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PXT3003 clinical trial

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LabTAG™ Is A Full Service, One Stop Shop For Your Lab Labeling Needs. Shop Now Build your Career in Healthcare, Data Science, Web Development, Business, Marketing & More. Learn from anywhere, anytime. Flexible, 100% online learning. Join & get 7-day free trial PXT3003 is a fixed dose combination of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol selected via a Systems Biology approach and developed by Pharnext, with the aim to limit the production of PMP22 and protect/improve axonal function in patients with CMT1A

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  1. This international, multi-center, randomized, double-blind, placebo-controlled, Phase III clinical study is designed to evaluate PXT3003 versus placebo in male and non-pregnant female subjects with genetically confirmed CMT1A of mild-to-moderate severity (CMTNS-V2 score >2 and ≤18) aged 16 to 65 years
  2. PXT3003 is a rational design, fixed combination of low-dose (RS) baclofen, naltrexone hydrochloride and D-sorbitol. The use of PXT3003 in a multicenter, randomised, placebo controlled phase II study (CLN-PXT3003-01) was well-tolerated and safe in patients with CMT1A for the three dose-levels investigated (Attarian et al., 2014)
  3. The present trial is a randomized, placebo-controlled study evaluating 3 different doses of PXT3003 in patients with CMT1A disease. Condition or disease Intervention/treatmen
  4. An international Phase 3 clinical trial investigating Pharnext's investigational therapy PXT3003 for treating Charcot-Marie-Tooth disease type 1A (CMT1A) is on track and expected to enroll its first participants in March, the company announced.. Dubbed PREMIER, the trial will include about 350 patients with mild-to-moderate CMT1A across 50 clinical sites
  5. ary efficacy profile of PXT3003 given over 12 months was first characterized in a multicenter, randomized, double-blind, placebo-controlled Phase 2 study performed in France in 80.
  6. PXT3003 may also have beneficial effects on other cellular targets, such as muscle cells and the junction between muscle and nerve. PXT3003 in clinical trials Results of a Phase 2 clinical trial ( NCT01401257 ), published in the Orphanet Journal of Rare Diseases , showed that PXT3003 was a safe and well-tolerated treatment for adults with CMT1A

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The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1 A (CMT1A). This double-blind study will assess in parallel groups 2 doses of PXT3003 compared to Placebo in CMT1A patients treated for 15 months.. Clinical Trials Registry. ICH GCP CMT1A Clinical Trial Update: Pharnext PREMIER trial of PXT3003 The PREMIER Trial, which is being conducted in patients with mild-to-moderate CMT1A, is expected to enroll approximately 350 subjects ages 16-65 with a confirmed genetic diagnosis of CMT1A.... Accelerate Clinical Trials in Charcot-Marie-Tooth Disease (ACT-CMT) Calling CMT1A Patients. The present trial is a randomized, placebo-controlled study evaluating 3 different doses of PXT3003 in patients with CMT1A disease.. Clinical Trials Registry. ICH GCP

Continuation of the clinical trial for PXT3003 in Charcot-Marie-Tooth disease type 1A (CMT1A) with a first date set year-end 2017 During the first half of 2017, Pharnext continued the Phase 3 clinical trial of PXT3003, PLEODRUGTM candidate, for the treatment of CMT1A. Patient enrolment was completed in compliance with the established roadmap Today, French pharmaceutical company Pharnext SA announced positive topline results from its pivotal Phase 3 clinical trial (PLEO-CMT) evaluating two doses of PXT3003 compared to placebo during 15 months for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A).. PLEO-CMT was a pivotal, 15-month, double-blind Phase 3 study that assessed the efficacy and safety of PXT3003 compared to.

