Clinical trials and the research pipeline

At a glanceABS-0871 is the only drug developed against the cause of CMT2C that has already been tested in people [4]. As of October 2026, its first patient trial is planned but not yet recruiting [34]. Future trial sites in Europe have also been proposed, with no dates yet (see the update below). Five other drugs act on different parts of the problem, such as muscle strength and stressed nerve fibers, and could one day be used alongside it. We need ABS-0871 to stop the cause, and these drugs to help with the damage that is already there.

Why we need ABS-0871 alongside the others

ABS-0871 is designed to block the overactive TRPV4 channel that causes CMT2C [4]. The other drugs on this page act on different steps: one helps muscles respond to a weakened nerve signal, others aim to protect or support stressed nerve fibers. That matters, because damage that has already happened will not undo itself. Only ABS-0871 aims to stop new damage at its source, so the most hopeful future is one in which it is used together with these drugs, each doing a different job. Why ABS-0871 means so much to us.

• ABS-0871 Built for CMT2C

  Stops the cause [4]

  • Aims at the overactive TRPV4 channel Yes [4]
  • Helped animals with a CMT2C change Company data say yes [16]; a similar blocker helped mice [15]
  • Tested in people with CMT2 Patient study planned [34]

• Ignaseclant Already open to CMT2

  Strengthens muscle response [45]

  • Aims at the overactive TRPV4 channel No, a later step
  • Helped animals with a CMT2C change Not tested yet
  • Tested in people with CMT2 Yes, for 3 weeks [45]

• RTX-117 Could work

  Calms nerve cell stress [189]

  • Aims at the overactive TRPV4 channel No, a later step
  • Helped animals with a CMT2C change Not tested yet
  • Tested in people with CMT2 Not yet

• AGT-100216 Could work

  Restarts transport in nerve fibers [191]

  • Aims at the overactive TRPV4 channel No, a later step
  • Helped animals with a CMT2C change Not tested yet
  • Tested in people with CMT2 Not yet

• SARM1 inhibitors Could work

  Protects nerve fibers [192]

  • Aims at the overactive TRPV4 channel No, a later step
  • Helped animals with a CMT2C change Not tested yet
  • Tested in people with CMT2 Not yet

• EN001 Could work

  Sends repair signals [198]

  • Aims at the overactive TRPV4 channel No, a later step
  • Helped animals with a CMT2C change Not tested yet
  • Tested in people with CMT2 Not yet

A similar TRPV4 blocker nearly fully rescued survival and movement in mice with CMT2C changes, even when started after symptoms began [15]. None of the other drugs have been tested in TRPV4 disease yet. That is why the ABS-0871 patient study matters so much to us, and why we would love to see the others tested in TRPV4 too, so they are ready to use alongside it.

The pipeline at a glance

Six drugs in human trials could matter for CMT2C. Only ABS-0871 is built for it, and each of the others is labeled by how close it is to CMT2C. The CMT Research Foundation's 2026 report says more drug candidates are in clinical trials for CMT than ever before [99].

Pre­clin­icalPhase 1Phase 2Phase 3

• ABS-0871 Built for CMT2CPhase 1a done; patient study planned
• Ignaseclant Already open to CMT2Phase 2a done
• RTX-117 Could workPhase 1
• AGT-100216 Could workPhase 1
• SARM1 inhibitors Could workPhase 1 to 2
• EN001 Could workPhase 1b/2a

What you can do now

Each group of drugs calls for something different. Pick the card that fits what you want to do.

• Built for CMT2C

  Get ready for the ABS-0871 patient study

  • Join or stay in the Johns Hopkins TRPV4 natural history study, which follows people over time to learn how the disease behaves. For now it is open only to people in the United States and Canada, and its participants are already known to TRPV4 researchers [101]. How to sign up.
  • Check the CMT Research Foundation trial tracker each month [34].
  • If you live in Europe, watch for news of the proposed European trial sites; I will add dates here as soon as they are announced (see the update below).
  • Keep a copy of your genetic report. In lab tests, some TRPV4 changes needed a much higher dose of a blocker, so trials may screen people by variant [28].

