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

166

项与 N-乙酰-L-亮氨酸相关的文献（医药）

2024-05-01·European Journal of Paediatric Neurology

Efficacy and safety of N-acetyl-L-leucine in patients with ataxia telangiectasia: A randomized, double-blind, placebo-controlled, crossover clinical trial

Article

作者: Saberi-Karimian, Maryam ; Tavallaie, Shima ; Houra, Mahsa ; Sarvghadi, Pooria ; Jamialahmadi, Tannaz ; Sahebkar, Amirhosein ; Beyraghi-Tousi, Mehran ; Bagheri, Reza ; Gumpricht, Eric

BACKGROUNDAtaxia telangiectasia (AT) is an autosomal recessive multisystem disorder. Most patients have progressive cerebellar ataxia, oculocutaneous telangiectasia, frequent pulmonary infection, and an increased risk of malignancies. Although N-acetyl-dl-leucine (ADLL) has shown some efficacy in patients with AT, its more pharmacologically active enantiomer, N-acetyl-l-leucine (NALL), has just recently been investigated in ataxic individuals. The current study assessed the efficacy of NALL in patients with AT.METHODSThis 2 × 2 crossover, double-blind, randomized clinical trial was conducted on 20 patients with AT. After excluding four patients, 16 subjects (eight females, eight males; mean age 9.8 ± 3.5 years) with a definitive genetic diagnosis of AT were randomly assigned to one of two study groups, with one group receiving 1-4 g/day NALL or a placebo for six weeks. Subjects then had a 4-week washout before crossing over to the other treatment for an additional six weeks. The Spinocerebellar Ataxia Functional Index (SCAFI) and the Scale for Assessment and Rating of Ataxia (SARA) score assessed patients' motor function. Quality of life (QOL) was evaluated by a specialist using the PedsQL questionnaire. Fasting blood samples were taken from all subjects before and after each intervention to determine potential side effects.RESULTSAlthough patients' nausea and constipation were improved, the results failed to reveal any significant benefits of NALL treatment on ataxia symptoms. NALL treatment had no significant effects on SARA, SCAFI-9HPT (9-hole peg test) nondominant, SCAFI-9HPT dominant, or SCAFI-8WMT (8 m walking time) (p > 0.05). Our patient's Physical Health score in Child self-report and Parent proxy-report did not significantly change in the treatment group compared to the placebo (p > 0.05). Furthermore, there were no significant changes in energy and macronutrient intake after NALL treatment. None of the volunteers reported serious or moderate side effects.CONCLUSIONSTo the best of our knowledge, this was the first placebo-controlled, randomized clinical trial exploring NALL's potential effects for treating AT. Despite improvements in some symptomss, NALL intervention failed to improve motor function significantly. However, patients' nausea and constipation were improved by NALL, which can be a relevant benefit clinically.

2024-02-01·New England Journal of Medicine1区 · 医学

Trial of N -Acetyl- l -Leucine in Niemann–Pick Disease Type C

1区 · 医学

Article

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

BACKGROUNDNiemann-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.METHODSIn 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.RESULTSA 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.CONCLUSIONSAmong 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·New England Journal of Medicine1区 · 医学

N -Acetyl- l -Leucine and Neurodegenerative Disease

1区 · 医学

作者: Tifft, Cynthia J

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.

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
GM2神经节苷脂贮积症	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	美国	IntraBio, Inc.	2019-06-07
山德霍夫病	临床2期	英国	IntraBio, Inc.	2019-06-07
山德霍夫病	临床2期	西班牙	IntraBio, Inc.	2019-06-07
泰-萨克斯病	临床2期	西班牙	IntraBio, Inc.	2019-06-07
GM2神经节苷脂贮积症	临床1期	德国	IntraBio, Inc.	2019-06-07
GM2神经节苷脂贮积症	临床1期	西班牙	IntraBio, Inc.	2019-06-07
GM2神经节苷脂贮积症	临床1期	英国	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


NCT00768287 (CTgov)	临床2/3期	血友病B	77	IB1001 (IB1001)	蓋獵選顧蓋簾選繭醖選(醖廠選製遞襯廠醖鏇觸) = 鹹製窪餘構築艱壓襯蓋鬱廠遞選憲鹹鑰鬱網鏇 (遞醖繭網製淵齋鹽網製, 齋餘壓蓋鏇窪獵獵衊淵 ~ 願構觸艱顧壓淵築糧積) 更多	-	2018-09-10
NCT00768287 (CTgov)	临床2/3期	血友病B	77	nonacog alfa (Nonacog Alfa)	蓋獵選顧蓋簾選繭醖選(醖廠選製遞襯廠醖鏇觸) = 鹹襯鏇襯襯蓋鬱夢簾顧鬱廠遞選憲鹹鑰鬱網鏇 (遞醖繭網製淵齋鹽網製, 襯觸願獵糧鬱鬱醖膚齋 ~ 鹹積積範膚鑰網壓範窪) 更多	-	2018-09-10
NCT01271868 (CTgov)	临床3期	血友病B	9	IB1001	衊憲願簾觸遞鏇衊製構(憲齋鏇選積衊壓構遞襯) = 網構願鏇衊積憲襯醖憲顧齋窪壓壓窪鹹積鹽範 (顧餘窪齋顧夢鹽鏇糧製, 網製蓋廠製遞積齋製壓 ~ 夢糧觸選製願繭構憲鬱) 更多	-	2019-04-11
NCT03759665 (Accesswire) 人工标引	临床2期	GM2神经节苷脂贮积症	30	IB1001	選鑰積憲衊鏇鏇築衊網(網選獵選顧繭鏇壓積鏇) = statistically significant change. 90% CI: 0.00, 1.50, p-value = 0.039 顧鏇壓憲鹹膚憲糧艱鬱 (壓範夢願遞襯糧鑰願蓋 ) 更多	积极	2021-09-14