N-acetyl-D-mannosamine(N-acetyl-D-mannosamine) - 在研适应症：远端性肌病，局灶性节段性肾小球硬化症，遗传性包涵体肌病_专利_临床

概要

基本信息

药物类型

小分子化药

别名

2-Actamido-2-deoxy-D-mannose、D-ManNAc、LB-301 - Leadiant Biosciences

+ [7]

靶点-

作用机制

糖基化刺激剂、蛋白调节剂

治疗领域

神经系统疾病遗传病与畸形皮肤和肌肉骨骼疾病+ [2]

在研适应症

远端性肌病局灶性节段性肾小球硬化症遗传性包涵体肌病

非在研适应症-

原研机构

National Institutes of Health

在研机构

National Human Genome Research Institute

National Institutes of Health

Leadiant Biosciences, Inc.

非在研机构

National Institute of Diabetes & Digestive & Kidney Diseases

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-

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结构

分子式C8H15NO6

InChIKeyMBLBDJOUHNCFQT-WCTZXXKLSA-N

CAS号3615-17-6

关联

9

项与 N-acetyl-D-mannosamine 相关的临床试验

NCT06664814 / Not yet recruiting临床2期IIT

An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Primary Focal Segmental Glomerulosclerosis

Background:
Focal segmental glomerulosclerosis (FSGS) is a disease that causes scarring in parts of the kidneys that filter waste. This can lead to protein loss in the urine, which can worsen kidney function. The kidneys may fail over time, and dialysis or a kidney transplant may be needed. Other treatments for this disease do not always work and often have adverse effects. Better treatments for FSGS are needed.
Objective:
To test a study drug (ManNAc) in people with FSGS.
Eligibility:
People aged 18 years and older with FSGS.
Design:
Participants will have 6 to 7 clinic visits over 14 weeks. Two of the visits will require overnight stays for 2 or 3 nights.
ManNAc is a white powder that comes in a sachet. It is dissolved in water and taken twice a day by mouth. Participants will take their first dose at the clinic. They will learn how to store ManNAc and prepare each dose. They will record their doses in a diary. They will also write down any adverse effects or troubles they have using the drug at home.
During clinic visits, participants will have physical exams with blood and urine tests. They will complete questionnaires about their health, sleep habits, and fatigue symptoms.
During overnight visits, participants will also have 24-hour urine collection.
A study team member will call participants 1 week after the first dose to check on their health. Follow-up phone calls will then be every 2 weeks after each clinic visit.
Participants may meet with a dietitian to discuss nutrition while taking the ManNAc.
Participants may choose to have genetic tests.

开始日期2024-11-26

申办/合作机构

National Human Genome Research Institute

ACTRN12623000609651 / RecruitingN/AIIT

An open-label, randomised, three-arm crossover study to examine the comparative pharmacokinetics of ManNAc administered as an oral solution and as two novel oral tablet formulations in healthy adult males

开始日期2023-07-13

申办/合作机构

University of South Australia

ACTRN12622000394741 / CompletedN/AIIT

An open-label, randomised, two-arm crossover study to determine the absolute oral bioavailability of ManNAc in healthy adult males

开始日期2022-11-17

申办/合作机构

University of South Australia

100 项与 N-acetyl-D-mannosamine 相关的临床结果

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100 项与 N-acetyl-D-mannosamine 相关的转化医学

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100 项与 N-acetyl-D-mannosamine 相关的专利（医药）

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156

项与 N-acetyl-D-mannosamine 相关的文献（医药）

2024-12-31·Gut microbes

Integrative analysis with microbial modelling and machine learning uncovers potential alleviators for ulcerative colitis

Article

作者: Zhu, Jinlin ; Chen, Wei ; Lu, Wenwei ; Hu, Mingyi ; Zhang, Hao ; Chen, Jing ; Yin, Jialin ; Wang, Hongchao

Ulcerative colitis (UC) is a challenging form of inflammatory bowel disease, and its etiology is intricately linked to disturbances in the gut microbiome. To identify the potential alleviators of UC, we employed an integrative analysis combining microbial community modeling with advanced machine learning techniques. Using metagenomics data sourced from the Integrated Human Microbiome Project, we constructed individualized microbiome community models for each participant. Our analysis highlighted a significant decline in both α and β-diversity of strain-level microbial populations in UC subjects compared to controls. Distinct differences were also observed in the predicted fecal metabolite profiles and strain-to-metabolite contributions between the two groups. Using tree-based machine learning models, we successfully identified specific microbial strains and their associated metabolites as potential alleviators of UC. Notably, our experimental validation using a dextran sulfate sodium-induced UC mouse model demonstrated that the administration of Parabacteroides merdae ATCC 43,184 and N-acetyl-D-mannosamine provided notable relief from colitis symptoms. In summary, our study underscores the potential of an integrative approach to identify novel therapeutic avenues for UC, paving the way for future targeted interventions.

