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Tuberous SclerosisPhase 3NCT02635789

Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)

A Phase 3 study testing NPC-12G gel in people with Tuberous Sclerosis and Angiofibroma, run by Nobelpharma at 1 site in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of this trial is to evaluate the efficacy and safety of NPC-12G gel (topical formulation of sirolimus) versus placebo gel to facial angiofibroma and other skin lesions in patients with tuberous sclerosis complex (TSC) Read more about this study

Who is it for?
Ages 3 years and older
What is being tested?
NPC-12G gel
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
62 participants
Where
1 siteJapan
Study dates
Dec 2015to Oct 2016
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 papules of angiofibroma ( >= 2 mm in diameter with redness in each) on the face at screening tests
  • Patients who are not suitable for therapy with laser or surgery, or who do not want therapy with laser or surgery
  • Patients or his/her guardian who give a written informed consent in understanding and willingness after having received enough explanation of the test drug and the current trial plan

Who cannot join

  • Patients who are hard to apply the test drug topically with keeping compliance
  • 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 who are hard to be taken pictures of their lesions adequately in such cases that they may not follow instruction of stillness
  • Patients with a history of hypersensitivity to alcohol or allergy to sirolimus
  • Patients who have complications such as malignant tumor, infection, serious heart disease, hepatic function disorder, renal function disorder or blood disorders which severity are considered by investigator as grade 2 or more severe with reference to ''Concerning classification criteria for seriousness of adverse drug reactions of medical agents''
  • Patients who have complications such as diseases unsuitable for the trial participation, for examples, uncontrolled diabetes (fasting blood glucose level >140 mg/dL or postprandial blood glucose level > 200 mg/dL), dyslipidemia (cholesterol level > 300 mg/dL or > 7.75 mmol/L, triglycerides level > 300 mg/dL or > 3.42 mmol/L), etc.
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  • Patients who have taken drugs with mTOR inhibitory action including sirolimus, everolimus or temsirolimus within 12 months before the initial registration
  • Patients who have applied topical tacrolimus on the lesion of angiofibroma within 3 months before the initial registration
  • Patients who have received therapy with laser or surgery to the lesion of angiofibroma within 6 months before the initial registration
  • Female patients who may be pregnancy or are lactating
  • Patients who cannot agree to take appropriate measures of contraception until completion of post-treatment phase or follow-up period after discontinuation from informed consent
  • Patients who have participated in other clinical trial and have taken a trial drug within 6 months before the initial registration
  • Others, patients who are considered by the investigator as unsuitable for participation in the trial

Trial locations and contacts

1 site in 1 country.

Japan1 site

  • Graduate School of Medicine, Osaka University

    Suita, Osaka, 565-0871

Other trials in Japan

About this study

Official title: A Double-blind, Randomized, Placebo-controlled Phase III Trial to Investigate the Efficacy and Safety of NPC-12G Gel (Topical Formulation of Sirolimus) to Angiofibroma and Other Skin Lesions in Patients With Tuberous Sclerosis Complex

The purpose of this trial is to evaluate the efficacy and safety of NPC-12G gel (topical formulation of sirolimus) versus placebo gel to facial angiofibroma and other skin lesions in patients with tuberous sclerosis complex (TSC)

Read the detailed description

Tuberous Sclerosis Complex (TSC) is an autosomal dominant hereditary disease that causes benign tumors on the almost whole body (including skin, brain, kidney, lung and heart), behavior disorder as autism, mental retardation and neurologic symptom as epilepsy. Angiofibroma is TSC-specific facial skin lesions, and hamartoma caused by increase of the component of skin connective tissues and blood vessels. Other skin lesions due to TSC are white macule(hypomelanotic macule), plaque, shagreen patch and ungual fibromas. Current therapeutic methods for angiofibroma are laser or surgical treatments, but there are problems as many relapses, deficiency of evidence, change of pigment, scar and risk of infection.

This will be a multicenter, double-blind, randomized, placebo-controlled parallel group trial. The trial has three phases; the screening phase, double-blinded treatment phase, and post-treatment phase. The screening phase comprises a screening visit where subject's initial eligibility will be evaluated. During double-blinded treatment phase, patients who meet all entry criteria for the trial will be randomized into two groups, and they will apply 0.2% NPC-12G gel or placebo gel topically twice a day for 12 weeks.

