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AzTrials

Huntington DiseasePhase 3INVEST-HDNCT07326709

A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease

A Phase 3 study testing Votoplam (blinded) in people with Huntington Disease, run by Novartis Pharmaceuticals at 117 sites in 27 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington's disease (HD) compared to the control arm. Read more about this study

Who is it for?
Ages 21 to 70 years
Conditions the study is for
Huntington Disease
What is being tested?
Votoplam (blinded)
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
770 participants
Where
117 sitesin 27 countries
Study dates
24 Mar 2026to 30 Apr 2030
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 21 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Signed informed consents must be obtained prior to participation in the study
  • Ambulatory male or female participants between 21 to 70 years of age, inclusive, on the day of Informed Consent signature
  • Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) repeat length of 40 or above. Participants must have prior genetic confirmation and known CAG repeat length obtained prior to screening.
  • Meets all of the following criteria:
  • UHDRS IS score ≥90
  • UHDRS TFC score = 13
  • UHDRS TMS score = 7-25, inclusive
  • CAP100 ≥ 70 Calculation: CAP = Age at study entry × (CAG length - 30) / 6.49

Who cannot join

  • History of gene therapy or cell transplantation or any other experimental brain surgery for the treatment of HD
  • Serologic evidence for active viral hepatitis as indicated by:
  • positive anti-HBc IgM
  • positive anti-HBc IgG confirmed by positive HBsAg and/or HBV DNA
  • positive HCV ab test confirmed by positive HCV RNA
  • Immunodeficiency diseases, including a positive human immunodeficiency virus (HIV) test result
Show all 13Show fewer
  • History or current diagnosis of ECG or cardiac abnormalities indicating significant risk of safety for participants such as:
  • Concomitant clinically significant cardiac arrhythmias, e.g., sustained ventricular tachycardia, and clinically significant second- or third-degree AV block without a pacemaker
  • History of familial long QT syndrome or known family history of Torsade de Pointes
  • Women of childbearing potential, defined as all women physiologically capable of becoming pregnant from menarche until becoming post-menopausal, unless they have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy or bilateral salpingectomy at least six weeks before taking study treatment. In the case of oophorectomy alone, the reproductive status of the woman needs to have been confirmed by follow-up hormone level assessment.
  • o WOCBP are excluded unless they are using highly effective methods of contraception (failure rate < 1% per year) while taking study treatment and for 8 months after stopping study treatment.
  • Pregnant or nursing (breastfeeding) women
  • Other protocol defined inclusion/exclusion criteria may apply

Trial locations and contacts

117 sites in 27 countries. Contact the study team or a site near you to ask about taking part.

