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Name:
UMIN ID:

Recruitment status Open public recruiting
Unique ID issued by UMIN UMIN000033083
Receipt No. R000037719
Scientific Title A Phase I/IIa double blind single institute trial of low dose sirolimus for Pendred syndrome/ DFNB4
Date of disclosure of the study information 2018/06/21
Last modified on 2018/06/21

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Basic information
Public title A Phase I/IIa double blind single institute trial of low dose sirolimus for Pendred syndrome/ DFNB4
Acronym PENDLRA trial
Scientific Title A Phase I/IIa double blind single institute trial of low dose sirolimus for Pendred syndrome/ DFNB4
Scientific Title:Acronym PENDLRA trial
Region
Japan

Condition
Condition Pendred syndrome/ DFNB4
Classification by specialty
Oto-rhino-laryngology
Classification by malignancy Others
Genomic information NO

Objectives
Narrative objectives1 Safety, tolerability and therapeutic effects of NPC-12T for Pendred syndrome / DFNB 4 (PDS) will be explored.
Basic objectives2 Safety
Basic objectives -Others
Trial characteristics_1 Exploratory
Trial characteristics_2
Developmental phase Phase I,II

Assessment
Primary outcomes Incidences of adverse events/reactions and their grades
Key secondary outcomes Clinical effects of NPC-12T upon symptoms (hearing level and vertigo/dizziness) of PDS patients.

Base
Study type Interventional

Study design
Basic design Parallel
Randomization Randomized
Randomization unit Individual
Blinding Double blind -all involved are blinded
Control Placebo
Stratification NO
Dynamic allocation NO
Institution consideration Institution is not considered as adjustment factor.
Blocking YES
Concealment Central registration

Intervention
No. of arms 2
Purpose of intervention Treatment
Type of intervention
Medicine
Interventions/Control_1 sirolimus
Interventions/Control_2 placebo-control
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit
7 years-old <=
Age-upper limit
50 years-old >
Gender Male and Female
Key inclusion criteria Outpatients who are diagnosed as PDS by genetic tests for SLC26A4 gene, suffered fluctuating symptoms of inner ear disorders and meet following criteria:
1) 7-50 years old
2) Diagnosed by genetic tests.
3) Symptoms of inner ear disorders are not fixed.
4) Subjective symptom of episodes of hearing loss and/or dizziness/vertigo within one year prior to the case registration.
5) At least three times of inner-ear function tests, including standard pure-tone audiometry, were performed with an interval of one day or longer, within six months prior to the case registration.
6) Written informed consent by himself/herself or the subject's legally acceptable representative.
Key exclusion criteria 1) Past usage of mTOR inhibitors or other molecular target drugs relating mTOR pathway.
2) History of allergy to Sirolimus, Sirolimus derivatives or additive substance.
3) Patients with HBV, with the history of HBV or with active type-C hepatitis.
4) Severe blood abnormalities or hepatic dysfunction
AST or ALT 100 IU/L or higher
Hct below 30%
Platelet below 80,000/mm3
Neutrophils below 1,000/mm3
Total WBC below 3,000/mm3
5) Dyslipidemia: Serum triglyceride of 500 mg/dL or higher or LDL cholesterol level of 190 mg/dL or higher in spite of the treatments.
6) Severe renal failure. eGFR < 30 mL/min/1.73 m^2.
7) Immunodeficiency including HIV or primary immunodeficiency.
8) Patients who are not able to take tablet, or who have gastrointestinal disorders possibly leading to insufficient absorption of Sirolimus.
9) Patients who had surgery during 8 weeks before the registry (any surgery that requires invasion of the body or suture with three or more stitches).
10) Pregnant, probably pregnant, or breast-feeding women. Patients who do not agree birth control during clinical trial.
11) Patients who need drugs that influence CYP3A4 activity after trials are started.
12) Patients with pulmonary infiltrations.
13) Hearing loss is fixed rather than fluctuating diagnosed by physicians with audiograms.
14) Participation in other clinical research and/or trials within six months prior to the date of consent.
15) Others not suitable judged by investigators.
Target sample size 16

Research contact person
Name of lead principal investigator
1st name
Middle name
Last name Masato FUJIOKA
Organization Keio University School of Medicine
Division name Department of Otolaryngology, Head and Neck Surgery
Zip code
Address 35 Shinanomachi, Shinjuku-ku, Tokyo, 160-8582
TEL 03-5363-3827
Email mtfuji@keio.jp

Public contact
Name of contact person
1st name
Middle name
Last name Yuto FUJIKI
Organization Keio University Hospital
Division name Clinical and Translational Research Center
Zip code
Address 35 Shinanomachi, Shinjuku-ku, Tokyo, 160-8582
TEL 03-5315-4294
Homepage URL
Email pmo@ccr.med.keio.ac.jp

Sponsor
Institute Keio University
Institute
Department

Funding Source
Organization Japan Agency Medical Research and Development
Organization
Division
Category of Funding Organization Japanese Governmental office
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization
Address
Tel
Email

Secondary IDs
Secondary IDs NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions 慶應義塾大学病院(東京都)

Other administrative information
Date of disclosure of the study information
2018 Year 06 Month 21 Day

Related information
URL releasing protocol
Publication of results Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status Open public recruiting
Date of protocol fixation
2018 Year 03 Month 19 Day
Date of IRB
Anticipated trial start date
2018 Year 03 Month 27 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date
2018 Year 06 Month 21 Day
Last modified on
2018 Year 06 Month 21 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000037719

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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