2024-09-04·RSC Advances

Soft X-ray spectromicroscopy of human fibroblasts with impaired sialin function.

Article

作者: Miinalainen, Ilkka ; Darin, Niklas ; Kangas, Salla M ; Uusimaa, Johanna ; Patanen, Minna ; Angervaniva, Pia ; Blomqvist, Maria ; Huttula, Marko ; Hinttala, Reetta ; Mansikkala, Tuomas

Salla disease (SD) is a lysosomal storage disease where free sialic acid (SA) accumulates in lysosomes due to the impaired function of a membrane protein, sialin. Synchrotron radiation-based scanning transmission soft X-ray spectromicroscopy (STXM) was used to analyze both SD patients' fibroblasts and normal human dermal fibroblasts (NHDF) from healthy controls. Both cell lines were also cultured with N-acetyl-d-mannosamine monohydrate (ManNAc) to see if it increased SA concentration in the cells. The STXM technique was chosen to simultaneously observe the morphological and chemical changes in cells. It was observed that free SA did not remain in the lysosomes during the sample processing, leaving empty vacuoles to the fibroblasts. The total cytosol and entire cell spectra, however, showed systematic differences between the SD and NHDF samples, indicating changes in the relative macromolecular concentrations of the cells. The NHDF cell lines contained a higher relative protein concentration compared to the SD cell lines, and the addition of ManNAc increased the relative protein concentration in both cell lines. In this study, two sample preparation methods were compared, resin-embedded thin sections and cells grown directly on sample analysis grids. While the samples grown on the grids exhibited clean, well-resolved spectra not masked by embedding resin, the low penetration depth of soft X-rays hindered the analysis to only the thin region of the microfilaments away from the thick nucleus.

2024-08-01·Clinical Pharmacology in Drug Development

Impact of Food on the Oral Absorption of N‐Acetyl‐D‐Mannosamine in Healthy Men and Women

Article

作者: Gahl, William A. ; Meola, Tahlia R. ; Fornasini, Gianfranco ; Reuter, Stephanie E. ; Evans, Allan M. ; Polasek, Thomas M. ; Huizing, Marjan

Abstract:

N‐Acetyl‐D‐mannosamine (ManNAc) is an endogenous monosaccharide and precursor of N‐acetylneuraminic acid (Neu5Ac), a critical sialic acid. ManNAc is currently under clinical development to treat GNE myopathy, a rare muscle‐wasting disease. In this randomized, open‐label, 2‐sequence, crossover study, 16 healthy women and men were administered a single oral dose of ManNAc under fasting and fed conditions. Blood samples were collected for 48 hours after dosing for quantification of plasma ManNAc and Neu5Ac concentrations. Noncompartmental pharmacokinetic and deconvolution analyses were performed using baseline‐corrected plasma concentration data. Administration of ManNAc in the fed state resulted in a 1.6‐fold increase in ManNAc exposure, compared to fasting conditions. A concurrent increase in Neu5Ac exposure was observed in the presence of food. Deconvolution analysis indicated that the findings were attributed to prolonged absorption rather than an enhanced rate of absorption. The impact of food on ManNAc pharmacokinetics was greater in women than men (fed/fasted area under the concentration‐time curve from time 0 to infinity mean ratio: 198% compared to 121%). It is hypothesized that the presence of food slows gastric emptying, allowing a gradual release of ManNAc into the small intestine, translating into improved ManNAc absorption. The results suggest that taking ManNAc with food may enhance its therapeutic activity and/or reduce the daily dosage requirement.

