A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick disease type C (NPC). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.

开始日期2022-06-30

申办/合作机构

IntraBio, Inc.

NCT03759678 / Recruiting临床2期

Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study

This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Ataxia-Telangiectasia (A-T).
There are two phases to this study: the Parent Study, and the Extension Phase.
The Parent Study evaluates the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the symptomatic treatment of A-T.
The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of A-T.

开始日期2020-01-08

申办/合作机构

IntraBio, Inc.

NCT03759639 / Completed临床2期

Effects of N-Acetyl-L-Leucine on Niemann Pick Type C Disease: A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study.

This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Niemann-Pick type C disease (NPC).
There are two phases to this study: the Parent Study, and the Extension Phase.
The Parent Study evaluates the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the symptomatic treatment of NPC.
The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of NPC.

开始日期2019-09-04

申办/合作机构

IntraBio, Inc.

100 项与 N-乙酰-L-亮氨酸相关的临床结果

登录后查看更多信息

100 项与 N-乙酰-L-亮氨酸相关的转化医学

登录后查看更多信息

100 项与 N-乙酰-L-亮氨酸相关的专利（医药）

登录后查看更多信息

138

项与 N-乙酰-L-亮氨酸相关的文献（医药）

2024-02-01·The New England journal of medicine1区 · 医学

1区 · 医学

Article

作者: Brands, Marion ; Mengel, Eugen ; Foltan, Tomas ; Kolnikova, Miriam ; Gowing, Francesca ; Arash-Kaps, Laila ; Bremova-Ertl, Tatiana ; Wibawa, Pierre ; Marquardt, Thorsten ; Walterfang, Mark ; Strupp, Michael ; Gautschi, Matthias ; Schneider, Susanne A ; Ramaswami, Uma ; Jones, Simon ; Martakis, Kyriakos ; Reichmannová, Stella ; Kay, Richard ; Hahn, Andreas ; Sivananthan, Siyamini ; Gissen, Paul ; Park, Julien H

BACKGROUND:

Niemann-Pick disease type C is a rare lysosomal storage disorder. We evaluated the safety and efficacy of N-acetyl-l-leucine (NALL), an agent that potentially ameliorates lysosomal and metabolic dysfunction, for the treatment of Niemann-Pick disease type C.

METHODS:

In this double-blind, placebo-controlled, crossover trial, we randomly assigned patients 4 years of age or older with genetically confirmed Niemann-Pick disease type C in a 1:1 ratio to receive NALL for 12 weeks, followed by placebo for 12 weeks, or to receive placebo for 12 weeks, followed by NALL for 12 weeks. NALL or matching placebo was administered orally two to three times per day, with patients 4 to 12 years of age receiving weight-based doses (2 to 4 g per day) and those 13 years of age or older receiving a dose of 4 g per day. The primary end point was the total score on the Scale for the Assessment and Rating of Ataxia (SARA; range, 0 to 40, with lower scores indicating better neurologic status). Secondary end points included scores on the Clinical Global Impression of Improvement, the Spinocerebellar Ataxia Functional Index, and the Modified Disability Rating Scale. Crossover data from the two 12-week periods in each group were included in the comparisons of NALL with placebo.

RESULTS:

A total of 60 patients 5 to 67 years of age were enrolled. The mean baseline SARA total scores used in the primary analysis were 15.88 before receipt of the first dose of NALL (60 patients) and 15.68 before receipt of the first dose of placebo (59 patients; 1 patient never received placebo). The mean (±SD) change from baseline in the SARA total score was -1.97±2.43 points after 12 weeks of receiving NALL and -0.60±2.39 points after 12 weeks of receiving placebo (least-squares mean difference, -1.28 points; 95% confidence interval, -1.91 to -0.65; P<0.001). The results for the secondary end points were generally supportive of the findings in the primary analysis, but these were not adjusted for multiple comparisons. The incidence of adverse events was similar with NALL and placebo, and no treatment-related serious adverse events occurred.

CONCLUSIONS:

Among patients with Niemann-Pick disease type C, treatment with NALL for 12 weeks led to better neurologic status than placebo. A longer period is needed to determine the long-term effects of this agent in patients with Niemann-Pick disease type C. (Funded by IntraBio; ClinicalTrials.gov number, NCT05163288; EudraCT number, 2021-005356-10.).

