Interventional, randomised, partial double-blind, placebo- and positive controlled, multiple-dose, 4-way-cross-over trial Investigating the effect of arimoclomol on cardiac repolarization in healthy men. - CS0344 Orphazyme TQT

开始日期2020-06-02

申办/合作机构-

NCT03836716

/ Terminated临床3期

Open Label, Non-randomized Extension Trial to Assess Long Term Safety and Efficacy of Arimoclomol in Subjects With Amyotropic Lateral Sclerosis Who Have Completed the ORARIALS-01 Trial

A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS) who have completed the ORARIALS-01 trial.

开始日期2019-09-19

申办/合作机构-

NCT04049097

/ Terminated临床3期

An Open-label, Non-randomized Trial to Investigate the Efficacy and Safety of Early Versus Delayed Start of Arimoclomol in Patients With Sporadic Inclusion Body Myositis Who Have Completed the IBM4809 Trial

A multicenter, nonrandomized, open-label, uncontrolled clinical extension trial designed to compare the efficacy and safety of early versus delayed start of arimoclomol in the treatment of Inclusion Body Myositis (IBM)

开始日期2019-05-20

申办/合作机构

University College London

[+1]

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100 项与 Arimoclomol 相关的转化医学

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100 项与 Arimoclomol 相关的专利（医药）

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项与 Arimoclomol 相关的文献（医药）

2026-06-03·Expert Review of Neurotherapeutics

Current and emerging therapeutic advancements for Niemann-Pick disease type C: where are we now and what does the future hold?

Review

作者: Hahn, Andreas

INTRODUCTION:

Niemann-Pick Disease Type C (NPD Type C) is a rare lysosomal disease characterized by progressive neurovisceral manifestations due to abnormal lipid storage resulting from biallelic mutations in the NPC1 or NPC2 gene. Until recently, treatment was mainly symptomatic.

AREAS COVERED:

This review summarizes the results of newer clinical trials evaluating efficacy and safety of new therapeutics, e.g. miglustat, cyclodextrins, arimoclomol, and N-Acetyl-L Leucine (NALL) for the treatment of NPD Type C. The PubMed database and the clinicaltrials.gov website were used for literature search and newest results posted.

EXPERT OPINION:

Arimoclomol and NALL showed significant improvements of neurological symptoms and reasonable safety profiles in phase II/III trials. The recent approval of arimoclomol in combination with miglustat and NALL for patients with NPD type C has the potential to modify the standard of care and will hopefully improve the prognosis. However, the collection of real-world data is warranted to explore the real clinical value of these new medicaments. Cyclodextrins are also promising therapeutics that principally allow to correct basic pathophysiological mechanisms in NPD type C, but further research is needed.

2026-06-01·CURRENT THERAPEUTIC RESEARCH-CLINICAL AND EXPERIMENTAL

Understanding Pharmacokinetic-Drug Interactions With Drugs Approved by the US Food and Drug Administration in 2024 to Better Manage the Risk of Drug Interactions With Concomitant Medications: A Review of Clinical Data From New Drug Applications

Review

作者: Owens, Katie ; Argon, Sophie ; Yu, Jingjing ; Ragueneau-Majlessi, Isabelle ; Wang, Yan

Objective:

This analysis aimed to provide a mechanistic understanding and an evaluation of the clinical relevance of pharmacokinetic drug-drug interactions (DDIs) associated with drugs approved by the Food and Drug Administration in 2024.

Methods:

Drug metabolism, transport, and absorption-based DDI data available in New Drug Applications (NDAs) of small molecular drugs approved (n = 34) was analyzed. The mechanism and clinical outcome of these interactions were characterized based on in vitro, in silico, and clinical data.

