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

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

NCT ID: NCT05495425

Condition: Tuberous Sclerosis Complex

Conditions: Official terms:
Tuberous Sclerosis
Sclerosis

Conditions: Keywords:
Tuberous sclerosis complex
Sirolimus
Rapalimus gel
mTOR inhibitor

Study type: Interventional

Study phase: Phase 3

Overall status: Active, not recruiting

Study design:

Allocation: Randomized

Intervention model: Parallel Assignment

Primary purpose: Treatment

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Intervention:

Intervention type: Drug
Intervention name: NPC-12Y gel
Description: 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.
Arm group label: NPC-12Y gel

Intervention type: Drug
Intervention name: NPC-12Y placebo gel
Description: 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.
Arm group label: NPC-12Y placebo gel

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.

Detailed description: This is a Phase 3, placebo-controlled comparative study of NPC-12Y gel in patients with skin lesions associated with tuberous sclerosis complex. Patients who meet all entry criteria for this study will apply NPC-12Y gel or placebo twice a day for 12 weeks. After the double-blind evaluation period, all patients will apply NPC-12Y gel twice a day for 52 weeks. Approximately 40 eligible patients will be enrolled

Criteria for eligibility:
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 reddish papules of angiofibroma ( >= 2 mm in diameter) on the face at screening tests 4. 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 5. 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. 6. Patients who (or whose guardian) give a written informed consent in understanding and willingness after having received enough explanation regarding the study participation. Exclusion Criteria: 1. Patients who (or whose guardian) are hard to apply the investigational drug topically with keeping compliance 2. Patients who are hard to be taken pictures of their lesions adequately in such cases that they may not follow instruction of stillness 3. 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 4. Patients with a history or complication of allergy to the component of the investigational drug (sirolimus) 5. 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. 6. Patients who have complications such as diseases unsuitable for the trial participation, for examples, uncontrolled diabetes, dyslipidemia, etc. 7. 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 8. Patients who used mTOR inhibitors (oral or injectable) other than everolimus within 4 weeks before the initial registration 9. Female patients who are pregnant, may be pregnant, or are lactating 10. 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) 11. 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 12. Other patients who are considered by the investigator as unsuitable for participation in the clinical study

Gender: All

Minimum age: 3 Years

Maximum age: N/A

Healthy volunteers: No

Locations:

Facility:
Name: Fujita Health University Hospital

Address:
City: Toyoake
Country: Japan

Facility:
Name: Gunma University Hospital

Address:
City: Maebashi
Country: Japan

Facility:
Name: Osaka University Hospital

Address:
City: Suita
Country: Japan

Facility:
Name: Seirei Hamamatsu General Hospital

Address:
City: Hamamatsu
Country: Japan

Facility:
Name: National Hospital Organization Nishi-Niigata Chuo Hospital

Address:
City: Niigata
Country: Japan

Start date: June 1, 2022

Completion date: October 31, 2024

Lead sponsor:
Agency: Nobelpharma
Agency class: Industry

Source: Nobelpharma

Record processing date: ClinicalTrials.gov processed this data on November 12, 2024

Source: ClinicalTrials.gov page: https://clinicaltrials.gov/ct2/show/NCT05495425