2024-02-01·The New England journal of medicine1区 · 医学

N -Acetyl- l -Leucine and Neurodegenerative Disease

1区 · 医学

作者: Tifft, Cynthia J

A review.Lysosomal storage disorders, although individually rare, have a collective incidence of 1 in 5000 live births.Moreover, 70% of the nearly 70 known lysosomal storage disorders affect the central nervous system.These monogenic disorders cause perturbation in lysosome function (see Key Concepts), leading to metabolic instability, dysregulation of mammalian target of rapamycin (mTOR; normally, mTOR suppresses inflammation), impaired autophagy, and neuronal cell death.Several therapies that address the underlying pathol. processes of lysosomal storage disorders are approved or under development; these include enzyme-replacement therapy, substrate-reduction therapy, mol. chaperone therapy, gene therapy, gene editing, and neuroprotective therapies.

2024-01-10·Journal of audiology & otology

Review

作者: Meireles, Luís ; Pinto, Ana Nóbrega ; Sousa, Francisco Alves de ; Rego, Ângela Reis ; Alves, Clara Serdoura

There have been few investigations on the epidemiology, etiology, and medical management of acute unilateral vestibulopathy (AUV). Short-term pharmaceutical resolutions include vestibular symptomatic suppressants, anti-emetics, and some cause-based therapies. Anticholinergics, phenothiazines, antihistamines, antidopaminergics, benzodiazepines, and calcium channel antagonists are examples of vestibular suppressants. Some of these medications may show their effects through multiple mechanisms. In contrast, N-acetyl-L-leucine, Ginkgo biloba, and betahistine improve central vestibular compensation. Currently, AUV pathophysiology is poorly understood. Diverse hypotheses have previously been identified which have brought about some causal treatments presently used. According to some publications, acute administration of anti-inflammatory medications may have a deleterious impact on both post-lesional functional recovery and endogenous adaptive plasticity processes. Thus, some authors do not recommend the use of corticosteroids in AUV. Antivirals are even more contentious in the context of AUV treatment. Although vascular theories have been presented, no verified investigations employing anti-clotting or vasodilator medications have been conducted. There are no standardized treatment protocols for AUV to date, and the pharmacological treatment of AUV is still questionable. This review addresses the most current developments and controversies in AUV medical treatment.

项与 N-乙酰-L-亮氨酸相关的新闻（医药）

2024-03-26

·BioSpace

IntraBio Announces U.S. FDA Accepts New Drug Application for IB1001 for the Treatment of Niemann-Pick Disease Type C