Results:

As objects, 11 drugs were identified as clinical substrates. Of these, 7 drugs were substrates of CYP3A, 3 of CYP2C9, one of CYP1A2, and one of CYP2C8, including the sensitive substrates vanzacaftor (CYP3A) and vorasidenib (CYP1A2). As precipitants, 6 drugs (acoramidis, cefepime/enmetazobactam, givinostat, lazertinib, mavorixafor, and resmetirom) were clinical inhibitors of CYP enzymes (2C8, 2C9, 2D6, 2E1, and 3A), with mavorixafor being a CYP2D6 strong inhibitor. Two drugs (elafibranor and tovorafenib) showed weak induction of CYP3A. Regarding transporter data, 3 drugs were substrates of transporters, including seladelpar (BCRP and OAT3), sulopenem (OAT3), and vadadustat (OAT1/3), and 8 drugs (arimoclomol, danicopan, givinostat, lazertinib, mavorixafor, resmetirom, vadadustat, and vazacaftor/tezacaftor/deutivacaftor) were inhibitors of transporters. All clinical DDIs with AUC changes ≥ 2-fold triggered labeling recommendations. Several DDIs with an AUC change <2 also had labeling recommendations, pertaining most often to the concomitant use of drugs with a narrow therapeutic index.

Conclusions:

Mechanistic DDI insights from newly approved therapies can be extrapolated to inform the management of commonly co-administered drugs, supporting a safer and more effective use of new drug products in the context of polypharmacy.

2026-06-01·JOURNAL OF NEUROCHEMISTRY

Effects of Lysine Deacetylation Inhibition Alone or in Combination With Arimoclomol on TDP ‐43 Proteinopathy

Article

作者: Andrea Calvo ; Monica Favagrossa ; Alfredo Cagnotto ; Mario Salmona ; Serena Scozzari ; Giovanni De Marco ; Massimo Tortarolo ; Valentina Bonetto ; Laura Pasetto ; Stefano Fabrizio Columbro ; Caterina Bendotti

ABSTRACT:

Cytoplasmic inclusions containing TAR DNA‐binding protein 43 kDa (TDP‐43) are recognized as a major pathological feature of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia. Peptidyl‐prolyl cis‐trans isomerase A (PPIA) interacts with TDP‐43 and influences its aggregation and function. This interaction is facilitated by PPIA Lys‐acetylation. Here, we investigated whether restoring lysine acetylation homeostasis exerts protective effects on TDP‐43 proteinopathy in vitro and in vivo and how this relates with PPIA. We found that vorinostat/SAHA, a broad‐spectrum histone deacetylase (HDAC) inhibitor that increases PPIA acetylation, is able to reverse TDP‐43 mislocalization in a cellular model of TDP‐43 proteinopathy. We confirmed its effects in peripheral blood mononuclear cells from ALS patients and explored its impact on TDP‐43 proteinopathy and PPIA acetylation in the Thy1‐hTDP‐43 mouse model. Thy1‐hTDP‐43 mice treated with SAHA showed a delayed onset of TDP‐43 pathology, associated with PPIA nucleus‐cytoplasm redistribution, lower neurodegeneration and neuroinflammation, and improved neuromuscular function markers. However, these effects were transient. When combined with arimoclomol, a heat shock protein co‐inducer, a mitigation of the neurodegeneration was sustained. A synergistic effect was observed in periphery, greatly enhancing tubulin acetylation and reducing phosphorylated TDP‐43 accumulation in the sciatic nerve and acetylcholine receptor γ‐subunit expression in gastrocnemius muscle. This study suggests that HDAC inhibition could be beneficial in restoring TDP‐43 localization and function through multiple mechanisms, including modulation of PPIA acetylation. The combination of lysine deacetylation inhibition and arimoclomol shows a synergistic effect in vivo and has potential as a therapeutic approach for patients.image

249

项与 Arimoclomol 相关的新闻（医药）

2026-06-23

·BioSpace

Zevra Therapeutics Announces Publication of Positive MIPLYFFA® Data from Pediatric Substudy in Infants with Niemann-Pick Disease Type C (NPC)

