Every two months, cmt.blog’s Trial Watch lists and comments on the clinical trials that are currently testing treatments for CMT. Between issues of Trial Watch, significant new trials and papers will appear on the Research Watch page as I review them.
This is the first Trial Watch post for cmt.blog. To catch up with the field, it covers the CMT drug trials underway or changed since 2024, from a Novartis CMT1A study recruiting only in Canada to a cancelled govorestat trial, with a plain-English note on each one. Skip down to The Baseline and then the Trial Status Table to get right into it.
Trial Watch looks at what each trial is testing, where it stands, and what changed. It includes the trials that were withdrawn or failed, because those are part of the record too. I first checked registry records on October 14, 2026, then reviewed the backlog once I’d calibrated Trial Watch’s custom search tool.
In the past I did a few, infrequent roundups of clinical trials in a completely manual process. Trial Watch is powered by a WordPress plugin that finds and follows the relevant needles within the essential open data haystacks of ClinicalTrials.gov and papers indexed by Europe PMC.
The Baseline: What Matters Now
This is the first issue, so it sets a baseline rather than tracking changes. The Research Watch search returned 99 registry records on October 14. Of those records, 29 were classified as trials of a drug, cell therapy or gene therapy; these are search-specific counts, not a census of all CMT research. The remaining records observe people with CMT, test devices or exercise, or develop ways to measure the disease. The table below covers the drug trials that are underway or that changed since 2024.
The biggest changes since mid-2025:
- New trials started. Novartis began a first-in-human trial of EDK060 for CMT1A, in Canada only, in September 2025. Elpida began a gene therapy trial for CMT4J in August 2026. Two “one-patient” antisense drugs, for CMT2S and CMT2D, are being tested.
- A major interruption and mixed results. Applied stopped its active govorestat studies and expanded access in April 2026; the planned CMT-SORD confirmatory trial was withdrawn in May before enrolment. Ignaseclant missed its primary walking endpoint but produced secondary signals that its sponsor says support further development.
- A small positive result. A Phase 3 trial of PXT3003 in China met its main goal, published in August 2026. The difference from placebo was small, and an earlier Phase 3 trial of the same drug did not confirm a benefit.
“Progress” means different things here. PXT3003 has a modest positive result from a randomized Phase 3 trial, weighed against conflicting earlier evidence. CLZ-2002 has an early published safety and exploratory efficacy study, without a control group. EDK060 and ELP-02 entering human testing is an important development, but neither has demonstrated clinical benefit. The single-participant antisense studies test highly individualized treatments; they are not evidence that the same approach will work for everyone with that subtype.
A further published development is CLZ-2002’s Phase 1 study: nine people with CMT1A or CMT1B received a cell product injected into muscle. The authors reported acceptable tolerability and improvements on exploratory measures. With nine participants, no placebo group and open-label assessment, efficacy and disease modification remain unproven.
Trial Status Table
Each row gives the drug and sponsor, what it targets and what kind of treatment it is, the trial phase, the registry number (linked), the status the registry shows and a plain-English note. Statuses are the sponsor’s own; “unknown” means the sponsor hasn’t updated the record in more than two years.
| Drug (sponsor) | Target and type | Phase | Trial | Status | Plain-English note |
|---|---|---|---|---|---|
| EDK060 (Novartis) | CMT1A; an oligonucleotide, according to a drug-industry database. Novartis hasn’t published its target | 1 | NCT07140614 | Recruiting | First test in people: one IV dose at one of four levels, against placebo, with planned enrolment of 28 adults aged 18 to 60. Measures safety and how the body handles the drug, not whether it works. Sites in Ottawa, Montreal and Sherbrooke only. Primary completion February 2028. |
| EN001 (ENCell) | CMT1A; donor stem cells from umbilical cord tissue (Wharton’s jelly), per a related registry record (NCT06218134) | 1b/2a | NCT06328712 | Recruiting | Two IV doses four weeks apart with planned enrolment of 27 adults in South Korea: an open-label dose-finding part, then low or high dose against placebo. In an earlier 9-person study the company reported no serious side effects and exploratory improvements; that was a company release, not a peer-reviewed paper. Primary completion December 31, 2026. |
| ELP-02 (Elpida Therapeutics) | CMT4J; gene therapy (AAV9) that delivers a working FIG4 gene | 1/2 | NCT07447557 | Recruiting | One dose into the spinal fluid with planned enrolment of 8 children and young people aged 3 to 20, open label. Started August 2026; recruiting in Palo Alto, with an Iowa City site not yet recruiting. Primary completion December 2032. |
