A Phase 2a Multicenter Randomized Controlled Trial Evaluating the Safety and Efficacy of Polyethylene Glycol (PEG) Mediated Fusion (PEG Fusion) Compared to Standard of Care in the Repair of Mixed Motor-sensory Acute Peripheral Nerve Injuries (PNI) for Rapid and Immediate Improvement in Outcome

The overall objective of this study is to determine the safety of PEG fusion when used with primary repair or reconstruction in patients with an acute upper extremity peripheral nerve injury. PEG is safe and effective for extending the half-life of circulating pharmaceutical products, when used in conjunction with a topical hemostatic agent in surgical wounds, and when used as a colon cleanser for endoscopic surgical procedures. However, PEG fusion has not been rigorously tested as a safe reagent to promote nerve regeneration in humans. Therefore, the goal of this Phase 2a clinical trial is to establish safety data and to examine the effect of PEG fusion on clinical outcomes including recovery of sensory and motor function. Results will be externally validated using data collected in the DoD funded prospective NERVE study and will provide preliminary evidence to power a larger phase II efficacy trial.

开始日期2023-06-01

申办/合作机构

Defense Language Institute Foreign Language Center

NCT05293522 / Terminated临床2期

A Phase 2a, Multicenter, Randomized, Subject and Evaluator Blinded, Controlled Study Evaluating the Safety and Efficacy of NTX-001 Compared to Standard of Care in the Treatment and Prevention of Facial Paralysis Requiring Surgical Repair.

This study involves the use of an Investigational Product called NTX-001. It is a product used in the repair of nerve injuries. It is used in the operating room. The main purposes of this study are to 1) see how safe NTX-001 is when used in nerve repair and, 2) see if your nerve becomes functional in a shorter period of time when compared to what is normally done to treat nerve injuries.

开始日期2022-07-12

申办/合作机构

Neuraptive Therapeutics, Inc.初创企业

NCT04572906 / Active, not recruiting临床2期

Phase 2a, Multicenter, Randomized, Patient and Evaluator Blinded, Controlled Study Evaluating the Safety and Efficacy of NTX-001 Compared to SOC in the Treatment of Acute Single Transected Peripheral Nerve Injury Occurring Below the Distal Border of the Brachial Plexus Requiring Surgical Repair.

NTX-001 is a single use surgical product intended for use in conjunction with standard suture neurorrhaphy of severed nerves in patients between 16 and 80 years of age.

开始日期2020-09-25

申办/合作机构

Neuraptive Therapeutics, Inc.初创企业

100 项与 NTX-001 相关的临床结果

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100 项与 NTX-001 相关的转化医学

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100 项与 NTX-001 相关的专利（医药）

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项与 NTX-001 相关的文献（医药）

2020-11-01·Epilepsia

Progress report on new antiepileptic drugs: A summary of the Fifteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XV). I. Drugs in preclinical and early clinical development

作者: Perucca, Emilio ; Johannessen, Svein I. ; White, H. Steve ; Levy, René H. ; Koepp, Matthias J. ; Bialer, Meir ; Tomson, Torbjörn ; Perucca, Piero

Abstract:

Since 1992, the Eilat Conferences have provided a forum for all stakeholders in the epilepsy community to appraise the latest data on new antiepileptic drugs and emergency seizure treatments, including, in recent years, updates on progress with the development of novel monitoring and therapeutic devices. Because of the COVID‐19 pandemic, the Fifteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XV) was held as a fully virtual conference on July 27‐30, 2020 for the sessions on drugs and on August 3, 2020 for the sessions on devices, and was attended during the 5 days by >500 participants from 63 countries. This progress report summarizes key preclinical and initial (phase 1) clinical data on eight investigational treatments that are currently in early development, including 2‐deoxy‐D‐glucose, GAO‐3‐02, JNJ‐40411813, NBI‐921352, NTX‐001, sec‐butylpropylacetamide, XEN1101, and XEN496. This report provides an overview of current scenarios in the area of treatment discovery and development. The information presented illustrates a variety of innovative strategies, including exploration of compounds with novel mechanisms of action, transplantation of interneurons into epileptogenic brain regions, and the targeting of rare, previously neglected syndromes.

