Tuberous Sclerosis ComplexPhase 3NCT05495425
Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC
A Phase 3 study testing NPC-12Y gel in people with Tuberous Sclerosis Complex, run by Nobelpharma at 5 sites in Japan.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Tuberous Sclerosis Complex
- What is being tested?
- NPC-12Y gel
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 43 participants
- Where
- 5 sitesJapan
- Study dates
- 1 Jun 2022to 30 Oct 2024
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
Japan5 sites
Seirei Hamamatsu General Hospital
Hamamatsu, Shizuoka
Gunma University Hospital
Maebashi, Gunma
National Hospital Organization Nishi-Niigata Chuo Hospital
Niigata
Osaka University Hospital
Suita, Osaka
Fujita Health University Hospital
Toyoake, Aichi-ken
About this study
Official title: 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.
Read the 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
- Conditions
- Tuberous Sclerosis Complex
- Being tested
- NPC-12Y gel, NPC-12Y placebo gel
- Keywords
- Tuberous sclerosis complex, Sirolimus, Rapalimus gel, mTOR inhibitor
- Medical subject headings
- Tuberous Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NPC-12Y gelDrug
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 placebo gelDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| NPC-12Y gel | Experimental | NPC-12Y gel is containing 0.2% Sirolimus Receives: Drug: NPC-12Y gel |
| NPC-12Y placebo gel | Placebo comparator | Placebo gel matched NPC-12Y gel Receives: Drug: NPC-12Y placebo gel |
What is being measured
The primary outcome is the main question the study is designed to answer.
Improvements in angiofibroma
Improvements comparing with baseline is assessed using photograph by IRC
Measured: 12 weeks
Secondary outcomes (2)
Improvements in angiofibroma, color and size
Improvements comparing with baseline is assessed using photograph by IRC
Measured: 12 weeks
Index of Facial Angiofibromas (IFA) score
Changes in Index of Facial Angiofibromas (IFA) score assessed by investigators
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Aug 2022
- Study start1 Jun 2022
- Primary completion30 Oct 2024
- Study completion30 Oct 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Tuberous Sclerosis ComplexStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 43 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nobelpharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05495425
- Sponsor's protocol ID
- NPC-12Y-1
- Status last verified
- Nov 2024
- First submitted
- 4 Aug 2022
- Last updated
- 22 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Placebo-controlled Comparative Study of NPC-12Y Gel in Patients With Skin Lesions Associated With Tuberous Sclerosis Complex
- ClinicalTrials.gov ID
- NCT05495425
- Acronym / sponsor ID
- NPC-12Y-1
- Conditions
- Tuberous Sclerosis Complex
- Keywords
- Tuberous sclerosis complex, Sirolimus, Rapalimus gel, mTOR inhibitor
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 43 participants (actual)
- Lead sponsor
- NobelpharmaIndustry (pharma, biotech, devices)
- Study start
- 1 Jun 2022 (actual)
- Primary completion
- 30 Oct 2024 (actual)
- Study completion
- 30 Oct 2024 (actual)
- First posted
- 10 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NPC-12Y gel | Experimental | Drug: NPC-12Y gelNPC-12Y gel is containing 0.2% Sirolimus |
| NPC-12Y placebo gel | Placebo comparator | Drug: NPC-12Y placebo gelPlacebo gel matched NPC-12Y gel |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NPC-12Y gelNPC-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 gel |
| NPC-12Y placebo gelNPC-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. | Drug | NPC-12Y placebo gel |
Primary outcome
Improvements in angiofibroma
Time frame: 12 weeks
Improvements comparing with baseline is assessed using photograph by IRC
2 secondary outcomes
Improvements in angiofibroma, color and size
Time frame: 12 weeks
Index of Facial Angiofibromas (IFA) score
Time frame: 12 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Nov 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing NPC-12Y gel in people with Tuberous Sclerosis Complex, run by Nobelpharma at 5 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Improvements in angiofibroma (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Nov 2024.