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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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20 February 2023 |
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Main ID: |
NCT02926066 |
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Date of registration:
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03/10/2016 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
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Scientific title:
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A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion (NTUH-AADC-011) |
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Date of first enrolment:
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November 9, 2016 |
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Target sample size:
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12 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02926066 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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Taiwan
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Contacts
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Name:
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Yin-Hsiu Chien, MD.,PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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National Taiwan University Hospital |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. With a confirmed diagnosis of AADC, including cerebrospinal fluid analysis to show
reduced levels of neurotransmitter metabolites, HVA and 5-HIAA, and higher L-Dopa, or
with more than one mutation within AADC gene, etc.
2. Classical clinical characteristics of AADC deficiency, such as oculogyric crises,
hypotonia and developmental retardation.
3. The child patient has to be over 2 years old or a thickness of skull enough for
surgery.
4. The child patient has to be under 6 years old (72 months) before being treated with
study drugs.
5. Participating patients must cooperate completely for all evaluations and examinations
before, during and after the whole trial.
6. Parents or guardians must sign to agree on this informed consent.
Exclusion criteria:
1. Significant brain structure abnormality determined by the physician.
2. Patients with any health or neurological doubts that may increase the risk of surgery
cannot join this trial. PI has the right to evaluate the feasibility of subjects for
this trial based on his/her health condition.
3. Patients with anti-AAV2 neutralizing antibody titer over 1,200 folds or an ELISA OD
over 1 cannot be recruited into this trial.
4. Subjects participating in this trial cannot take any medications that may affect this
clinical trial, which do not apply to those drugs used at specified duration as
mentioned in this protocol.
Age minimum:
N/A
Age maximum:
6 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Aromatic Amino Acid Decarboxylase Deficiency
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Intervention(s)
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Drug: AAV2-hAADC
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Primary Outcome(s)
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Evaluation of therapeutic effect
[Time Frame: 13 months]
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Secondary Outcome(s)
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Exploratory endpoint
[Time Frame: 13 months]
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Evaluation of secondary therapeutic effects
[Time Frame: 13 months]
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Evaluation for the treatment safety
[Time Frame: 13 months]
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Secondary ID(s)
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201511036MIND
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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