RPGR遺伝子のバリアントに関連するX連鎖網膜色素変性症の治療のための遺伝子治療試験
RPGR遺伝子のバリアントに関連するX連鎖性網膜色素変性症の治療のためのAAV5-RPGRの第3相ランダム化比較試験
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
調査の概要
研究の種類
入学 (実際)
段階
- フェーズ 3
連絡先と場所
研究場所
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California
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La Jolla、California、アメリカ、92093 0946
- Shiley Eye Institute Jacobs Retina Center
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Los Angeles、California、アメリカ、90027
- Childrens Hospital
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Palo Alto、California、アメリカ、94303
- Stanford Health Care
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Florida
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Gainesville、Florida、アメリカ、32607
- VitreoRetinal Associates, PA
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Georgia
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Atlanta、Georgia、アメリカ、30322
- Emory University
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Massachusetts
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Boston、Massachusetts、アメリカ、02114
- Massachusetts General Hospital - Center for Celiac Research and Treatment
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Michigan
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Ann Arbor、Michigan、アメリカ、48105
- Univ of Michigan Medical Center
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North Carolina
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Durham、North Carolina、アメリカ、27705
- Duke Eye Center
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Pennsylvania
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Pittsburgh、Pennsylvania、アメリカ、15213
- University of Pittsburgh Medical Center (UPMC)
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Texas
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Bellaire、Texas、アメリカ、77401
- Retina Consultants of Houston
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Edinburgh、イギリス、EH3 9HA
- NHS Lothian
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Glasgow、イギリス、G12 0YN
- Gartnavel General Hospital
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Leeds、イギリス、LS9 7TF
- St James University Hospital
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London、イギリス、EC1V 2PD
- Moorfields Eye Hospital
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Jerusalem、イスラエル、91120
- Hadassah Medical Center
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Florence、イタリア、50134
- Azienda Ospedaliero Universitaria Careggi
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Milan、イタリア、20142
- Ospedale San Paolo
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Naples、イタリア、80131
- Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli
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Roma、イタリア、00198
- IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS
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Amsterdam、オランダ、1105AZ
- VUMC Amsterdam
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Nijmegen、オランダ、6525EX
- Radboudumc
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Ontario
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Toronto、Ontario、カナダ、M5G 1X8
- Hospital For Sick Children
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Basel、スイス、4031
- University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical
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Lausanne、スイス、1004
- Universite de Lausanne, Hopital ophtalmique Jules-Gonin
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Madrid、スペイン、28040
- Hosp Univ Fund Jimenez Diaz
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Glostrup Municipality、デンマーク、2600
- Rigshospitalet Glostrup
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Paris、フランス、75012
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
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Ghent、ベルギー、9000
- UZ Gent
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参加基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
説明
Inclusion Criteria:
- Male or female
- 3 years of age or older
- Has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory
Exclusion Criteria:
- Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration
- Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule
- Has undergone prior retinal surgery involving the macula, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery
研究計画
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：処理
- 割り当て：ランダム化
- 介入モデル：並列代入
- マスキング：独身
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
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実験的：Experimental - Immediate Treatment
Intermediate dose. |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
他の名前：
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
|
他の：Deferred Treatment
Deferred Treatment |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
他の名前：
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
|
実験的：Experimental Immediate Treatment
Low dose. |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
他の名前：
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Change From Baseline to Week 52 in Vision-guided Mobility Assessment (VMA) as Measured by the Ability of the Participant to Navigate Through a VMA Maze
時間枠：From Baseline to 52 Weeks
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Change from baseline to Week 52 in VMA as measured by the ability of the participant to navigate through a VMA maze. |
From Baseline to 52 Weeks
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (Mean Retinal Sensitivity Within the Central 10 Degree Excluding Scotoma in Static Perimetry [MRS10]) in Static Perimetry at Week 52
時間枠：From Baseline to Week 52
|
Change from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry at Week 52 will be assessed. |
From Baseline to Week 52
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Change From Baseline in Mean Retinal Sensitivity of Worse-seeing Eye Within the Central 10 Degrees Excluding Scotoma in Static Perimetry (MRS10) at Week 52
時間枠：From Baseline to Week 52
|
Change from baseline in mean retinal sensitivity of worse-seeing eye within the central 10 degrees excluding scotoma in static perimetry (MRS10) at Week 52 will be assessed. |
From Baseline to Week 52
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Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52
時間枠：From Baseline to Week 52
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Pointwise response in full visual field at Week 52 will be assessed. |
From Baseline to Week 52
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Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52
時間枠：From Baseline to Week 52
|
Pointwise response in worse-seeing eye in full visual field at Week 52 will be assessed. |
From Baseline to Week 52
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Change in Retinal Function as Assessed by Pointwise Response in the Central 30 Degrees Visual Field at Week 52
時間枠：From Baseline to Week 52
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Pointwise response in the central 30 degrees visual field at Week 52 will be assessed. |
From Baseline to Week 52
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Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52
時間枠：From Baseline to Week 52
|
Pointwise response in worse-seeing eye in the central 30 degrees visual field at Week 52 will be assessed. |
From Baseline to Week 52
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Change From Baseline in Retinal Function as Assessed by Mean Retinal Sensitivity Within the Full Visual Field (MRS90) in Static Perimetry at Week 52
時間枠：From Baseline to Week 52
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Change from baseline in retinal function as assessed by mean retinal sensitivity within the full visual field (MRS90) in static perimetry at Week 52 will be assessed. |
From Baseline to Week 52
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Change in Functional Vision by Using Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" at Week 52
時間枠：From Baseline to Week 52
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Change in functional vision by using VMA assessment in the "Worse-seeing Eye" at Week 52. |
From Baseline to Week 52
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Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52
時間枠：From Baseline to Week 52
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Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52. |
From Baseline to Week 52
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Change From Baseline in Visual Function as Assessed by Monocular Low Luminance Visual Acuity Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter score at Week 52
時間枠：From Baseline to Week 52
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Change from baseline in visual function as assessed by monocular low luminance visual acuity using the ETDRS chart letter score at Week 52. |
From Baseline to Week 52
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Change From Baseline in Visual Function as Assessed by monocular Best Corrected Visual Acuity (BCVA) Using the ETDRS Chart Letter Score at Week 52
時間枠：From Baseline to Week 52
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Change From Baseline in visual function as assessed by monocular BCVA using the ETDRS chart letter score at Week 52. |
From Baseline to Week 52
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Change From Baseline in Visual Function as Assessed by Low Luminance Visual Acuity Using the ETDRS Chart Letter Score in Worse-seeing Eye at Week 52
時間枠：From Baseline to Week 52
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Change from baseline in visual function as assessed by low luminance visual acuity using the ETDRS chart letter score in worse-seeing eye at Week 52. |
From Baseline to Week 52
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Number of Participants with Ocular and Non-ocular Adverse Events
時間枠：Day 1 - Week 52
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Number of participants with ocular and non-ocular adverse events will be assessed. |
Day 1 - Week 52
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Number of Participants With Abnormalities in Laboratory Assessments
時間枠：Day 1 - 52 Weeks
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Number of participants with abnormalities in laboratory assessments will be assessed. |
Day 1 - 52 Weeks
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協力者と研究者
スポンサー
捜査官
- スタディディレクター：Study Director、MeiraGTx Ocular UK Ltd
研究記録日
主要日程の研究
研究開始 (実際)
一次修了 (実際)
研究の完了 (実際)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
その他の研究ID番号
- CR109258
- 2020-002873-88 (EudraCT番号)
- MGT-RPGR-021 (その他の識別子：MeiraGTx Ocular UK Ltd)
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
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