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Hope for Huntington's: experimental drug targets root cause

NCT ID NCT05686551

Disease control Sponsor: Hoffmann-La Roche 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Aug 11, 2026 · Updated 4 times

Summary

This phase 2 trial tests an experimental drug called tominersen in 301 people with early-stage Huntington's disease. The drug aims to lower levels of the harmful mutant protein that causes the disease. Researchers will compare tominersen against a placebo to see if it can slow down symptoms and check for safety issues like brain inflammation.

What this could mean

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

Active substance
tominersen
What this could lead to
If it works, this could slow or delay the progression of Huntington's disease, giving people more years of independence.
What could go wrong
This is a mid-stage trial with only 301 participants. The drug may not show enough benefit or could cause side effects like brain swelling or inflammation.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

301 people

The number who actually took part.

Started

Feb 2023

Expected to finish

Oct 2026

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

25 to 50 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

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.

Inclusion Criteria: DB Period: * HD gene expansion mutation carrier status with a cytosine-adenine-guanine-age product (CAP) score of 400-500 inclusive * Either: * Prodromal HD (defined as Diagnostic Confidence Level (DCL) 2 to 3, Independence Scale (IS) ≥70, and TFC ≥8); Or * Early manifest HD (defined as DCL 4, IS ≥70, and TFC ≥8) * Total body weight \> 40 kilograms (kg) and a body mass index (BMI) within the range of 18-32 kilograms per meter square (kg/m\^2) * Study companion OLE Period: * Participants must have completed the DB treatment period * Participants must remain in the DB Safety follow-up period until OLE period starts Exclusion Criteria: DB Period: * Current or previous use of an antisense oligonucleotide (ASO) (including small interfering ribonucleic acid \[RNA\]) or any HTT lowering therapy (including tominersen) * Anti-platelet or anticoagulant therapy within 14 days prior to screening or anticipated use during the study, including, but not limited to, aspirin (unless ≤ 81 milligrams per day \[mg/day\]), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban, apixaban, and heparin * History of gene therapy, cell transplantation, or brain surgery * Hydrocephalus * Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 5 months after the final dose of study drug * History of attempted suicide or suicidal ideation with plan (i.e., active suicidal ideation) that required hospital visit and/or change in level of care within 12 months prior to screening OLE Period: * Early discontinuation from the DB treatment and the safety follow-up (SFU) periods * Pregnant or breastfeeding, or with the intention of becoming pregnant during the study or within the timeframe in which contraception is required * Current or previous use of an ASO other than tominersen (including small interfering RNA) or any other HTT-lowering therapy * Hydrocephalus * Received any active investigational treatment other than tominersen during or since completion of the DB treatment period Key inclusions/exclusion criteria are listed here. Other protocol-defined I/E criteria may apply.

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Conditions

The condition(s) this trial relates to.

Huntington disease

As listed by the trial registrant

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

Huntington disease

Contacts and locations

Locations

  • Addenbrookes Hospital

    Cambridge, CB2 0QQ, United Kingdom

  • Azienda Ospedaliera Sant'Andrea

    Rome, Lazio, 00189, Italy

  • Barrow Neurological Institute

    Phoenix, Arizona, 85013, United States

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215, United States

  • CHU Angers, Batiement Larrey 2, Neurologie

    Angers, 49933, France

  • CHU Strasbourg Hpital Hautepierre

    Strasbourg, 67098, France

  • CHU de la Timone - Hopital d Adultes

    Marseille, 13005, France

  • CHU toulouse - Hôpital Purpan

    Toulouse, 31059, France

  • CINME

    Buenos Aires, C1056ABI, Argentina

  • CNS - Campus Neurológico

    Torres Vedras, 2560-280, Portugal

  • CenExel Rocky Mountain Clinical Research, LLC

    Englewood, Colorado, 80113, United States

  • Chapel Allerton Hospital

    Leeds, LS7 4SA, United Kingdom

  • Charité - Universitätsmed. Berlin, Klinik für Psychiatrie und Psychotherapie

    Berlin, 10117, Germany

  • Dent Neurological Institute

    Amherst, New York, 14226, United States

  • EvergreenHealth Investigational Drug Services

    Kirkland, Washington, 98034, United States

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, Lombardy, 20133, Italy

  • Georgetown University

    Washington D.C., District of Columbia, 20007, United States

  • German Center for Neurodegenerative Diseases (DZNE)