2019-12-01·BMC ophthalmology4区 · 医学

Efficacy and safety of a novel naltrexone treatment for dry eye in type 1 diabetes

4区 · 医学

ArticleOA

作者: Sassani, Joseph W ; Titunick, Michelle B ; Zagon, Ian S ; McLaughlin, Patricia J

BACKGROUND:

Dry eye disease (DED) is a prevalent complication of diabetes and presents as reduced tear production and/or increased corneal surface sensitivity often with secondary ocular surface changes. This study examined the safety and efficacy of a proprietary new eye drop formulation for topical treatment of DED.

METHODS:

Type 1 diabetes (T1D) was established in male Sprague-Dawley rats to study the efficacy and safety of the investigational compound that contained 20 μg/ml of naltrexone (NTX). Tear production was measured by the Schirmer's 1 test, and ocular surface sensitivity was measured using an aesthesiometer. Diabetic rats received twice daily applications of a single drop (~ 0.02 ml) of the proprietary formulation (NTX-001) or vehicle onto one eye. For comparison, some diabetic rats received eye drops containing NTX in sterile Vigamox®. Safety was monitored by assessment of ocular histopathology in naïve male rats and naïve male rabbits receiving twice daily treatment of two drops for 30 days.

RESULTS:

Dry eye in T1D rats was reversed within hours of a single treatment of NTX-001, and over a period of 10 days NTX-001 restored corneal sensitivity and reversed dry eye relative to values measured in diabetic rats receiving vehicle. In comparison to NTX dissolved in Vigamox®, the proprietary NTX-001 was more effective at reversing dry eye. Safety studies in naïve rats and rabbits revealed no visible ocular pathology after 30 days of treatment.

CONCLUSIONS:

An investigational new eye drop containing 20 μg/ml NTX effectively reversed tear film deficits and restored corneal surface sensitivity in diabetic animals without causing toxic side effects.

项与 NTX-001 相关的新闻（医药）

2024-02-22

·BioSpace

Neuraptive Therapeutics, Inc. Announces Positive 24-Week Data for NTX-001 in the Phase 2 NEUROFUSE Study, Reinforcing Previously Announced Proof of Concept 12-Week Data

