Erythropoietic ProtoporphyriaPhase 3NCT04578496

A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)

A Phase 3 study testing Afamelanotide in people with Erythropoietic Protoporphyria, run by Clinuvel Pharmaceuticals Limited.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The main objectives of the study were to evaluate the safety and tolerability of afamelanotide by measuring treatment emergent adverse events (AEs) and to determine whether afamelanotide can improve the quality of life of EPP patients. Read more about this study

Who is it for?: Ages 18 to 75 years
Conditions the study is for: Erythropoietic Protoporphyria
What is being tested?: Afamelanotide
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 16 participants
Where: No sites listed yet
Study dates: 11 Jul 2011to 27 Feb 2014
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 subjects with a positive diagnosis of EPP who successfully completed the CUV017 or CUV029 studies;
aged 18-75 years (inclusive);
provide written informed patient consent prior to the performance of any study-specific procedure.

Who cannot join

any serious adverse event considered to be related to afamelanotide or the polymer contained in the implant;
any allergy to lignocaine or other local anaesthetic to be used during the administration of the 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 evidence of clinically significant organ dysfunction or any clinically significant deviation from normal in the clinical or laboratory determinations;

Show all 12Show fewer

acute history of drug or alcohol abuse (in the last 12 months);
female who is pregnant (confirmed by positive serum β-Human Chorionic Gonadotropin (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 for 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.

About this study

Official title: A Phase III, Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)

Conditions: Erythropoietic Protoporphyria
Being tested: Afamelanotide
Medical subject headings: Protoporphyria, Erythropoietic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

AfamelanotideDrug

Study groups in NCT04578496
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 Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL).
The EPP-QoL is presented as 15 questions aiming at measuring how the condition has affected the patient over the last 2 months. Each question is answered on a categorical scale. The maximum score is calculated by summing the score of each question resulting in a maximum of 100 and a minimum of 0. The higher the score, the better the quality of life.
Measured: Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)
Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI)
The DLQI questionnaire consists of 10 questions, each answered on a categorical scale. The DLQI is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired.
Measured: Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

11 Jul 201127 Feb 2014

First posted8 Oct 2020
Study start11 Jul 2011
Primary completion27 Feb 2014
Study completion27 Feb 2014

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Erythropoietic ProtoporphyriaStudies 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 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 16 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04578496
Sponsor's protocol ID: CUV037
Status last verified: Nov 2020
First submitted: 5 Oct 2020
Last updated: 19 Nov 2020
Results posted: Yes, on ClinicalTrials.gov

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

Enrolling by invitationPhase 2/3
HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
Erythropoietic Protoporphyria
RecruitingPhase 2/3
A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Active, not recruitingPhase 3
Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
EPP, XLP
RecruitingObservational study
Longitudinal Study of the Porphyrias
Acute Porphyrias, Cutaneous Porphyrias
RecruitingObservational study
Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)
Erythropoietic Protoporphyria (EPP), X-Linked Porphyria (XLP)

All Erythropoietic Protoporphyria trials

Study at a glance

From the registry record, last updated by the sponsor on 19 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase III, Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)
ClinicalTrials.gov ID: NCT04578496
Acronym / sponsor ID: CUV037
Conditions: Erythropoietic Protoporphyria
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 16 participants (actual)
Lead sponsor: Clinuvel Pharmaceuticals LimitedIndustry (pharma, biotech, devices)
Study start: 11 Jul 2011 (actual)
Primary completion: 27 Feb 2014 (actual)
Study completion: 27 Feb 2014 (actual)
First posted: 8 Oct 2020 (actual)
Results: Posted 2 Nov 2020

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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 (3 listed)

male or female subjects with a positive diagnosis of EPP who successfully completed the CUV017 or CUV029 studies;
aged 18-75 years (inclusive);
provide written informed patient consent prior to the performance of any study-specific procedure.

Key exclusion criteria (12 listed)

any serious adverse event considered to be related to afamelanotide or the polymer contained in the implant;
any allergy to lignocaine or other local anaesthetic to be used during the administration of the 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;

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
Afamelanotide	Drug	Afamelanotide

Primary outcome

Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL).
Time frame: Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)
The EPP-QoL is presented as 15 questions aiming at measuring how the condition has affected the patient over the last 2 months. Each question is answered on a categorical scale. The maximum score is calculated by summing the score of each question resulting in a maximum of 100 and a minimum of 0. The higher the score, the better the quality of life.
Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI)
Time frame: Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)
The DLQI questionnaire consists of 10 questions, each answered on a categorical scale. The DLQI is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired.

Results and safety

Results posted to the registry on 2 Nov 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Afamelanotide	16	12

Primary outcome: Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL).

Time frame: Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable). Median (Full Range). Unit: EPP-QoL score

Measure	Afamelanotide n = 13
Day 60	27.8 (-56 to 75)
Day 120	59.7 (11 to 85)
Day 180 or ET	56.9 (8 to 67)

Population analysed: Intent-to-treat population

Primary outcome: Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI)

Time frame: Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable). Median (Full Range). Unit: DLQI score

Measure	Afamelanotide n = 13
Day 60	-5 (-17 to 1)
Day 120	-4.5 (-17 to 0)
Day 180 or ET	-5 (-17 to 0)

Population analysed: Intent-to-treat population

Adverse events

Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Afamelanotide	0 of 16 (0%)	1 of 16 (6.3%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 19 Nov 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Erythropoietic Protoporphyria

Enrolling by invitationPhase 2/3
HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
Erythropoietic Protoporphyria
RecruitingPhase 2/3
A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Active, not recruitingPhase 3
Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
EPP, XLP
RecruitingObservational study
Longitudinal Study of the Porphyrias
Acute Porphyrias, Cutaneous Porphyrias
RecruitingObservational study
Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)
Erythropoietic Protoporphyria (EPP), X-Linked Porphyria (XLP)

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.

Pilar Bilbao study director

Clinuvel Pharmaceuticals Limited

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Afamelanotide in people with Erythropoietic Protoporphyria, run by Clinuvel Pharmaceuticals Limited.

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 3 inclusion and 12 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: finished, as of the sponsor's last update on 19 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.

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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 2 Nov 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL). (time frame: Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04578496.

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 19 Nov 2020.