Our Mission
HNFâs mission is advancing and supporting clinical research and trials for CMT. HNF has been instrumental in advancing clinical trials by providing valuable patient insight, innovative data collection and regulatory initiatives to assist in industry sponsored clinical trial design and to inform the FDA for decision making and oversight during drug development and commercialization of drugs and gene therapies for CMT.
How we advance Clinical Trials
HNF continues to be instrumental in advancing clinical trials by:
- Providing valuable patient insight
- Developing innovative data collection
- Consulting industry-sponsored clinical trial designÂ
- Informing the FDA for decision-making and oversight during drug development and commercialization of drugs and gene therapies for CMT.
- Advocating for regulatory initiatives
Learn about current clinical trials and how you can help prepare for future participation in vital research!
The Key Role CMT Patients Play in Research
CMT Thrive Webinar
Presented by: Joy Aldrich, HNF GRIN Patient Registry Coordinator
Current Clinical Trials
- Pharnext PXT3003Â PREMIERÂ Trial for CMT1A
- Impact of CMT in the Real World â CMT&Me App
- Applied Therapeutics AT007Â INSPIREÂ Trial for SORD Deficiency
- Global Registry for Inherited Neuropathies (GRIN)Â
How has HNF advanced & supported clinical trials for CMT?
HNF has developed a collaborative team of experienced CMT experts, patient advocates and clinical trial resources to support clinical trials for all types of CMT.
HNFâs Global Registry for Inherited Neuropathies (GRIN) helps to promote the needs and priorities of patients through IRB-approved studies.
These studies have and continue to generate robust data on genotype (CMT subtypes) correlated to phenotypes (signs & symptoms) from our Natural History Study as well as generated data from ClinGen surveys that are recognized as valid sources of patient data by FDA.
What are our Industry Partners saying?
âInitiatives such as the HNF registry bring together critical data from patients, caregivers and clinicians, including genetic tests and electronic health records. This information can help inform clinical trials of therapies for the 150,000 people in the US who are living with Charcot-Marie-Tooth disease and have no treatment options available,â
âGRIN data has been essential in clinical trial protocol design, site selection, and efficient patient recruitment for the pivotal Phase III clinical study of our lead asset PXT3003, the PREMIER trial, in development for the most common type of CMT, CMT1A.Â
Latest on HNF Industry Partnerships Clinical Trials
SORD Deficiency
A clinical-stage biopharmaceutical company that has developed a novel molecule to treat the newly discovered type 2 CMT gene which causes SORD deficiency. Experts believe that SORD deficiency could very well become the most common subtype of CMT 2. HNF has been instrumental in helping to facilitate and accelerate the approval of their clinical lead product AT-007, a Aldose Reductase Inhibitor (ARI) currently in clinical trials.
âInitiatives such as the HNF registry bring together critical data from patients, caregivers and clinicians, including genetic tests and electronic health records. This information can help inform clinical trials of therapies for the 150,000 people in the US who are living with Charcot-Marie-Tooth disease and have no treatment options available,â said Shoshana Shendelman, CEO & Founder of Applied Therapeutics.
Pharnext Pharmaceuticals
A late stage clinical biopharmaceutical company has developed a novel synergistic drug combination to treat the most common type of CMT, CMT1A. The drug Syngility is a fixed-dose in combination of baclofen, naltrexone and sorbitol to downregulate the overexpression of PMP22 protein, leading to improvement of neuronal signaling in dysfunctional peripheral nerves which is the primary pathophysiology problem for CMT1A. HNF has been in partnership with Pharnext since 2012 and is a key contributor to supporting the development and commercialization of Syngility. Expected results of their pivotal phase III is expected by Q4, 2023.
âGRIN data has been essential in clinical trial protocol design, site selection, and efficient patient recruitment for the pivotal Phase III clinical study of our lead asset PXT3003, the PREMIER trial, in development for the most common type of CMT, CMT1A. In addition, the robust data collected in GRIN, together with the ongoing CMT&Me digital survey, will enable a better understanding of the burden of disease, comorbidities, and critical patient demographics as we move forward to a potential commercialization of PXT3003 if approved by the FDA. Topline data of the PREMIER trial are expected in the last quarter of 2023.â Xavier Paoli M.Sc. Pharnext, Chief Operating Officer
We believe this clinical trial has the potential to be a crucial turning point in the effort to finally provide an efficacious treatment for patients suffering from CMT1A,â said Daniel Cohen, M.D., Ph.D., co-founder and CEO of Pharnext. âTodayâs therapeutic options are very limited and mostly palliative in nature. Our PLEODRUGÂź PXT3003 has already demonstrated safety, tolerability and improvements in CMT1A patient disability in a Phase 2 trial. Given this positive safety assessment by the DSMB, we are hopeful to bring this much needed potential therapy to patients suffering from this debilitating condition upon completion of this Phase 3 trial.â
Join GRIN!
Without your participation, researchers wonât have the essential and necessary patient information to develop drugs, gene therapies, and clinical trials for Charcot-Marie-Tooth and other Inherited Neuropathies!
Genetic Testing â CMT Genie
Wish there was an easy guide for genetic testing?
Now there is with the CMT Genie!
HNFâs CMT Genie is designed to assist you and your health care provider determine what the best option is for you!
Learn more on this topic
Latest News and Related Blog Posts
Positioning HNF to Translate Treatments to Clinical Trials
In June 2013, HNF made the important decision to refine the organizationâs mission. We now place more importance on new initiatives that take our research discoveries and translate them to the next phase in the drug discovery process.
We divide our research initiatives into 3 areas: