Ixoberogene Soroparvovec (Ixo-vec) Contralateral Dosing Study in Participants With Neovascular Age-related Macular Degeneration
Evaluation of the Safety and Tolerability of Ixoberogene Soroparvovec (Ixo-vec) Intravitreal Gene Therapy in the Second (Contralateral) Eye of Participants With Bilateral Neovascular Age-related Macular Degeneration.
The purpose of this study is to evaluate safety, effectiveness and durability of a gene therapy called Ixo-vec (Ixoberogene soroparvovec) when administered to the contralateral (second) eye of adult participants (≥ 50 years of age) who have been diagnosed with bilateral neovascular (wet) age related macular degeneration (nAMD). The study will enroll adults with nAMD in both eyes, including participants who previously received Ixo-vec treatment in one (initial) eye and/or participants who will receive Ixo-vec treatment for the first time. This study focuses on how the treatment works when both eyes are treated at different times and how effective and long-lasting Ixo-vec treatment is in the second (contralateral) eye.
Participants will receive a single administration of Ixo-vec in the contralateral eye and will be followed for approximately 5 years to evaluate safety, efficacy and durability of contralateral treatment. Secondary objectives include assessments that will evaluate clinical activity, including visual and anatomic outcomes, as well as the need for supplemental anti-VEGF therapy.
The study is intended to provide additional information on the safety, tolerability, and use of Ixo-vec in bilateral treatment.
調査の概要
詳細な説明
This is a Phase 2, single-arm, open-label, multi-center study in participants who are ≥ 50 years old with a diagnosis of bilateral choroidal neovascularization (CNV) secondary to neovascular (wet) age related macular degeneration (nAMD). This study is designed to evaluate the safety, tolerability, and efficacy of Ixo-vec dosing in the contralateral (second) eye.
The study includes participants who have previously received Ixo-vec in one eye in a prior clinical study, as well as Ixo-vec-naïve participants eligible for sequential bilateral administration. The study will evaluate the use of Ixo-vec when both eyes are treated at different time points.
Neovascular AMD is a progressive retinal disease characterized by the growth of abnormal blood vessels originating from the choroid, which can lead to vision loss. Current standard of care includes repeated intravitreal (IVT) administration of anti-vascular endothelial growth factor (anti-VEGF) therapies.
Ixo-vec (also known as Ixoberogene soroparvovec, ADVM-022 or AAV.7m8-aflibercept) is an adeno-associated virus (AAV)-based gene therapy designed to enable sustained intraocular expression of an anti-VEGF protein following a single IVT administration.
The primary objective of this study is to assess the safety and tolerability of Ixo-vec administered to the contralateral eye. Secondary objectives include assessments that will evaluate clinical activity, including visual and anatomic outcomes, as well as the need for supplemental anti-VEGF therapy over time.
研究の種類
入学 (推定)
段階
- フェーズ2
連絡先と場所
研究連絡先
- 名前：Adverum Study Contact
- 電話番号：650-656-9323
- メール：ADVM02214ClinOp@adverum.com
研究場所
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Arizona
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Phoenix、Arizona、アメリカ、85053
- 募集
- Adverum site 126
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California
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Bakersfield、California、アメリカ、93309
- 募集
- Adverum site 109
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Fullerton、California、アメリカ、92835
- 募集
- Adverum site 169
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Florida
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Deerfield Beach、Florida、アメリカ、33064
- 募集
- Adverum site 124
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Fort Lauderdale、Florida、アメリカ、33308
- 募集
- Adverum site 176
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Jacksonville、Florida、アメリカ、32216
- 募集
- Adverum site 168
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Hawaii
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‘Aiea、Hawaii、アメリカ、96701
- 募集
- Adverum site 149
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Illinois
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Lemont、Illinois、アメリカ、60439
- 募集
- Adverum site 179
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New Jersey
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Teaneck、New Jersey、アメリカ、07666
- 募集
- Adverum site 171
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South Carolina
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West Columbia、South Carolina、アメリカ、29169
- 募集
- Adverum site 122
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Texas
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The Woodlands、Texas、アメリカ、77384
- 募集
- Adverum site 107
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The Woodlands、Texas、アメリカ、77384
- 募集
- Adverum site 231
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参加基準
適格基準
就学可能な年齢
- 大人
- 高齢者
健康ボランティアの受け入れ
説明
INCLUSION CRITERIA:
- Male or female, aged ≥ 50 years at Screening Visit 1.
- Must agree to use an acceptable form of contraception.
- Have diagnosis of bilateral CNV secondary to nAMD.
Meet ETDRS BCVA letter score criteria:
- For Ixo-vec-experienced participants: Contralateral eye has an ETDRS BCVA letter score within a protocol-defined range appropriate for nAMD clinical trials.
- For Ixo-vec-naïve participants: Both the initial eye and contralateral eye have ETDRS BCVA letter score within a protocol-defined range appropriate for nAMD clinical trials
- Have prior treatment history consistent with protocol-defined requirements, including prior anti-VEGF therapy and, where applicable, prior Ixo-vec exposure.