Novartis Pharmaceuticals contact

1-888-669-6682

novartis.email@novartis.com

Novartis Pharmaceuticals contact

+41613241111

117 sites in 27 countries

United States40 sites, 40 recruiting

  • Albany Medical College

    Albany, New York, 12208

    Alicia Leader, +1 518-262-5938, downeya@amc.edu

    Era Kaur Hanspal

    Recruiting

  • Grady Memorial Hospital and Clinics

    Atlanta, Georgia, 30303

    Patricia Noren, +1 404 616 7636, pnoren@gmh.edu

    Chantale Octavia Branson

    Recruiting

  • Johns Hopkins University

    Baltimore, Maryland, 21287

    Etta Bernard, +1 412 533 2264, egoldm17@jhmi.edu

    Jee Bang

    Recruiting

  • University of Alabama Birmingham

    Birmingham, Alabama, 35233

    Candace Cromer, +1 205 934 8314, candacecromer@uabmc.edu

    Marissa Dean

    Recruiting

  • Beth Israel Deaconess Med Ctr

    Boston, Massachusetts, 02215

    Caroline Wolff, cwolff@bidmc.harvard.edu

    Samuel Arthur Frank

    Recruiting

  • UBMD Neurology

    Buffalo, New York, 14202

    Sonia Joseph, +1 716 932 6080, joseph53@buffalo.edu

    Kelly Andrzejewski

    Recruiting

  • The University of Vermont MC

    Burlington, Vermont, 05401

    Nasim Nehzat, +1 802 847 4256, nasim.nehzat@uvmhealth.org

    James Thomas Boyd

    Recruiting

  • University Of NC At Chapel Hill

    Chapel Hill, North Carolina, 27599-9500

    Alexandra Seemungal, +1 984 974 5790, Alexandra_Seemungal@med.unc.edu

    Claudia Testa

    Recruiting

  • Parkinsons-Movement Disorder Ctr

    Chicago, Illinois, 60611

    Zsa Zsa Brown, +1 312 503 5706, zsazsa.brown@northwestern.edu

    Danny Bega

    Recruiting

  • Rush University Medical Center

    Chicago, Illinois, 60612

    Tyler Svymbersky, +1 312 942 8011, Tyler_Svymbersky@rush.edu

    Deborah Hall

    Recruiting

  • Cleveland Clinic Foundation

    Cleveland, Ohio, 44195

    Margaret Andorf, +1 216 442 5232, andorfm@ccf.org

    Adam Margolius

    Recruiting

  • Henry Ford Health System

    Detroit, Michigan, 48202

    Blagica Veselinova, bveseli1@hfhs.org

    Bisena Bulica

    Recruiting

  • Duke University Health System

    Durham, North Carolina, 27710

    Lisa Gauger, lisa.gauger@duke.edu

    Burton Scott

    Recruiting

  • CenExcel Rocky Mtn Clin Research

    Englewood, Colorado, 80113

    Liza Heap, +1 303 762 6674, e.heap@cenexel.com

    Meagen Salinas

    Recruiting

  • University of Kansas Hospital

    Fairway, Kansas, 66205

    Cameron Zoraghchi, czoraghchi2@kumc.edu

    Richard Dubinsky

    Recruiting

  • U Conn Health

    Farmington, Connecticut, 06030

    Sumith Abraham Varghese, +1 860 679 3265, abrahamvarghese@uchc.edu

    Shakaib Khan

    Recruiting

  • TX Movement Disorder Spec PLLC

    Georgetown, Texas, 78628

    Wendy White, wendy@txmds.net

    Michael Soileau

    Recruiting

  • Trinity Health Michigan

    Grand Rapids, Michigan, 49503

    Melissa Brown Culbertson, melissa.brown-culbertson@trinity-health.org

    Danette C Taylor

    Recruiting

  • U of TX Health Science Ct

    Houston, Texas, 77030

    Dane Weinig, +1 832 325 7080, Dane.R.Weinig@uth.tmc.edu

    Erin Furr Stimming

    Recruiting

  • Indiana University Health

    Indianapolis, Indiana, 46202

