UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000017802 |
|---|---|
| Receipt number | R000020624 |
| Scientific Title | Clinical research of the gene therapy for AADC deficiency |
| Date of disclosure of the study information | 2015/06/04 |
| Last modified on | 2023/06/08 14:45:43 |
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Basic information
Public title
Clinical research of the gene therapy for AADC deficiency
Acronym
Gene therapy for AADC deficiency
Scientific Title
Clinical research of the gene therapy for AADC deficiency
Scientific Title:Acronym
Gene therapy for AADC deficiency
Region
| Japan |
Condition
Condition
AADC deficiency
Classification by specialty
| Pediatrics |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
To investigate the safety and improve the motor function of the patients with aromatic L-amino acid decarboxylase (AADC) deficiency, after injection of AADC gene inserted into the adeno- associated virus (AAV) vector type 2 (AAV-hAADC-2) into the patients' striatum (Putamen).
Basic objectives2
Safety
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
Safety of the AAV-hAADC-2 injection therapy into the Putamen of the patients with AADC deficiency.
･ Adverse event
･Clinical records of seizure, physical findings, and neurological findings.
･Laboratory findings, cerebro- spinal fluid examination, cranial MRI, and EEG.
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
| Gene |
Interventions/Control_1
Subjects will be hospitalized before Day -14 and conducted the baseline examination. The target putmen for AAV-hAADC-2 infusion is identified on MRI image that has been taken prior to the operation, and then subjects will be bilaterally infused with a total volume of 200 micro L at a total of 4 sites (2 sites in left putamen, 2 sites in right putamen; 50 micro L per site) at a flow rate of 3 micro L per minute on Day 0.
After the infusion is complete, the cannula devices will be removed, and the surgical incision will be seamed in accordance with usual trephination. After that, cranial CT scan will be performed so as to confirm whether there are complications such as the occurrence of intracranial bleeding or not. Subjects stay in a hospital for 14 days after infusion of AAV-hAADC-2.
Data and Safety Monitoring Board (DSMB) will be evaluated the efficacy of all subjects and safety of the treatment.
At the time of 6 months after the infusion, investigator assesses the treatment effect of AAV-hAADC-2 on the basis of clinical assessment and FMT-PET imaging.
The investigator also assesses the safety for 5 years after the baseline examination. Long-term follow up study is additionally conducted for 10 years in reference to guideline of FDA.
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
| 4 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
1.Typical AADC deficiency patients who was unable to stand with motor disturbance and dystonia. Diagnosis was confirmed from the findings of CSF analysis, enzyme activity or genetic analysis.
2.Age; => 4 years at the time of medical treatment. No restriction of age upper limit.
3.No findings suggestive of CNS Degenerative Disease are found.
4.To be able to comply with the requirements of this study, including the frequent clinical examination after medical treatment.
5.To keep the therapeutic medicine for AADC deficiency for at least 2 months prior to participation in this study.
6.Written informed consent from patient' s parental authorities.
Key exclusion criteria
1.Mild AADC deficiency patients who can stand and walk.
2.Patients with history of significant cardiovascular disease including cerebrovascular accident.
3.Malignant neoplasm in the brain, clinically significant neurological disease.
4.History of malignancy, with the exception of treated carcinoma cutaneum, within 5 years.
5.Uncontrolled hypertension: systolic blood pressure over 160 mmHg.
6.Coagulopathy or need for anticoagulant therapy.
7.Clinically significant immune dysfunction (for example, the case who require the use of immunosuppresive drugs).
8.Unable to scan MRI.
9.Cases without abnormal finding in FMT-PET.
10.Past medical history of serious drug allergy.
11.Patients who have participated in other clinical trial within 6 months.
12.Patients who meet any of the following criteria:
a) Serious renal disorder (Cr; => 2.0 mg/dl, and BUN; => 25mg/dl)
b) Serious hepatic disorder AST (GOT) / ALT (GPT) ; => 2.5 x upper limit of normal (ULN)
c) Serious diabetes (casual blood glucose or fasting blood glucose; => 200 mg/dl and HbA1c; => 9 %)
13.Seriously ill patients
14.Any other patients judged by investigators to be inappropriate for the subject of this study.
Target sample size
5
Research contact person
Name of lead principal investigator
| 1st name | Karin |
| Middle name | |
| Last name | Kojima |
Organization
Jichi Medical University
Division name
Department of Pediatrics
Zip code
329-0498
Address
3311-1 Yakushiji, Shimotsuke, Tochigi
TEL
0285-58-7366
karinkojima@jichi.ac.jp
Public contact
Name of contact person
| 1st name | Michiyo |
| Middle name | |
| Last name | Osawa |
Organization
Jichi Medical University
Division name
Clinical Research Center
Zip code
329-0498
Address
3311-1 Yakushiji, Shimotsuke, Tochigi
TEL
0285-58-8960
Homepage URL
m.osawa@jichi.ac.jp
Sponsor or person
Institute
JIchi Medical University
Institute
Department
Personal name
Funding Source
Organization
Japan Agency for 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
Jichi Medical University
Address
3311-1 Yakushiji, Shimotsuke, Tochigi
Tel
0285-44-2111
jmu-crb2020@jichi.ac.jp
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
| 2015 | Year | 06 | Month | 04 | Day |
Related information
URL releasing protocol
Publication of results
Partially published
Result
URL related to results and publications
Number of participants that the trial has enrolled
8
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
No longer recruiting
Date of protocol fixation
| 2015 | Year | 01 | Month | 13 | Day |
Date of IRB
| 2014 | Year | 07 | Month | 14 | Day |
Anticipated trial start date
| 2015 | Year | 06 | Month | 04 | Day |
Last follow-up date
| 2023 | Year | 11 | Month | 26 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2015 | Year | 06 | Month | 04 | Day |
Last modified on
| 2023 | Year | 06 | Month | 08 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000020624
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| Registered date | File name |
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