• Already open to CMT2

  Ask about ignaseclant's next trial

  • Its Phase 2a trial enrolled adults with any genetically confirmed CMT1 or CMT2 [45].
  • The next trial is not registered yet [187]. Ask NMD Pharma to keep people with CMT2 eligible.

• Could work

  Ask the makers to test their drugs in TRPV4 disease

  • RTX-117, AGT-100216, SARM1 inhibitors and EN001 have never been tried in TRPV4 disease.
  • A mouse carrying a CMT2C change already exists [15]. Use the email template to ask.

Make your voice heard: the Charcot-Marie-Tooth Association (CMTA) has organized a private FDA Patient Listening Session for the CMT2C/TRPV4 community on 20 October 2026, where FDA decision-makers hear directly from people living with CMT2C [104]. The speakers for this session have already been chosen. To be invited to future opportunities like this, as well as surveys and focus groups, sign up for CMTA Patients as Partners in Research [54]. How to sign up.

Sign up: studies and lists open to you now

No drug trial is open to people with CMT2C right now. These four studies and lists are open, and each one puts you on the radar of the researchers planning CMT2C trials. Sign-up details were checked on 10 October 2026.

• Recruiting

  Johns Hopkins TRPV4 natural history study

  For people aged 3 to 80 with a TRPV4 change, for now only in the United States and Canada. One two-day visit a year for six years; travel and tests are covered [101].

  To sign up: contact the study coordinators listed on the study record, Betty Hu (xhu38@jhu.edu, 443-250-4699) or Simone Thomas (sthom125@jhmi.edu, 410-614-4188) [101].

  See the study record

• Open to join

  CMTA Patients as Partners in Research

  The CMTA's list for early notice of clinical trials, surveys and focus groups [54]. The CMTA points people here for future CMT2C/TRPV4 opportunities like the FDA listening session [104].

  Sign up with the CMTA

• Recruiting

  INC 6601 natural history study

  For any type of CMT, with yearly visits at 21 sites in the US, Canada, the UK, Italy and Australia [52].

  To sign up: contact the coordinator at your nearest site, listed on the study page.

  If you have a TRPV4 change: ask for the extra TRPV4-specific survey that is part of joining INC. Not every INC clinic knows about it, so it is easy to miss.

  Find your nearest site

• Open to join

  GRIN registry (Hereditary Neuropathy Foundation)

  A free registry open worldwide to anyone with CMT or another inherited neuropathy. It collects genetic and survey-based natural history data [55].

  Join GRIN

Living outside the US and Canada

The Johns Hopkins TRPV4 study cannot enroll you yet. The closest option is INC 6601, which has sites in the UK and Italy. Stephanie Carmody, who started the TRPV4 patient group on Facebook, especially recommends the London site, where Dr. Mary M. Reilly sees patients at the Queen Square Centre for Neuromuscular Diseases. The GRIN registry is open to people anywhere in the world [55].

No sign-up yet

• ABS-0871: the patient study is not yet recruiting and has no public registry record [34]. Patients as Partners, the TRPV4 Facebook group and the natural history study are the best ways to hear news first.
• Ignaseclant: the CMT trial is finished [203], and the next one is not registered yet [187].
• EN001 is recruiting only people with CMT1A, in Korea [198]. The SARM1 inhibitor trials enroll only people who are having chemotherapy or who have ALS [196].
• RTX-117 and AGT-100216 are testing healthy volunteers only [99].

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How each drug acts on CMT2C

In CMT2C, the TRPV4 channel lets in too much calcium. In flies and nerve cells, that stalls mitochondria, the cell's power supply, on their way down the axon (the nerve fiber) until the axon degenerates [26]. In mice with a CMT2C change, the damage seemed to start in the lining of small blood vessels in the spinal cord [15]. Each drug acts at a different step.