Data published in Molecular Genetics and Metabolism Reports suggest MIPLYFFA (arimoclomol) was well tolerated in pediatric patients with no new safety signals observed BOSTON, June 23, 2026 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on bringing life-changing therapeutics to people living with rare diseases, announced the publication of “Arimoclomol in infants with Niemann-Pick disease type C: Results from the phase 2/3 open-label pediatric substudy” in Molecular Genetics and Metabolism Reports . This multicenter, open-label pediatric substudy evaluated arimoclomol, in addition to concomitant miglustat, in five children with Niemann-Pick disease type C (NPC) aged 12 to <24 months at enrollment, assessing safety, tolerability, and pharmacokinetics over up to 36 months of treatment. “The publication of these data adds to the growing body of clinical evidence supporting MIPLYFFA across age groups in NPC,” said Christine i Dali, Zevra’s Vice President, Clinical Science and an author of the publication. “Understanding the safety and pharmacokinetic pro arimoclomol in these young children is critical as we continue to explore the potential role of earlier treatment initiation for patients living with this devastating disease.” Arimoclomol was generally well tolerated, with no new safety signals, and pharmacokinetic results were consistent with older pediatric populations. Developmental assessments showed variable delays, reflecting underlying phenotypic heterogeneity. Although limited by small sample size, the study provides preliminary evidence supporting initiation of MIPLYFFA in this age group. About MIPLYFFA ® (arimoclomol) MIPLYFFA (arimoclomol) is Zevra’s approved therapy for the treatment of Niemann-Pick disease type C (NPC). Approved by the U.S. Food and Drug Administration on Sep. 20, 2024, MIPLYFFA (arimoclomol) increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. In the pivotal phase 3 trial, MIPLYFFA halted disease progression compared to placebo over the one-year duration of the trial when measured by the only validated disease progression measurement tool, the NPC Clinical Severity Scale. MIPLYFFA has also received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC. The extensive data generated for MIPLYFFA has shown long-term, meaningful clinical outcomes with more than 5 years of patient experience across more than 270 NPC patients worldwide through a Phase 2/3 clinical trial, Open-Label Extension (OLE) study, Expanded Access Programs (EAP), and a pediatric sub-study, which is the most expansive clinical development program in NPC to date. Zevra has submitted a Marketing Authorization Application to the European Medicines Agency for the evaluation of arimoclomol for the treatment of Niemann-Pick disease type C. INDICATIONS AND USAGE MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. IMPORTANT SAFETY INFORMATION Hypersensitivity Reactions: Hypersensitivity reactions such as urticaria and angioedema have been reported in patients treated with MIPLYFFA during Trial 1: two patients reported both urticaria and angioedema (6%) and one patient (3%) experienced urticaria alone within the first two months of treatment. Discontinue MIPLYFFA in patients who develop severe hypersensitivity reactions. If a mild or moderate hypersensitivity reaction occurs, stop MIPLYFFA and treat promptly. Monitor the patient until signs and symptoms resolve. Embryofetal Toxicity: MIPLYFFA may cause embryofetal harm when administered during pregnancy based on findings from animal reproduction studies. Advise pregnant females of the potential risk to the fetus and consider pregnancy planning and prevention for females of reproductive potential. Increased Creatinine without Affecting Glomerular Function: Across clinical trials of MIPLYFFA, mean increases in serum creatinine of 10% to 20% compared to baseline were reported. These increases occurred mostly in the first month of MIPLYFFA treatment and were not associated with changes in glomerular function. During MIPLYFFA treatment, use alternative measures that are not based on creatinine to assess renal function. Increases in creatinine reversed upon MIPLYFFA discontinuation. The most common adverse reactions in Trial 1 (≥15%) in MIPLYFFA-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight. Three (6%) of the MIPLYFFA-treated patients had the following adverse reactions that led to withdrawal from Trial 1: increased serum creatinine (one patient), and progressive urticaria and angioedema (two patients). Serious adverse reactions reported in MIPLYFFA-treated patients were hypersensitivity reactions including urticaria and angioedema. To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. toll-free at 1-844-600-2237 or FDA at 1-800-FDA-1088 or . Drug Interaction(s): Arimoclomol is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates. When MIPLYFFA is used concomitantly with OCT2 substrates, monitor for adverse reactions and reduce the dosage of the OCT2 substrate . Use in Females and Males of Reproductive Potential: Based on animal findings, MIPLYFFA may impair fertility and may increase post-implantation loss and reduce maternal, placental, and fetal weights. Renal Impairment: The recommended dosage of MIPLYFFA, in combination with miglustat, in patients with an eGFR ≥15 mL/minute to <50 mL/minute is lower than the recommended dosage (less frequent dosing) in patients with normal renal function. MIPLYFFA capsules for oral use are available in the following strengths: 47 mg, 62 mg, 93 mg, and 124 mg. For more information, please see the full Prescribing Information , including Instructions for Use About Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with rare diseases. The Company is focused on broadening access through geographic expansion opportunities, progressing its pipeline toward key milestones, and delivering meaningful therapeutics. The commercialization of its lead product, marketed in the U.S. for Niemann-Pick disease type C (NPC), a rare, progressive neurodegenerative disease, provides a strong corporate foundation and validates its ability to advance therapies from development to market. Zevra's vision is realized through disciplined execution of its strategic plan and core values — patient centricity, integrity, accountability, innovation, and courage — which guide its efforts to deliver long-term value. For more information, please visit or follow us on X and LinkedIn . Caution Concerning Forward-Looking Statements This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding the potential use of arimoclomol in infants with NPC. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the "Risk Factors" section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2025, filed on March 9, 2026, as well as Zevra’s other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release. Investor Contact Nichol Ochsner +1 (732) 754-2545 nochsner@zevra.com Media Contact Julie Downs +1 (508) 246-3230 jdowns@zevra.com