IntraBio's IB1001 New Drug Application was accepted and granted priority review PDUFA date set for September 24th, 2024 Application based on positive results of the IB1001-301 Phase 3 Pivotal Trial which was recently published in the New England Journal of Medicine on February 1st, 2024 AUSTIN, TX / ACCESSWIRE / March 26, 2024 / IntraBio Inc today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for IB1001 for the treatment of Niemann-Pick disease Type C (NPC). The application has been granted Priority Review and was given a Prescription Drug User Fee Act (PDUFA) target action date of September 24th, 2024. The NDA is based on the results of a Pivotal Phase 3 trial (IB1001-301) for adult and pediatric patients with NPC that met all primary and key secondary endpoints and showed IB1001 improved neurological signs and symptoms, functioning, and quality of life versus placebo. The findings from the study were recently published in the New England Journal of Medicine on February 1st, 2024. The NDA also included data from a positive multinational Phase IIb trial of IB1001 for NPC, which also met its primary and secondary endpoints and showed improvement in symptoms, functioning, and quality of life in pediatric and adult patients with NPC with the drug being well-tolerated. Sean Kassen, Director of the Ara Parseghian Medical Research Fund commented: "We are absolutely thrilled and filled with hope with the news of IntraBio's NDA filing acceptance. NPC has an extremely high unmet medical need and the data from the IB1001 clinical trials demonstrated that this therapy would bring relief and improved quality of life to those affected by Niemann-Pick Type C disease. We are excited and committed to working with and supporting the NPC community and IntraBio and look forward to the eventual approval of IB1001 so all patients will benefit from this therapy." "The FDA's acceptance of IntraBio's NDA submission for IB1001 and granting of priority review brings us one step closer to our ultimate goal of delivering new, effective treatments to patient communities like NPC who have extremely high unmet medical needs," said Mallory Factor, IntraBio's Executive Chairman. "IntraBio remains dedicated to advancing therapies and addressing unmet medical needs, and we are optimistic IB1001 will be approved and made rapidly available for all NPC patients." Professor Elizabeth Berry-Kravis, Rush University Medical Center, Chicago, IL, commented: "We are tremendously excited to have potential new treatment options for our NPC patient community. Once approved, we intend to treat all our NPC patients who meet the labelling criteria with IB1001 and to carefully track long-term improvement relative to baseline with whatever therapies they are on to help elucidate the long-term effects." IntraBio also today announced it has recently closed an equity financing round, in which it raised over $40 million US, to support the commercialization and launch of IB1001 subject to FDA approval. About IB1001 IB1001 is an orally administered therapy that is taken up by monocarboxylate transporters, which are expressed ubiquitously and deliver IB1001 to all tissues, including across the blood-brain barrier. Inside cells, IB1001's underlying mechanism of action is multi-modal and targets the major drivers of disease pathophysiology in rare and common neurological disorders. About IntraBio IntraBio Inc, a US biopharmaceutical company, is focused on the development of novel drugs addressing rare and common neurological diseases. IntraBio's platform technologies result from decades of research and collaboration with universities and institutions worldwide. Its clinical programs are based upon the expertise in lysosomal function and intracellular signalling of its scientific founders from the University of Oxford and the University of Munich. For further information, please contact: Cass Fields Vice-President of External Affairs Email: ccfields@intrabio.com SOURCE: IntraBio Inc. View the original press release on accesswire.com

临床结果申请上市临床2期优先审批临床3期

100 项与 N-乙酰-L-亮氨酸相关的药物交易

登录后查看更多信息

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
C型尼曼匹克病	申请上市	美国	IntraBio, Inc.	2024-03-26
共济失调毛细血管扩张	临床2期	美国	IntraBio, Inc.	2020-01-08
共济失调毛细血管扩张	临床2期	德国	IntraBio, Inc.	2020-01-08
共济失调毛细血管扩张	临床2期	西班牙	IntraBio, Inc.	2020-01-08
共济失调毛细血管扩张	临床2期	英国	IntraBio, Inc.	2020-01-08
山德霍夫病	临床2期	美国	IntraBio, Inc.	2019-06-07
山德霍夫病	临床2期	德国	IntraBio, Inc.	2019-06-07
山德霍夫病	临床2期	西班牙	IntraBio, Inc.	2019-06-07
山德霍夫病	临床2期	英国	IntraBio, Inc.	2019-06-07
泰-萨克斯病	临床2期	美国	IntraBio, Inc.	2019-06-07

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03759665 (Accesswire) 人工标引	临床2期	GM2神经节苷脂贮积症	30	IB1001	範構選齋繭選襯夢蓋觸(鏇糧遞積獵廠遞鑰範蓋) = statistically significant change. 90% CI: 0.00, 1.50, p-value = 0.039 積夢壓醖蓋顧繭糧鑰構 (艱膚窪窪淵獵窪顧簾醖 ) 更多	积极	2021-09-14
NCT03759639 (Pubmed \| J Neurol) 人工标引	临床2期	C型尼曼匹克病	32	N-acetyl-L-leucine	獵築觸糧網構衊構遞遞(構鹹膚網廠餘鬱醖範範) = NALL met the CI-CS primary endpoint (mean difference 0.86, SD = 2.52, 90% CI 0.25, 1.75, p = 0.029) 顧選窪蓋襯醖衊製觸蓋 (艱壓網鏇衊醖衊憲窪願 )	积极	2021-08-13
NCT01271868 (CTgov)	临床3期	血友病B	9	IB1001	糧遞簾簾襯夢齋夢廠齋(構築願窪選範襯鏇願簾) = 繭簾選膚憲夢遞餘鑰選憲廠獵鑰鏇衊廠壓顧獵 (遞夢繭憲鬱鑰觸顧鬱積, 鏇餘製顧艱積醖網醖鑰 ~ 襯衊襯願鏇蓋淵獵遞衊) 更多	-	2019-04-11