Clinical Trials Logo

Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01605136
Other study ID # CUV039
Secondary ID
Status Completed
Phase Phase 3
First received
Last updated
Start date May 2012
Est. completion date July 2013

Study information

Verified date September 2019
Source Clinuvel Pharmaceuticals Limited
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This is a randomized placebo-controlled study to be conducted in two parallel study arms for a six month period (three doses). Between 75 and 100 eligible patients will be enrolled. Patients will receive afamelanotide (16 mg implants) or placebo according to the following dosing regimen:

- Group A will be administered afamelanotide implants on Days 0, 60 and 120

- Group B will be administered placebo implants on Days 0, 60 and 120

The number and severity of phototoxic reactions, the type and duration of sun exposure, treatment-emergent adverse events and the use of concomitant medication will be recorded by patients in study diaries between Days 0 and 180. Quality of life will be measured using the DLQI and EPP-QoL at Days 0, 60, 120 and 180. Participants will visit the clinic on Days 60, 120 and 180 for assessments of adverse events.

A subset of patients will be photoprovoked on the lower back and dorsal surface of the hand and the minimal symptom dose (MSD) will be determined on Days 0, 30, 60, 90 and 120.


Description:

Afamelanotide is a man-made drug being studied for use as a preventative medication for Erythropoietic Protoporphyria (EPP) sufferers. It is a synthetically produced analogue of human alpha melanocyte stimulating hormone (alpha-MSH) and is available in Europe.

The purpose of this study is to look at the type and duration of sun exposure when patients are exposed to light. This study will also look at how the drug is tolerated when taken by people with EPP.

The study will involve the use of an implant, which comes in the form of a small rod to be administered under the skin. The implant may contain the study drug afamelanotide or a placebo (inactive medication).

Over 620 subjects have been treated with afamelanotide to date with no serious safety concerns identified. For this study, afamelanotide has been formulated as a controlled release depot injection (implant). This means that the afamelanotide will be released slowly into the body over a few days.

This study aims to confirm the photoprotective properties if afamelanotide demonstrated in the earlier Phase II and phase III studies.


Recruitment information / eligibility

Status Completed
Enrollment 93
Est. completion date July 2013
Est. primary completion date July 2013
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria:

- Male or female subjects with characteristic symptoms of EPP phototoxicity and a biochemically-confirmed diagnosis of EPP.

- Aged 18 years old and above (inclusive).

- Able to understand and sign the written Informed Consent Form.

- Willing to take precautions to prevent pregnancy until completion of the study (Day 180).

Exclusion Criteria:

- Any allergy to afamelanotide or the polymer contained in the implant or to lidocaine or other local anesthetic 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 other photodermatosis such as polymorphic light eruption, actinic prurigo, discoid lupus erythematosus, chronic actinic dermatitis or solar urticaria.

- Any evidence of clinically significant organ dysfunction or any clinically significant deviation from normal in the clinical or laboratory determinations.

- Acute history of drug or alcohol abuse (in the last 6 months).

- Patient assessed as not suitable for the study in the opinion of the Investigator (e.g. noncompliance history, allergic to local anesthetics, faints when given injections or giving blood).

- 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, including drugs that cause photosensitivity or skin pigmentation.

- Female who is pregnant (confirmed by positive serum ß-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).

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Afamelanotide
One 16mg subcutaneous implant every 2 months for 6 months.
Placebo
One placebo subcutaneous implant every 2 months for 6 months

Locations

Country Name City State
United States University of Alabama Birmingham Alabama
United States Carolina's Medical Center Cannon Research Charlotte North Carolina
United States Henry Ford Medical Center Detroit Michigan
United States University of Texas Galveston Texas
United States Mt. Sinai New York New York
United States University of Utah Salt Lake City Utah
United States University of California, San Francisco San Francisco California

Sponsors (1)