1. TRPV4 mutationChannel stuck open; RhoA brake weakened.

   What could act there:

   • ABS-0871 Built for CMT2CBlocks TRPV4: aims at the cause
2. Too much calcium entersIn nerve and blood vessel lining cells.
3. The axon comes under stressMitochondria stall in the axon. A stress alarm (ISR) may stay on. Energy supply and repair falter.

   What could act there:

   • RTX-117 Could workSwitches the stress alarm off
   • AGT-100216 Could workGets mitochondria moving again
   • EN001 (stem cells) Could workReleases repair signals
4. The axon self-destructsSARM1 dismantles it; muscle loses its nerve.

   What could act there:

   • SARM1 inhibitors Could workBlock the self-destruct switch
5. The muscle gets a weak signalWeakness in feet, hands, voice, breathing.

   What could act there:

   • Ignaseclant Already open to CMT2Eases the ClC-1 brake on muscle

ABS-0871: the first drug designed for CMT2C

What it is. ABS-0871 is described by its developer as “a potent and selective orally available TRPV4 small molecule inhibitor”, designed to inhibit the overactive channel caused by gain-of-function TRPV4 mutations [4]. In the company's preclinical CMT2C models it produced “marked improvements in motor function and mobility, compared to untreated controls” [16]. The company has not published the species, doses or numbers behind that claim.

What the research says

• The plan. In June 2025 the company said it would expand into a Phase 1b study in patients with TRPV4-positive CMT2C in 2026 [42]. No design, sites or registry number are public yet, and no Phase 1a results have been released [34].
• TRPV4 blockers have been given to people before. An earlier blocker, GSK2798745, was studied in healthy volunteers and in people with heart failure [49] and chronic cough [51]. Its first study reported no significant safety issues [49]. It was never tested in CMT2C.
• A blocker rescued the CMT2C mouse. A TRPV4 blocker started after symptoms began nearly fully rescued survival and movement in mice with CMT2C changes [15]. These mice were severely affected, so the result may not carry over directly to adults.
• One caveat. CMT2C changes also loosen TRPV4's grip on a protein called RhoA, and in cells this stunted nerve growth. A channel blocker did not fix that growth problem, while blocking RhoA did [24]. If this holds in people, a channel blocker may not fix everything on its own.
• The latest company update. The CMT Research Foundation's convention agenda lists David Goldstein (Actio Biosciences) as a speaker in the clinical trials session on 26 September 2026 [179]. I will add a summary when one is published.

Timeline so far

Date	Milestone	Source
June 2023	CMT2C patients and families took part in a virtual focus group with the CMTA and Actio Biosciences	[39]
8 August 2024	FDA Orphan Drug and Rare Pediatric Disease designations announced	[40]
3 March 2025	First participant dosed in a Phase 1 trial in healthy volunteers (randomized, double-blind, placebo-controlled, single and multiple ascending doses)	[16]
18 June 2025	$66 million Series B financing announced; Fast Track designation listed; Phase 1b study in CMT2C patients planned for 2026	[42]
3 December 2025	Actio Biosciences, leading CMT2C experts and the CMTA hold a clinical trial update meeting for the CMT2C/TRPV4 community. The CMTA posted the recording on YouTube	[103]
By 2026	Company website lists the program at “End of Phase 1”, with the Phase 1a healthy-volunteer study completed	[4]
10 August 2026	Jazz Pharmaceuticals announces it will acquire Actio Biosciences. ABS-0871 is to move into a new independent company funded by existing investors, with Jazz holding a minority stake	[37]
15 September 2026	Jazz completes the acquisition; Actio spins out a new private company holding its other programs	[178]
September 2026	The CMT Research Foundation's 2026 annual research report says Actio launched its Phase 1 trial for CMT2C (the healthy-volunteer study) in fiscal year 2025, and that the foundation expects the program's next steps in fiscal year 2027	[99]
26 September 2026	Company update at the CMT Research Foundation's 2026 Global CMT Research Convention	[179]
4 October 2026	The CMT Research Foundation's trial tracker lists ABS-0871 under planned trials: Phase 1, “Not yet recruiting”, sites “To be announced”	[34]
October 2026	The company developing ABS-0871 has a new name, coming soon. Future clinical trial sites in Europe are proposed; the timing is not yet known	Update from Klaudia
20 October 2026	FDA Patient Listening Session for the CMT2C/TRPV4 community, organized by the CMTA: a private, 90-minute virtual meeting where FDA decision-makers hear from people living with CMT2C. The speakers have already been chosen	[104]

