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

NCT02635789 | Completed Phase 3

• 1 other identifier: NPC-12G-1
• Study Type: interventional
• Target: 62
• Locations: 1 country
• Sites: 1

Brief 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)

Conditions

Tuberous Sclerosis; Angiofibroma; Hypomelanotic Macule; Plaque

Keywords

Sirolimus; Skin lesions; TSC; Tuberous Sclerosis Complex; NPC-12G; Angiofibroma; Hypomelanotic macule; Plaque

Outcome Measures

Primary Outcomes (1)

• Improvements in angiofibroma

  Improvements comparing with baseline is assessed using photograph by the central photo-judgement committee

  12 weeks

Secondary Outcomes (11)

• Improvements in angiofibroma

  Week 4 and 8 and follow-up Week 16

• Improvements in angiofibroma

  Week 4, 8, 12 and follow-up Week 16

• Improvements in redness of angiofibroma

  Week 4, 8, 12 and follow-up Week 16

• Improvements in hypomelanotic macule and plaque of upper neck

  Week 4, 8, 12 and follow-up Week 16

• 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

  Week 4, 8, 12 and follow-up Week 16

Study Arms (2)

NPC-12G gel

EXPERIMENTAL

NPC-12G gel is containing 0.2% Sirolimus

Drug: NPC-12G gel

Placebo gel

PLACEBO COMPARATOR

Placebo gel is matched ingredient with NPC-12G gel

Drug: Placebo gel

Interventions

NPC-12G gel DRUG

NPC-12G gel is administered topically twice a day for 12 weeks

NPC-12G gel

Placebo gel DRUG

NPC-12G gel placebo is administered topically twice a day for 12 weeks

Placebo gel

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patients 3 years old or greater at the time of informed consent
• Patients who are diagnosed as definite diagnosis according to diagnostic criteria for tuberous sclerosis complex (International Tuberous Sclerosis Complex Consensus Conference 2012)
• Patients with three or more papules of angiofibroma ( \>= 2 mm in diameter with redness in each) on the face at screening tests
• Patients who are not suitable for therapy with laser or surgery, or who do not want therapy with laser or surgery
• 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

You may not qualify if:

• Patients who are hard to apply the test drug topically with keeping compliance
• 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
• Patients who are hard to be taken pictures of their lesions adequately in such cases that they may not follow instruction of stillness
• Patients with a history of hypersensitivity to alcohol or allergy to sirolimus
• 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''
• 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.
• Patients who have taken drugs with mTOR inhibitory action including sirolimus, everolimus or temsirolimus within 12 months before the initial registration
• Patients who have applied topical tacrolimus on the lesion of angiofibroma within 3 months before the initial registration
• Patients who have received therapy with laser or surgery to the lesion of angiofibroma within 6 months before the initial registration
• Female patients who may be pregnancy or are lactating
• 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
• Patients who have participated in other clinical trial and have taken a trial drug within 6 months before the initial registration
• Others, patients who are considered by the investigator as unsuitable for participation in the trial

Sponsors & Collaborators

• Nobelpharma: lead

Study Sites (1)

Graduate School of Medicine, Osaka University

Suita, Osaka, 565-0871, Japan

Location

Related Publications (1)

• Wataya-Kaneda M, Ohno Y, Fujita Y, Yokozeki H, Niizeki H, Ogai M, Fukai K, Nagai H, Yoshida Y, Hamada I, Hio T, Shimizu K, Murota H. Sirolimus Gel Treatment vs Placebo for Facial Angiofibromas in Patients With Tuberous Sclerosis Complex: A Randomized Clinical Trial. JAMA Dermatol. 2018 Jul 1;154(7):781-788. doi: 10.1001/jamadermatol.2018.1408.

  PMID: 29800026 DERIVED

MeSH Terms

Conditions

Tuberous Sclerosis; Angiofibroma; Plaque, Amyloid

Condition Hierarchy (Ancestors)

Hamartoma; Neoplasms; Neoplasms, Multiple Primary; Neoplastic Syndromes, Hereditary; Malformations of Cortical Development, Group I; Malformations of Cortical Development; Nervous System Malformations; Nervous System Diseases; Neurocutaneous Syndromes; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Neoplasms, Vascular Tissue; Neoplasms by Histologic Type; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Study Officials

• Mari Wataya-Kaneda, MD, PhD

  Department of Dermatology, Graduate School of Medicine, Osaka University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 15, 2015
• First Posted: December 21, 2015
• Study Start: December 1, 2015
• Primary Completion: October 1, 2016
• Study Completion: October 1, 2016
• Last Updated: February 2, 2017

Record last verified: 2017-02

Locations

JP (1)