NTX-001 continues to be generally safe and well tolerated, with no additional adverse effects NTX-001 demonstrated a clinically and statistically significant improvement in function at 24 weeks. NTX-001 demonstrated a clinically and statistically significant improvement in pain at 24 weeks. WAYNE, Pa.--(BUSINESS WIRE)-- Neuraptive Therapeutics, Inc., a pioneer in nerve repair and regeneration, is proud to announce new 24-week data from the ongoing NEUROFUSE Study for NTX-001, further solidifying the product's status following the previously announced proof of concept results based on the 12-week data in January 2024. This press release features multimedia. View the full release here: Evan L. Tzanis, COO and EVP Head of R&D, Neuraptive Therapeutics, Inc. (Photo: Business Wire) The latest data from the multicenter, randomized, evaluator-blinded, Phase 2a NEUROFUSE Study underscores NTX-001’s sustained safety and efficacy as an adjunct treatment for transected peripheral nerves. At the 24-week mark, NTX-001 continues to demonstrate an encouraging safety profile, with lower treatment-emergent adverse event rates than the standard of care. Regarding efficacy, NTX-001 showed a statistically significant improvement in hand function and symptomatology as measured by the Michigan Hand Questionnaire (MHQ) compared to the standard of care alone, with clinically and statistically meaningful changes observed at eight and 24-week intervals. The secondary endpoints, including the Numerical Pain Rating Scale, also showed statistically significant improvements, favoring NTX-001 at the 24-week timepoint. These results validate the early clinical response reported at the 12-week interim time point and exhibit consistent, supportive trends across multiple secondary endpoints, including multiple assessments of sensory recovery. “Current techniques for the repair of traumatic peripheral nerve injuries often result in disappointing functional outcomes with the potential for significant neuropathic pain. This Phase 2a randomized clinical trial utilizing NTX-001 in nerve repair demonstrates its safety for use in the coaptation of nerves and shows a significant improvement in patient-reported pain scores and patient-reported functional outcome measures. Its potential to improve the care of patients with these devastating injuries using this novel technique is intriguing and holds promise for future clinical application,” said David M. Brogan, MD MSc, Associate Professor of Orthopedic Surgery, Washington University in St. Louis and Lead Author of the study. “We are excited about the results and are rapidly preparing to present these top-line results at an upcoming scientific conference, and we anticipate an End of Phase 2 meeting with the FDA in the near future to discuss late-phase development and the approval pathway,” said Evan Tzanis, Chief Operating Officer and EVP of R&D at Neuraptive Therapeutics, Inc. “We are thankful to the patients, investigators, and their staff for their participation and commitment to this study.” With the continued advancement of NTX-001, Neuraptive Therapeutics, Inc. remains at the forefront of developing novel solutions for a significant number of patients are affected by peripheral nerve injuries globally. About Neuraptive Therapeutics, Inc. Neuraptive Therapeutics, Inc. is dedicated to the innovation and development of medical products and therapeutics to repair and regenerate peripheral nerves. The company is focused on addressing the unmet medical needs of patients and physicians dealing with the complex challenges of nerve injuries. Notice Regarding Forward-Looking Statements This press release contains forward-looking statements subject to various risks and uncertainties. Actual results could materially differ from those anticipated in the forward-looking statements due to various factors. Neuraptive Therapeutics, Inc. disclaims any obligation to update the information contained in these forward-looking statements.

临床2期临床结果

2024-01-16

·Pharmaceutical Technology

FDA grants orphan drug status to Neuraptive Therapeutics’ NTX-001

The NTX-001 treatment kit is intended for peripheral nerve injury repair. Credit: namtipStudio/Shutterstock.com. The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Neuraptive Therapeutics’ NTX-001, a treatment kit containing fusogen, for peripheral nerve injury repair. The designation is a crucial step in the development of the company’s lead development product, which is poised to potentially enhance functional outcomes rapidly for patients with peripheral nerve injuries. NTX-001 is the only surgical technology designed to prevent Wallerian degeneration by polyethylene glycol-fusing severed axons through an improved treatment sequence within a controlled nerve isolation chamber. Neuraptive Therapeutics chief medical officer Dr Seth Schulman stated: “Receiving the orphan drug designation for NTX-001 is a testament to our commitment to advancing healthcare solutions for patients impacted by peripheral nerve injuries who today have limited options.” The US FDA grants ODD to therapies for the treatment of rare diseases or ailments in the region. Neuraptive Therapeutics research and development, employee value proposition and chief operating officer Evan Tzanis stated: “The granting of orphan drug designation by the FDA comes at an important time, as we prepare to meet with regulators in the coming months to seek their input on the future development work and the ultimate path to approval. “Following this designation, Neuraptive Therapeutics plans to accelerate the development of NTX-001, aiming to bring this promising treatment to patients as soon as possible.” The ODD comes with benefits including marketing exclusivity for seven years to approved orphan products’ sponsors, a federal tax credit for clinical research expenditures within the US and a Prescription Drug User Fee Act fee waiver. It gives the potential to compete for grants for research from the Office of Orphan Products Development and the opportunity to obtain regulatory assistance from the US regulator.