Being tested
NPC-12G gel, Placebo gel
Keywords
Sirolimus, Skin lesions, TSC, Tuberous Sclerosis Complex, NPC-12G, Angiofibroma, Hypomelanotic macule, Plaque
Medical subject headings
Tuberous Sclerosis, Angiofibroma, Plaque, Amyloid

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NPC-12G gelDrug

    NPC-12G gel is administered topically twice a day for 12 weeks

  • Placebo gelDrug

    NPC-12G gel placebo is administered topically twice a day for 12 weeks

Study groups in NCT02635789
Study groupRoleWhat they receive
NPC-12G gelExperimentalNPC-12G gel is containing 0.2% Sirolimus
Receives: Drug: NPC-12G gel
Placebo gelPlacebo comparatorPlacebo gel is matched ingredient with NPC-12G gel
Receives: Drug: Placebo gel

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Improvements in angiofibroma

    Improvements comparing with baseline is assessed using photograph by the central photo-judgement committee

    Measured: 12 weeks

Secondary outcomes (11)

  1. Improvements in angiofibroma

    Improvements comparing with baseline is assessed using photograph by the central photo-judgement committee

    Measured: Week 4 and 8 and follow-up Week 16

  2. Improvements in angiofibroma

    Improvements comparing with baseline is assessed by the investigator

    Measured: Week 4, 8, 12 and follow-up Week 16

  3. Improvements in redness of angiofibroma

    Improvement comparing with baseline is assessed by the central photo-judgement committee and the investigator

    Measured: Week 4, 8, 12 and follow-up Week 16

  4. Improvements in hypomelanotic macule and plaque of upper neck

    Improvement comparing with baseline is assessed by the central photo-judgement committee and the investigator

    Measured: Week 4, 8, 12 and follow-up Week 16

  5. The rate of patients who are evaluated as ''improvement'' or more (improvement rate) in primary outcome measure and in secondary outcome measures above outcome 1 to 5

    Measured: Week 4, 8, 12 and follow-up Week 16

  6. Change in total score from baseline for DLQI and CDLQI

    DLQI for subjects 16 years old and greater, or CDLQI for children of less than 16 years old is assessed by patients

    Measured: Week 4, 8, 12 and follow-up Week 16

  7. Adverse events

    Adverse events during the study period

    Measured: 16 weeks

  8. Serious adverse events

    Serious adverse events during the study period

    Measured: 16 weeks

  9. Laboratory findings

    Laboratory findings during the study period

    Measured: 16 weeks

  10. Vital sign

    Vital sign during the study period

    Measured: 16 weeks

  11. Sirolimus blood concentration

    Blood concentration of Sirolimus is assessed by drug monitoring

    Measured: Baseline, Week 4 and Week 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Dec 2015 (estimated)
  2. Study startDec 2015
  3. Primary completionOct 2016
  4. Study completionOct 2016

Resources

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
62 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02635789
Sponsor's protocol ID
NPC-12G-1
Status last verified
Feb 2017
First submitted
15 Dec 2015
Last updated
2 Feb 2017
Results posted
No
Data monitoring committee
No

Publications

  • Wataya-Kaneda M, Ohno Y, Fujita Y, Yokozeki H, Niizeki H, Ogai M, Fukai K, Nagai H, Yoshida Y, Hamada I, Hio T, Shimizu K, Murota H. Sirolimus Gel Treatment vs Placebo for Facial Angiofibromas in Patients With Tuberous Sclerosis Complex: A Randomized Clinical Trial. JAMA Dermatol. 2018 Jul 1;154(7):781-788. doi: 10.1001/jamadermatol.2018.1408.

    PubMed 29800026

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing NPC-12G gel in people with Tuberous Sclerosis and Angiofibroma, run by Nobelpharma at 1 site 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 5 inclusion and 13 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 2 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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?

On the ClinicalTrials.gov record for NCT02635789, which also lists 1 linked publication.

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 2 Feb 2017.

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