    Jill Buck, jilmbuck@iu.edu

    Christopher James

    Recruiting

  • University of Iowa Health Care

    Iowa City, Iowa, 52242-1091

    Harika Anedal Kekinagath, +1 319 335 7555, harika-anedalkekinagath@uiowa.edu

    Annie Killoran

    Recruiting

  • University of CA San Diego

    La Jolla, California, 92037

    Krisha Bagga, +1 858 249 0574, kbagga@health.ucsd.edu

    Jody Corey-Bloom

    Recruiting

  • Univ of Arkansas for Med Sciences

    Little Rock, Arkansas, 72205

    Anja Rassmann, +1 501 686 5838, arassmann@uams.edu

    Rohit Dhall

    Recruiting

  • Uni Of Wisconsin Hosp And Clinic

    Madison, Wisconsin, 53705-3611

    Amy Nagel, +1 608 263 4505, anagel@neurology.wisc.edu

    Kathleen Shannon

    Recruiting

  • Veracity Neuroscience LLC

    Memphis, Tennessee, 38157

    Faye Ward, +1 901 237 3561, faye@veracityneuroscience.com

    Mark Steven LeDoux

    Recruiting

  • UM Department Of Neurology

    Miami, Florida, 33136

    Wilfredo Rodriguez- Somoza, 3052436732, wxr205@med.miami.edu

    Henry Moore

    Recruiting

  • Vanderbilt University Medical CenterX

    Nashville, Tennessee, 37221

    Elisa Martinez Cancino, +1 615 878 7403, Elisa.cancino@vumc.org

    Amy Brown

    Recruiting

  • Columbia University Irving Medical

    New York, New York, 10032

    Sandra Rizer, +1 212 305 1718, sz2667@cumc.columbia.edu

    Karen Marder

    Recruiting

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19107

    Gwenivere Toti, gwenivere.toti@pennmedicine.upenn.edu

    Aaron Lasker

    Recruiting

  • University Of Pittsburgh

    Pittsburgh, Pennsylvania, 15213

    Patty Conlon, +1 412 648 0776, patricia.conlon@pitt.edu

    Valerie Suski

    Recruiting

  • Oregon Health Sciences University

    Portland, Oregon, 97239

    Emily Leonard, +1 503 494 5742, leonarde@ohsu.edu

    Lauren Talman

    Recruiting

  • VCU Health Systems MCV

    Richmond, Virginia, 23298

    Brooke Foley, +1 804 828 9000, brooke.foley@vcuhealth.org

    Stephanie Bissonnette

    Recruiting

  • Universi of Rochester School of Med

    Rochester, New York, 14609

    Hannah Mesmer, +1 585 275 6412, hannah_mesmer@urmc.rochester.edu

    Frederick Marshall

    Recruiting

  • UC Davis Health System

    Sacramento, California, 95817

    Isabella Knott, igknott@health.ucdavis.edu

    Alexandra Duffy

    Recruiting

  • UCSF

    San Francisco, California, 94115

    Zach Lamson, +1 415 476 2900, zach.lamson@ucsf.edu

    Cameron Dietiker

    Recruiting

  • University of Washington Medical Center

    Seattle, Washington, 98195

    Debra Del Castillo, +1 206 616 7117, debradel@uw.edu

    Suman Jayadev

    Recruiting

  • Stanford Healthcare

    Stanford, California, 94305-5152

    Jessica Yankulova, +1 650 723 6469, jessky@stanford.edu

    Hengameh Zahed Kargaran

    Recruiting

  • University Of South Florida

    Tampa, Florida, 33612

    Ruth Cajuste, +1 813 974 6378, rsc@usf.edu

    Theresa Zesiewicz

    Recruiting

  • Georgetown University

    Washington D.C., District of Columbia, 20007

    Robin Kuprewicz, (202) 687-0100, rk1028@georgetown.edu

    Karen Anderson

    Recruiting

  • Hereditary Neurological Disease Centre

    Wichita, Kansas, 67226