- Demonstrate a meaningful anatomic response to anti-VEGF in the contralateral eye (all participants) and in the initial eye (Ixo-vec-naïve participants only). Meaningful anatomic response to prior anti-VEGF therapy as defined in the protocol.
- Able to comply with study procedures according to the Investigator's judgment.
Other protocol-defined INCLUSION CRITERIA apply.
EXCLUSION CRITERIA:
A/ General Exclusion Criteria
- Have history of a medical condition (systemic disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding) giving reasonable suspicion of a condition that contraindicates the use of Ixo-vec, compromises the participant's ability to comply with the planned study assessments, or that might affect the interpretation of the results of the study or render the participant at high risk for treatment complications in the opinion of the Investigator. History of severe coronavirus disease of 2019 (COVID-19) infection may meet this exclusion criterion if, in the opinion of the Investigator, it is likely to lead to any of the complications listed above.
- Received prior gene therapy (other than Ixo-vec, where applicable)
- Currently receiving steroids at Screening Visit 1 in the previously treated eye (Ixo-vec-experienced participants) or in either eye (Ixo-vec-naïve participants).
- Received any non-gene IMP or medical device in the contralateral eye (and/or initial eye for Ixo-vec-naïve participants) within 3 months of Screening Visit 1
- Have history of allergy, hypersensitivity, or contraindications to the use of any product or its excipients administered as part of this study.
- Have evidence of poorly controlled diabetes or glycated hemoglobin (HbA1c) ≥ 8.0%
- Have history or evidence of cardiovascular diseases and ongoing bleeding disorders as defined by the protocol.
- Use of systemic immunosuppressive therapy within a protocol-defined period.
- Received systemic anti-VEGF therapy within a protocol-defined period.
Have history of malignancy within the 5 years prior to Screening Visit 1, except for adequately treated malignancies as defined by the protocol.
B/Ocular Exclusion Criteria
- Have any active ocular or periocular infection in the contralateral eye in the contralateral eye (all participants) or in the initial eye (Ixo-vec-naïve participants only).
- Have any history or evidence of a concurrent intraocular condition in the contralateral eye (all participants) or in the initial eye (Ixo-vec-naïve participants only), including retinal diseases other than nAMD, that, in the judgment of the Investigator, could reduce the potential for visual improvement, confound assessment of the macula or require medical or surgical intervention during the study.
- Have prior ocular surgery or treatment that may confound assessment or increase risk.
- Have any history or evidence of retinal detachment (with or without repair) or retinal pigment epithelium rip/tear in the contralateral eye (all participants) or in the initial eye (Ixo-vec-naïve participants only), as determined by the Investigator.
- Have uncontrolled ocular hypertension or glaucoma as defined by the protocol.
- Have any history of inflammatory anterior chamber cell or vitreous cell ≥ 2+ in the initial eye, that, in the opinion of the Investigator and Sponsor, predisposes the contralateral eye to a heightened risk of intraocular inflammation. Enrollment of a participant with prior anterior chamber cell or vitreous cell ≥2+ in the initial eye requires discussion with the medical monitor.
Other protocol-defined EXCLUSION CRITERIA apply.
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：なし
- 介入モデル：単一グループの割り当て
- マスキング：なし（オープンラベル）
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：Ixo-vec dosing in Contralateral Eye
Population of Ixo-vec-Experienced or Ixo-vec Naïve Participants.
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Ixo-vec (6 × 10^10 vg/eye) will be administered intravitreally
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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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Incidence of ocular adverse events (AEs)
時間枠：Baseline through Week 28
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The number of participants who experience an ocular adverse event will be summarized.
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Baseline through Week 28
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Incidence of ocular serious adverse events (SAEs).
時間枠：Baseline through Week 28
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The number of participants who experience ocular serious adverse event will be summarized.
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Baseline through Week 28
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Incidence of non-ocular serious adverse events (SAEs).
時間枠：Baseline through Week 28
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The number of participants who experience non-ocular serious adverse event will be summarized.
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Baseline through Week 28
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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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Mean change from baseline in best corrected visual acuity (BCVA) over time through Week 28.
時間枠：Baseline through Week 28
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BCVA will be measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart.
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Baseline through Week 28
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Mean change from baseline in central subfield thickness (CST) over time through Week 28.
時間枠：Baseline through Week 28
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CST as measured by spectral domain optical coherence tomography (SD-OCT).
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Baseline through Week 28
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Mean number of supplemental aflibercept IVT injections received from Week 4 through Week 28
時間枠：Week 4 through Week 28
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Efficacy will be measured by the number of supplemental aflibercept injections post Ixo-vec administration.
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Week 4 through Week 28
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Percentage of participants who are supplemental aflibercept injection-free through Week 28.
時間枠：Baseline through Week 28
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Efficacy will be measured as the percentage of participants who will not received supplemental aflibercept injections post Ixo-vec administration.
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Baseline through Week 28
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協力者と研究者
捜査官
- スタディディレクター：Adam Turpcu, PhD、Adverum Biotechnologies, Inc.
研究記録日
主要日程の研究
研究開始 (実際)
一次修了 (推定)
研究の完了 (推定)
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
キーワード
その他の研究ID番号
- ADVM-022-14
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?
IPD プランの説明
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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