| IGHMBP2 gene therapy (Nationwide Children’s Hospital) | CMT2S and SMARD1; gene therapy (AAV9) that delivers a working IGHMBP2 gene | 1/2 | NCT05152823 | Enrolling by invitation | Up to 10 participants in Columbus, Ohio. Started 2021; primary completion July 2028. |
| VCA-894A (Vanda Pharmaceuticals) | CMT2S; antisense drug made for one person’s IGHMBP2 variant | 1/2 | NCT07223632 | Active, not recruiting | One participant. No results reported. |
| nL-GARS1-001 (n-Lorem Foundation) | CMT2D; antisense drug made for one person’s GARS1 deletion | 1/2 | NCT07226297 | Enrolling by invitation | One participant, in Houston. A nonprofit’s one-patient drug, alongside Vanda’s. Primary completion October 2027. |
| Drug (sponsor) | Target and type | Phase | Trial | Status | Plain-English note |
|---|---|---|---|---|---|
| Ignaseclant, formerly NMD670 (NMD Pharma) | CMT1 and CMT2; pill that blocks a muscle chloride channel (ClC-1) | 2a | NCT06482437 | Completed | 81 adults enrolled; 80 were randomized to the drug or placebo for 21 days. In February 2026 the company said it missed its main goal, the 6-minute walk, and reported gains on some secondary measures. Detailed results were presented at the March 2026 MDA conference; a full peer-reviewed report was not identified in this review. |
| PXT3003 (Tasly GeneNet) | CMT1A; oral solution combining baclofen, naltrexone and sorbitol | 3 | NCT05092841 | Completed | Published August 2026: over 15 months the drug group’s disability score (ONLS, 0 to 12) improved slightly while the placebo group’s stayed level, a difference of 0.25 points. The adjusted difference was −0.249 points (95% CI −0.467 to −0.030). It met the primary endpoint, but this is an average group difference, not a quarter-point improvement in every participant; its clinical importance remains uncertain. Significant secondary findings included the leg subscore and ankle-dorsiflexion strength. The share of people who improved by a point or more wasn’t significantly different, nor were most other measures. Side effects were similar to placebo. |
| PXT3003 PREMIER (Pharnext) | CMT1A; same combination | 3 | NCT04762758 | Unknown | The company’s December 2023 topline results did not confirm a benefit. The registry hasn’t been updated since April 2024, so its status shows as “unknown.” Had sites in Ottawa, Toronto, Montreal, Québec City and Chicoutimi. |
| Govorestat, AT-007 (Applied Therapeutics) | CMT-SORD; aldose reductase inhibitor that lowers sorbitol | 2/3 | NCT05397665 | Completed | Missed its main goal in 2025. In November 2025 the company listed open questions from the FDA. Active studies and expanded access were stopped in April 2026 after Cycle acquired Applied; the follow-up Phase 3 was withdrawn in May (see below). |
| Govorestat confirmatory study (Applied Therapeutics) | CMT-SORD | 3 | NCT07191912 | Withdrawn | Withdrawn in May 2026 before anyone enrolled. |
| scAAV1.tMCK.NTF3 (Nationwide Children’s Hospital) | CMT1A; gene therapy delivering the nerve growth factor NT-3 to muscle | 1/2a | NCT03520751 | Withdrawn | Withdrawn in July 2026, before enrolment, because the lead investigator retired. |
| Engensis, VM202 (Helixmith) | CMT1A; non-viral plasmid DNA expressing hepatocyte growth factor (HGF), injected into muscle | 1/2 | NCT05361031 | Completed | 12 people in South Korea in an uncontrolled safety study, finished in 2022. Registry results are posted; the sponsor reported exploratory improvements, which do not establish efficacy. See our 2020 coverage and 2026 follow-up. |
| CLZ-2002 (Cellatoz Therapeutics) | CMT1; Schwann cell-like product derived from human tonsillar mesenchymal stem cells, injected into muscle | 1 | NCT05947578 | Completed | 9 people in South Korea (5 CMT1A, 4 CMT1B), finished August 2024. Phase 1 results are published, although no results are posted to the registry. No treatment-related serious adverse events were reported; exploratory improvements need confirmation in a larger controlled trial. |
| Epalrestat (Third Xiangya Hospital) | CMT-SORD; aldose reductase inhibitor | 2 | NCT05777226 | Unknown | Planned 30 people at five hospitals in China and to finish in April 2026. Not updated since April 2023. |
Mixed Results and Setbacks
Govorestat for CMT-SORD. Applied Therapeutics’ Phase 2/3 trial, INSPIRE (NCT05397665), missed its main goal in 2025. In September 2025 the company registered a confirmatory Phase 3 for about 155 people (NCT07191912). In November 2025 it reported that the FDA had raised open questions, including how well sorbitol levels stand in for clinical benefit, and that it had $11.9 million in cash at the end of September. Cycle completed its acquisition of Applied on February 3, 2026. On April 17, Applied announced that it was discontinuing all active Applied-sponsored govorestat studies and ending expanded access, citing program planning rather than new safety findings. This affected people already receiving the investigational drug, not just future recruits. See the acquisition announcement and Applied’s April 17 program update. The November cash figure is historical context, not an established explanation for the shutdown. In May 2026 the confirmatory trial was withdrawn before anyone enrolled; the registry says only that “the sponsor decided not to proceed with study initiation.”