What is not yet known (as of October 2026)

• The new names of the company and the drug, and the company's website. I will add them as soon as they are public.
• When and where the proposed clinical trial sites in Europe will open.
• Whether the patient trial has opened for recruitment. When I last checked, the tracker listed it as not yet recruiting [34].
• The trial's sites, eligibility criteria and registry number. I could not confirm a registry number for any ABS-0871 trial.
• Results of the Phase 1 healthy-volunteer study. No published results were found.

Where to check for updates

• The TRPV4 Facebook group. Stephanie Carmody, who started the TRPV4 Related Hereditary Neuropathies (CMT2C, SPSMA & CDSMA) group, will share news about the trial there once it starts enrolling. She will also organize Zoom meetings for the patient community with the CMTA, the company developing ABS-0871, and the TRPV4 researchers Dr. Charlotte Sumner and Dr. Brett McCray, to go through the details. You may need to sign in to Facebook and ask to join the group.
• CMTA Patients as Partners in Research. Stephanie also recommends signing up, because the CMTA uses it to alert people when new trials open for their type of CMT. Members are among the first to hear about clinical trials and studies [54], and the sign-up form asks for your CMT type and subtype, with CMT2C on the list. See how to sign up.
• The CMT Research Foundation's clinical trials tracker [34].
• ClinicalTrials.gov, which the FDA recommends for finding trials [43]. Search for “TRPV4” and “ABS-0871”, and for the new name once it is announced.
• Please note that ActioTrials.com covered only a different Actio program (ABS-1230, for KCNT1 epilepsy) and did not mention CMT2C when checked in October 2026 [44].

A personal note: I follow this program closely. I will update this page when a patient trial opens.

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Ignaseclant: already open to CMT2

Ignaseclant (NMD670) is not aimed at TRPV4, but it is the only drug here whose trial has already accepted people with CMT2, so people with CMT2C were eligible [45]. It may help muscles respond to a weakened nerve signal, something ABS-0871 is not designed to do, while ABS-0871 aims to slow the nerve damage itself. The two could complement each other.

Why CMT2 may respond more than CMT1

• CMT2 nerves drop more signals. In a nerve test study of 21 people with CMT, signals failed to cross from nerve to muscle in 75% of CMT2 recordings, compared with 10% in CMT1. Only 5 people had CMT2, and the study was sponsored by NMD Pharma [180].
• It worked in a CMT2 mouse. In CMT2D mice, one dose raised muscle force by 41 to 45%. No TRPV4 mouse has been tested [180].
• Voice that tires with use. In CMT2C, voice weakness can get worse with exercise or excitement [18]. Weakness that gets worse with use can point to signals not crossing well from nerve to muscle. This is my own hypothesis, not a finding, but it is worth putting to NMD Pharma.

Read with care: one company release calls a day-28 CMT function score result significant, but the conference abstract gives p=0.06, which is not statistically significant [183]. Nearly all ignaseclant data so far come from the company or its investigators.

Could work alongside ABS-0871: ask the researchers

These four drugs act on damage that CMT2C may share with other types of CMT, so each could add something ABS-0871 cannot. None of them have been tested in TRPV4 disease yet, and a request from a patient is one of the few ways to change that.

The fastest test. A mouse carrying a CMT2C change already exists [15]. The most concrete thing a patient can do is ask each company whether it would test its drug in that mouse, or work with the team that built it.