临床结果上市批准孤儿药临床3期

2026-06-19

·BioSpace

Denali sells FDA voucher for $195M to support neurodegenerative pipeline

iStock, d1sk Denali Therapeutics is the latest company to sell a priority review fast pass for an elevated price after the program was renewed earlier this year. Denali Therapeutics has sold a priority review voucher for $195 million, marking a rise in value for the coveted regulatory fast passes. Denali snagged the ticket from the approval of rare disease drug Avlayah in March 2026. The drug was cleared by the FDA for use in patients with Hunter syndrome. The buyer was not disclosed in Denali’s June 18 announcement . The biotech intends to use the proceeds from the sale to support a pipeline of assets generated using its TransportVehicle technology, which aids the passage of drugs across the blood-brain barrier. Denali is developing therapies for lysosomal storage disorders and neurodegenerative diseases. The FDA awards priority review vouchers (PRVs) upon approval of a drug that has been granted a rare pediatric disease tag. Companies can either use them for a speedy approval of another drug or sell them, generating non-dilutive capital. C-suite Denali CEO’s ‘greatest professional moment’ arrives as rare disease drug launches After receiving the FDA’s greenlight for Hunter syndrome drug Avlayah, Denali Therapeutics CEO Ryan Watts saw the culmination of 20 years of hard work unraveling the mysteries of the blood-brain barrier. April 22, 2026 · 7 min read · Annalee Armstrong Read more When the program began in 2014, the passes sold for as high as $350 million. More recently, the going rate has been $150 million. Zevra Therapeutics sold one last year for that much after the approval of Miplyffa. But Jazz received $200 million for one in January this year as the Senate debated renewal of the priority review program, which lapsed in December 2024. After months of delay, the rare pediatric disease priority review program was in fact renewed in February . Avlayah was the first new drug approved for the rare disease Hunter syndrome in 20 years. The disease causes sugar molecules to build up in the body, affecting physical and mental development. Patients typically die by age 20. With Avlayah approved and the launch underway, Denali is now turning its attention to its remaining pipeline. This includes DNL126 for Sanfilippo syndrome type A, DNL593 for GRN-related frontotemporal dementia, DNL952 for Pompe disease and DNL628 for Alzheimer’s disease, in addition to a clutch of preclinical programs. Rare diseases Rare Disease Biotechs Left in a Lurch as Congress Fails To Renew Priority Review Program Congress did not reauthorize the rare pediatric disease priority review program at the end of 2024. Advocates say the ripple effect is already being felt across biopharma. March 5, 2025 · 9 min read · Annalee Armstrong Read more