Ignaseclant (NMD670). NMD Pharma’s Phase 2a trial (NCT06482437) did not improve the 6-minute walk after 21 days. The company pointed to improvements on secondary measures of function, hand strength and patient-reported physical function. These secondary findings need confirmation and do not overturn the primary endpoint miss. Detailed data were subsequently presented at the March 2026 MDA conference, and the sponsor said it intended to continue development; this is a mixed result, not an established failure of the program. See the sponsor’s announcement and conference presentation.
NT-3 gene therapy for CMT1A. A Phase 1/2a trial at Nationwide Children’s Hospital (NCT03520751) was withdrawn in July 2026 without enrolling anyone, because its lead investigator retired.
PXT3003. Pharnext’s PREMIER trial (NCT04762758) reported in December 2023 that it did not confirm a benefit, and its registry record has been left at “unknown” since. The Chinese trial (NCT05092841) later met its main goal, with a small effect. In a September 2026 review, Joshua Burns describes PXT3003 as re-emerging with renewed evidence of benefit after years of formulation problems, interrupted development, regulatory uncertainty and disputed efficacy, and argues that judging it depends on how much of the evidence is public.
Older stops. Acceleron ended its ACE-083 trials in 2020 (NCT03124459 and its extension NCT03943290) because the drug didn’t improve function. Flex Pharma ended FLX-787 in 2018 (NCT03254199), and a French trial of ulipristal for CMT1A (NCT02600286) was terminated without a stated reason. No trial in the ClinicalTrials.gov search used for this issue was listed as suspended; that does not rule out pauses elsewhere or delays not recorded by sponsors. See our report on ACE-083’s functional endpoint failures.
For the longer development and commercial history of govorestat, PXT3003 and Helixmith’s Engensis, see Three “Treatable CMT” Stories, and What the Market Taught Us. Our 2020 PXT3003 report explains why another Phase 3 was needed. Néovacs has also reported a proposed transaction concerning PXT3003 rights; that announcement does not establish a completed sale, a new trial or regulatory approval. The same Neovacs release, dated September 9, 2026, says Tasly applied for marketing authorization of PXT3003 in China on April 11, 2026, and cites the public list of applications accepted by China’s Center for Drug Evaluation. I couldn’t find the application in that list on October 15, searching by Tasly’s name and by PXT3003’s ingredients and code name, so the filing isn’t independently confirmed. We’re watching for it.
Before Trials in People With CMT
Condition-field searches can miss programs being tested first in healthy volunteers. Actio announced healthy-volunteer Phase 1 dosing of ABS-0871 in March 2025. This oral TRPV4 inhibitor is intended for CMT2C; that trial tests safety and pharmacology, not benefit in people with CMT. ABS-0871 is changing hands. In August 2026, Jazz Pharmaceuticals agreed to buy Actio for its epilepsy drug, ABS-1230. Under the deal, ABS-0871 was to go to a new private company spun out of Actio at closing, funded by Actio’s existing investors, with Jazz taking a minority stake. Jazz completed the purchase on September 15, 2026, but that announcement doesn’t name the new company or mention ABS-0871. We’re watching for who now develops ABS-0871 and what happens to its trial plans.
Augustine announced healthy-volunteer Phase 1 dosing of AGT-100216 in May 2025, an HDAC6 inhibitor intended for CMT. Its current website describes a differently named lead, AGT-100XXX, at the IND-enabling stage. The relationship between these programs and AGT-100216’s current status needs clarification; the earlier announcement is not proof of continued progress.
