rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease
Bu çalışma, tipik Canavan hastalığı (Canavan disease) olan çocuklarda rAAV-Olig001-ASPA adlı bir gen terapisini incelemektedir. Çalışmaya, bir nörolog tarafından kesin tipik Canavan hastalığı tanısı konmuş çocuklar yaş gruplarına göre katılabilmektedir. Faz 1/Faz 2 aşamasında yürütülmekte ve davetle katılım almaktadır.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
Inclusion Criteria: * Definitive diagnosis of typical CD by a board certified neurologist. * Written informed consent from parent(s)/guardian(s). Consent to enroll into the study will include a written agreement to comply with all the conditions of the study, including attendance at follow-up visits. * For cohort 1: age more than 36 months and up to 60 months. * For cohort 2: age between 15 months and 36 months. * For cohort 3: age less than 15 months. Exclusion Criteria: * At the discretion of the PI, any significant chronic medical condition, including, but not limited to neurological, cardiac, hepatic, renal, hematological, gastrointestinal, endocrine, pulmonary, or infectious disease, which would put the subject at increased risk during surgery or which would interfere with participation in the study, interpretation of safety monitoring, or the integrity of the study data. * History of severe allergic reaction or anaphylaxis. * Past participation in gene therapy trials or receipt of any other investigational product within 6 months prior to enrollment. * Prior intracranial surgery. * Any absolute contraindication to immunosuppression. * Any absolute contraindication to MRI. * Any vaccination less than 1 month prior to gene therapy. * Anticipated life expectancy of less than 12 months for any reason. * GMFM-88 total raw score \>35%. * Clinically significant out-of-range lab values, at the discretion of clinical PI.