    Gregory Suter, gregory@hndcentre.com

    William Mallonee

    Recruiting

Other trials in United States

China10 sites, 10 recruiting

  • Novartis Investigative Site

    Beijing, 100053

    Recruiting

  • Novartis Investigative Site

    Beijing, 100070

    Recruiting

  • The Second Xiangya Hospital of Central South University

    Changsha, Hunan, 410011

    Chunyu Wang

    Recruiting

  • West China Hospital of Sichuan Uni

    Chengdu, Sichuan, 610041

    Huifang Shang

    Recruiting

  • Novartis Investigative Site

    Fuzhou, Fujian, 350000

    Recruiting

  • Novartis Investigative Site

    Guangzhou, Guangdong, 510080

    Recruiting

  • Novartis Investigative Site

    Guangzhou, Guangdong, 510120

    Recruiting

  • The Second Affiliated Hosp Of Zhejiang Univ School Of Medicine

    Hangzhou, Zhejiang, 310002

    Zhiying Wu

    Recruiting

  • Novartis Investigative Site

    Shanghai, 200233

    Recruiting

  • Tongji Hos Affiliated Tongji Medical College of Huazhong

    Wuhan, Hubei, 430030

    Shanshan Huang

    Recruiting

Other trials in China

United Kingdom10 sites, 10 recruiting

  • Novartis Investigative Site

    Aberdeen, Grampian Region, AB25 2ZN

    Recruiting

  • Novartis Investigative Site

    Cambridge, CB2 0QQ

    Recruiting

  • Novartis Investigative Site

    Cardiff, CF14 4XW

    Recruiting

  • Novartis Investigative Site

    Exeter, Devon, EX2 5DW

    Recruiting

  • Novartis Investigative Site

    Glasgow, EH9 1LF

    Recruiting

  • Novartis Investigative Site

    Leeds, LS1 3EX

    Recruiting

  • University College Hospital

    London, NW1 2BU

    Kate Fayer, 02031087483, k.fayer@ucl.ac.uk

    Sarah Tabrizi

    Recruiting

  • Novartis Investigative Site

    London, SW17 0QT

    Recruiting

  • Novartis Investigative Site

    Oxford, OX3 9DU

    Recruiting

  • Southampton General Hospital

    Southampton, SO16 6YD

    Giorgia Cortorillo, 02381206132, Neuro-DResearchTeam@uhs.nhs.uk

    Christopher Kipps

    Recruiting

Other trials in United Kingdom

Germany5 sites, 5 recruiting

  • Novartis Investigative Site

    Aachen, 52074

    Recruiting

  • Novartis Investigative Site

    Bochum, 44791

    Recruiting

  • Novartis Investigative Site

    Münster, 48149

    Recruiting

  • Novartis Investigative Site

    Tübingen, 72076

    Recruiting

  • Novartis Investigative Site

    Ulm, 89081

    Recruiting

Other trials in Germany

Spain5 sites, 5 recruiting

  • Novartis Investigative Site

    Barcelona, 08035

    Recruiting

  • Novartis Investigative Site

    Barcelona, 08041

    Recruiting

  • Novartis Investigative Site

    Madrid, 28034

    Recruiting

  • Novartis Investigative Site

    Palma, Balearic Islands, 07120

    Recruiting

  • Novartis Investigative Site

    Seville, 41009

    Recruiting

Other trials in Spain

Argentina4 sites, 4 recruiting

  • Novartis Investigative Site

    Buenos Aires, 1060

    Recruiting

  • Instituto Argentino de Investigación Neurológica (IADIN)

    CABA, Buenos Aires, 1425

    Nelida Susana Garretto, +54-9-11-6510-5613, nsgarretto@gmail.com

    Nelida Susana Garretto

    Recruiting

  • Centro de Investigaciones Metabólicas (CINME)