    Bonn, 53127, Germany

  • Groupe Hospitalier Pellegrin

    Bordeaux, 33076, France

  • Henry Ford Hospital

    Detroit, Michigan, 48202, United States

  • Hopital Gui de Chauliac

    Montpellier, 34295, France

  • Hopital Henri Mondor

    Créteil, 94010, France

  • Hopital Roger Salengro Service de Neurologie

    Lille, DUMMY_VALUE, France

  • Hospital Britanico de Buenos Aires

    Ciudad Autonoma Buenos Aires, C1284AEB, Argentina

  • Hospital Ramon y Cajal

    Madrid, 28034, Spain

  • Hospital Ramos Mejía

    CABA, C1221ADC, Argentina

  • Hospital Universitario Virgen Macarena

    Seville, 41009, Spain

  • Hospital Universitario de Badajoz

    Badajoz, 06080, Spain

  • Hospital Universitario de Burgos. Servicio de Neurología

    Burgos, 09006, Spain

  • Hospital Universitario la Fe

    Valencia, 46026, Spain

  • Hospital de Cruces

    Barakaldo, Vizcaya, 48903, Spain

  • Hospital de Santa Maria

    Lisbon, 1649-035, Portugal

  • Hospital de la Santa Creu i Sant Pau

    Barcelona, 08041, Spain

  • INEBA

    Capital Federal, C1192AAX, Argentina

  • Inland Northwest Research

    Spokane, Washington, 99202, United States

  • John Hopkins University School of Medicine

    Baltimore, Maryland, 21287, United States

  • John Radcliffe Hospital

    Oxford, OX3 9DU, United Kingdom

  • Krakowska Akademia Neurologii Sp z o.o. Centrum Neurologii K

    Krakow, 31-505, Poland

  • Monash Medical Centre

    Clayton, Victoria, 3168, Australia

  • Montreal Neurological Inst

    Montreal, Quebec, H3A 2B4, Canada

  • Neurozentrum Siloah

    Gümligen, 3073, Switzerland

  • New Zealand Brain Research Institute

    Christchurch, 8011, New Zealand

  • Northwestern University

    Chicago, Illinois, 60611, United States

  • Ospedale Bellaria

    Bologna, Emilia-Romagna, 40139, Italy

  • Perron Institute for Neurological and Translational Science

    Nedlands, Western Australia, 6009, Australia

  • Rigshospitalet, Hukommelsesklinikken

    København Ø, 2100, Denmark

  • Royal Melbourne Hospital

    Parkville, Victoria, 3050, Australia

  • Southampton University Hospitals NHS Trust

    Southampton, SO16 6YD, United Kingdom

  • St. Josef-Hospital, Neurologische Klinik der Ruhr-Uni

    Bochum, 44791, Germany

  • Szpital Sw. Wojciecha

    Gdansk, 80-462, Poland

  • The University of Texas Health Science Center at Houston

    Houston, Texas, 77030, United States

  • UCL Hospital NHS Trust

    London, NW1 2PG, United Kingdom

  • Uab Medicine

    Birmingham, Alabama, 35294, United States

  • Uniklinik RWTH Aachen

    Aachen, 52074, Germany

  • Uniklinik fuer Neurologie, Medizinische Universitaet Innsbruck

    Innsbruck, 6020, Austria

  • University Hospitals Birmingham NHS Foundation Trust

    Birmingham, B15 2TH, United Kingdom

  • University of Alberta Hospital

    Edmonton, Alberta, T6G 2G3, Canada

  • University of California Davis Medical System

    Sacramento, California, 95817, United States

  • University of Florida

    Gainesville, Florida, 32608, United States

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242, United States

  • University of Pittsburgh

    Pittsburgh, Pennsylvania, 15213, United States

  • University of South Florida

    Tampa, Florida, 33612, United States

  • Universitätsklinikum Erlangen, Abteilung Molekulare Neurologie

    Erlangen, 91054, Germany

  • Universitätsklinikum Schleswig-Holstein / Campus Lübeck

    Lübeck, 23538, Germany

  • Universitätsklinikum Ulm

    Ulm, 89081, Germany

  • Universitätsspital Basel

    Basel, 4031, Switzerland

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37212, United States

  • Waikato Hospital

    Hamilton, 3240, New Zealand

  • Wojskowy Instytut Medycyny Lotniczej

    Warsaw, 01-755, Poland

  • kbo - Isar-Amper-Klinikum Taufkirchen

    Taufkirchen, 84416, Germany

More trials for these conditions

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