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更新于：2026-09-08
Gildeuretinol acetate
更新于：2026-09-08
概要
基本信息
非在研机构- |
最高研发阶段临床3期 |
首次获批日期- |
最高研发阶段(中国)- |
特殊审评快速通道 (美国)、孤儿药 (美国)、罕见儿科疾病 (美国)、孤儿药 (欧盟)、突破性疗法 (美国) |
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结构/序列
分子式C22H32O2 |
InChIKeyQGNJRVVDBSJHIZ-BMSJAHLVSA-N |
CAS号118139-36-9 |
关联
5
项与 Gildeuretinol acetate 相关的临床试验NCT07419334
A Phase 3 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Safety, Tolerability, and Efficacy of Gildeuretinol Acetate (ALK 001) on the Progression of Stargardt Disease (STGD)
NCT04239625
A Phase 2 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Long Term Safety, Tolerability, Pharmacokinetics and Effects of ALK-001 on the Progression of Stargardt Disease
NCT03845582
A Phase 2/3 Multicenter, Randomized, Double-masked, Parallel-group, Placebo-controlled Study to Investigate the Safety, Pharmacokinetics, Tolerability, and Efficacy of ALK-001 in Geographic Atrophy Secondary to Age-related Macular Degeneration
100 项与 Gildeuretinol acetate 相关的临床结果
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100 项与 Gildeuretinol acetate 相关的转化医学
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100 项与 Gildeuretinol acetate 相关的专利（医药）
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15
项与 Gildeuretinol acetate 相关的文献（医药）2026-06-01Ophthalmology Retina
Gildeuretinol Acetate versus Placebo for Geographic Atrophy Secondary to Age-Related Macular Degeneration
Article
作者: DeBartolomeo, Gabrielle M ; O'Malley, Adrian E ; Ferrone, Philip J ; Boyer, David S ; Graham, Kenneth B ; Kay, Christine N ; Jiang, Honghua ; Khan, Seemi ; Melamud, Alexander ; Harris, Lindsey D ; Saad, Leonide ; Washington, Ilyas
OBJECTIVE:
To assess the effects of gildeuretinol acetate (C20D3-vitamin A) in geographic atrophy (GA).
DESIGN:
A 24-month, double-masked, multicenter, randomized, placebo-controlled trial.
PARTICIPANTS:
Participants aged ≥60 years with GA.
INTERVENTIONS:
Randomized 2:1 to oral gildeuretinol acetate 14 mg daily or placebo.
MAIN OUTCOME MEASURES:
The primary endpoint was GA growth rate from baseline to 24 months. A prespecified sensitivity analysis accounted for a 6-month delay in onset of therapeutic effect. Secondary endpoints included change from baseline in low-luminance visual acuity (LLVA), best-corrected visual acuity (BCVA), Visual Function Questionnaire-25, and Functional Reading Independence (FRI) Index.
RESULTS:
Among 198 randomized participants (mean [standard deviation {SD}] age, 78.4 [7.0] years; 68.7% women), 69.2% completed the 24-month study. In the gildeuretinol and placebo groups, baseline mean (SD) total GA lesion area in mm2 was 7.72 (6.10) and 7.09 (4.16); BCVA letter scores were 56.2 (21.3) (Snellen equivalent: ∼20/80) and 58.8 (18.0) (∼20/70); and LLVA letter scores were 31.1 (19.0) (∼20/200) and 31.6 (17.8) (∼20/250), respectively. Mean GA growth rates were 1.62 mm2/yr with gildeuretinol and 1.87 mm2/yr with placebo (least squares [LSs] mean difference: -0.25 mm2/yr; 95% confidence interval [CI]: -0.53 to 0.03; P = 0.075), a 13.4% relative reduction for gildeuretinol versus placebo. The LS mean (standard error) GA growth rates from 6 to 24 months were 1.90 (0.12) mm2/yr (placebo) and 1.61 (0.08) mm2/yr (gildeuretinol), a 15.3% relative reduction (-0.29 mm2/yr; 95% CI: -0.576 to -0.004; nominal P = 0.047). At 24 months, LLVA declined 3.9 and 8.3 ETDRS letters in the gildeuretinol and placebo groups, respectively (difference: 4.4 letters; 95% CI: 0.40-8.41; nominal P = 0.031). Best-corrected visual acuity declined 6.9 letters with gildeuretinol and 10.2 letters with placebo (difference: 3.3 letters; 95% CI: -0.63 to 7.23; P = 0.099). Visual Function Questionnaire-25 scores declined 4.81 fewer points with gildeuretinol than placebo (95% CI: 0.87-8.76; nominal P = 0.017), and FRI scores declined 0.35 fewer points (95% CI: 0.11-0.58; nominal P = 0.004).
CONCLUSIONS:
The prespecified primary endpoint was not met. Prespecified piecewise and secondary functional and patient-reported analyses favored gildeuretinol with nominal P values; these exploratory findings support further evaluation in adequately powered trials.
TRIAL REGISTRATION:
NCT03845582.
FINANCIAL DISCLOSURE(S):
Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.
2026-05-01CURRENT OPINION IN OPHTHALMOLOGY
Nutritional supplements: current evidence for retinitis pigmentosa and Stargardt disease
Review
作者: Gregori, Ninel Z. ; Barthelemy, Normila ; Sengillo, Jesse D. ; Lee, Winston ; Lam, Byron L.
Purpose of review:
Inherited retinal diseases (IRDs) are genetically and phenotypically heterogeneous disorders that cause progressive vision loss and lack broadly effective disease-modifying therapies. Increasing evidence implicates metabolic stress, oxidative injury, and photoreceptor–retinal pigment epithelium (RPE) dysfunction in IRD pathophysiology. This review evaluates the evidence and limitations surrounding nutritional and metabolic interventions for retinitis pigmentosa and Stargardt disease.A narrative review of preclinical studies, randomized controlled trials, and contemporary genetic re-analyses was performed. Nutritional interventions reviewed include vitamin A, vitamin E, N-acetylcysteine (NAC), omega-3 fatty acids (docosahexaenoic acid), carotenoids and apocarotenoids, and the deuterated vitamin A analog C20-D3-retinyl acetate (ALK-001).
Recent findings:
Initial studies suggesting a protective effect of vitamin A in retinitis pigmentosa were not confirmed in subsequent trials or genetic re-analyses, which identified baseline differences between groups as a possible contributor to the original findings. In contrast, supplemental vitamin E may accelerate disease progression. NAC demonstrated acceptable tolerability and modest short-term improvements in visual function in early-phase trials, with a phase 3 study ongoing. Omega-3 fatty acids, carotenoids, and apocarotenoids have not shown consistent clinically meaningful benefit in retinitis pigmentosa or Stargardt disease despite strong mechanistic rationale. In Stargardt disease, ALK-001 reduces lipofuscin accumulation in animal models and clinical trials are ongoing to assess efficacy in patients.
Summary:
Most nutritional supplements studied to date have not demonstrated durable or clinically meaningful benefit in IRDs. Vitamin A supplementation in retinitis pigmentosa is no longer supported by current evidence, while NAC remains a promising metabolic therapy under investigation. High-quality, genotype-informed clinical trials with clinically relevant endpoints are needed before nutritional interventions can be incorporated into IRD management guidelines.
2021-09-01The Journal of biological chemistry2区 · 生物学
Vitamin A cycle byproducts impede dark adaptation
2区 · 生物学
Article
作者: Dan Zhang ; Kiera Robinson ; Leonide Saad ; Ilyas Washington
Impaired dark adaptation (DA), a defect in the ability to adjust to dimly lit settings, is a universal hallmark of aging. However, the mechanisms responsible for impaired DA are poorly understood. Vitamin A byproducts, such as vitamin A dimers, are small molecules that form in the retina during the vitamin A cycle. We show that later in life, in the human eye, these byproducts reach levels commensurate with those of vitamin A. In mice, selectively inhibiting the formation of these byproducts, with the investigational drug C20D3-vitamin A, results in faster DA. In contrast, acutely increasing these ocular byproducts through exogenous delivery leads to slower DA, with otherwise preserved retinal function and morphology. Our findings reveal that vitamin A cycle byproducts alone are sufficient to cause delays in DA and suggest that they may contribute to universal age-related DA impairment. Our data further indicate that the age-related decline in DA may be tractable to pharmacological intervention by C20D3-vitamin A.
100 项与 Gildeuretinol acetate 相关的药物交易
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外链
| KEGG | Wiki | ATC | Drug Bank |
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| D12713 | - | - | - |
研发状态
10 条进展最快的记录, 后查看更多信息
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 斯塔加特病 | 临床3期 | 美国 | 2026-06-03 | |
| 干性年龄相关性黄斑病变 | 临床3期 | 美国 | 2019-05-07 | |
| 地图样萎缩 | 临床3期 | 美国 | 2019-05-07 | |
| II型糖原贮积病 | 临床3期 | 美国 | 2019-05-07 | |
| 视网膜变性疾病 | 临床1期 | - | 2014-04-01 |
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临床结果
临床结果
适应症
分期
评价
查看全部结果
临床2期 | 198 | 襯築醖獵獵窪繭觸選糧(夢築膚選遞願繭醖築積): Difference = 3.3 (95.0% CI, -0.63 ~ 7.23), P-Value = 0.099 更多 | 不佳 | 2026-06-01 | |||
Placebo | |||||||
临床2/3期 | 200 | (ALK-001) | 窪鏇繭憲糧膚選築廠願(簾觸襯鏇壓積築鏇鏇艱) = 積廠憲膚製鏇顧積繭廠 鹽餘網範願簾鬱築艱網 (鹽淵齋遞淵蓋壓膚齋齋, 0.081) 更多 | - | 2025-10-16 | ||
Placebo oral capsule (Placebo) | 窪鏇繭憲糧膚選築廠願(簾觸襯鏇壓積築鏇鏇艱) = 網齋鏇廠壓齋網繭蓋鬱 鹽餘網範願簾鬱築艱網 (鹽淵齋遞淵蓋壓膚齋齋, 0.116) 更多 | ||||||
临床1/2期 | 5 | 艱鏇範窪窪鑰製襯網鑰(簾齋鹹廠繭獵窪鹹淵蓋) = 膚淵鹽鹹範憲糧築築鬱 壓網範鹹製積蓋觸襯鑰 (衊壓糧膚醖膚築齋積網 ) | 积极 | 2025-08-07 | |||
临床3期 | 198 | 築廠遞鑰鏇鹽願製築廠(襯簾鹽糧觸觸構築膚壓) = 憲夢淵蓋繭襯積選憲憲 遞醖構顧選鬱壓窪鏇夢 (鏇襯顧膚淵糧鏇餘夢積 ) 更多 | 积极 | 2025-05-04 | |||
Placebo | - | ||||||
临床3期 | 198 | 簾鑰顧製鏇願夢願艱獵(觸淵餘壓願構憲壓蓋廠) = 鬱餘網獵夢鏇積願簾壓 糧壓鏇築衊鏇衊獵鏇蓋 (醖獵鏇鑰衊獵繭夢觸製 ) 更多 | 积极 | 2024-10-23 | |||
Placebo | 壓窪築鏇鹹鬱鹽鏇範繭(襯襯鹽艱夢蓋範製範夢) = 鹹齋網艱築壓艱選鹹鑰 製齋齋鬱廠壓簾鹹獵餘 (夢廠鏇範鏇築顧簾艱膚 ) | ||||||
临床3期 | - | 餘膚觸觸選網構製膚願(糧願顧餘積鬱鹹醖膚顧) = demonstrated a reduction of 0.25 sqmm/year vs. placebo (p=0.07) 鑰壓鑰夢鑰簾膚鬱壓艱 (繭窪鬱淵膚膚範積積繭 ) 更多 | 积极 | 2024-09-17 | |||
Placebo | |||||||
N/A | 5 | 鑰艱襯鏇鬱顧鏇鬱齋製(憲築鑰憲壓積襯構獵簾) = A total of five patients have completed the 24-month study. Following the study, these patients have remained on treatment and continue to show no disease progression, with the longest duration of therapy to date being more than seven years. 繭顧廠廠範選網構鹹製 (顧構餘壓簾觸齋夢醖廠 ) | 积极 | 2024-01-10 | |||
临床2期 | 50 | 廠鹹製繭鹹鬱夢鑰觸膚(蓋製網艱繭製遞鬱繭築): Difference(%) = -21, P-Value = <0.001 更多 | 积极 | 2023-11-03 | |||
Placebo | |||||||
临床2期 | 斯塔加特病 ABCA4 gene | 50 | 鹽積壓廠顧簾壓獵夢襯(選廠衊壓窪蓋艱夢鹽鬱) = 淵積網鏇鏇觸繭積積製 積網選壓艱構壓淵齋網 (餘顧繭築繭廠鏇醖窪齋, 8 ~ 91) 更多 | - | 2017-06-01 | ||
临床2期 | 50 | 餘醖衊網膚獵鏇構衊衊(網夢範蓋鹹鑰顧醖範製) = 糧築廠願襯餘糧選鏇憲 憲築鏇遞鬱鏇構憲淵窪 (鑰糧獵積鑰鑰窪製鏇積 ) 更多 | 积极 | 2016-09-01 |
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