    CABA, Buenos Aires, C1056ABI

    Maria Graciela Cersosimo, +54-9-11-6364-6774, mcersosimo@cinme.com.ar

    Maria Graciela Cersosimo

    Recruiting

  • Novartis Investigative Site

    Ciudad de, Buenos Aires, C1425DND

    Recruiting

Other trials in Argentina

Italy4 sites, 4 recruiting

  • Novartis Investigative Site

    Bologna, BO, 40139

    Recruiting

  • Novartis Investigative Site

    Brescia, BS, 25123

    Recruiting

  • Novartis Investigative Site

    Milan, MI, 20133

    Recruiting

  • Novartis Investigative Site

    Milan, MI, 20149

    Recruiting

Other trials in Italy

South Korea4 sites, 4 recruiting

  • Inje University Busan Paik Hospital

    Busan, 47392

    Min A. Son, +82-51-890-5781, son122141@naver.com

    Recruiting

  • Novartis Investigative Site

    Daejeon, Korea, 35015

    Recruiting

  • SMG SNU Boramae Medical Center

    Dongjak Gu, Seoul, 07061

    Yun Won Choi, 02-870-3696, 0428crc@naver.com

    Recruiting

  • Seoul National University Hospital

    Seoul, Seoul, 03080

    Seonmin Park, 82 2 2072 2193, psmin0409@snu.ac.kr

    Recruiting

Other trials in South Korea

Canada3 sites, 3 recruiting

  • Centre de recherche du CHUM

    Montreal, Quebec, H2W 1T8

    Martine Comeau, +1514 890 8000, martine.comeau.chum@ssss.gouv.qc.ca

    Alby Richard

    Recruiting

  • CUSM Montreal Neurological Institute

    Montreal, Quebec, H3A 2B4

    Yara Nasr, yara.nasr@mcgill.ca

    Anne-Louise Lafontaine

    Recruiting

  • North York General Hospital

    North York, Ontario, M2K1E1

    Clare Gibbons, +416 756 6788, clare.gibbons@nygh.on.ca

    Ragini Srinivasan, MD

    Recruiting

Other trials in Canada

France3 sites, 3 recruiting

  • Novartis Investigative Site

    Angers, France, 49933

    Recruiting

  • Novartis Investigative Site

    Marseille, 13885

    Recruiting

  • Novartis Investigative Site

    Montpellier, 34090

    Recruiting

Other trials in France

Japan3 sites, 3 recruiting

  • Novartis Investigative Site

    Bunkyo Ku, Tokyo, 1138655

    Recruiting

  • Novartis Investigative Site

    Kashihara, Nara, 6348522

    Recruiting

  • Novartis Investigative Site

    Sagamihara, Kanagawa, 2520392

    Recruiting

Other trials in Japan

Taiwan3 sites, 3 recruiting

  • Novartis Investigative Site

    Changhua, 50006

    Recruiting

  • Novartis Investigative Site

    Taipei, 11217

    Recruiting

  • Novartis Investigative Site

    Taoyuan, 33305

    Recruiting

Other trials in Taiwan

Australia2 sites, 2 recruiting

  • Novartis Investigative Site

    Caulfield South, Victoria, 3162

    Recruiting

  • Novartis Investigative Site

    Westmead, New South Wales, 2145

    Recruiting

Other trials in Australia

Bulgaria2 sites, 2 recruiting

  • Novartis Investigative Site

    Sofia, 1113

    Recruiting

  • Novartis Investigative Site

    Sofia, 1431

    Recruiting

Other trials in Bulgaria

Czechia2 sites, 2 recruiting

  • Novartis Investigative Site

    Brno, 602 00

    Recruiting

  • Novartis Investigative Site

    Prague, 128 08

    Recruiting

Other trials in Czechia

Hungary2 sites, 2 recruiting

  • Novartis Investigative Site

    Budapest, 1083

    Recruiting

  • Novartis Investigative Site

    Szeged, 6725

    Recruiting

Other trials in Hungary

Netherlands2 sites, 2 recruiting

  • Novartis Investigative Site

    Leiden, South Holland, 2333 ZA

    Recruiting

  • Novartis Investigative Site

    Maastricht, Limburg, 6229 HX

    Recruiting

Other trials in Netherlands

Portugal2 sites, 2 recruiting

  • Novartis Investigative Site

    Lisbon, 1649-035

    Recruiting

  • Novartis Investigative Site

    Porto, 4099-001

    Recruiting

Other trials in Portugal

Romania2 sites, 2 recruiting

  • Novartis Investigative Site

    Bucharest, 040215

    Recruiting

  • Novartis Investigative Site

    Târgu Mureş, 540136

    Recruiting

Other trials in Romania

Switzerland2 sites, 2 recruiting

  • Novartis Investigative Site

    Basel, 4031

    Recruiting

  • Novartis Investigative Site

    Muri bei Bern, 3073

    Recruiting

Other trials in Switzerland

Belgium1 site, 1 recruiting

  • Novartis Investigative Site

    Leuven, Vlaams Brabant, 3000

    Recruiting

Other trials in Belgium

Colombia1 site, 1 recruiting

  • Novartis Investigative Site

