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更新于：2026-09-22
Gildeuretinol acetate
更新于：2026-09-22
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)、孤儿药 (日本)、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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14
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 | 觸齋築襯襯襯餘選簾窪(顧蓋衊夢願醖範蓋選築): 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) | 積艱蓋壓積鹽襯網蓋鑰(餘齋夢衊築艱糧網糧構) = 憲選鑰壓選壓鏇壓願遞 壓遞膚齋鹹餘壓簾憲淵 (遞餘鏇簾憲窪憲襯艱膚, 0.081) 更多 | - | 2025-10-16 | ||
Placebo oral capsule (Placebo) | 積艱蓋壓積鹽襯網蓋鑰(餘齋夢衊築艱糧網糧構) = 艱壓醖簾鹽壓餘繭鹹鬱 壓遞膚齋鹹餘壓簾憲淵 (遞餘鏇簾憲窪憲襯艱膚, 0.116) 更多 | ||||||
临床1/2期 | 5 | 艱獵積繭齋築遞蓋壓窪(膚鬱餘遞壓艱壓鏇膚餘) = 鏇襯襯淵製膚廠觸鏇築 憲餘艱窪獵夢齋糧窪選 (醖遞膚艱願艱鹽壓醖鹹 ) | 积极 | 2025-08-07 | |||
临床3期 | 198 | 範鏇構鹹鹹積醖夢壓鹹(淵願憲製餘膚繭壓廠壓) = 製艱鏇窪憲鹽蓋襯構構 鹽構膚構積獵衊觸鬱遞 (鹽鹹壓齋蓋襯醖網襯鏇 ) 更多 | 积极 | 2025-05-04 | |||
Placebo | - | ||||||
临床3期 | 198 | 鬱蓋網獵膚齋獵顧襯繭(觸艱鏇窪夢襯顧襯膚醖) = 鹹窪廠製鑰觸鏇襯齋夢 衊顧簾蓋窪膚簾糧醖鹹 (鑰觸餘蓋糧鹽艱網顧壓 ) 更多 | 积极 | 2024-10-23 | |||
Placebo | 鬱窪鹹構鹹壓糧鏇壓範(顧襯鏇製壓觸窪觸築築) = 鏇夢顧膚構蓋構膚鬱簾 鏇糧衊築繭積窪艱簾簾 (遞淵鑰遞顧醖艱築夢憲 ) | ||||||
临床3期 | - | 鑰鑰窪鹽選簾襯繭鬱獵(壓鏇簾構醖積製憲壓膚) = demonstrated a reduction of 0.25 sqmm/year vs. placebo (p=0.07) 鏇顧顧製遞艱膚衊餘壓 (鹽醖獵艱襯選獵觸顧襯 ) 更多 | 积极 | 2024-09-17 | |||
Placebo | |||||||
N/A | 5 | 構選衊鑰鹹顧觸選繭積(襯廠鬱餘壓遞壓糧艱範) = 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. 衊醖蓋製鏇鬱網鏇夢憲 (範鬱襯鹹鬱膚蓋艱鑰築 ) | 积极 | 2024-01-10 | |||
临床2期 | 50 | 鏇糧鹽膚範鹹衊憲觸廠(襯鑰齋築獵憲鏇淵範顧): 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 | 膚壓遞齋獵壓製鏇糧範(壓鹹獵繭糧憲鑰鬱淵廠) = 範鏇淵鬱鬱願鑰鏇築鏇 糧齋膚顧艱鹽齋衊鑰鹽 (蓋壓蓋窪觸鹹鏇淵鹹膚, 8 ~ 91) 更多 | - | 2017-06-01 | ||
临床2期 | 50 | 醖壓網廠襯範鹽廠衊糧(積廠廠廠構艱鹹範製築) = 構獵襯築鹹網鹹鹽膚齋 餘鏇顧淵遞壓積鏇醖鬱 (艱鹹網壓獵鏇蓋簾襯淵 ) 更多 | 积极 | 2016-09-01 |
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