Trials Recruiting in Canada
Novartis’s EDK060 study (NCT07140614) is the only CMT drug trial identified as recruiting in Canada in the sources checked for this issue. It is enrolling adults aged 18 to 60 with CMT1A at sites in Ottawa, Montreal and Sherbrooke. Two observational studies are also recruiting at a Toronto site: a natural history study of several CMT types (NCT01193075) and a study of genetic modifiers of CMT1A and new causes of CMT2 (NCT01193088).
The Government of Canada’s clinical trial search portal lists four CMT trials, and all four are also on ClinicalTrials.gov: the PREMIER trial and Pharnext’s long-term extension study of PXT3003 (NCT03023540), EDK060 and the ACE-083 extension.
How This Table Is Kept
The list starts from an automatic search of ClinicalTrials.gov run by Research Watch, a tool I built for this site. It searches the registry’s condition field for CMT, its older names, its subtype codes (CMT1A, CMT2S and so on), HNPP, SORD deficiency and distal hereditary motor neuropathy, and flags selected changes in status, phase, interventions, locations and registry results availability. It does not capture every change, and a registry results flag does not tell us whether a journal paper or conference report exists. I then read each trial record myself and check results against company announcements and published papers. For this issue I also searched the Canadian trial portal and the EU’s Clinical Trials Information System by hand; neither had a CMT trial missing from ClinicalTrials.gov. Five of the trials underway (EDK060, EN001, ELP-02 and the two one-patient antisense drugs) also have their own Research Watch entries, which I review when new information arrives; upstream registry changes do not automatically alter their approved public summaries.
Research Watch also collects journal records and explicitly labelled preprints from Europe PMC. Its literature search is separate from its trial search; it does not automatically match every publication to a trial or monitor sponsor news, regulatory decisions and conference disclosures. For each issue, the registry baseline needs a separate search by drug name, aliases, sponsor and trial ID, including this site’s earlier reporting. Planned enrolment is not actual enrolment, and an estimated primary completion date is not a promised results announcement. Completion refers to finishing the relevant data collection, not proving a treatment works.
The limits: trials registered only in other national registries, such as China’s, Korea’s or Japan’s, can be missed. A registry status is whatever the sponsor last entered, so it can be out of date. A company press release is not peer review, and I say which results come from one. If you know of a trial that’s missing, tell me.
Sources
- Actio Biosciences. “Actio Biosciences Announces First Participant Dosed in Phase 1 Clinical Trial of ABS-0871, a Novel TRPV4 Inhibitor, for the Treatment of Charcot Marie Tooth Disease 2C.” Actio Biosciences (press release). March 3, 2025. https://actiobiosciences.com/actio-biosciences-announces-first-participant-dosed-in-phase-1-clinical-trial-of-abs-0871-a-novel-trpv4-inhibitor-for-the-treatment-of-charcot-marie-tooth-disease-2c/.
- Applied Therapeutics. “Program Update: Govorestat Development and Clinical Studies.” Applied Therapeutics. April 17, 2026. Archived May 21, 2026, at the Wayback Machine. https://web.archive.org/web/20260521131918/https://www.appliedtherapeutics.com/.
- Arnold, W. David, David Herrmann, Kristl G. Claeys, Henning Andersen, Gregory Carter, Yessar Hussein, Thomas S. Grønnebæk, et al. “Efficacy and Safety of Ignaseclant in Adults with Charcot-Marie-Tooth Disease: Topline Results of a Phase 2a Study.” Poster presented at the MDA Conference, March 2026. https://static1.squarespace.com/static/60015fa94b905b61b3febe42/t/69b12f04531ec327a0603ce4/1773219588844/NMD%2BPharma_CMT%2Btopline%2Bresults_MDA%2BConference_.pdf.
- Augustine Therapeutics. “Augustine Therapeutics Announces First Patient Dosed in Phase I Clinical Trial Evaluating Lead Candidate AGT-100216 for the Treatment of Charcot-Marie-Tooth Disease.” Augustine Therapeutics (press release). May 27, 2025. https://www.augustinetx.com/media/augustine-therapeutics-announces-first-patient-dosed-in-phase-i-clinical-trial-evaluating-lead-candidate-agt-100216-for-the-treatment-of-charcot-marie-tooth-disease.
- Augustine Therapeutics. “Augustine Therapeutics: Novel HDAC6 Inhibitors for Chronic Disease.” Accessed October 15, 2026. https://www.augustinetx.com/.
- Burns, Joshua. “PXT3003: Lessons for Therapeutic Development in Charcot-Marie-Tooth Disease Type 1A and Related Neuropathies.” iScience 29, no. 10 (2026): 117568. https://doi.org/10.1016/j.isci.2026.117568.
