A randomized, double-blind, vehicle-controlled, multicenter trial of topically administered LDE225 cream [0.75% bid] to evaluate clearance of Basal Cell Carcinoma in adult patients with Nevoid Basal Cell Carcinoma Syndrome - ND

Active, not recruiting

• Conditions: Basal Cell Carcinoma in adult patients with Nevoid Basal Cell Carcinoma Syndrome; MedDRA version: 12.1Level: LLTClassification code 10004146Term: Basal cell carcinoma

• Registration Number: EUCTR2010-019856-30-IT
• Lead Sponsor: OVARTIS FARMA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-06-21
• Last Update Posted: 10 February 2014

Recruitment & Eligibility

• Status: Not Recruiting
• Sex: All
• Target Recruitment: 109

Inclusion Criteria

1 to 6 must be met at the Screening Visit. Criteria 7 and 8 must be met at the Baseline Visit. 1. Written informed consent, which includes consent for PTCH1 genetic mutation testing, consent for at least 3 and up to 12 2-mm punch biopsies, and three surgical excisions must be obtained before any study-related assessment is performed. 2. Male or female patient, of any race or ethnicity, at least 18 years of age. 3. Patient is able to understand and communicate with the investigator and comply with the requirements of the study. If not, a caregiver or legal representative who is able to understand, communicate with the investigator and comply with the requirements of the study must be present at all visits. 4. Typical presentation of NBCCS in the opinion of the investigator, based on clinical criteria such as Kimonis et al (Kimonis et al 1997). 5. At least one of the criteria below characteristic of NBCCS: • Odontogenic keratocysts of the jaws • Palmar or plantar pits (3 or more in any combination) • Bilamellar calcification of the falx cerebri • Bifid, fused or markedly splayed ribs • First degree relatives with NBCCS (a biologically related parent, sibling or child) • PTCH1 mutation identified in DNA from non-tumor tissue. 6. Presence of multiple BCCs at the Screening Visit (and the Rescreening Visit, if applicable) to be biopsied, including: o At least three BCCs that appear to be of superficial type clinically of at least (=) 8 mm and less (<) than 20 mm in their longest dimension, that are located in a body location in which a surgical excision with a peri-tumoral margin of approximately 4 mm is technically feasible. These BCCs must not be recurring tumors, and must not have been previously treated (and must not be treated until Baseline). There must be at least 20 mm healthy looking surrounding skin bordering each tumor that will be biopsied. 7. Presence of multiple BCCs at the Baseline Visit, including: a. At least two superficial BCCs confirmed histologically from tumors biopsied at the Screening/Rescreening Visits. At the Baseline Visit, the longest dimension of each superficial BCC (Target BCC) must be less (<) than 20 mm and must not have decreased from the measurement prior to the biopsy performed during the screening period. The Target BCCs must not have been treated between Screening and Baseline. b. Preferably one superficial and partly nodular BCC confirmed histologically (Nodular BCC) from the tumors biopsies at the Screening/Rescreening Visits. At the Baseline Visit, the longest dimension of the Nodular BCC must be less (<) than 20 mm and must not have decreased from the measurement prior to the biopsy performed during the screening period. The Nodular BCC must not have been treated between Screening and Baseline. c. Preferably up to 8 Additional superficial BCCs of less (<) than 20 mm at Baseline in their longest dimension. These BCCs must clinically appear to be of superficial type, and must not be recurring tumors, and must not have been previously treated. If they have been biopsied, the type can be either of superficial or superficial and partly nodular histologically. 8. The BCCs where biopsies were taken at the Screening (or Rescreening) Visit and that will be treated during the study must be completely healed at the Baseline Visit.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for

Exclusion Criteria

Patients fulfilling any of the following criteria are not eligible for inclusion in this study: 1. BCCs to be treated must not be: superficial BCCs = 20 mm, any type of BCC other than superficial or superficial and partly nodular, including sclerodermiform, clinically unclear diagnosis of BCC, such as hyperkeratotic lesion that could correspond to a metatypic BCC or Squamous Cell Carcinoma or Actinic Keratosis. 2. Any concomitant dermatological disease that could confound the evaluations, based on the discretion of the investigator. 3. Participation in a prior LDE225 study in which active LDE225 was a treatment arm. The patient can not have been exposed to LDE225 prior to this study. 4. Use of systemic treatment for BCC (e.g., retinoid, chemotherapy, celecoxib, …) in the 4 weeks prior to Baseline. 5. Use of any topical treatment (imiquimod or 5-fluorouracil (5-FU)) in the Treated Area of any of the BCCs (area of BCC + 10 mm surrounding BCC) to be treated in the 12 weeks prior to Baseline. 6. Use of PDT in the Treated Area (area of BCC + 10 mm surrounding skin) of any of the BCCs in the 12 weeks prior to Baseline. 7. Use of other investigational drugs at Baseline, or within 30 days or 5 half-lives prior to Baseline, whichever is longer. 8. Clinically significant medical condition, as per judgment of the investigator, including metastatic BCC. 9. History of hypersensitivity to any of the ingredient of the study drug. 10. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive ßhCG laboratory test (> 5 mIU/mL). 11. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, including women whose career, lifestyle, or sexual orientation precludes intercourse with a male partner and women whose partners have been sterilized by vasectomy or other means, UNLESS they are using two birth control methods from the time they sign the informed consent form. The double method contraception can be a double-barrier method or a barrier method plus a hormonal method. o Adequate barrier methods of contraception include: diaphragm, condom (by the partner), intrauterine device (copper or hormonal), sponge or spermicide. Hormonal contraceptives include any marketed contraceptive agent that includes an estrogen and/or a progestational agent. o Reliable contraception should be maintained throughout the treatment period and for at least 6 weeks after study drug discontinuation. o Woman are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or six months of spontaneous amenorrhea with serum FSH levels > 40 mIU/mL [for US only: and estradiol < 20 pg/mL] or have had surgical bilateral oophorectomy (with or without hysterectomy) at least six weeks ago. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment. 12. Fertile males, defined as all males physiologically capable of conceiving offspring UNLESS the patient and his partner agree to comply with acceptable double method contraception defined above from the time the male patient signs the informed consent form. Double-method contraception is not required if the partne

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified