Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC
Study Purpose
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.
Recruitment Criteria
Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
No
Study Type
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
Searching Both is inclusive of interventional and observational studies.
Interventional
Eligible Ages
3 Years and Over
Gender
All
More Inclusion & Exclusion 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
Trial Details
Trial ID:
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
Phase 3
Lead Sponsor
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
Nobelpharma
Principal Investigator
The person who is responsible for the scientific and technical direction of the entire clinical study.
N/A
Principal Investigator Affiliation
N/A
Agency Class
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
Industry
Overall Status
Recruiting
Countries
Japan
Conditions
The disease, disorder, syndrome, illness, or injury that is being studied.
Tuberous Sclerosis Complex
Additional Details
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
Arms & Interventions
Arms
Experimental: NPC-12Y gel
NPC-12Y gel is containing 0.2% Sirolimus
Placebo Comparator: NPC-12Y placebo gel
Placebo gel matched NPC-12Y gel
Interventions
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.
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.
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.
International Sites
Fujita Health University Hospital, Toyoake, Aichi, Japan
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