Clinical Trials

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Clinical trials are medical research studies that test a new treatment, device, or procedure to see if it is safe and effective.

Researchers test a new intervention or treatment, device, or procedure to see if it is safe and effective.

Participants are assigned to receive the investigational treatment or a comparison (such as the current standard of care or a placebo).
Trials go through phases (I–III) to answer questions about safety, effectiveness, and how the treatment compares to current options.
Why they matter: They provide the evidence the FDA and other regulators need to decide if a new therapy can be approved. Complete our interest form to be notified of changes in trial status.

KCNT1-Targeted Clinical Studies

Trial name & link	Sponsor	Modality	Phase	Overall Status*	Countries / Active sites	Coordinator / Contact
[ABS-1230]	Actio Biosciences	Small molecule	I (safety in healthy humans) -nearing completion	Not recruiting. (Screening to begin soon)	US	See webpage for more info
[KANDLE Study]	Servier	ASO	Ib/II	Recruiting (site-dependent)	Spain, France ( Italy, US, Japan opening soon)	scientificinformation@servier.com
[ATL-201]	Atalanta	di-siRNA	TBD	Preclinical	NA	NA
[KCNT1 International Registry]	Multicenter	Observational	–	Not yet recruiting	US /international	Amanda.A@kcnt1epilepsy.org

*Overall status reflects registry labels (e.g., Not yet recruiting, Recruiting, Active—not recruiting). Individual hospitals enroll only after site activation (contracts, ethics, training, drug on site).

Clinical trial information is shared for awareness only. The KCNT1 Epilepsy Foundation does not screen participants or manage enrollment. Eligibility and participation decisions are made by the study sponsor and research sites.

Other Clinical Trials (not KCNT1-specific)

	Sponsor	Therapy Name (link)	Phase	Class of therapeutic	Delivery Target	Status
Lundbeck	Bexicaserin	Phase 3	Small molecule	Oral	5-HT2C (not KCNT1)	Enrolling

Frequently Asked Questions About Clinical Trials

Participation Basics

Can I participate in more than one study at the same time?

You can often participate in multiple observational studies, because they don’t involve experimental therapies.
For clinical trials, you must ask the trial’s Primary Investigator. Some studies prohibit participation in another trial at the same time.

Who is eligible to participate in a clinical trial?

If I am eligible for a trial, how do I enroll?

What if I don’t qualify for a clinical trial?

What to Expect During a Study

What is informed consent?

Will my medical care change if I join a study?

What happens during a clinical trial?

How long do clinical trials last?

Safety & Ethics

How do I know if a study is safe?

What are my rights and responsibilities as a participant?

Can I share my experience publicly?

Finding Trials

Where can I find out about clinical trials?

Can I join a clinical trial if I live outside the United States?

Do studies cost money to join?

Want to Learn More? Visit our clinical trials education page!

Disclaimer: We gratefully acknowledge many organizations for educational resources we have provided here: SCN2aFamilies, Global Genes, Dravet Syndrome Foundation, International Rett Syndrome Foundation, Angelman Clinical Trials, LGS Foundation, NIH, HHS, FDA, and others.

KCNT1 Clinical Trial Interest Form

This brief form helps the KCNT1 Epilepsy Foundation understand who is interested in learning about KCNT1 clinical trials and what kind of educational resources might be helpful.

Study-Specific Permissions

Please select the study or studies for which you authorize us to share your name and contact information only with the enrolling site(s), and with the sponsor or CRO if applicable, for the sole purpose of contacting you about that study.

Please select the study

Actio Biosciences ACT-1230 (TBD)

Server: A Phase Ib/II first-in-human, multicentre, open-label, multiple ascending dose study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamic effect of intrathecal S230815 in pediatric participants with KCNT1-related Developmental and Epileptic Encephalopathy

I authorize the KCNT1 Epilepsy Foundation and its staff and representatives to share my name and contact information with the enrolling clinical trial site(s) so they may contact me about this study.

Name

First Name

Last Name

Your country of residence

State

Province/Region

Important Notes

No medical or health information will be shared.
Eligibility and enrollment decisions are made solely by the study team.
This permission applies only to the study or studies selected above.
Your contact information will not be used for other studies without your separate permission.
You may withdraw this permission at any time by contacting trials@kcnt1epilepsy.org.

This form is not part of a research study and is not reviewed by an Institutional Review Board (IRB). It does not collect medical information or determine eligibility. Contact information is shared only at your request and only for the study or studies you select.

Confirmation

I confirm that I am voluntarily providing this permission.

Captcha

If you are human, leave this field blank.

Your answers do not enroll you in a clinical trial, and the Foundation does not provide medical advice or decide who is eligible. With your permission, we may share your contact information with an approved trial site coordinator so they can contact you if there is a potential match.