Phase III Trial Assessing the Efficacy and Safety of

  1. e whether PXT3003 is effective and well tolerated in patients with CMT1A. PXT3003, developed.
  2. Pharnext's first-in-class PLEODRUG™ PXT3003, developed using Pharnext's R&D platform, PLEOTHERAPY™, is a novel oral fixed-dose combination of baclofen, naltrexone and sorbitol, with Orphan Drug Designation in the U.S. and E.U. PXT3003, Pharnext's lead PLEODRUG™, has shown positive results both in non-clinical pharmacology and.
  3. The first subject has been enrolled in the pivotal Phase III clinical study ('PREMIER trial') of PXT3003 in the U.S. at the Austin Neuromuscular Center (Texas). PXT3003 is Pharnext's lead program to treat Charcot-Marie-Tooth disease type 1A ('CMT1A'), an indication with currently no existing approved therapies. The trial will enroll approximately 350 subjects with mild-to-moderate.

The PREMIER trial is a randomized, double-blind, two-arm placebo-controlled, pivotal Phase III study, evaluating the efficacy and safety of PXT3003 versus placebo in CMT1A patients, over a 15-month period. The dose of PXT3003 tested in the PREMIER trial corresponds to the high dose tested in the prior Phase III trial ('PLEO-CMT') The other drugs are baclofen and sorbitol. (Sorbitol is a simple sugar alcohol that can be obtained without a prescription.) They are doing trials on three different dosages. Low Dose - .6mg baclofen, .07mg naltrexone, 21mg sorbitol. Intermediate Dose - 1.2mg baclofen, .14 naltrexone, 42 sorbitol

SA was the coordinator of the clinical trial CLN-PXT3003-01 and the PI of the Marseille's site. JMV, PMG, AL, YP and OD were principal investigators at study centres: Limoges, Lyon, Lille, Nantes and Paris respectively. LM, BF, JP, JMic, JF, MNL, KG, MAM, VT, MP, AMag, LLV and TS were local, principal co-investigators at study centres CLN-PXT3003-02: National Competent Authority: Spain - AEMPS: Clinical Trial Type: EEA CTA: Trial Status: Completed: Date on which this record was first entered in the EudraCT database: 2015-10-30: Trial results: View result In preclinical studies, PXT3003 inhibited the overexpression of the PMP22 gene, improved myelination of peripheral nerves and motor / sensory impairments. In a Phase 2 clinical trial in 80 adult patients with CMT1A, PXT3003 improved multiple efficacy endpoints beyond stabilization, particularly the ONLS scale Status of the IMP to be used in the clinical trial : D.2.1: IMP to be used in the trial has a marketing authorisation: No : D.2.5: The IMP has been designated in this indication as an orphan drug in the Community: Yes : D.2.5.1: Orphan drug designation number: EU/3/14/1260: D.3 Description of the IMP: D.3.2: Product code : PXT3003 dose 1: D.3.4. Easy to use and user-friendly trial registry with advanced search and download. Accepting all clinical research studies - interventional and observationa

Description. PXT3003 is a rational design, fixed combination of low-dose (RS) baclofen, naltrexone hydrochloride and D-sorbitol. The use of PXT3003 in a multicenter, randomised, placebo controlled phase II study (CLN-PXT3003-01) was well-tolerated and safe in patients with CMT1A for the three dose-levels investigated (Attarian et al., 2014) The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1 A (CMT1A). This d. Sign In Create an Account. Find a Trial. Find a Trial PXT3003 is formulated using an approach called pleotherapy™ which systemizes the identification and development of new synergistic combinations of repositioned drugs for diseases with high unmet medical needs. In the phase III clinical trial PLEO-CMT (NCT02579759)2, PXT3003 is administered twice daily as an oral solution for 15 months PXT3003 could also have a positive effect on other cellular types of the motor unit such as the axon (direct protection), neuromuscular junctions or muscle cells. PXT3003 has shown promising and consistent results across preclinical and clinical studies in Phase II and Phase III (PLEO-CMT)

