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Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Clinuvel Pharmaceuticals Limited
ClinicalTrials.gov Identifier:
NCT01097044
First received: March 30, 2010
Last updated: March 21, 2013
Last verified: March 2013
History of Changes
  Purpose

This is a randomized placebo-controlled study to be conducted in two parallel study arms for a six month period (three doses). Approximately 10 eligible patients per center will be enrolled and 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

To determine eligibility for study inclusion, patients will undergo a screening evaluation 7 to 14 days prior to the administration of the first dose. The number and severity of phototoxic reactions will be determined Days 60, 120, and 180. Quality of life will be measured every 7 days, beginning at Day 0 until Day 180. Participants will visit the clinic on Days 60, 120 and 180 for assessments of adverse events.


Condition Intervention Phase
Erythropoietic Protoporphyria
 Drug: Afamelanotide
Drug: Placebo
 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase II, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients With Erythropoietic Protoporphyria (EPP)

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Clinuvel Pharmaceuticals Limited:

Primary Outcome Measures:
  • Severity of phototoxic reaction measured by visual analogue scale [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Number of phototoxic reactions [ Time Frame: 6 months ] [ Designated as safety issue: No ]
  • Quality of life measured by patient completed questionnaire [ Time Frame: 6 months ] [ Designated as safety issue: No ]
  • Free protoporphyrin IX level [ Time Frame: 6 months ] [ Designated as safety issue: No ]
  • Treatment emergent adverse events [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]

Enrollment: 77
Study Start Date: April 2010
Study Completion Date: April 2011
Primary Completion Date: April 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Afamelanotide Drug: Afamelanotide
One 16mg subcutaneous implant every 2 months for 6 months.
Placebo Comparator: Placebo Drug: Placebo
One placebo subcutaneous implant every 2 months for 6 months

Detailed Description:

Afamelanotide is a man-made drug being studied for use as a preventative medication for EPP sufferers. It is a synthetically produced analogue of human alpha melanocyte stimulating hormone (alpha-MSH) and is not yet available on the market.

The purpose of this study is to look at whether afamelanotide can reduce the number and severity of EPP symptoms 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 450 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. Once inserted, the implant will remain in the body after afamelanotide has been released and will slowly dissolve.

This study will help to provide more information about afamelanotide. This information will be used to determine the safety and efficacy (the ability of the drug to produce an effect) of this drug in EPP sufferers.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female subjects with characteristic photosensitivity of EPP symptoms and positive diagnosis of EPP confirmed by laboratory result of elevated total protoporphyrin IX.
  • 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 PLE, DLE 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 within 60 days prior to the screening visit.
  • Female who is pregnant (confirmed by positive serum β-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).
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01097044

Locations
United States, Alabama
University of Alabama
Birmingham, Alabama, United States, 35294
United States, California
University of California, San Francisco
San Francisco, California, United States, 94143
United States, New York
Mt. Sinai
New York, New York, United States, 10029
United States, North Carolina
Carolina's Medical Center Cannon Research
Charlotte, North Carolina, United States, 29203
United States, Texas
University of Texas
Galveston, Texas, United States, 77555
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84132
Sponsors and Collaborators
Clinuvel Pharmaceuticals Limited
Investigators
Principal Investigator: Robert Desnick, MD Mt. Sinai
  More Information

No publications provided

Responsible Party: Clinuvel Pharmaceuticals Limited
ClinicalTrials.gov Identifier: NCT01097044     History of Changes
Other Study ID Numbers: CUV030
Study First Received: March 30, 2010
Last Updated: March 21, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Clinuvel Pharmaceuticals Limited:
Erythropoietic Protoporphyria
EPP
Afamelanotide

Additional relevant MeSH terms:
Protoporphyria, Erythropoietic
Porphyrias, Hepatic
Liver Diseases
Digestive System Diseases
Porphyrias
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Skin Diseases, Genetic
 Skin Diseases
Skin Diseases, Metabolic
Metabolic Diseases
Alpha-MSH
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 19, 2013