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

Sponsor

Nobelpharma (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05495425

Collaborator

(none)

Enrollment

Locations

Arms

Anticipated Duration (Months)

Patients Per Site

0.3

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Tuberous Sclerosis Complex	Drug: NPC-12Y gel Drug: NPC-12Y placebo gel	Phase 3

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

Study Design

Study Type:

Interventional

Anticipated Enrollment :

40 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

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

Primary Purpose:

Treatment

Official Title:

Placebo-controlled Comparative Study of NPC-12Y Gel in Patients With Skin Lesions Associated With Tuberous Sclerosis Complex

Actual Study Start Date :

Jun 1, 2022

Anticipated Primary Completion Date :

Nov 30, 2023

Anticipated Study Completion Date :

Sep 30, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: NPC-12Y gel NPC-12Y gel is containing 0.2% Sirolimus	Drug: NPC-12Y gel 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.
Placebo Comparator: NPC-12Y placebo gel Placebo gel matched NPC-12Y gel	Drug: NPC-12Y placebo gel 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

Intervention/Treatment

Experimental: NPC-12Y gel

NPC-12Y gel is containing 0.2% Sirolimus

Drug: NPC-12Y gel

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.

Placebo Comparator: NPC-12Y placebo gel

Placebo gel matched NPC-12Y gel

Drug: NPC-12Y placebo gel

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.

Outcome Measures

Primary Outcome Measures

Improvements in angiofibroma [12 weeks]
Improvements comparing with baseline is assessed using photograph by IRC

Secondary Outcome Measures

Improvements in angiofibroma, color and size [12 weeks]
Improvements comparing with baseline is assessed using photograph by IRC
Index of Facial Angiofibromas (IFA) score [12 weeks]
Changes in Index of Facial Angiofibromas (IFA) score assessed by investigators

Eligibility Criteria

Criteria

Ages Eligible for Study:

3 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

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.

Exclusion Criteria:

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

Contacts and Locations

Locations

	Site	City	State	Country
1	Fujita Health University Hospital	Toyoake	Aichi	Japan
2	Gunma University Hospital	Maebashi	Gunma	Japan
3	Osaka University Hospital	Suita	Osaka	Japan
4	Seirei Hamamatsu General Hospital	Hamamatsu	Shizuoka	Japan
5	National Hospital Organization Nishi-Niigata Chuo Hospital	Niigata		Japan