Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New hope for rare dementia: experimental drug DNL593 enters human trials

NCT ID NCT05262023

Disease control Sponsor: Denali Therapeutics Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests an experimental drug called DNL593 in healthy volunteers and people with frontotemporal dementia (FTD), a rare brain disease that causes personality changes and memory loss. The trial aims to check if the drug is safe, how the body processes it, and whether it might help. It includes a small number of participants and an optional long-term follow-up.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
DNL593
What this could lead to
If successful, this could point toward a treatment that slows or manages frontotemporal dementia, a currently untreatable disease.
What could go wrong
This is an early-phase trial with only 85 participants, so results may not confirm effectiveness. The drug may cause side effects or fail to show benefit.
Why investors are watching

Denali Therapeutics is testing DNL593, an experimental drug for frontotemporal dementia (FTD), a brain disease with no approved treatment. The trial first checks safety in healthy volunteers, then measures whether the drug is safe and shows signs of working in 85 people with FTD over 25 weeks. For a small company like Denali, this readout is a key test of whether DNL593 deserves further development, and a positive signal could support its pipeline value.

If it works: If DNL593 proves safe and shows meaningful effects in people with FTD, Denali could advance the drug to later-stage trials. That result would strengthen the company's position in a disease area with few options, which could attract partnership interest or support further investment in the program.

If it fails: If the drug fails to show a clear benefit or causes safety problems, Denali may need to stop or redesign the program, wasting years of research and money. Trials in brain diseases often fail, so a negative result is a real possibility that could hurt the company's prospects.

AI-written from the trial record. Speculative, and not investment advice.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

85 people

The number who actually took part.

Started

Feb 2022

Expected to finish

Nov 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 80 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: Part A: * Women of non-childbearing potential (surgically sterilized or post menopausal) or men, aged ≥18 to ≤ 55 years * BMI of ≥ 18 to ≤ 32 kg/m² * When engaging in sex with a woman of child bearing potential, two forms of birth control are required Part B: * Women of non-childbearing potential (surgically sterilized or post menopausal) or men, aged ≥18 to ≤ 80 years. Women who are of childbearing potential but on highly effective, low user dependent contraceptive methods will be allowed. * BMI of ≥ 18 to ≤ 32 kg/m² * Have a Clinical Dementia Rating® plus National Alzheimer's Coordinating Center frontotemporal lobar degeneration global score ≥ 0.5 * Have confirmed granulin (GRN) mutation via genetic testing or historical records available for review by investigator * When engaging in sex with a woman of child bearing potential, both the male participant and his female partner must use highly effective contraception Part C: * All participants who completed Part B of this trial are eligible for an 18-month OLE if the participant has no unresolved clinically significant TEAEs, where continued dosing may represent a risk to participant safety. Key Exclusion Criteria: * Have any history of clinically significant neurologic, psychiatric, endocrine, pulmonary, cardiovascular, gastrointestinal, hepatic, pancreatic, renal, metabolic, hematologic, immunologic, or allergic disease, or other major disorders * Have a history of malignancy, except fully resected basal cell carcinoma or other malignancies at low risk of recurrence * Have a clinically significant history of stroke, cognitive impairment due to causes other than FTD, seizure within 5 years of screening, or head trauma with loss of consciousness within 2 years of screening * Have a positive serum pregnancy test or are currently lactating or breastfeeding

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Frontotemporal dementia are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

frontotemporal dementia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Frontotemporal dementia

Contacts and locations

Locations

  • ASST degli Spedali Civili di Brescia

    Brescia, Italy

  • Azienda Ospedaliera Cardinale G Panico

    Tricase, Italy

  • Azienda Ospedaliera Universitaria Careggi

    Florence, Italy

  • CHU Rouen

    Rouen, France

  • CHU Toulouse

    Toulouse, France

  • CHU de Nantes

    Nantes, France

  • Campus Neurológico Sénior

    Torres Vedras, Portugal

  • Erasmus University Medical Center

    Rotterdam, Netherlands

  • Faculdade de Medicina Da Universidade de São Paulo

    São Paulo, Brazil

  • Fakultni nemocnice v Motole

    Prague, Czechia

  • Grupo de Neurosicencias de la Universidad de Antioquia

    Medellín, Colombia

  • Hospital Clinic de Barcelona

    Barcelona, Barcelona, 8036, Spain

  • Hospital Universitario San Ignacio

    Bogotá, Colombia

  • Hospital Universitario Virgen del Rocio

    Seville, Sevilla, 41013, Spain

  • Hospital Universitario de Donostia

    Donostia / San Sebastian, Guipúzcoa, 20014, Spain

  • Hospital de Braga

    Braga, Portugal

  • Hospital of the University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • IRCCS Istituto Auxologico Italiano

    Milan, Italy

  • Istanbul University Istanbul Medical Faculty

    Istanbul, Turkey (Türkiye)

  • John Hopkins University

    Baltimore, Maryland, 21218, United States

  • L2IP - Instituto de Pesquisas Clinicas LTDA

    Brasília, Brazil

  • Ondokuz Mayis University Hospital

    Samsun, Turkey (Türkiye)

  • Simbec Orion

    Merthyr Tydfil, Wales, CF48 4DR, United Kingdom

  • UZ Leuven

    Leuven, Belgium

  • University of Antwerp

    Antwerp, Belgium

  • University of California San Francisco

    San Francisco, California, 94143, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.