No gene-targeted therapy for TRPV4 has been found in clinical or published preclinical development; the published research focuses on small-molecule channel blockers [3].

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Using them together

We need ABS-0871 to stop the cause, and the other drugs could help with what it cannot reach, such as damage that is already done. That is why the most hopeful plans combine them. No pair has been tested together yet, and for now the only way to take two experimental drugs together would be in a trial designed for it.

Four possible pairings

Combination	The idea	Strength of the case	What has to be true first
ABS-0871 + ignaseclant	Stop new damage at the nerve, and get more strength from the connections that are left	Strongest	ABS-0871 works on its own, and the two are safe together. In rats, ignaseclant added to the effect of other signal-boosting drugs rather than replacing it [181]
ABS-0871 + AGT-100216	Stop the cause, and clear the mitochondria traffic jam it already made	Promising	AGT-100216 helps in a TRPV4 model
ABS-0871 + RTX-117	Stop the calcium flood, and switch off a lingering stress alarm	Possible	Evidence that the stress alarm is on in TRPV4 nerves
ABS-0871 + a SARM1 inhibitor	Stop the cause, and protect stressed axons	Needs a TRPV4 test	A positive TRPV4 test, after no benefit in three CMT2 mouse types [194]

Only with your neurologist. Never put a combination together yourself. Exercise and physical therapy stay the foundation.

Why combining could pay off, and what to be careful about

Why combining could pay off

• Stopping damage is not undoing it. Even if ABS-0871 halts progression, nerve fibers lost years ago may not regrow. An add-on could help the remaining connections do more.
• Nerve and muscle are different places. Drugs that act on different tissues are less likely to work against each other.
• Short trials favor fast drugs. CMT2C changes slowly, about 0.81 points a year on one exam score in the registry [3], so a drug like ignaseclant that helps within weeks is easier to test.

What to be careful about

• No safety data on any pair. Two drugs can interact, or their side effects can add up, in ways neither shows alone.
• Trials usually exclude other experimental drugs. Combinations would come after approval, or through a trial built for add-on use.

What other diseases have shown

Disease	Combination	What happened	Lesson for CMT2C
Spinal muscular atrophy	Apitegromab, a muscle drug, added to gene-targeted treatment	Approved by the FDA as Isembyld on 11 September 2026 for people already on gene-targeted treatment [200]	The closest model for ABS-0871 plus ignaseclant
Duchenne muscular dystrophy	Givinostat added to steroids	Approved on 21 March 2024 [201]	Add-ons are tested on top of the standard drug
ALS	Relyvrio, a two-drug pill	Withdrawn in April 2024 after a large trial failed [202]	A small early win is not proof

What to watch for

Five trial results are due by March 2027, but none of them come from people with CMT2C. The ABS-0871 patient study, which matters most, has no public start date.

Oct
2026Dec
2026Feb
2027Apr
2027

• ABS-0871 patient study Built for CMT2CPlanned for 2026; not yet openExpected: not announced (the company said 2026; the CMT Research Foundation expects next steps in its fiscal year 2027)
• Ignaseclant: SMA results Already open to CMT2Phase 2 in spinal muscular atrophyExpected: Oct 2026 to Dec 2026
• Ignaseclant: MG results Already open to CMT2Phase 2b in myasthenia gravisExpected: Jan 2027 to Mar 2027
• RTX-117 Phase 1 ends Could workA CMT Phase 2 would followExpected: Dec 2026
• TNV108 safety data Could workPhase 1 of a SARM1 blockerExpected: Oct 2026 to Dec 2026
• EN001 Phase 1b/2a ends Could workCMT1A stem cell resultsExpected: Dec 2026

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Natural history studies: the foundation for trials

