Huntington DiseasePhase 2NCT05686551
GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease
A Phase 2 study testing Tominersen in people with Huntington Disease, run by Hoffmann-La Roche at 70 sites in 15 countries.
Trial summary
This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease (HD). Read more about this study
- Who is it for?
- Ages 25 to 50 years
- Conditions the study is for
- Huntington Disease
- What is being tested?
- Tominersen
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 301 participants
- Where
- 70 sitesin 15 countries
- Study dates
- 3 Feb 2023to 31 Oct 2026
- Phase
- Phase 2
- 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 25 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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)
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- 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
Who cannot join
- 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
Show all 14Show fewer
- 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.
Trial locations and contacts
70 sites in 15 countries.
70 sites in 15 countries
United States18 sites
Dent Neurological Institute
Amherst, New York, 14226
John Hopkins University School of Medicine
Baltimore, Maryland, 21287
Uab Medicine
Birmingham, Alabama, 35294
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Northwestern University
Chicago, Illinois, 60611
Henry Ford Hospital
Detroit, Michigan, 48202
CenExel Rocky Mountain Clinical Research, LLC
Englewood, Colorado, 80113
University of Florida
Gainesville, Florida, 32608
The University of Texas Health Science Center at Houston
Houston, Texas, 77030
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
EvergreenHealth Investigational Drug Services
Kirkland, Washington, 98034
Vanderbilt University Medical Center
Nashville, Tennessee, 37212
Barrow Neurological Institute
Phoenix, Arizona, 85013
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
University of California Davis Medical System
Sacramento, California, 95817
Inland Northwest Research
Spokane, Washington, 99202
University of South Florida
Tampa, Florida, 33612
Georgetown University
Washington D.C., District of Columbia, 20007
France8 sites
CHU Angers, Batiement Larrey 2, Neurologie
Angers, 49933
Groupe Hospitalier Pellegrin
Bordeaux, 33076
Hopital Henri Mondor
Créteil, 94010
Hopital Roger Salengro Service de Neurologie
Lille, DUMMY_VALUE
CHU de la Timone - Hopital d Adultes
Marseille, 13005
Hopital Gui de Chauliac
Montpellier, 34295
CHU Strasbourg Hpital Hautepierre
Strasbourg, 67098
CHU toulouse - Hôpital Purpan
Toulouse, 31059
Germany8 sites
Uniklinik RWTH Aachen
Aachen, 52074
Charité - Universitätsmed. Berlin, Klinik für Psychiatrie und Psychotherapie
Berlin, 10117
St. Josef-Hospital, Neurologische Klinik der Ruhr-Uni
Bochum, 44791
German Center for Neurodegenerative Diseases (DZNE)
Bonn, 53127
Universitätsklinikum Erlangen, Abteilung Molekulare Neurologie
Erlangen, 91054
Universitätsklinikum Schleswig-Holstein / Campus Lübeck
Lübeck, 23538
kbo - Isar-Amper-Klinikum Taufkirchen
Taufkirchen, 84416
Universitätsklinikum Ulm
Ulm, 89081
Spain7 sites
Hospital Universitario de Badajoz
Badajoz, 06080
Hospital de Cruces
Barakaldo, Vizcaya, 48903
Hospital de la Santa Creu i Sant Pau
Barcelona, 08041
Hospital Universitario de Burgos. Servicio de Neurología
Burgos, 09006
Hospital Ramon y Cajal
Madrid, 28034
Hospital Universitario Virgen Macarena
Seville, 41009
Hospital Universitario la Fe
Valencia, 46026
United Kingdom6 sites
University Hospitals Birmingham NHS Foundation Trust
Birmingham, B15 2TH
Addenbrookes Hospital
Cambridge, CB2 0QQ
Chapel Allerton Hospital
Leeds, LS7 4SA
UCL Hospital NHS Trust
London, NW1 2PG
John Radcliffe Hospital
