Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)

• Status: Completed
• Trial end date: 2016-10-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this trial is to evaluate the efficacy and safety of NPC-12G gel (topical formulation of sirolimus) versus placebo gel to facial angiofibroma and other skin lesions in patients with tuberous sclerosis complex (TSC)

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Nobelpharma
• Treatments: Everolimus; Sirolimus

Criteria

Inclusion Criteria:

1. Male or female patients 3 years old or greater at the time of informed consent

2. Patients who are diagnosed as definite diagnosis according to diagnostic criteria for tuberous sclerosis complex (International Tuberous Sclerosis Complex Consensus Conference 2012)

3. Patients with three or more papules of angiofibroma ( >= 2 mm in diameter with redness in each) on the face at screening tests

4. Patients who are not suitable for therapy with laser or surgery, or who do not want therapy with laser or surgery

5. Patients or his/her guardian who give a written informed consent in understanding and willingness after having received enough explanation of the test drug and the current trial plan

Exclusion Criteria:

1. Patients who are hard to apply the test drug topically with keeping compliance

2. Patients with clinical findings such as erosion, ulcer and eruption on or around the lesion of angiofibroma, which may affect assessment of safety or efficacy

3. Patients who are hard to be taken pictures of their lesions adequately in such cases that they may not follow instruction of stillness

4. Patients with a history of hypersensitivity to alcohol or allergy to sirolimus

5. Patients who have complications such as malignant tumor, infection, serious heart disease, hepatic function disorder, renal function disorder or blood disorders which severity are considered by investigator as grade 2 or more severe with reference to ''Concerning classification criteria for seriousness of adverse drug reactions of medical agents''

6. Patients who have complications such as diseases unsuitable for the trial participation, for examples, uncontrolled diabetes (fasting blood glucose level >140 mg/dL or postprandial blood glucose level > 200 mg/dL), dyslipidemia (cholesterol level > 300 mg/dL or > 7.75 mmol/L, triglycerides level > 300 mg/dL or > 3.42 mmol/L), etc.

7. Patients who have taken drugs with mTOR inhibitory action including sirolimus, everolimus or temsirolimus within 12 months before the initial registration

8. Patients who have applied topical tacrolimus on the lesion of angiofibroma within 3 months before the initial registration

9. Patients who have received therapy with laser or surgery to the lesion of angiofibroma within 6 months before the initial registration

10. Female patients who may be pregnancy or are lactating

11. Patients who cannot agree to take appropriate measures of contraception until completion of post-treatment phase or follow-up period after discontinuation from informed consent

12. Patients who have participated in other clinical trial and have taken a trial drug within 6 months before the initial registration

13. Others, patients who are considered by the investigator as unsuitable for participation in the trial