In a prior study (84-C-0039) oral isotretinoin, administered in high dose (2 mg/kg/day), was effective in preventing the appearance of new skin cancers in patients with xeroderma pigmentosum. The incidence of new skin cancers in patients with the nevoid basal cell carcinoma syndrome also improved. Some patients responded to doses as low as 0.5 mg/kg/day. The purpose of this protocol is to continue to follow those patients previously treated and to study new patients to further define the cancer preventative effects of isotretinoin in these genodermatoses.

Condition
Basal Cell Nevus Syndrome Xeroderma Pigmentosum

Further Study Details: 

Expected Total Enrollment:  30

In a prior study (84-C-0039) oral isotretinoin, administered in high dose (2 mg/kg/day), was effective in preventing the appearance of new skin cancers in patients with xeroderma pigmentosum. The incidence of new skin cancers in patients with the nevoid basal cell carcinoma syndrome also improved. Some patients responded to doses as low as 0.5 mg/kg/day. The purpose of this protocol is to continue to follow those patients previously treated and to study new patients to further define the cancer preventative effects of isotretinoin in these genodermatoses.

Genders Eligible for Study:  Both

Criteria

Patients with autosomal recessive disorder xeroderma pigmentosum or the autosomal dominant NBCC syndrome:
Patients with xeroderma pigmentosum the diagnosis must be documented by the clinical signs and symptoms listed in standard text books: sun sensitivity, increased numbers of freckles and other pigmentary lesions, cutaneous atrophy and telangiectasia, actinic keratoses, and skin cancers. Ocular abnormalities such as photophobia, conjunctivitis, and keratitis or premalignant or malignant tumors of the eye or lids may be present. Some patients may have neurological abnormalities such as progressive hearing loss, diminished reflexes, or progressive mental deterioration.
Patients with the nevoid basal cell carcinoma syndrome the diagnosis must be documented on the basis of clinical signs and symptoms listed in standard text books. These include basal carcinomas, palmar pits, skeletal abnormalities, and calcification of the falx.
Laboratory tests of ultraviolet inducted killing of cultured cells or of DNA repair may be performed.
An accurate assessment of the total number of skin cancers that the patient developed during the past 2 years must be documented by pathology reports.
During 2 years prior to beginning oral isotretinoin therapy, the patient must have had a minimum of two documented skin cancers per year.
Patients must be clear of all skin cancers before beginning oral therapy.
Patients (or parent, guardian, surrogate where appropriate) must give written informed consent.
Patients must be available for and agreeable to regular follow-up examinations in the clinic for clinical evaluation and blood tests.
Patients must be willing and able to participate for the duration of the study (2 or more years of outpatient treatment).
Patients who have not had prior isotretinoin therapy.
Patients must obtain appropriate treatment for skin cancers that arise during the study.
Fertile women must use effective means of birth control during the time of use of isotretinoin and for 1 month after discontinuing treatment.
No evidence of metastatic cancer.
Patients with persistently abnormal (SGOT or SGPT greater than 3 times the upper limit of normal) liver function tests are ineligible.
Patients with persistent pre-treatment hypertriglyceridemia (greater than 300 mg/dl) are ineligible.
Patients with persistently abnormal (creatinine greater than 3 times the upper limit of normal) renal function tests are ineligible.
No proven active malignancy other than skin cancer.
No severe coronary artery disease (Class III-IV criteria of New York Heart Association).
Patients with recent, chronic high dose vitamin A use (greater than 30,000 IU/day) are ineligible.
Patients with hypersensitivity to parabens which are in the soft gelatin capsule of Accutane are ineligible.

Maryland
      National Cancer Institute (NCI), 9000 Rockville Pike,  Bethesda,  Maryland,  20892,  United States

Study ID Numbers:  910161; 91-C-0161
Record last reviewed:  January 11, 2002
Last Updated:  December 16, 2002
Record first received:  May 12, 2000
ClinicalTrials.gov Identifier:  NCT00005661
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-04-08