To gather up-to-2-year data to estimate the long term effect of PXT3003 on clinical, functional and electrophysiological endpoints; - To compare the effect of PXT3003 in patients receiving PXT3003 active dose from the start of the primary study to patients assigned to a delayed start (i.e., who received placebo during the primary study) in order to show if PXT3003 slows the progression of the. The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1A. This double-blind study will assess in parallel groups 1 dose of PXT3003 compared to Placebo in CMT1A patients treated for 15 months Pharnext's first-in-class PLEODRUG(TM) PXT3003, developed using Pharnext's R&D platform, PLEOTHERAPY(TM), is a novel oral fixed-dose combination of baclofen, naltrexone and sorbitol, with Orphan. A Multi-center, Randomized, Double-blind, Placebo-controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth Type 1A (CMT1A) Investigators: Gregory T. Carter, MD; Alicia Fuhrman, M Pharnext's first-in-class PLEODRUG(TM) PXT3003, developed using Pharnext's R&D platform, PLEOTHERAPY(TM), is a novel oral fixed-dose combination of baclofen, naltrexone and sorbitol, with Orphan Drug Designation in the U.S. and E.U. PXT3003, Pharnext's lead PLEODRUG(TM), has shown positive results both in non-clinical pharmacology and clinical.

The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1 A (CMT1A). This double-blind study will assess in parallel groups 2 doses of PXT3003 compared to Placebo in CMT1A patients treated for 15 months PXT3003 is a novel drug candidate for the treatment of CMT1A and has been granted both Orphan Drug Designation and Fast Track Designation by the US Food and Drug Administration ('FDA'). In Pharnext's previous interactions with FDA, the agency provided guidance that an additional Phase III study would be required Dr. David Horn Solomon, CEO of Pharnext discusses PXT3003 which is currently focused on the treatment of Charcot Marie-Tooth (CMT) disease Type 1A. Charcot-Marie-Tooth is a degenerative nerve disease that usually appears in adolescence or early adulthood. CMT affects the Peripheral Nervous System. It is a progressive nerve disease PARIS, FRANCE / ACCESSWIRE / June 10, 2020 / Pharnext SA (FR0011191287 - ALPHA), a biopharmaceutical company pioneering new approaches to developing innovative drug combinations based on big genomics data and artificial intelligence, today provided an update on the regulatory and clinical status of PXT3003, its lead program in Charcot-Marie-Tooth Type 1A (CMT1A). PXT3003 is a novel drug. Results of a phase 2 clinical trial (NCT01401257), published in the Orphanet Journal of Rare Diseases, showed that PXT3003 was a safe and well-tolerated treatment for adults with CMT1A. The trial enrolled 80 CMT1A patients who were randomly assigned to a low, medium, or high dose of PXT3003 or a placebo for 12 months

To date, there is no approved pharmacologic treatment for any form of Charcot-Marie-Tooth disease (CMT). However, some clinical or preclinical trials for CMT1A have been undertaken, for example Neurotrophin-3, PXT3003, and neuregulin-1. Gene therapy for CMT1X, CMT2F and Giant axonal neuropathy using Pharnext Provides Regulatory and Clinical Update on PXT3003 Phase III Study for the Treatment of Charcot-Marie-Tooth Type 1A June 10, 2020 GMT US Food and Drug Administration has agreed with Pharnext and provided clear guidance on the regulatory pathway to approval for PXT3003, including key design elements of a single pivotal Phase III. PXT3003 is an oral fixed-low dose combination of baclofen, naltrexone and sorbitol, and is currently being evaluated in a multi-center, randomized, 15-month, double-blind, placebo-controlled phase 3 clinical study. The trial has enrolled patients aged 16 and older with mild-to-moderate CMT1A in 30 sites across Europe, the US, and Canada The forthcoming Phase II randomized, comparative, placebo-controlled clinical trial will evaluate the safety, efficacy, pharmacodynamics and pharmacokinetics of PXT3003 in patients suffering from.