优先审批上市批准

2026-06-11

·allsci.com

Beren attracts USD 300m in financing ahead of potential Niemann-Pick disease drug launch

Thousand Oaks, California-based Beren Therapeutics P.B.C. has secured USD 300 million in combined financing to support the potential commercial launch of adrabetadex, its lead candidate for infantile-onset Niemann-Pick disease type C (I-NPC), a rare and rapidly fatal pediatric neurodegenerative disease. The funding comprises a USD 135 million equity round alongside up to USD 165 million in non-dilutive capital from Hercules Capital, Inc. (NYSE: HTGC), structured as a senior secured debt facility and royalty financing. The transaction is designed to position Beren as financially self-sustaining through a potential commercial launch, with the US FDA having accepted the company’s New Drug Application (NDA) for adrabetadex under Priority Review. The FDA recently extended its review time for the molecule, issuing a PDUFA target action date of November 17, 2026. The equity round brought together Wellington Partners, JIC Venture Growth Investments (JIC VGI), Founders Fund, Narya Capital, Eisai Co., Ltd., and other institutional investors. The non-dilutive component with Hercules Capital includes up to USD 110 million in term loans tied to regulatory and revenue milestones — of which USD 30 million has been drawn — and a USD 55 million royalty financing tranche to be funded upon FDA approval of adrabetadex. The royalty financing carries a 7.5% royalty on US net sales and 5% on net sales outside the US, capped at 1.75x through 2031, with an option to redeem at a lower multiple in the first two years. Beren said proceeds will support commercial readiness activities including accelerating diagnosis to enable early intervention, establishing a single point of contact for families, ensuring local access to sites of care, and generating longitudinal real-world evidence. The company and its subsidiary Mandos LLC have supported the NPC community since 2021 through an Expanded Access Program. No prior funding rounds were disclosed in the press release or available data for Beren Therapeutics. Adrabetadex and the Case for I-NPC Treatment I-NPC is the most severe form of Niemann-Pick disease type C, a rare autosomal-recessive disorder caused by pathogenic variants in the NPC1 or NPC2 genes. These mutations impair intracellular cholesterol trafficking, leading to progressive neurological decline. Patients with early infantile-onset — defined as neurological symptom onset before age two — have a mean age of death of approximately 5.6 years. For late-infantile onset, the mean age of death is approximately 13.4 years. Adrabetadex is a proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers. Data suggest the compound re-establishes intracellular cholesterol trafficking, directly addressing the underlying pathology of NPC rather than managing downstream symptoms. The US FDA has granted adrabetadex Breakthrough Therapy Designation. If approved, it would represent the only therapy specifically authorized for I-NPC and the only agent designed to act on accumulated intracellular cholesterol driving the disease. The main adverse events associated with adrabetadex in clinical and expanded access data include hearing impairment — manageable with hearing aids — and post-dose fatigue and/or ataxia. The NPC treatment landscape is becoming increasingly competitive. Zevra Therapeutics (Nasdaq: ZVRA) markets Miplyffa (arimoclomol), which received US FDA approval in September 2024 and is indicated in combination with miglustat for neurological manifestations of NPC in patients two years and older — a broader label that includes adult and pediatric patients beyond the infantile-onset population. Rafael Holdings (NYSE: RFL) has also completed last patient last visit in its pivotal Phase III TransportNPC study of Trappsol Cyclo (hydroxypropyl beta-cyclodextrin), with topline data expected in the second half of 2026 and an NDA submission also anticipated in that period. Adrabetadex is distinguished by its infantile-onset-specific indication and its direct mechanism targeting the cholesterol trafficking defect, positioning it as complementary to rather than directly competitive with MIPLYFFA in the near term. Beren’s single disclosed program is adrabetadex in I-NPC. The company has not publicly described additional pipeline assets or platform programs beyond its focus on cholesterol trafficking biology and cyclodextrin-based therapeutics. The November 2026 PDUFA date represents the company’s near-term inflection point, with commercial launch preparations contingent on a positive regulatory decision. This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: https://allsci.com/news/ Your email address will not be published. Required fields are marked * Comment * Name * Email * Website Copyright © 2026. AllSci Corp. All rights reserved.

