Alternate Title
Basic Trial Information
Objectives
Entry Criteria
Expected Enrollment
Outline
Published Results
Trial Contact Information
Registry Information

Alternate Title

Isotretinoin in Preventing Skin Cancer

Basic Trial Information

Phase	Type	Status	Age	Sponsor	Protocol IDs
No phase specified	Prevention	Completed	Over 2	NIAMS	NIAMS-91-AR-0161 NCI-91-C-0161, NCT00025012

Objectives

I. Determine the lowest effective dose of oral isotretinoin for long-term 
therapy that is capable of reducing the rate of formation of new skin cancers 
in patients with xeroderma pigmentosum or nevoid basal cell carcinoma syndrome.

II. Determine the possible side effects associated with long-term use of 
isotretinoin in this patient population.

Entry Criteria

Disease Characteristics:

Diagnosis of autosomal recessive disorder xeroderma pigmentosum 
 Diagnosis must be documented by the clinical signs and symptoms listed in
  standard text books, e.g.:
   Sun sensitivity
   Increased number of freckles and other pigmentary lesions
   Cutaneous atrophy and telangiectasia
   Actinic keratoses
   Skin cancers
 Ocular abnormalities (e.g., photophobia, conjunctivitis, keratitis, or
  premalignant or malignant tumors of the eye or lid) allowed
 Neurologic abnormalities (e.g., progressive hearing loss, diminished
  reflexes, or progressive mental deterioration) allowed

OR

Diagnosis of autosomal dominant nevoid basal cell carcinoma syndrome
 Diagnosis must be documented by the clinical signs and symptoms listed in
  standard text books, e.g.:
   Basal cell carcinomas
   Palmar pits
   Skeletal abnormalities
   Falx calcification

History of at least 2 documented skin cancers a year during the 2 years before
study, but currently clear of all skin cancer 

Patients not previously treated with isotretinoin must agree to undergo a 1
year follow-up period without isotretinoin (to facilitate observation of any
chronic toxicity and observe for new tumors) 

Must undergo appropriate treatment for any skin cancers that arise during
study 

No evidence of metastatic cancer

Prior/Concurrent Therapy:

See Disease Characteristics

No recent, chronic, high-dose vitamin A use (greater than 30,000 IU/day)

No concurrent supplemental vitamin A

No other concurrent therapy for the skin (except sunscreens) unless approved
by the investigators 

Patient Characteristics:

Age:
 Over 2

Hematopoietic:
 Complete blood cell counts normal

Hepatic:
 SGOT or SGPT less than 3 times upper limit of normal (ULN)
 Triglycerides less than 200 mg/dL

Renal:
 Creatinine less than 3 times ULN

Cardiovascular:
 No New York Heart Association class III or IV heart disease

Other:
 No hypersensitivity to parabens (used in drug formulation)
 No proven active malignancy except skin cancer
 Not pregnant
 Negative pregnancy test
 Fertile patients must use effective contraception during and for 1 month
  after study therapy

Expected Enrollment

Approximately 30 patients will be accrued for this study.

Outline

Patients are stratified according to disease type (xeroderma pigmentosum vs 
nevoid basal cell carcinoma syndrome).

Patients not previously treated with isotretinoin receive oral isotretinoin 
daily for 2 years and then are followed without receiving isotretinoin for 1 
year. After the follow-up period, treatment may be resumed if the rate of new 
skin tumor formation reaches 2 per year provided original eligibility criteria 
are met. Treatment may be resumed during the follow-up period if the rate of 
new skin tumor formation increases to the rate observed before study.

Patients previously treated with oral isotretinoin continue treatment and are 
followed to evaluate any long-term effects of treatment.

Kraemer KH, DiGiovanna JJ, Moshell AN, et al.: Prevention of skin cancer in xeroderma pigmentosum with the use of oral isotretinoin. N Engl J Med 318 (25): 1633-7, 1988.[PUBMED Abstract]

Trial Contact Information

Trial Lead Organizations

National Institute of Arthritis and Musculoskeletal and Skin Diseases

John DiGiovanna, MD, Protocol chair		Ph: 301-402-1607 Email: jdigiova@mail.nih.gov

Registry Information
Official Title		Use Of Isotretinion For Prevention Of Skin Cancer In Patients With Xeroderma Pigmentosum Or Nevoid Basal Cell Carcinoma Syndrome
Trial Start Date		1991-06-01
Registered in ClinicalTrials.gov		NCT00025012
Date Submitted to PDQ		1991-06-01
Information Last Verified		2007-11-08

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