life.
Measured: From Baseline to Day 238.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Dec 2021
- Study start19 Oct 2021
- Primary completionMar 2024 (estimated)
- Study completionOct 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Xeroderma PigmentosumStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Clinuvel Europe Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05159752
- Sponsor's protocol ID
- CUV156
- Status last verified
- Jun 2023
- First submitted
- 9 Nov 2021
- Last updated
- 18 Jun 2023
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT05159752
- Acronym / sponsor ID
- CUV156
- Conditions
- Xeroderma Pigmentosum
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Clinuvel Europe LimitedIndustry (pharma, biotech, devices)
- Study start
- 19 Oct 2021 (actual)
- Primary completion
- Mar 2024 (estimated)
- Study completion
- Oct 2024 (estimated)
- First posted
- 16 Dec 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The CUV156 study will evaluate the safety of afamelanotide in XP-C patients, as well as the drug's ability to assist reparative processes following ultraviolet (UV) provoked DNA damage of the skin. It will assess whether SCENESSE® increases the amount of UV light needed to cause DNA damage of skin cells, as well as the extent of skin repair before and after treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Afamelanotide | Experimental | Drug: Afamelanotide |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Male or female patient with a molecular-genetically confirmed diagnosis of XP-C;
- Aged 18-75 years.
Key exclusion criteria (9 listed)
- 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;
- Female who is pregnant (confirmed by positive urine beta-Human chorionic gonadotropin pregnancy test) or lactating;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AfamelanotidePatients will receive afamelanotide every two weeks for twelve weeks and undergo a follow up visit 6 months later. | Drug | Afamelanotide |
Primary outcome
Change in minimal erythema dose (MED).
Time frame: From Baseline to Day 76.
MED is the lowest dose of UV light that causes reddening of the skin.
7 secondary outcomes
Change in UV-induced DNA damage and repair capacity.
Time frame: From Baseline to Day 76.
Change in skin disease severity (A).
Time frame: From Baseline to Day 238.
Change in skin disease severity (B).
Time frame: From Baseline to Day 238.
Change in skin disease severity (C).
Time frame: From Baseline to Day 238.
Change in dermal melanin density.
Time frame: From Baseline to Day 238.
Change in quality of life assessed by a disease specific tool (A)
Time frame: From Baseline to Day 238.
Change in quality of life assessed by a validated global quality of life tool (B)
Time frame: From Baseline to Day 238.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Afamelanotide in people with Xeroderma Pigmentosum, run by Clinuvel Europe Limited at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 18 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Head of Clinical Operations (+441372860765, mail@clinuvel.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in minimal erythema dose (MED). (time frame: From Baseline to Day 76.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jun 2023.