Oxford, OX3 9DU
Southampton University Hospitals NHS Trust
Southampton, SO16 6YD
Argentina4 sites
CINME
Buenos Aires, C1056ABI
Hospital Ramos Mejía
CABA, C1221ADC
INEBA
Capital Federal, C1192AAX
Hospital Britanico de Buenos Aires
Ciudad Autonoma Buenos Aires, C1284AEB
Australia3 sites
Monash Medical Centre
Clayton, Victoria, 3168
Perron Institute for Neurological and Translational Science
Nedlands, Western Australia, 6009
Royal Melbourne Hospital
Parkville, Victoria, 3050
Italy3 sites
Ospedale Bellaria
Bologna, Emilia-Romagna, 40139
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Lombardy, 20133
Azienda Ospedaliera Sant'Andrea
Rome, Lazio, 00189
Poland3 sites
Szpital Sw. Wojciecha
Gdansk, 80-462
Krakowska Akademia Neurologii Sp z o.o. Centrum Neurologii K
Krakow, 31-505
Wojskowy Instytut Medycyny Lotniczej
Warsaw, 01-755
Canada2 sites
University of Alberta Hospital
Edmonton, Alberta, T6G 2G3
Montreal Neurological Inst
Montreal, Quebec, H3A 2B4
New Zealand2 sites
New Zealand Brain Research Institute
Christchurch, 8011
Waikato Hospital
Hamilton, 3240
Portugal2 sites
Hospital de Santa Maria
Lisbon, 1649-035
CNS - Campus Neurológico
Torres Vedras, 2560-280
Switzerland2 sites
Universitätsspital Basel
Basel, 4031
Neurozentrum Siloah
Gümligen, 3073
Austria1 site
Uniklinik fuer Neurologie, Medizinische Universitaet Innsbruck
Innsbruck, 6020
About this study
Official title: A Phase II, Randomized, Double-blind, Placebo-controlled, Dose-finding Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen in Individuals With Prodromal and Early Manifest Huntington's Disease
This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease (HD).
- Conditions
- Huntington Disease
- Being tested
- Tominersen, Placebo
- Keywords
- Prodromal and Early Manifest Huntington's Disease
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TominersenDrug
Also called RO7234292;, RG6042
Tominersen will be administered at the dose and schedule specified in the protocol.
PlaceboDrug
Matching placebo administered IT, Q16W during the DB period.
| Study group | Role | What they receive |
|---|---|---|
| Tominersen 60 milligrams (mg) | Experimental | 60 mg tominersen administered intrathecally (IT) every 16 weeks (Q16W). Tominersen will be administered in the DB period and the OLE period. Receives: Drug: Tominersen |
| Placebo | Placebo comparator | Placebo will be administered IT, Q16W in the DB period. Receives: Drug: Placebo |
| Tominersen 100 mg | Experimental | 100 mg tominersen administered IT, Q16W. Tominersen will be administered in the DB period and the OLE period. Receives: Drug: Tominersen |
What is being measured
The primary outcome is the main question the study is designed to answer.
DB Period: Incidence and Severity of Adverse Events (AEs), With Severity Determined According to the AE Severity Grading Scale
Measured: Up to approximately 36 months
DB Period: Change From Baseline in Clinical Laboratory Results - Cerebrospinal Fluid (CSF) White Blood Cell (WBC)
Measured: Baseline visit (Day 1), and Months 4, 8, 9, 12, 16
DB Period: Change From Baseline in Clinical Laboratory Results - CSF Protein
Measured: Baseline visit (Day 1), and Months 4, 8, 9, 12, 16
DB Period: Change From Baseline in Structural Magnetic Resonance Imaging (MRI) Assessing Any New Abnormalities Including Radiographic Features Consistent With Hydrocephalus and Other Relevant MRI Safety Findings
Measured: Baseline, Months 4, 8, 12, 16 and up to approximately 36 months
DB Period: Percentage Change From Baseline in Geometric Means of CSF Mutant Huntingtin (mHTT) Protein Levels at Month 9
Measured: Baseline, Month 9
DB Period: Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores (non-U.S. Sites) at 16 Months
Change in scores on the scale.