    Medellín, 050001

    Recruiting

Other trials in Colombia

Denmark1 site, 1 recruiting

  • Novartis Investigative Site

    Copenhagen, 2100

    Recruiting

Other trials in Denmark

Israel1 site, 1 recruiting

  • Tel Aviv Sourasky Med Ctr Ichilov

    Tel Aviv, 6423906

    Tanya Gurevich, tanyag@tlvmc.gov.il

    Tanya Gurevich

    Recruiting

Other trials in Israel

Poland1 site, 1 recruiting

  • Novartis Investigative Site

    Krakow, POL, 31-505

    Recruiting

Other trials in Poland

Saudi Arabia1 site, 1 recruiting

  • Novartis Investigative Site

    Riyadh, 11211

    Recruiting

Other trials in Saudi Arabia

Slovakia1 site, 1 recruiting

  • Novartis Investigative Site

    Košice, 041 90

    Recruiting

Other trials in Slovakia

About this study

Official title: A Randomized, Placebo-controlled, Double-blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease

The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington's disease (HD) compared to the control arm.

HTT227 - current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is Novartis code under Novartis sponsorship.

Read the detailed description

This study will have a variable double-blind treatment duration of up to 36 months. As part of the study design, not every participant will complete 36 months of treatment.

The study consists of 3 periods:

  • Screening Period: A period of up to 42-days to assess participants eligibility
  • Double-blind Treatment Period: This period will have variable individual treatment duration, up to 36 months. The double-blind treatment period concludes when ≥50% patients complete Month 36. The maximum treatment duration for an individual participant is 36 months.
  • Safety Follow-up Period: A period consisting of one safety follow-up visit, conducted on site or by phone call, for all participants not continuing treatment in the separate open-label extension study or discontinuing early. The visit/phone call will take place 30 days after End of Study (EOS)
Being tested
Votoplam (blinded), Placebo
Keywords
HD, INVEST-HD, HTT227, VOTOPLAM, HUNTINGTON DISEASE, UHDRS, mHTT, NfL
Medical subject headings
Huntington Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Votoplam (blinded)Drug

    Also called HTT227

    Votoplam (blinded) active treatment

  • PlaceboDrug

    Placebo

Study groups in NCT07326709
Study groupRoleWhat they receive
VotoplamExperimentalVotoplam (blinded) taken orally, randomized in a 3:2 ratio (Votoplam: Placebo)
Receives: Drug: Votoplam (blinded)
PlaceboPlacebo comparatorPlacebo (blinded) taken orally, randomized in a 3:2 ratio (Votoplam: Placebo)
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change from Baseline in cUHDRS score

    The Composite Unified Huntingtons Disease Ratings Scale (cUHDRS) is an improved composite measure to assess multi-domain clinical progression in HD. The cUHDRS is a combined weighted score of measures of motor function (TMS), cognition (SDMT and SWRT) and overall functional capacity (TFC):

    cUHDRS = [(TFC-10.4)/1.9 - (TMS-29.7)/14.9 + (SDMT-28.4)/11.3 + (SWRT - 66.1)/20.1] + 10.

    In conjunction with one another, these measures provide a comprehensive, sensitive and specific tool for monitoring disease progression, with lower scores indicating more severe disease. cUHDRS score range from -8 to 25.

    Measured: Baseline, Month 36

Secondary outcomes (9)

  1. Change from Baseline in UHDRS-TFC

    The UHDRS-Total Functional Capacity (TFC) focuses on the Investigator's assessment of the participant's capacity to perform a range of activities including occupation, finances, self-care, domestic chores and activities of daily living. The responses are derived from interview with the participant and/or companion, if applicable. Scores range from 0 to 13, with higher scores representing better functioning.

    Measured: Baseline, Month 36

  2. Change from Baseline in UHDRS-IS

    The UHDRS-Independence Scale (IS) measures a patient's overall level of functional independence in activities of daily living. It provides a single score ranging from 10 (total dependence/bedbound) to 100 (no special care needed) in intervals of 10, with higher scores indicating better functioning. Scores can also be assigned in increments of 5 for intermediate levels of function.