In many respects, 2020 was a year of rebuilding for Pharnext following the disruption in its previous Phase III clinical study of PXT3003 for the treatment of Charcot-Marie-Tooth disease, type 1A (CMT1A). While showing positive results, the study's high dose arm was prematurely discontinued due to a manufacturing issue. In 2020, Pharnext completed gathering data from the study and met with. Completing enrollment of our pivotal Phase 3 trial of PXT3003 is a significant milestone and highlights the strength of our clinical operations and management teams, as well as the support of. Interim Analysis from the Ongoing Open-Label Phase III Extension Study Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A ('CMT1A') New results suggest good safety profile and sustained efficacy of PXT3003 as measured with the Overall Neuropathy Limitation Scale ('ONLS'), after 4.5 years of total trial time. A live conference call and webcast will. PXT3003 is a novel drug candidate for the treatment of CMT1A and has been granted both Orphan Drug Designation and Fast Track Designation by the US Food and Drug Administration (FDA) Studies of PXT3003 with an ONLS Data Readout at 54 M including in respect of timing of and prospects for clinical trials and regulatory submissions of the Company's product candidates as.

Search by clinical trial title International, multi-center, double blind 9-month FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A (Phase III) - E Adrian Hepner, MD, PhD, Chief Medical Officer of Pharnext,said: 'PXT3003 has already shown an encouraging response in our prior Phase II and Phase III ('PLEO-CMT') trials using the ONLS, and we place high hope that the efficacy and safety of PXT3003 will be further demonstrated in our PREMIER trial. CMT1A is a severe, debilitating, chronic. Pharnext Announces First Patient Enrolled in the PREMIER Trial, its Pivotal Phase III Clinical Development Program of PXT3003 in Charcot-Marie-Tooth Disease Type 1A ('CMT1A' PXT3003 is the Company's lead program to treat Charcot-Marie-Tooth disease type 1A ('CMT1A'), an indication with currently no existing approved therapies. The trial will enroll approximately 350 subjects with mild-to-moderate CMT1A in 50 centers in the U.S., Canada, Europe, and Israel. The PREMIER trial is a randomized, double-blind, two-arm.

Adrian Hepner, MD, PhD, Chief Medical Officer of Pharnext, said: PXT3003 has already shown an encouraging response in our prior Phase II and Phase III ('PLEO-CMT') trials using the ONLS, and we place high hope that the efficacy and safety of PXT3003 will be further demonstrated in our PREMIER trial. CMT1A is a severe, debilitating, chronic. I have been part of a Phase III clinical trial of 300 CMT Type 1A patients at 30 sites world wide. I have been going to St. Paul for my visits with Dr. Walk and his staff at the University of Minnesota. The drug in the trial is Pharnext PXT 3003. It is a mixture of baclofen, naltrexone and D-sorbital Found 178 clinical trials. We call this infoimagery. It is unique to every trial and is generated based on a trials specific data eg. therapeutic area, study type, phase etc. and the centerwatch system associates the information to a matching set of related symbols and icons Adrian Hepner, MD, PhD, Chief Medical Officer of Pharnext,said: PXT3003 has already shown an encouraging response in our prior Phase II and Phase III ('PLEO-CMT') trials using the ONLS, and we. This Phase 3 clinical trial is highly significant for patients suffering from CMT1A where only supportive care is available today, said Daniel Cohen, M.D., Ph.D., Co-Founder and Chief Executive Officer of Pharnext. We believe that our PLEODRUG™ PXT3003, if successful, has the potential to transform the treatment of CMT1A in adults..

PXT3003 is the Company's lead program to treat Charcot-Marie-Tooth disease type 1A ('CMT1A'), an indication with currently no existing approved therapies. ('PLEO-CMT') trials using the ONLS. The results of the trial will start to gather evidence on whether PXT3003 is effective and safe in the treatment of patients with Charcot-Marie-Tooth type 1A. If successful, the study will also determine what is the best dosage of PXT3003 for testing in a larger phase 3 trial. Contact Details Interim Analysis from the Ongoing Open-Label Phase III Extension Study Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A ('CMT1A'). New results suggest good safety profile and sustained efficacy of PXT3003 as measured with the Overall Neuropathy Limitation Scale ('ONLS'), after 4.5 years of total trial time.. A live conference call and webcast. CLINICAL TRIALS. Oregon Neurology is excited to offer interested patients access to clinical trials, in addition to standard treatments. Participating in a clinical trial is a unique opportunity to help further medical research and potentially receive new medications before they are available to everyone