Measured: Baseline to 16 months
DB Period: Change From Baseline in Total Functional Capacity (TFC) Scores (U.S. Sites) at 16 Months
Change in scores on the scale.
Measured: Baseline to 16 months
OLE Period: Incidence and Severity of AEs, With Severity Determined According to the AE Severity Grading Scale
Measured: Up to approximately 29 months
OLE Period: Change Over Time in Clinical Laboratory Results - CSF WBC
Measured: Up to approximately 24 months
OLE Period: Change Over Time in Clinical Laboratory Results - CSF Protein
Measured: Up to approximately 24 months
OLE Period: Change From Baseline in Structural MRI Assessing Any New Abnormalities, Including Radiographic Features Consistent With Hydrocephalus and Other Relevant MRI Safety Findings
Measured: Up to approximately 29 months
Secondary outcomes (18)
DB Period: Change From Baseline in Montreal Cognitive Assessment (MoCA) Scores
Measured: Baseline, Months 4, 8, 12, 16 and up to approximately 36 months
DB Period: Percentage of Participants With Suicidal Ideation or Behavior (I/B), as Assessed by C-SSRS Score at Each Visit, Including Detailed Focus on Any Individual Cases Identified as Having Severe I/B During the Study Conduct
C-SSRS=Columbia-suicide Severity Rating Scale
Measured: Up to approximately 36 months
DB Period: Change From Baseline at 16 Months in TFC (non-U.S. Sites) Scores
Measured: Baseline to 16 months
DB Period: Change From Baseline at 16 Months in cUHDRS (U.S. Sites) Scores
Measured: Baseline to 16 months
DB Period: Change From Baseline at 16 Months in Symbol Digit Modalities Test (SDMT) Scores
Measured: Baseline to 16 months
DB Period: Change From Baseline at 16 Months in Stroop Word Reading (SWR) Score
Measured: Baseline to 16 months
DB Period: Change From Baseline at 16 Months in Total Motor Score (TMS)
Measured: Baseline to 16 months
DB Period: Change From Baseline in CSF Neurofilament Light Chain (NfL) Levels at 16 Months
Measured: Baseline to 16 months
DB Period: Incidence of Anti-drug Antibodies (ADAs) at Specified Timepoints Relative to the Prevalence of ADAs at Baseline
Measured: Baseline up to approximately 36 months
DB Period: Titers Determined if ADAs are Identified
Measured: Baseline up to approximately 36 months
OLE Period: Change Over Time in TFC Score
Measured: Up to approximately 29 months
OLE Period: Change Over Time in cUHDRS Score
Measured: Up to approximately 29 months
OLE Period: Change Over Time in SDMT Score
Measured: Up to approximately 29 months
OLE Period: Change Over Time in TMS
Measured: Up to approximately 29 months
OLE Period: Change Over Time in SWR Score
Measured: Up to approximately 29 months
OLE Period: Change Over Time in MoCA Score
Measured: Up to approximately 29 months
OLE Period: Percentage of Participants With Suicidal I/B, as Assessed by C-SSRS Score at Each Visit, Including Detailed Focus on Any Individual Cases Identified as Having Severe I/B During the Study Conduct
Measured: Up to approximately 29 months
OLE Period: Incidence of ADAs at Specified Timepoints
Measured: Up to approximately 29 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jan 2023
- Study start3 Feb 2023
- Primary completion14 May 2026
- Study completion31 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 301 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05686551
- Sponsor's protocol ID
- BN42489
- Other IDs
- Other, 2022-001991-32, 2023-503928-10-00, 2023
- Status last verified
- Aug 2026
- First submitted
- 16 Dec 2022
- Last updated
- 10 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing
Publications
McColgan P, Thobhani A, Boak L, Schobel SA, Nicotra A, Palermo G, Trundell D, Zhou J, Schlegel V, Sanwald Ducray P, Hawellek DJ, Dorn J, Simillion C, Lindemann M, Wheelock V, Durr A, Anderson KE, Long JD, Wild EJ, Landwehrmeyer GB, Leavitt BR, Tabrizi SJ, Doody R; GENERATION HD1 Investigators. Tominersen in Adults with Manifest Huntington's Disease. N Engl J Med. 2023 Dec 7;389(23):2203-2205. doi: 10.1056/NEJMc2300400. No abstract available.