    Measured: Baseline, Month 36

  3. The time to decline in TFC score by at least one or IS score by at least 10

    The UHDRS-Total Functional Capacity (TFC) focuses on the Investigator's assessment of the participant's capacity to perform a range of activities including occupation, finances, self-care, domestic chores and activities of daily living. The responses are derived from interview with the participant and/or companion, if applicable. Scores range from 0 to 13, with higher scores representing better functioning.

    The UHDRS-Independence Scale (IS) measures a patient's overall level of functional independence in activities of daily living. It provides a single score ranging from 10 (total dependence/bedbound) to 100 (no special care needed) in intervals of 10, with higher scores indicating better functioning. Scores can also be assigned in increments of 5 for intermediate levels of function.

    Measured: Baseline to end of treatment up to 36 months

  4. Change from Baseline in UHDRS-TMS

    The UHDRS-Total Motor Score (TMS) is the cumulative sum of the individual motor ratings obtained during the administration of the motor assessment portion of the UHDRS. It includes items related to eye movements, speech, limb movements (including hand taps and pronation-supination), dystonia, chorea, and gait/balance. Scores range from 0 to 124, with higher scores indicating greater motor impairment.

    Measured: Baseline, Month 36

  5. Change from Baseline in SDMT

    The Symbol Digit Modality Test (SDMT) is used to assess attention, working memory, psychomotor speed and visual perceptual processing. Participants are presented with a translation key of specific numbers paired with unique abstract symbols. Below the translation key is an array of symbols paired with empty spaces, the participant's task is to match the number for each symbol as quickly as possible in a given time frame. Scores are based on number of correctly paired items and range from 0 to 110, with higher scores representing better performance.

    Measured: Baseline, Month 36

  6. Change from Baseline in SWRT

    The Stroop Word Reading Test (SWRT) is a measure of processing and psychomotor speed. Participants are presented with a list or words (color names) printed in black ink and asked to read aloud as many words as possible within a set timeframe. Scores are based on the number of words read correctly and range from 0 to 60, with higher scores representing better performance.

    Measured: Baseline, Month 36

  7. Percent change from Baseline in blood mHTT protein

    The mutant huntington protein (mHTT) which is detectable both in blood and cerebrospinal fluid (CSF), represents a reliable biomarker for HD due to its direct involvement in disease pathology.

    Blood mHTT is a direct product of the pathogenic HTT gene mutation and represents a proximal marker of Huntington's disease (HD) biology.

    Measured: Baseline to steady state and up to 36 months in blood mHTT protein

  8. Change from Baseline in serum NfL

    Serum Neurofilament light chain NfL is a component of the neuronal cytoskeleton, released into CSF and blood after neuro-axonal damage, serving as a marker of neurodegeneration and disease progression in HD.

    Measured: Baseline, Month 36

  9. Incidence and severity of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and TEAEs leading to participant withdrawal

    Number of participants with AEs and SAEs, including notable findings on physical examination, changes in vital signs, laboratory parameters, ECG, etc.

    Measured: Baseline to up to 36 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Jan 2026
  2. Study start24 Mar 2026
  3. Primary completion2 Apr 2030 (estimated)
  4. Study completion30 Apr 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
770 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07326709
Sponsor's protocol ID
CHTT227A12301
Acronym
INVEST-HD
Status last verified
Sep 2026
First submitted
7 Jan 2026
Last updated
22 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Votoplam (blinded) in people with Huntington Disease, run by Novartis Pharmaceuticals at 117 sites in 27 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 22 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 117 sites in United States, China, United Kingdom, Germany, Spain and 22 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Novartis Pharmaceuticals (1-888-669-6682, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change from Baseline in cUHDRS score (time frame: Baseline, Month 36). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07326709.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Sep 2026.

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