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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02635789
• Date of registration: 15/12/2015
• Prospective Registration: No
• Primary sponsor: Nobelpharma
• Public title: Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)
• Scientific title: A Double-blind, Randomized, Placebo-controlled Phase III Trial to Investigate the Efficacy and Safety of NPC-12G Gel (Topical Formulation of Sirolimus) to Angiofibroma and Other Skin Lesions in Patients With Tuberous Sclerosis Complex
• Date of first enrolment: December 2015
• Target sample size: 62
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02635789
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Japan

Contacts

• Name: Mari Wataya-Kaneda, MD, PhD
• Affiliation: Department of Dermatology, Graduate School of Medicine, Osaka University

Key inclusion & exclusion 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



Age minimum: 3 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Hypomelanotic Macule

Plaque

Angiofibroma

Tuberous Sclerosis

Intervention(s)

Drug: Placebo gel

Drug: NPC-12G gel

Primary Outcome(s)

Improvements in angiofibroma [Time Frame: 12 weeks]

Secondary Outcome(s)

Sirolimus blood concentration [Time Frame: Baseline, Week 4 and Week 12]

Adverse events [Time Frame: 16 weeks]

Improvements in angiofibroma [Time Frame: Week 4, 8, 12 and follow-up Week 16]

Improvements in redness of angiofibroma [Time Frame: Week 4, 8, 12 and follow-up Week 16]

Serious adverse events [Time Frame: 16 weeks]

The rate of patients who are evaluated as ''improvement'' or more (improvement rate) in primary outcome measure and in secondary outcome measures above outcome 1 to 5 [Time Frame: Week 4, 8, 12 and follow-up Week 16]

Laboratory findings [Time Frame: 16 weeks]

Change in total score from baseline for DLQI and CDLQI [Time Frame: Week 4, 8, 12 and follow-up Week 16]

Improvements in angiofibroma [Time Frame: Week 4 and 8 and follow-up Week 16]

Improvements in hypomelanotic macule and plaque of upper neck [Time Frame: Week 4, 8, 12 and follow-up Week 16]

Vital sign [Time Frame: 16 weeks]

Secondary ID(s)

NPC-12G-1

Source(s) of Monetary Support

Please refer to primary and secondary sponsors