PubMed 38055260
Study at a glance
From the registry record, last updated by the sponsor on 10 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Randomized, Double-blind, Placebo-controlled, Dose-finding Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen in Individuals With Prodromal and Early Manifest Huntington's Disease
- ClinicalTrials.gov ID
- NCT05686551
- Acronym / sponsor ID
- BN42489
- Conditions
- Huntington Disease
- Keywords
- Prodromal and Early Manifest Huntington's Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 301 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 3 Feb 2023 (actual)
- Primary completion
- 14 May 2026 (actual)
- Study completion
- 31 Oct 2026 (estimated)
- First posted
- 17 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease (HD).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Tominersen 60 milligrams (mg) | Experimental | Drug: Tominersen60 mg tominersen administered intrathecally (IT) every 16 weeks (Q16W). Tominersen will be administered in the DB period and the OLE period. |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo will be administered IT, Q16W in the DB period. |
| Tominersen 100 mg | Experimental | Drug: Tominersen100 mg tominersen administered IT, Q16W. Tominersen will be administered in the DB period and the OLE period. |
Who may qualify
- Ages
- 25 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- HD gene expansion mutation carrier status with a cytosine-adenine-guanine-age product (CAP) score of 400-500 inclusive
- Either:
- 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
- Participants must have completed the DB treatment period
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TominersenAlso: RO7234292;, RG6042Tominersen will be administered at the dose and schedule specified in the protocol. | Drug | Tominersen 100 mg, Tominersen 60 milligrams (mg) |
| PlaceboMatching placebo administered IT, Q16W during the DB period. | Drug | Placebo |
Primary outcome
DB Period: Incidence and Severity of Adverse Events (AEs), With Severity Determined According to the AE Severity Grading Scale
Time frame: Up to approximately 36 months
DB Period: Change From Baseline in Clinical Laboratory Results - Cerebrospinal Fluid (CSF) White Blood Cell (WBC)
Time frame: Baseline visit (Day 1), and Months 4, 8, 9, 12, 16
DB Period: Change From Baseline in Clinical Laboratory Results - CSF Protein
Time frame: Baseline visit (Day 1), and Months 4, 8, 9, 12, 16
DB Period: Change From Baseline in Structural Magnetic Resonance Imaging (MRI) Assessing Any New Abnormalities Including Radiographic Features Consistent With Hydrocephalus and Other Relevant MRI Safety Findings
Time frame: Baseline, Months 4, 8, 12, 16 and up to approximately 36 months
DB Period: Percentage Change From Baseline in Geometric Means of CSF Mutant Huntingtin (mHTT) Protein Levels at Month 9
Time frame: Baseline, Month 9
DB Period: Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores (non-U.S. Sites) at 16 Months
Time frame: Baseline to 16 months
Change in scores on the scale.
DB Period: Change From Baseline in Total Functional Capacity (TFC) Scores (U.S. Sites) at 16 Months
Time frame: Baseline to 16 months
Change in scores on the scale.
