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

November 20, 2024 updated by: Nobelpharma

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
43

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Japan
      • Niigata, Japan
        • National Hospital Organization Nishi-Niigata Chuo Hospital
    • Aichi
      • Toyoake, Aichi, Japan
        • Fujita Health University Hospital
    • Gunma
      • Maebashi, Gunma, Japan
        • Gunma University Hospital
    • Osaka
      • Suita, Osaka, Japan
        • Osaka University Hospital
    • Shizuoka
      • Hamamatsu, Shizuoka, Japan
        • Seirei Hamamatsu General Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

9 months and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Improvements in angiofibroma
Time Frame: 12 weeks
Improvements comparing with baseline is assessed using photograph by IRC
12 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Improvements in angiofibroma, color and size
Time Frame: 12 weeks
Improvements comparing with baseline is assessed using photograph by IRC
12 weeks
Index of Facial Angiofibromas (IFA) score
Time Frame: 12 weeks
Changes in Index of Facial Angiofibromas (IFA) score assessed by investigators
12 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Nobelpharma

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 1, 2022

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 30, 2024

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 30, 2024

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 4, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 8, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 10, 2022

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 22, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 20, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • NPC-12Y-1

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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