Assessing Long Term Safety and Tolerability of PXT3003 in

The PLEO-CMT-FU trial is part of the PXT3003 Phase III clinical programme, which includes another Phase III trial PLEO-CMT. Pharnext chief medical officer René Goedkoop said: The initiation of this second international Phase III trial marks an important milestone for the whole PXT3003 clinical development programme as it aims to confirm the long-term safety and tolerability profile of. A phase 3 trial of Pharnext's PXT3003 in a rare, muscle wasting disease has met its primary endpoint. The trial linked the fixed-dose combination to a statistically significant improvement on a. Phase 2 clinical trials showed PXT3003 to be a promising treatment for CMT1A, and that it was safe. The results were published in Orphanet Journal of Rare Diseases. The company is also conducting a Phase 3 trial of the therapy. The international, randomized, double-blind, placebo-controlled trial (NCT02579759) is called PLEO-CMT PXT3003-treated CMT1A rats continuously gained muscle strength until study end to 6.6 ± 1.5 N for PXT3003-1, 6.7 ± 1.2 N for PXT3003-3, and 6.9 ± 1.8 N for PXT3003-4 (Figure 2a and Figure S1c). Analysis of the change from baseline shows the significant therapy effects for PXT3003-3 and PXT3003-4 groups at study end (Figure 2b )

Phase II, Randomized, Placebo-controlled Trial in Patients

Pivotal Phase 3 Trial of PXT3003 to Begin CMT1A Patient

About the PREMIER Trial. The PREMIER trial is an international, randomized, double-blind, two-arm placebo-controlled, pivotal Phase III study, evaluating the efficacy and safety of PXT3003 versus. for clinical trials (Fledrich et al., 2014; Meyer zu Horste et al., 2007), failed in clinical testing (Lewis et al., 2013; Pareyson et al., 2011) or have not been translated into patients yet (Fledrich et al., 2018; Sociali et al., 2016; Zhao et al., 2018). PXT3003 is a fixed-ratio combination of baclofen (BCL), naltrex PXT3003 Update. Pharnext has released a bit more data from their clinical trial for PXT3003, the experimental drug to help treat the symptoms of CMT type 1A. The data continues to be encouraging for the higher dose in particular showing modest improvement in ability to function Since PXT3003 has a strong safety profile and is currently undergoing a phase III trial in CMT1A patients, our results suggest that PXT3003 therapy may be a bona fide translatable therapy option for children and young adolescent patients suffering from CMT1A. (CMTPedS) which is a sensitive outcome measure for clincal trials . Despite. The Phase 2 randomized clinical trial involved 80 adult patients with mild-to-moderate CMT1A at six hospital sites in France. The participants received twice-daily placebos or one of the three increasing doses of PXT3003 over one year. The safety and tolerability of PXT3003 was found to be good

Pharnext - PXT300

CMT1A is the most common inherited peripheral neuropathy. There is currently no approved treatment. We performed a meta-analysis including four randomized, double-blind, Placebo-controlled clinical trials to assess the disease progression after one year under Placebo, Ascorbic Acid (AA) or PXT3003, a combination of three repurposed drugs PXT3003 is a rational design, fixed combination of low-dose (RS) baclofen, naltrexone hydrochloride and D-sorbitol. The use of PXT3003 in a multicenter, randomised, placebo controlled phase II study (CLN-PXT3003-01) was well-tolerated and safe in patients with CMT1A for the three dose-levels investigated (Attarian et al., 2014). The intermediate and high dose of PXT3003 demonstrated an.