OLE Period: Incidence and Severity of AEs, With Severity Determined According to the AE Severity Grading Scale
Time frame: Up to approximately 29 months
OLE Period: Change Over Time in Clinical Laboratory Results - CSF WBC
Time frame: Up to approximately 24 months
OLE Period: Change Over Time in Clinical Laboratory Results - CSF Protein
Time frame: Up to approximately 24 months
OLE Period: Change From Baseline in Structural MRI Assessing Any New Abnormalities, Including Radiographic Features Consistent With Hydrocephalus and Other Relevant MRI Safety Findings
Time frame: Up to approximately 29 months
18 secondary outcomes
DB Period: Change From Baseline in Montreal Cognitive Assessment (MoCA) Scores
Time frame: Baseline, Months 4, 8, 12, 16 and up to approximately 36 months
DB Period: Percentage of Participants With Suicidal Ideation or Behavior (I/B), as Assessed by C-SSRS Score at Each Visit, Including Detailed Focus on Any Individual Cases Identified as Having Severe I/B During the Study Conduct
Time frame: Up to approximately 36 months
DB Period: Change From Baseline at 16 Months in TFC (non-U.S. Sites) Scores
Time frame: Baseline to 16 months
DB Period: Change From Baseline at 16 Months in cUHDRS (U.S. Sites) Scores
Time frame: Baseline to 16 months
DB Period: Change From Baseline at 16 Months in Symbol Digit Modalities Test (SDMT) Scores
Time frame: Baseline to 16 months
DB Period: Change From Baseline at 16 Months in Stroop Word Reading (SWR) Score
Time frame: Baseline to 16 months
DB Period: Change From Baseline at 16 Months in Total Motor Score (TMS)
Time frame: Baseline to 16 months
DB Period: Change From Baseline in CSF Neurofilament Light Chain (NfL) Levels at 16 Months
Time frame: Baseline to 16 months
DB Period: Incidence of Anti-drug Antibodies (ADAs) at Specified Timepoints Relative to the Prevalence of ADAs at Baseline
Time frame: Baseline up to approximately 36 months
DB Period: Titers Determined if ADAs are Identified
Time frame: Baseline up to approximately 36 months
OLE Period: Change Over Time in TFC Score
Time frame: Up to approximately 29 months
OLE Period: Change Over Time in cUHDRS Score
Time frame: Up to approximately 29 months
OLE Period: Change Over Time in SDMT Score
Time frame: Up to approximately 29 months
OLE Period: Change Over Time in TMS
Time frame: Up to approximately 29 months
OLE Period: Change Over Time in SWR Score
Time frame: Up to approximately 29 months
OLE Period: Change Over Time in MoCA Score
Time frame: Up to approximately 29 months
OLE Period: Percentage of Participants With Suicidal I/B, as Assessed by C-SSRS Score at Each Visit, Including Detailed Focus on Any Individual Cases Identified as Having Severe I/B During the Study Conduct
Time frame: Up to approximately 29 months
OLE Period: Incidence of ADAs at Specified Timepoints
Time frame: Up to approximately 29 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Aug 2026.
- Derived from this study: PubMed 38055260McColgan P, Thobhani A, Boak L, Schobel SA, Nicotra A, Palermo G, Trundell D, Zhou J, Schlegel V, Sanwald Ducray P, Hawellek DJ, Dorn J, Simillion C, Lindemann M, Wheelock V, Durr A, Anderson KE, Long JD, Wild EJ, Landwehrmeyer GB, Leavitt BR, Tabrizi SJ, Doody R; GENERATION HD1 Investigators. Tominersen in Adults with Manifest Huntington's Disease. N Engl J Med. 2023 Dec 7;389(23):2203-2205. doi: 10.1056/NEJMc2300400. No abstract available.
- Individual participant dataSharing planned: Yes. For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data_sharing
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
70 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Tominersen in people with Huntington Disease, run by Hoffmann-La Roche at 70 sites in 15 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 10 inclusion and 14 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: under way, not looking for participants, as of the sponsor's last update on 10 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 70 sites in United States, France, Germany, Spain, United Kingdom and 10 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
DB Period: Incidence and Severity of Adverse Events (AEs), With Severity Determined According to the AE Severity Grading Scale (time frame: Up to approximately 36 months). The study lists 11 primary outcomes in all. 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 NCT05686551, which also lists 1 linked publication.
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 10 Aug 2026.