Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex

NCT02634931 | Completed Phase 3

• 1 other identifier: NPC-12G-2
• Study Type: interventional
• Target: 94
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this trial is to evaluate the safety and efficacy of long-term treatment with NPC-12G gel (0.2% sirolimus gel) to angiofibroma and other skin lesions in patients with tuberous sclerosis complex in the open-label trial.

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)

• The discontinuation rate due to adverse events

  The first discontinuation in each patient due to adverse events is assessed.Completion of week 26 and 52 are cut-off points for interim-analyses by Kaplan-Meier method

  52 weeks and longer

Secondary Outcomes (14)

• Adverse events and adverse events related to the test drug

  52 weeks and longer

• Adverse events related to the test drug leading to the discontinuation permanently

  52 weeks and longer

• Serious adverse events and serious adverse events related to the test drug

  52 weeks and longer

• Adverse events and adverse events related to the test drug leading to modification of dosage and administration

  52 weeks and longer

• Significant adverse events and significant adverse events related to the test drug

  52 weeks and longer

Study Arms (1)

NPC-12G gel

EXPERIMENTAL

NPC-12G gel is containing 0.2% Sirolimus

Drug: NPC-12G gel

Interventions

NPC-12G gel DRUG

NPC-12G gel is administered topically twice a day for 52 weeks or longer

NPC-12G 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 skin lesions such as angiofibroma, white macules or plaque upper neck associated with tuberous sclerosis complex at the screening visit or the baseline visit
• Patients or his/her guardian who agree to use the test drug (NPC-12G gel) or who want to participate in the trial again following participation in Phase III trial (NPC-12G-1)
• Patient who are considered to be an appropriate patient to participate in the trial by investigator
• 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 have offered to withdraw from Phase III trial (NPC-12G-1) and have been discontinued
• Patients who have not applied the test drug topically more than 25% of whole applications without appropriate reason for Phase III trial (NPC-12G-1)
• 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 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.
• Female patients who may be pregnancy or are lactating
• Patients who cannot agree to take appropriate measures of contraception until completion of the trial or follow-up period after discontinuation from informed consent
• Patients who have participated in other clinical trial other than Phase III trial (NPC-12G-1) and have taken a trial drug within 6 months before informed consent
• 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, Nagai H, Ohno Y, Yokozeki H, Fujita Y, Niizeki H, Yoshida K, Ogai M, Yoshida Y, Asahina A, Fukai K, Tateishi C, Hamada I, Takahata T, Shimizu K, Shimasaki S, Murota H. Safety and Efficacy of the Sirolimus Gel for TSC Patients With Facial Skin Lesions in a Long-Term, Open-Label, Extension, Uncontrolled Clinical Trial. Dermatol Ther (Heidelb). 2020 Aug;10(4):635-650. doi: 10.1007/s13555-020-00387-7. Epub 2020 May 8.

  PMID: 32385845 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: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 16, 2015
• First Posted: December 18, 2015
• Study Start: December 1, 2015
• Primary Completion: October 1, 2018
• Study Completion: October 1, 2018
• Last Updated: February 20, 2019

Record last verified: 2015-12

Locations

JP (1)