Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC

NCT05495425 | Completed Phase 3

• 1 other identifier: NPC-12Y-1
• Study Type: interventional
• Target: 43
• Locations: 1 country
• Sites: 5

Brief Summary

The purpose of this study is to investigate the efficacy and safety of NPC-12Y gel compared with placebo for skin lesions associated with tuberous sclerosis.

Conditions

Tuberous Sclerosis Complex

Keywords

Tuberous sclerosis complex; Sirolimus; Rapalimus gel; mTOR inhibitor

Outcome Measures

Primary Outcomes (1)

• Improvements in angiofibroma

  Improvements comparing with baseline is assessed using photograph by IRC

  12 weeks

Secondary Outcomes (2)

• Improvements in angiofibroma, color and size

  12 weeks

• Index of Facial Angiofibromas (IFA) score

  12 weeks

Study Arms (2)

NPC-12Y gel

EXPERIMENTAL

NPC-12Y gel is containing 0.2% Sirolimus

Drug: NPC-12Y gel

NPC-12Y placebo gel

PLACEBO COMPARATOR

Placebo gel matched NPC-12Y gel

Drug: NPC-12Y placebo gel

Interventions

NPC-12Y gel DRUG

NPC-12Y gel is applied twice a day for 12 weeks. After the double-blind evaluation period, NPC-12Y gel is applied twice a day for 52 weeks.

NPC-12Y gel

NPC-12Y placebo gel DRUG

NPC-12Y placebo gel is applied twice a day for 12 weeks. After the double-blind evaluation period, NPC-12Y gel is applied twice a day for 52 weeks.

NPC-12Y 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 reddish papules of angiofibroma ( \>= 2 mm in diameter) on the face at screening tests
• Patients who are not suitable for therapy with laser or surgery (including liquid nitrogen therapy and phototherapy) for angiofibroma, or who do not want therapy with laser or surgery
• Patients who are being treated with Rapalimus® gel 0.2% (NPC-12G Gel 0.2%) and who are able to and agree to a withdrawal of at least 4 weeks prior to enrollment in this study.
• Patients who (or whose guardian) give a written informed consent in understanding and willingness after having received enough explanation regarding the study participation.

You may not qualify if:

• Patients who (or whose guardian) are hard to apply the investigational drug topically with keeping compliance
• 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 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 or complication of allergy to the component of the investigational drug (sirolimus)
• Patients who have any infectious disease, cardiac disease, hepatic disease, pulmonary disease, renal disease, hematological disease, or malignant tumor that is considered inappropriate for participation in this clinical study.
• Patients who have complications such as diseases unsuitable for the trial participation, for examples, uncontrolled diabetes, dyslipidemia, etc.
• Patients who have participated in other clinical trial or clinical study, and have taken an investigational or clinical study drug within 6 months before the initial registration
• Patients who used mTOR inhibitors (oral or injectable) other than everolimus within 4 weeks before the initial registration
• Female patients who are pregnant, may be pregnant, or are lactating
• Patients who cannot agree to use appropriate contraception after the date of consent to participate in the clinical study and for the duration of the clinical study (including male patients with a partner of childbearing potential)
• Patients who have received therapy with laser or surgery (including liquid nitrogen therapy and phototherapy) to the lesion of angiofibroma within 6 months before the initial registration
• Other patients who are considered by the investigator as unsuitable for participation in the clinical study

Sponsors & Collaborators

• Nobelpharma: lead

Study Sites (5)

Fujita Health University Hospital

Toyoake, Aichi-ken, Japan

Location

Gunma University Hospital

Maebashi, Gunma, Japan

Location

Osaka University Hospital

Suita, Osaka, Japan

Location

Seirei Hamamatsu General Hospital

Hamamatsu, Shizuoka, Japan

Location

National Hospital Organization Nishi-Niigata Chuo Hospital

Niigata, Japan

Location

MeSH Terms

Conditions

Tuberous Sclerosis

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

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: August 4, 2022
• First Posted: August 10, 2022
• Study Start: June 1, 2022
• Primary Completion: October 30, 2024
• Study Completion: October 30, 2024
• Last Updated: November 22, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

JP (5)