Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex

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Brief Title

Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex

Official Title

A Long-term, Single-arm, Open-label Trial of NPC-12G (Topical Formulation of Sirolimus) to Angiofibroma and Other Skin Lesions in Patients With Tuberous Sclerosis Complex

Brief Summary

      The purpose of this trial is to evaluate the safety and efficacy of long-term treatment with
      NPC-12G gel (0.2% sirolimus gel) to angiofibroma and other skin lesions in patients with
      tuberous sclerosis complex in the open-label trial.
    

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 a TSC-specific facial skin lesion, 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 and surgical treatments, but there are
      problems as many relapses, deficiency of evidence, change of pigment, scar and risk of
      infection.

      This is a multicenter and open-label trial. The trial consists of two phase. In the first
      trial phase for 52 weeks, the efficacy as well as the safety is evaluated. For the second
      trial phase the trial will be continued until the date of approval of NDA for NPC-12G. The
      safety is evaluated during the second trial phase, but not the efficacy. Patients who meet
      all entry criteria for the trial apply 0.2% NPC-12G gel twice a day. Patients will visit at 4
      to 5-week intervals for the first 6 months of the first trial phase, and then 3 months
      intervals thereafter.
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

The discontinuation rate due to adverse events

Secondary Outcome

 Adverse events and adverse events related to the test drug

Condition

Tuberous Sclerosis

Intervention

NPC-12G gel

Study Arms / Comparison Groups

 NPC-12G gel
Description:  NPC-12G gel is containing 0.2% Sirolimus

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

94

Start Date

December 2015

Completion Date

October 2018

Primary Completion Date

October 2018

Eligibility 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 skin lesions such as angiofibroma, white macules or plaque upper neck
             associated with tuberous sclerosis complex at the screening visit or the baseline
             visit

          4. Patients or his/her guardian who agree to use the test drug (NPC-12G gel) or who want
             to participate in the trial again following participation in Phase III trial
             (NPC-12G-1)

          5. Patient who are considered to be an appropriate patient to participate in the trial by
             investigator

          6. 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

        Exclusion Criteria:

          1. Patients who have offered to withdraw from Phase III trial (NPC-12G-1) and have been
             discontinued

          2. Patients who have not applied the test drug topically more than 25% of whole
             applications without appropriate reason for Phase III trial (NPC-12G-1)

          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 of hypersensitivity to alcohol or allergy to sirolimus

          5. 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''

          6. 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.

          7. Female patients who may be pregnancy or are lactating

          8. Patients who cannot agree to take appropriate measures of contraception until
             completion of the trial or follow-up period after discontinuation from informed
             consent

          9. Patients who have participated in other clinical trial other than Phase III trial
             (NPC-12G-1) and have taken a trial drug within 6 months before informed consent

         10. Others, patients who are considered by the investigator as unsuitable for
             participation in the trial
      

Gender

All

Ages

3 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Mari Wataya-Kaneda,, MD, PhD, , 

Location Countries

Japan

Location Countries

Japan

Administrative Informations


NCT ID

NCT02634931

Organization ID

NPC-12G-2


Responsible Party

Sponsor

Study Sponsor

Nobelpharma


Study Sponsor

Mari Wataya-Kaneda,, MD, PhD, Principal Investigator, Department of Dermatology, Graduate School of Medicine, Osaka University


Verification Date

December 2015