Lead Sponsor Collaborator
Clinuvel Pharmaceuticals Limited

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Duration of Direct Sunlight Exposure Between 10:00 and 18:00 Hours on Days When no Pain Was Experienced (Pain Score of 0). The amount of direct sunlight exposure between 10:00 and 18:00 hours on days when no pain was experienced (e.g.11-point Likert pain score of 0). Time was recorded in a patient diary using 15 minute time blocks.
The pain score is measured by the 11-point Likert Pain scale with minimum of 0 and maximum of 10.
Likert Pain scale of 0 represents no pain and 10 represents worst imaginable pain.
Daily for 6 months
Secondary Combined Sun Exposure and Phototoxic Pain Time in direct sunlight exposure between 10:00 and 18:00 hours on days when no or mild pain was experienced (Likert scores of 0 to 3).
The pain score is measured by the 11-point Likert Pain scale with minimum of 0 and maximum of 10.
Likert Pain scale of 0 represents no pain and 10 represents worst imaginable pain.
Daily for 6 months
Secondary Sun Exposure Duration of direct sunlight exposure between 10:00 and 18:00 hours during the study. Daily for 6 months
Secondary Quality of Life Score The Quality of life of participant is measured using DLQI and EPP QoL. The Dermatology Life Quality Index (DLQI) is a simple practical measure for routine clinical use.
The DLQI ranges from 0 (no impact on life) to 30 (significant impact on life) . The Erthropoietic protoporphyria quality of life measure (EPP-QoL) scores range from 0 (worst imaginable QoL) to 100 (best possible QoL).
Day 60, Day 120, and Day 180 or early termination.
Secondary Photoprovocation A subset of subjects was photoprovoked on the dorsal surface of the hand (predilection place) and lower back and the minimum symptom dose (MSD) determined on Days 0, 30, 60, 90 and 120.
The amount of radiation required to provoke the first clinical symptom was recorded.
Day 0, Day 30, Day 60, Day 90 and Day 120.
Secondary Maximum Severity of Phototoxic Reaction Experienced by Participants The phototoxicity - phototoxic pain secondary endpoint has been divided into two secondary outcome measures.
The days on which the participant experienced pain as a result of phototoxic reactions (caused by exposure to natural light) was recorded in a study diary. On each day such a reaction occurred, the participant scored the level of pain using an 11-point Likert pain scale, with minimum of 0 and maximum of 10. The 11-point Likert pain scale with a value of 0 represents no pain and 10 represents worst imaginable pain.
The maximum severity of a phototoxic reaction was determined by the highest daily 11-point Likert scale score that occurred during that phototoxic reaction.
Daily for 6 months
Secondary Total Number Phototoxic Reactions Experienced by Participants The phototoxicity - phototoxic pain secondary endpoint has been divided into two secondary outcome measures.
The number of episodes was the endpoint. The days on which the participant experienced pain as a result of phototoxic reactions (caused by exposure to natural light) was recorded in a study diary. On each day such a reaction occurred, the participant scored the level of pain using an 11-point Likert pain scale, with minimum of 0 and maximum of 10. The 11-point Likert Pain scale with a value of 0 represents no pain and 10 represents worst imaginable pain.
The number of phototoxic reactions was determined by counting the number of episodes on which participants report a 11-point Likert scale score of 4 or more for one or more consecutive days.
Daily for 6 months
See also
  Status Clinical Trial Phase
Completed NCT00979745 - Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP) Phase 3
Active, not recruiting NCT05308472 - Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP Phase 2
Completed NCT00004831 - Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria N/A
Completed NCT05572125 - Iron Therapy in Erythropoietic Protoporphyria
Completed NCT01422915 - Sorbent Therapy of the Cutaneous Porphyrias Phase 2/Phase 3
Completed NCT04053270 - Multicentre Phase III Erythropoietic Protoporphyria Study Phase 3
Completed NCT00004940 - Phase III Study of L-Cysteine in Patients With Erythropoietic Protoporphyria Phase 3
Recruiting NCT06388642 - Pharmacokinetics of Afamelanotide in EPP Patients Phase 1/Phase 2
Recruiting NCT05020184 - Effect of Oral Cimetidine in the Protoporphyrias Phase 2
Completed NCT01688895 - Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact
Completed NCT04578496 - A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP) Phase 3
Recruiting NCT00206869 - Does Exercise and Heat Increase the Lightsensibility in Patients With Erythropoietic Protoporphyria N/A
Recruiting NCT05780840 - Protection Against Visible Light by Dihydroxyacetone in Erythropoietic Protoporphyria N/A
Completed NCT02979249 - Oral Iron for Erythropoietic Protoporphyrias N/A
Enrolling by invitation NCT05883748 - Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP Phase 2/Phase 3
Completed NCT01097044 - Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP) Phase 2
Completed NCT03682731 - Light Exposure Patterns and Symptoms Among Patients With Erythropoietic Protoporphyria