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治験 NCT03278873（対象：色覚異常）は中止です。詳細は治験レーダーのタイル表示と AI 発見ツールで確認するか、ここで質問してください。 | ||
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メイラGTxLong-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3 34 遺伝子治療 長期追跡
治験（臨床試験）の詳細は主に英語で提供されていますが、治験レーダーAIがサポートします！「治験解説」をクリックして、選択した言語で試験情報を表示し、議論してください。
治験番号 NCT03278873 は 観察研究 臨床試験 で、色覚異常 に関するものです。現在は 中止 で、2017年6月29日 から開始しています。34 名の参加者 の募集が計画されています。この試験は メイラGTx によって主導され、2024年4月4日 に完了予定です。ClinicalTrials.gov からの最新更新日は 2025年6月11日 です。
概要
This is a longer-term follow-up study of patients with achromatopsia associated with defects in CNGA3 who participated in a clinical trial in which they received AAV-CNGA3 retinal gene therapy, or of patients with achromatopsia associated with defects in CNGB3 who participated in a clinical trial in which they received AAV-CNGB3 retinal gene therapy.
詳細説明
The follow-up study is designed to collect data on the longer-term safety and efficacy of AAV-CNGA3 retinal gene therapy and AAV-CNGB3 retinal gene therapy.
公式タイトル
Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hCARp.hCNGB3 and AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3 or CNGA3
疾患名
色覚異常その他の研究識別子
- MGT007
- 2016-003856-59 (EudraCT番号)
NCT番号
開始日
2017-06-29
最終更新日
2025-06-11
終了予定日
2024-04-04
目標参加者数
34
試験の種類
観察研究
状況
中止
キーワード
Achromatopsia
CNGA3
CNGB3
CNGA3
CNGB3
群（アーム）／介入
| 参加グループ/群 | 介入/治療法 |
|---|---|
Low dose of AAV-CNGA3 or AAV-CNGB3 Subretinal administration of a single low dose of AAV-CNGA3 or AAV-CNGB3 | Prior exposure to AAV-CNGA3 or AAV-CNGB3 Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively. |
Intermediate dose of AAV-CNGA3 or AAV-CNGB3 Subretinal administration of a single intermediate dose of AAV-CNGA3 or AAV-CNGB3 | Prior exposure to AAV-CNGA3 or AAV-CNGB3 Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively. |
Other dose of AAV-CNGA3 or AAV-CNGB3 Subretinal administration of a single other dose (between the intermediate and high dose) of AAV-CNGA3 or AAV-CNGB3 | Prior exposure to AAV-CNGA3 or AAV-CNGB3 Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively. |
High dose of AAV-CNGA3 or AAV-CNGB3 Subretinal administration of a single high dose of AAV-CNGA3 or AAV-CNGB3 | Prior exposure to AAV-CNGA3 or AAV-CNGB3 Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively. |
主要評価項目
副次評価項目
| 評価指標 | 指標の説明 | 時間枠 |
|---|---|---|
Incidence of Adverse Events Related to the Treatment | The primary outcome measure is the longer-term safety of treatment with AAV-CNGA3 or AAV-CNGB3, assessed by the absence of IMP-related adverse events. | 5 Years |
| 評価指標 | 指標の説明 | 時間枠 |
|---|---|---|
Improvements in Visual Function as Assessed by Visual Acuity at Month 12 | Change from baseline to Month 12 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score in the treated eye. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time. | 12 months |
Improvements in Visual Function as Assessed by Visual Acuity at Month 60 | Change from baseline to Month 60 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score in the treated eye. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time. | 60 months |
Improvements in Retinal Function as Assessed by Static Perimetry at Month 12 | Change from baseline to Month 12 in contrast sensitivity in the treated eye. The direction of improvement is an increase in sensitivity. | 12 months |
Improvements in Retinal Function as Assessed by Static Perimetry at Month 60 | Change from baseline to Month 60 in contrast sensitivity in the treated eye. The direction of improvement is an increase in sensitivity. | 60 months |
Quality of Life at Month 12 Measured by QoL Questionnaires in Children and Adolescents | Change from baseline to Month 12 in EuroQol-5D-Y Visual Analogue Scale (EQ-VAS) in children and adolescents. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement, and a negative change from baseline reflects worsening. | 12 months |
Quality of Life at Month 60 Measured by QoL Questionnaires in Children and Adolescents | Change from baseline to Month 60 in EuroQol-5D-Y Visual Analogue Scale (EQ-VAS) in children and adolescents. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement, and a negative change from baseline reflects worsening. | 60 months |
Quality of Life at Month 12 Measured by QoL Questionnaires in Adults | Change from baseline to Month 12 in EuroQol-5D-5L Visual Analogue Scale (EQ-VAS) in adults. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement, and a negative change from baseline reflects worsening. | 12 months |
Quality of Life at Month 60 Measured by QoL Questionnaires in Adults | Change from baseline to Month 60 in EuroQol-5D-5L Visual Analogue Scale (EQ-VAS) in adults. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement, and a negative change from baseline reflects worsening. | 60 months |
適格基準
Inclusion in the study will be limited to individuals who:
- Are able to give informed consent or assent, with or without the guidance of their parent(s)/guardian(s), where appropriate
- Received AAV2/8-hCARp.hCNGB3 or AAV2/8-hG1.7p.coCNGA3 by intraocular administration in the prior open-label, Phase I/II, dose escalation study (EudraCT 2016-002290-35 or EudraCT 2018-003431-29)
- Are willing to adhere to the protocol and long-term follow-up
Individuals will be excluded who:
Are unwilling or unable to meet the requirements of the study
メイラGTx連絡先情報がありません。
2 2カ国の場所
Michigan
Kellogg Eye Center, Ann Arbor, Michigan, 48105, United States
Moorfields Eye Hospital NHS Foundation Trust, London, United Kingdom