临床3期申请上市上市批准优先审批突破性疗法

100 项与 Arimoclomol 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	Arimoclomol	-	DB05025

研发状态

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适应症	国家/地区	公司	日期
C型尼曼匹克病	美国	Zevra Denmark A/S	2024-09-20
C型尼曼匹克病	美国	Zevra Therapeutics, Inc.	2024-09-20

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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
包涵体肌炎	临床3期	美国	University College London	2019-05-20
包涵体肌炎	临床3期	英国	University College London	2019-05-20
肌萎缩侧索硬化	临床3期	美国	Zevra Denmark A/S	2018-07-31
肌萎缩侧索硬化	临床3期	比利时	Zevra Denmark A/S	2018-07-31
肌萎缩侧索硬化	临床3期	加拿大	Zevra Denmark A/S	2018-07-31
肌萎缩侧索硬化	临床3期	法国	Zevra Denmark A/S	2018-07-31
肌萎缩侧索硬化	临床3期	德国	Zevra Denmark A/S	2018-07-31
肌萎缩侧索硬化	临床3期	意大利	Zevra Denmark A/S	2018-07-31
肌萎缩侧索硬化	临床3期	荷兰	Zevra Denmark A/S	2018-07-31
肌萎缩侧索硬化	临床3期	波兰	Zevra Denmark A/S	2018-07-31

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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


