UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000033083 |
|---|---|
| Receipt number | 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 | 2021/04/13 17:59:35 |
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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
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
pmo@ccr.med.keio.ac.jp
Sponsor or person
Institute
Keio University
Institute
Department
Personal name
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
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
Completed
Date of protocol fixation
| 2018 | Year | 03 | Month | 19 | Day |
Date of IRB
| 2018 | Year | 03 | Month | 05 | Day |
Anticipated trial start date
| 2018 | Year | 03 | Month | 27 | Day |
Last follow-up date
| 2020 | Year | 07 | Month | 06 | Day |
Date of closure to data entry
| 2020 | Year | 07 | Month | 20 | Day |
Date trial data considered complete
| 2020 | Year | 08 | Month | 05 | Day |
Date analysis concluded
| 2021 | Year | 01 | Month | 08 | Day |
Other
Other related information
Management information
Registered date
| 2018 | Year | 06 | Month | 21 | Day |
Last modified on
| 2021 | Year | 04 | Month | 13 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000037719
| Research Plan | |
|---|---|
| Registered date | File name |
| Research case data specifications | |
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| Registered date | File name |
| Research case data | |
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| Registered date | File name |