孤儿药

2024-01-15

·BioSpace

Neuraptive Therapeutics Inc. Receives Orphan Drug Designation for its Lead Development Asset

WAYNE, Pa.--(BUSINESS WIRE)-- Neuraptive Therapeutics Inc., a leader in innovative medical solutions, is pleased to announce that it has received Orphan Drug Designation from the United States (US) Food and Drug Administration (FDA) for its fusogen contained in the NTX-001 treatment kit. This designation marks a significant milestone in developing NTX-001, a novel treatment for peripheral nerve injury requiring repair. Dr. Seth Schulman, Chief Medical Officer of Neuraptive Therapeutics Inc., stated, “Receiving the Orphan Drug Designation for NTX-001 is a testament to our commitment to advancing healthcare solutions for patients impacted by peripheral nerve injuries who today have limited options.” The Orphan Drug Designation is granted to drugs intended to treat rare diseases or conditions in the US. Highlights of Orphan Designation are: 7-year marketing exclusivity to sponsors of approved orphan products Federal tax credit for expenses incurred in conducting clinical research within the United States Waiver of Prescription Drug User Fee Act (PDUFA) fees for orphan drugs Ability to qualify to compete for research grants from the Office of Orphan Products Development (OOPD) to support clinical studies for orphan drugs Eligibility to receive regulatory assistance and guidance from the FDA in the design of an overall drug development plan. “The Granting of orphan drug designation by the FDA comes at an important time, as we prepare to meet with regulators in the coming months to seek their input on the future development work and the ultimate path to approval, “ said Evan L. Tzanis, Chief Operating Officer and EVP of R&D. “ Following this designation, Neuraptive Therapeutics plans to accelerate the development of NTX-001, aiming to bring this promising treatment to patients as soon as possible.” Said Mr. Tzanis. For more information about Neuraptive Therapeutics, Inc., and our work in peripheral nerve injury treatments, please visit Neuraptive.com. About NTX-001 NTX-001 is the only surgical technology that may rapidly improve functional outcomes in patients with peripheral nerve injuries by preventing Wallerian degeneration after PEG-fusing severed axons using an optimized and specific treatment sequence in a controlled nerve isolation chamber. About Neuraptive Therapeutics, Inc.: Neuraptive Therapeutics, Inc. is a biotechnology company dedicated to developing novel therapeutics and medical products to address the unmet needs of physicians and patients impacted by peripheral nerve injuries. The company is headquartered in Wayne, PA. For more information, see . This press release contains forward-looking statements that are subject to risks and uncertainties. Due to various factors, actual results may differ materially from those indicated in the forward-looking statements. The Company disclaims any obligation to update or revise forward-looking statements.

孤儿药

100 项与 NTX-001 相关的药物交易

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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
面瘫	临床2期	美国	Neuraptive Therapeutics, Inc.初创企业	2022-07-12
周围神经损伤	临床2期	美国	Neuraptive Therapeutics, Inc.初创企业	2020-09-25

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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


NCT04572906 (NEUROFUSE, Biospace) 人工标引	临床2期	周围神经损伤	-	NTX-001	壓製築願選餘襯網襯鹹(遞鏇餘繭願鑰鏇簾鏇繭) = clinically and statistically meaningful changes observed at eight and 24-week intervals. 鹽廠製膚齋網膚願鹽淵 (製襯鬱憲醖齋淵夢積製 ) 更多	积极	2024-02-22
				Standard of Care
NCT04572906 (NEUROFUSE, BusinessWire) 人工标引	临床2期	周围神经损伤	60	NTX-001	窪窪齋鏇積膚鏇選窪窪(選廠鏇憲餘夢構襯鑰選) = the MHQ change at baseline in Total Score at 12 weeks demonstrated that NTX-001 treatment was numerically improved over SOC but did not reach statistical significance for this global function assessment. 積獵鹽蓋窪夢窪鏇壓範 (淵願鏇製壓鬱選夢窪艱 ) 更多	积极	2024-01-08
				SOC