- Center for Drug Evaluation, National Medical Products Administration. “受理品种信息 (Accepted Applications).” Accessed October 15, 2026. https://www.cde.org.cn/main/xxgk/listpage/9f9c74c73e0f8f56a8bfbc646055026d.
- ClinicalTrials.gov. “Charcot-Marie-Tooth Studies.” Accessed October 14, 2026. https://clinicaltrials.gov/search?cond=Charcot-Marie-Tooth%20Disease.
- Cycle Pharmaceuticals. “Cycle Pharmaceuticals Completes Acquisition of Applied Therapeutics.” Cycle Pharmaceuticals (press release). February 3, 2026. https://cyclepharma.com/news/acquisition-of-applied-therapeutics/.
- ENCell. “ENCell Presents the Phase 1 Clinical Trial Results of Their Mesenchymal Stem Cell Therapy EN001 for Charcot Marie Tooth Disease at the PNS Conference.” Business Wire (press release). June 24, 2024. https://www.businesswire.com/news/home/20240624485930/en/ENCell-Presents-the-Phase-1-Clinical-Trial-Results-of-their-Mesenchymal-Stem-Cell-Therapy-EN001-for-Charcot-Marie-Tooth-Disease-at-the-PNS-conference.
- European Medicines Agency. “Clinical Trials in the European Union (CTIS).” Accessed October 14, 2026. https://euclinicaltrials.eu/.
- Fierce Biotech. “Analysts Skeptical Applied Tx Can Get Rare Disease Drug to Market as FDA Talks Drag On.” Fierce Biotech, November 14, 2025. https://www.fiercebiotech.com/biotech/analysts-question-if-applied-tx-can-ever-get-rare-disease-drug-market-fda-talks-drag.
- Government of Canada. “Canadian Clinical Trial Search Portal.” Accessed October 14, 2026. https://clinical-trials-search.canada.ca/en/home.
- Institut de Myologie. “Preliminary Results from the PREMIER Trial Do Not Confirm the Efficacy of PXT 3003 in CMT 1A.” Accessed October 14, 2026. https://www.institut-myologie.org/en/?p=41796.
- Jazz Pharmaceuticals. “Jazz Pharmaceuticals Completes Acquisition of Actio Biosciences.” Actio Biosciences (press release). September 15, 2026. https://actiobiosciences.com/jazz-pharmaceuticals-completes-acquisition-of-actio-biosciences/.
- Jazz Pharmaceuticals. “Jazz Pharmaceuticals to Acquire Actio Biosciences, Expanding Rare Epilepsy Portfolio.” Actio Biosciences (press release). August 10, 2026. https://actiobiosciences.com/jazz-pharmaceuticals-to-acquire-actio-biosciences-expanding-rare-epilepsy-portfolio/.
- Kim, Hyesun, Byung-Ok Choi, Hyunju Lee, Saeyoung Park, Jaeseung Lim, and Sung-Chul Jung. “A Phase 1 Clinical Trial to Evaluate the Safety and Tolerability of CLZ-2002 for the Treatment of Patients with Charcot–Marie–Tooth Disease Type 1.” Stem Cells Translational Medicine 15, no. 8 (2026): szag048. https://doi.org/10.1093/stcltm/szag048.
- Liu, Xiaoxuan, Wanqian Cao, Hongli Li, et al. “A Phase III Clinical Trial of PXT3003 in Chinese Patients with Charcot-Marie-Tooth Disease Type 1A.” iScience 29, no. 9 (2026): 117284. https://doi.org/10.1016/j.isci.2026.117284.
- Manalac, Tristan. “New Confirmatory Study Hints at ‘Alignment’ Between Applied Therapeutics, FDA for Rare Disease Drug.” BioSpace, September 29, 2025. https://www.biospace.com/drug-development/new-confirmatory-study-hints-at-alignment-between-applied-therapeutics-fda-for-rare-disease-drug.
- Neovacs. “Nouvelle publication scientifique des résultats de Phase III de PXT3003 en Chine.” Neovacs (regulatory information). September 9, 2026. https://www.neovacs.com/fr/investisseurs/information-reglementee/?ID=ACTUS-0-100134.
- NMD Pharma. “NMD Pharma Announces Topline Results from Its Phase 2a Study of Ignaseclant in Charcot-Marie-Tooth Disease Types 1 and 2.” NMD Pharma (press release). February 3, 2026. https://www.nmdpharma.com/news/topline-results.
- Patsnap Synapse. “EDK-060.” Accessed October 14, 2026. https://synapse.patsnap.com/drug/2e6d3c80517cbb27ba91d4d0c280d9e5.
Leave a Reply