The two studies, and why they matter

• Johns Hopkins TRPV4 natural history study (NCT05600764). It is open to children and adults aged 3 to 80 with a TRPV4 change, and was listed as recruiting. For now it is open only to people in the United States and Canada, and several Canadians are already taking part. Participants visit Johns Hopkins in Baltimore once a year for six years, with testing spread over two days; travel and hotel costs are reimbursed and all tests are free [101]. Testing includes neurological exams, nerve conduction studies, imaging, voice and vocal cord assessments, and tests of daily tasks such as getting up from a chair or walking for six minutes [101]. The study is led by Johns Hopkins, with the Muscular Dystrophy Association and Actio Biosciences as collaborators and funders [101]. Contact details are on the study listing [101].
• INC 6601. The Inherited Neuropathy Consortium's natural history study is open to people with any type of CMT. It involves yearly visits, was listed as recruiting, and runs at 21 sites in the US, Canada, the UK, Italy and Australia, including Johns Hopkins. To join, you contact the coordinator at a participating site [52]. The 2025 TRPV4 registry paper was built from this study [3]. If you have a TRPV4 change, ask for the extra TRPV4-specific survey when you enroll; not every INC clinic knows about it. If you live outside the US and Canada, the European INC sites are the closest route, especially London (Dr. Mary M. Reilly).
• Why they matter. FDA guidance explains that natural history data help decide which patients to enroll, which outcomes to measure and how long a trial needs to run [91]. The registry authors call for outcome measures that capture non-length-dependent weakness (weakness that does not simply affect the longest nerves first), vocal fold weakness and breathing involvement, because standard CMT scales miss them [3].

My own experience: I had nerve testing at Johns Hopkins in September 2026 as part of the TRPV4 natural history study.

Research that could speed up CMT2C trials

A blood test, blood vessel cells and shared trial rules

• A blood test to track CMT2C. The CMT Research Foundation is funding a new Johns Hopkins study, led by Dr. Jeremy Sullivan and Dr. Charlotte Sumner, to look for a blood biomarker for CMT2C. The idea is that damaged nerves leak specific proteins into the blood. The team will compare blood from treated and untreated lab models of CMT2C, looking for changes that track disease progression and response to treatment. A validated blood test would give trial sponsors a faster, simpler way to tell whether a TRPV4-blocking drug is working [99]. The report describes this as a new study, so there are no results yet.
• Blood vessel lining cells. Through its Emerging Researcher Award, the foundation funded a 2025 project at Johns Hopkins (Jonathan Alevy) to find changes in gene activity in the cells that line nerve blood vessels (neurovascular endothelial cells) in a lab model of CMT2C [99]. This follows earlier Johns Hopkins work on TRPV4 and the barrier between blood and nerves [30].
• Shared rules for CMT trials. In August 2026 a coalition called ToPIC, led mainly by the CMT Research Foundation and including other CMT organizations, patients, clinicians and drug companies, published shared recommendations on trial design, endpoints, biomarkers and enrollment in the Journal of the Peripheral Nervous System [99]. They cover all types of CMT, not CMT2C specifically.

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How to take part and reach out

The six steps for joining studies and registries are on the Community page. To ask a company to include CMT2C, write to it directly and copy a patient foundation, since foundations fund research and companies listen to them.

An email you can adapt

Subject: Would you consider CMT2C (TRPV4) for [drug name]?

Hello. I have CMT2C, caused by a TRPV4 gain-of-function variant. I read about [drug name] [where you read about it].

CMT2C shares [stressed nerve fibers / stalled mitochondria / weak nerve-to-muscle signals] with the types you are studying. Would you consider testing [drug name] in a TRPV4 model, or including CMT2 patients in your next study? A CMT2C mouse model already exists (Sullivan et al., Science Translational Medicine, 2024).

I would be glad to share my experience or connect you with the CMT2C community. Thank you for your work.

[Your name and email]

Expanded access (compassionate use)

Expanded access is a way to get an investigational drug (one that is not yet approved) outside a trial. The FDA's criteria are a serious or life-threatening disease, no satisfactory alternative therapy, inability to take part in a clinical trial, and a potential benefit that justifies the risks [43]. A licensed physician must make the request, and the manufacturer may decline [43]. I found no expanded access program for ABS-0871 as of October 2026.

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