PXT3003 - Charcot-Marie-Tooth New

Pharnext to Start Final Phase 3 Trial of PXT3003 for CMT1A

Press release content from Accesswire. The AP news staff was not involved in its creation The pharmaceutical company Pharnext is on track to start its phase III clinical trial, PREMIER in the USA, by the end of March 2021.. The PREMIER study will test the safety and efficacy of the drug PXT3003 for the treatment of people with Charcot Marie Tooth disease type 1A (CMT1A). PXT3003 is a combination of three different drugs and aims to reduce the levels of the PMP22 protein, which is. PXT3003 completed an international Phase 3 trial with positive topline results for the treatment of Charcot-Marie-Tooth disease type 1A and benefits from orphan drug status in Europe and the. Results of this research collaboration might inform the addition of new exploratory endpoints in our next Phase III trial of PXT3003 to be initiated in Q1 2021. We believe this alliance will enable us to accelerate our efforts in bringing a safe and effective therapeutic for this disease that currently has no viable treatment options

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Pharnext to Launch New Phase 3 Trial of PXT3003 for CMT1

DJ Pharnext Announces First Patient Enrolled in the PREMIER Trial, its Pivotal Phase III Clinical Development Program of PXT3003 in Charcot-Marie-Tooth Disease Type 1A ('CMT1A') Pharnext Pharnex However, the clinical effectiveness of PXT3003 was not so striking as preclinical data in phase 2 clinical trial and waiting for additional results from phase 3 clinical study. In this manuscript the authors analyzed the efficacy of single compound and the synergism of their combination PXT3003 will be tested versus placebo in mild-to-moderate CMT1A patients over a 15-month period. including in respect of timing of and prospects for clinical trials and regulatory submissions. This clinical trial is a Phase 2, open-label study to determine the anti-tumor activity of FLX475 in combination with ipilimumab in subjects with advanced melanoma previously treated with an anti-PD-1 or anti-PD-L1 agent. Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients

Pxt3003 - Cmta

PARIS, FRANCE / ACCESSWIRE / March 31, 2021 / Pharnext SA (FR0011191287 - ALPHA) (the 'Company'), an advanced late-stage clinical biopharmaceutical company pioneering new approaches to developing innovative drug combinations based on big genomics data and artificial intelligence using its PLEOTHERAPY(TM) platform, today announces that the first subject has been enrolled in its pivotal Phase. Dr. David Horn Solomon, CEO of Pharnext discusses PXT3003 which is currently focused on the treatment of Charcot Marie-Tooth (CMT) disease Type 1A.Charcot-Marie-Tooth is a TrialSite Staff January 13, 202 PXT3003 also improved axonal regeneration and remyelination in the murine nerve crush model. Based on these observations in preclinical models, a clinical trial of PTX3003 in CMT1A, a neglected orphan disease, is warranted. If the efficacy of PTX3003 is confirmed, rational polytherapy based on novel combinations of existing non-toxic drugs with. * announces that the dsmb recommends continuing the ongoing phase 3 trial of pxt3003 for charcot-marie-tooth disease type 1

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Our Focus on PXT3003 and Upcoming Pivotal Phase III Clinical Trial in CMT1A. We continue to focus most of our resources on developing PXT3003 for CMT1A with the goal of obtaining marketing approval from FDA and EMA. PXT3003 is a novel combination of naltrexone, baclofen and sorbitol, taken orally, and has Orphan Drug Designation from both FDA. Pharnext Pharnext Announces First Patient Enrolled in the PREMIER Trial, its Pivotal Phase III Clinical Development Program of PXT3003 in DAX® 15.607,97 +0,12% TecDAX® 3.562,98 +0,74% Dow. Key inclusion & exclusion criteria: Inclusion criteria: Period 1 • Patients under P and D1 must have completed 15 months of double-blind treatment in the primary study CLN-PXT3003-02, including all procedures required at the Study Termination visit (V6) or patients under D2 prematurely discontinued from CLN-PXT3003-02 (due to sponsor decision on September 18th, 2017) must have performed an.

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