NCT02612129 (NPC-002, Pubmed) 人工标引	临床2/3期	C型尼曼匹克病	50	arimoclomol (DB phase)	-	积极	2025-08-01
				arimoclomol (OLE phase)	繭繭鑰淵製衊獵鏇製窪(積範鏇構網鹹壓衊鏇淵) = 憲襯憲網壓築淵獵壓膚糧蓋範遞壓選廠廠遞齋 (顧鹽廠壓餘廠壓築襯獵, 4.8) 更多
NCT02612129 (FDA_CDER) 人工标引	临床3期	C型尼曼匹克病	50	MIPLYFFA with miglustatMIPLYFFA with miglustat	蓋窪憲築鹹艱鹽鬱廠壓(醖獵衊淵艱憲遞衊衊鬱) = 築顧網遞選艱淵繭顧選壓鏇繭壓淵衊築積膚鹹 (顧鏇襯醖糧觸艱壓選獵, 1)	积极	2024-09-20
				Placebo with miglustat	蓋窪憲築鹹艱鹽鬱廠壓(醖獵衊淵艱憲遞衊衊鬱) = 構襯範鑰壓繭膚範簾構壓鏇繭壓淵衊築積膚鹹 (顧鏇襯醖糧觸艱壓選獵, 3.4)
NCT04049097 (CTgov)	临床3期	包涵体肌炎	121	Arimoclomol	遞鑰願鹽製願簾鹽觸鬱(衊窪廠餘鑰艱壓顧簾選) = 獵窪簾獵獵蓋築餘選鹹餘獵選顧願淵遞構醖憲 (醖膚膚遞構醖願簾齋簾, 2.60) 更多	-	2023-09-15
NCT03836716 (CTgov)	临床3期	肌萎缩侧索硬化	120	Arimoclomol	選蓋鹹衊築蓋築構築糧 = 網選淵願積願構蓋鏇艱膚繭夢窪淵夢積鏇製鬱 (餘範顧淵選鏇淵鏇鏇鑰, 積蓋醖鏇築築憲獵繭蓋 ~ 獵獵遞構憲膚糧鏇鏇築) 更多	-	2023-08-24
NCT03491462 (ORARIALS-01, CTgov)	临床3期	肌萎缩侧索硬化	245	Arimoclomol (Arimoclomol (up to 76 Weeks))	築蓋製廠觸壓窪襯網鬱(齋淵築願選顧積選夢襯) = 網醖鏇齋鬱齋淵構構構構艱顧廠鏇衊獵艱夢選 (鑰蓋遞鹹願蓋繭鹹膚廠, 0.291) 更多	-	2023-08-24
				Placebo (Placebo (up to 76 Weeks))	築蓋製廠觸壓窪襯網鬱(齋淵築願選顧積選夢襯) = 餘範積壓襯蓋醖構積淵構艱顧廠鏇衊獵艱夢選 (鑰蓋遞鹹願蓋繭鹹膚廠, 0.283) 更多
NCT02612129 (NPC-002, CTgov)	临床2/3期	C型尼曼匹克病	50	arimoclomol (Arimoclomol (12-month Double-blind Phase))	鹽繭淵餘選壓廠艱顧淵(積鏇糧鑰積齋鑰憲憲糧) = 積簾獵糧憲壓鏇繭鏇襯襯鹹鬱顧築衊製餘鏇築 (築夢繭壓選願壓艱願鑰, 餘齋膚蓋鑰鬱選餘齋糧 ~ 鏇顧願製簾蓋蓋築積夢) 更多	-	2023-06-09
				Placebo (Placebo (12-month Double-blind Phase))	鹽繭淵餘選壓廠艱顧淵(積鏇糧鑰積齋鑰憲憲糧) = 願壓築鑰繭繭鹹築網鹹襯鹹鬱顧築衊製餘鏇築 (築夢繭壓選願壓艱願鑰, 鹽憲網齋製鹹窪願衊餘 ~ 廠壓廠顧淵糧齋壓糧膚) 更多
NCT02753530 (NCT02753530, CTgov)	临床2期	包涵体肌炎	152	Arimoclomol (Arimoclomol (20 Months))	願蓋醖蓋範網襯網鑰壓(夢膚範簾壓製鏇遞選選) = 衊餘鑰積築遞鹽夢膚蓋壓餘鑰壓鏇遞齋艱願壓 (繭獵壓鹽簾鹽窪壓夢夢, 憲淵範糧憲餘構網選繭 ~ 鹹夢廠蓋願積構壓鏇鏇) 更多	-	2023-05-10
				Placebo (Placebo (20 Months))	願蓋醖蓋範網襯網鑰壓(夢膚範簾壓製鏇遞選選) = 艱鏇齋窪鹹壓鹹獵憲艱壓餘鑰壓鏇遞齋艱願壓 (繭獵壓鹽簾鹽窪壓夢夢, 繭鏇選淵衊窪襯獵積鬱 ~ 夢壓範選製簾鹽醖範鑰) 更多
NCT02753530 (ACR2021) 人工标引	临床2期	包涵体肌炎	152	Arimoclomol	構築蓋淵壓醖鏇製襯窪(製鹹艱蓋壓餘顧築窪鏇) = 願淵窪構願壓膚構願積鏇顧遞觸獵糧築壓憲襯 (艱憲淵顧廠範夢積糧遞 ) 更多	不佳	2021-09-01
				Placebo	構築蓋淵壓醖鏇製襯窪(製鹹艱蓋壓餘顧築窪鏇) = 範膚顧製鑰願廠齋齋齋鏇顧遞觸獵糧築壓憲襯 (艱憲淵顧廠範夢積糧遞 ) 更多
NCT03491462 (ORARIALS-01, GlobeNewswire) 人工标引	临床3期	肌萎缩侧索硬化	245	arimoclomol 248 mg	齋遞築糧壓築願構網選(鏇獵膚製築鹽夢選糧鹹) = did not meet its primary endpoints 選選醖鑰糧蓋鹽糧襯顧 (餘鹹餘簾淵顧餘鏇窪襯 ) 更多	不佳	2021-05-07
				Placebo
NCT00706147 (Pubmed \| Ann Neurol \| Neurology) 人工标引	临床2/3期	肌萎缩侧索硬化	36	arimoclomol	獵廠襯觸獵築選齋鏇襯(簾構醖築醖蓋蓋齋壓願) = 繭觸構膚夢餘襯膚醖築鏇衊獵窪淵觸衊鏇衊構 (積鏇製夢齋憲淵膚蓋鏇, -0.63 ~ 1.63) 更多	积极	2018-02-13
				Placebo	-