Phase I Study of Isotretinoin in Patients With Recessive Dystrophic Epidermolysis Bullosa

This study is ongoing, but not recruiting participants.

First Received: April 10, 2001 Last Updated: June 23, 2005 History of Changes

Sponsors and Collaborators:	FDA Office of Orphan Products Development University of North Carolina
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00014729

Purpose

OBJECTIVES:

I. Determine the safety of isotretinoin in patients with recessive dystrophic epidermolysis bullosa.

Condition	Intervention	Phase
Epidermolysis Bullosa	Drug: isotretinoin	Phase I

Study Type:	Interventional
Study Design:	Treatment

Resource links provided by NLM:

Genetics Home Reference related topics: dystrophic epidermolysis bullosa epidermolysis bullosa simplex

Drug Information available for: Isotretinoin

U.S. FDA Resources

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment:	20
Study Start Date:	October 2000

Detailed Description:

PROTOCOL OUTLINE:

Patients receive oral isotretinoin daily for 8 months in the absence of disease progression or unacceptable toxicity.

Eligibility

Ages Eligible for Study:	15 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) RDEB Hallopeau-Siemens OR RDEB non-Hallopeau-Siemens
Concurrent enrollment in the National Epidermolysis Bullosa Registry
No regional or distant metastasis in patients with previous or concurrent squamous cell carcinoma

--Patient Characteristics--

Hepatic: No clinically significant hypertriglyceridemia No clinically significant hepatic dysfunction
Renal: No clinically significant renal dysfunction
Other: Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00014729

Sponsors and Collaborators

FDA Office of Orphan Products Development

University of North Carolina

Investigators

Study Chair:

Jo-David Fine

University of North Carolina

More Information

No publications provided

Study ID Numbers:	199/15738, UNCCH-FDR001796
Study First Received:	April 10, 2001
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00014729 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:

dermatologic disorders
epidermolysis bullosa
genetic diseases and dysmorphic syndromes
rare disease

Study placed in the following topic categories:

Skin Diseases
Skin Diseases, Vesiculobullous
Collagen Diseases
Rare Diseases
Skin Abnormalities
Epidermolysis Bullosa
Dystrophic Epidermolysis Bullosa

Genetic Diseases, Inborn
Connective Tissue Diseases
Isotretinoin
Epidermolysis Bullosa Dystrophica
Congenital Abnormalities
Skin Diseases, Genetic

Additional relevant MeSH terms:

Skin Diseases
Skin Diseases, Vesiculobullous
Collagen Diseases
Skin Abnormalities
Epidermolysis Bullosa
Pharmacologic Actions
Genetic Diseases, Inborn

Therapeutic Uses
Connective Tissue Diseases
Isotretinoin
Epidermolysis Bullosa Dystrophica
Congenital Abnormalities
Skin Diseases, Genetic
Dermatologic Agents

ClinicalTrials.gov processed this record on July 02, 2009