1

项与 N-acetyl-D-mannosamine 相关的新闻（医药）

2021-04-27

·BioSpace

LogicBio Therapeutics Announces Research Collaboration with Daiichi Sankyo

LEXINGTON, Mass., April 27, 2021 /PRNewswire/ -- LogicBio Therapeutics, Inc. (LogicBio - Nasdaq: LOGC), a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood, today announced the signing of a research collaboration and exclusive option agreement with Daiichi Sankyo Company, Limited (Daiichi Sankyo). Under the terms of the agreement, the companies will collaborate on the development of treatments for two undisclosed indications based on GeneRide™, LogicBio's proprietary gene insertion platform. The agreement also grants Daiichi Sankyo an option to negotiate and acquire worldwide licenses for LogicBio's development programs in these two indications. Financial terms of the collaboration are not disclosed. "I am very pleased to announce this collaboration as a new milestone in the validation of our GeneRide platform. In 2020, we cleared our first Investigational New Drug (IND) application for GeneRide for the treatment of pediatric methylmalonic acidemia (MMA) patients and initiated the Phase 1/2 SUNRISE clinical trial. We also formed our first research agreement with another major pharmaceutical company to develop a drug candidate for Crigler-Najjar syndrome," said Frederic Chereau, president and chief executive officer of LogicBio. "By partnering with Daiichi Sankyo, a global leader in developing innovative medicines, we are reinforcing our commitment to bring innovative therapeutics to people living with serious genetic conditions including many that can present symptoms as early as infancy." The GeneRide platform harnesses a cell's natural DNA repair process leading to durable therapeutic protein expression levels. GeneRide supports the development of a new generation of genetic medicine able to insert a DNA segment into the human genome while potentially avoiding certain risks associated with other gene editing technologies. The therapy uses a viral vector to deliver the therapeutic gene, known as a transgene, to the nuclei of a patient's cells via one-time infusion. LogicBio's two lead development programs based on the GeneRide platform are focused on rare pediatric conditions. The company is currently evaluating LB-001 in the Phase 1/2 SUNRISE trial in MMA and LB-301 in Crigler-Najjar syndrome through another research collaboration. LogicBio is also advancing the development of sAAVyTM, its next-generation AAV capsid platform, designed to overcome limitations with the current generation of AAV-based gene therapies. About LogicBio Therapeutics, Inc. LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood. The company's proprietary GeneRide™ platform is a new approach to precise gene insertion that harnesses a cell's natural DNA repair process leading to durable therapeutic protein expression levels. LogicBio's cutting-edge sAAVyTM capsid development platform is designed to support development of treatments in a broad range of indications and tissues. The company is based in Lexington, MA. For more information, visit . Bill Berry Berry & Company Public Relations W: 212-253-8881 C: 917-846-3862 bberry@berrypr.com Jenna Urban Berry & Company Public Relations W: 212-253-8881 C: 203-218-9180 jurban@berrypr.com Company Codes: NASDAQ-NMS:LOGC

合作

100 项与 N-acetyl-D-mannosamine 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB12536

研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
远端性肌病	临床3期	-	National Institutes of Health	-
远端性肌病	临床3期	-	Leadiant Biosciences, Inc.	-
局灶性节段性肾小球硬化症	临床2期	美国	National Human Genome Research Institute	2024-11-26
遗传性包涵体肌病	临床2期	美国	National Human Genome Research Institute	2015-02-05
肾脏疾病	临床1期	美国	National Institute of Diabetes & Digestive & Kidney Diseases	2016-07-07

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT02346461 (Pubmed \| Genet Med) 人工标引	临床2期	遗传性包涵体肌病	12	ManNAc	齋糧醖鬱範醖餘構製齋(糧簾淵餘膚選壓遞壓鑰) = 範獵齋鬱醖襯範蓋壓構選蓋繭齋選餘醖壓製膚 (鏇遞淵鹹壓夢襯膚鹽鬱 )	积极	2021-11-01
NCT02639260 (Phase 1 Study of N-Acetylmannosamine (ManNAc) for Glomerular Disease, ASN2020)	临床1期	肾小球疾病	7	ManNAc 3g single dose	鏇構鏇鬱襯鏇淵獵範淵(範艱獵醖鬱願艱選鬱鹽) = There were no serious adverse events. Most subjects (5 of 6) that received ManNAc twice daily showed a marked reduction in urine PCR (26-54%), which correlated with the degree of glomerular hyposialylation 廠廠構餘衊鹹廠艱鑰製 (繭網壓簾窪壓襯膚範顧 ) 更多	积极	2020-10-19
NCT02346461 (CTgov)	临床2期	遗传性包涵体肌病	12	ManNAc (ManNAc: Cohort A)	鬱鬱鹹衊艱襯鹽觸鹽獵(觸範膚糧願構憲積壓窪) = 積簾鏇齋憲鑰糧鏇鬱糧廠蓋遞鑰選築壓觸願艱 (積餘積築鑰糧壓憲餘淵, 鏇蓋鹹鹹獵艱齋鏇鹹淵 ~ 構積壓餘製鹹鑰獵膚糧) 更多	-	2019-04-16
NCT02346461 (CTgov)	临床2期	遗传性包涵体肌病	12	ManNAc (ManNAc: Cohort B)	鬱鬱鹹衊艱襯鹽觸鹽獵(觸範膚糧願構憲積壓窪) = 壓衊醖憲餘遞簾網齋襯廠蓋遞鑰選築壓觸願艱 (積餘積築鑰糧壓憲餘淵, 窪網醖構製築繭衊廠醖 ~ 鹹鑰衊範選糧夢範製鹹) 更多	-	2019-04-16
ASN2010	N/A	微小病变性肾病 Angptl4 \| NPHS2-Angptl4 \| PAN	-	ManNAc	願餘餘鏇壓鬱膚窪網鑰(遞製壓範願構鬱餘鑰醖) = 憲範鹽網鏇築膚夢齋範製顧醖糧範糧觸襯糧製 (遞構壓選窪構製憲繭獵 )	积极	2010-11-16