UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000009392 |
|---|---|
| Receipt number | R000011032 |
| Scientific Title | An Open-Label, Dose Escalation, Proof-of-Concept Clinical Trial of Chaperone therapy of Neuronopathic Gaucher Disease with Ambroxol |
| Date of disclosure of the study information | 2012/11/24 |
| Last modified on | 2012/11/24 13:53:31 |
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Basic information
Public title
An Open-Label, Dose Escalation, Proof-of-Concept Clinical Trial of Chaperone therapy of Neuronopathic Gaucher Disease with Ambroxol
Acronym
Chaperone therapy of Neuronopathic Gaucher Disease with Ambroxol
Scientific Title
An Open-Label, Dose Escalation, Proof-of-Concept Clinical Trial of Chaperone therapy of Neuronopathic Gaucher Disease with Ambroxol
Scientific Title:Acronym
Chaperone therapy of Neuronopathic Gaucher Disease with Ambroxol
Region
| Japan |
Condition
Condition
Neuronopathic Gaucher disease
Classification by specialty
| Endocrinology and Metabolism | Neurology | Pediatrics |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
Chaperone therapy is expected to amelior-
ate neurological symptoms in lysosomal
storage disorders, since small molecules can cross the blood-brain barrier.
Gaucher disease (GD) is characterized by a deficiency of the lysosomal enzyme, glucocerebrosidase (GBA).
Ambroxol (ABX), a commonly-used expector-ant, has been reported to have chaperone
activity on mutant GBA.
We aimed to investigate the effect of ABX
on neurological manifestations of GD in combination with enzyme replacement therapy.
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Exploratory
Trial characteristics_2
Developmental phase
Phase II,III
Assessment
Primary outcomes
Primary Outcome Measures: Safety
Safety will be based on physical exam,
vital signs, adverse event query, and
clinical pathology (includes chemistry,
hematology and urinalysis), asessed at
baseline and approximately biweekly
during the study.
Secondary Outcome Measures: Efficacy
Efficacy is based on biomarker (glucoce-
rebrosidase activities in lymphocytes),
changes in neurological or neurophysiol-
ogical assessments, activities of daily
living (ADLs).
Key secondary outcomes
Base
Study type
Interventional
Study design
Basic design
Single arm
Randomization
Non-randomized
Randomization unit
Blinding
Open -no one is blinded
Control
Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
1
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
Ambroxol at a dose level of 25 mg/kg/day (upper limit: 1g/day) will be given by mouth for 6 months.
Interventions/Control_2
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
| Not applicable |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
Written informed consent
Key exclusion criteria
1) Patients with critical illness
2) Pregnant or lactating woman
3) Within 3 months from other clinical
trial
4) Patients who are not appropriate to
participate to the trial.
Target sample size
5
Research contact person
Name of lead principal investigator
| 1st name | |
| Middle name | |
| Last name | Kousaku Ohno |
Organization
Tottori University
Division name
Child Neurology
Zip code
Address
36-1 Nishi-cho, Yonago, Tottori 683-8504, Japan
TEL
0859-38-6777
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | Aya Narita |
Organization
Tottori University
Division name
Child Neurology
Zip code
Address
36-1 Nishi-cho, Yonago, Tottori 683-8504, Japan
TEL
0859-38-6777
Homepage URL
aya.luce@nifty.com
Sponsor or person
Institute
Tottori University, Faculty of Medicine, Institute of Neurological Science
Institute
Department
Personal name
Funding Source
Organization
None
Organization
Division
Category of Funding Organization
Self funding
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
Other administrative information
Date of disclosure of the study information
| 2012 | Year | 11 | Month | 24 | 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
| 2010 | Year | 03 | Month | 01 | Day |
Date of IRB
Anticipated trial start date
| 2012 | Year | 11 | Month | 24 | 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
| 2012 | Year | 11 | Month | 24 | Day |
Last modified on
| 2012 | Year | 11 | Month | 24 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000011032
