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Trial record 2 of 3 for:    RG6042

A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Patients With Manifest Huntington's Disease

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ClinicalTrials.gov Identifier: NCT03761849
Recruitment Status : Recruiting
First Posted : December 3, 2018
Last Update Posted : May 22, 2019
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Brief Summary:
This study will evaluate the efficacy, safety, and biomarker effects of RO7234292 (RG6042) compared with placebo in patients with manifest Huntington's disease (HD).

Condition or disease Intervention/treatment Phase
Huntingtons Disease Drug: RO7234292 Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 660 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Multicenter, Double-Blind, Placebo-Controlled, Phase III Clinical Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Patients With Manifest Huntington's Disease
Actual Study Start Date : January 23, 2019
Estimated Primary Completion Date : March 1, 2022
Estimated Study Completion Date : August 1, 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: RO7234292 Q4W
RO4234292 is administered intrathecally every 4 weeks.
Drug: RO7234292
Intrathecal injection

Experimental: RO7234292 Q8W
RO7234292 is administered intrathecally every 8 weeks. Participants in this arm will also receive placebo at alternate weeks to keep the blind.
Drug: RO7234292
Intrathecal injection

Drug: Placebo
Intrathecal injection

Placebo Comparator: Placebo
Placebo will be administered every 4 weeks by IT injection.
Drug: Placebo
Intrathecal injection




Primary Outcome Measures :
  1. Change from Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) [ Time Frame: Baselined, Week 101 ]
  2. Change From Baseline in the TFC Score [ Time Frame: Baseline, Week 101 ]

Secondary Outcome Measures :
  1. Change From Baseline in Total Motor Score (TMS) [ Time Frame: Baseline, Week 101 ]
  2. Change From Baseline in Symbol Digit Modalities Test (SDMT) [ Time Frame: Baseline, Week 101 ]
  3. Change From Baseline in Stroop Word Reading (SWR) Test [ Time Frame: Baseline, Week 101 ]
  4. Change From Baseline in the Clinical Global Impression, Severity Scale (CGI-S) [ Time Frame: Baseline, Week 101 ]
  5. Percentage of Patients with a Decrease From Baseline of >=1 point on the TFC [ Time Frame: Baseline, Week101 ]
  6. Percentage of Patients With a Decline From Baseline of >=1.2 Points on the cUHDRS [ Time Frame: Baseline, Week 101 ]
  7. Percentage of Patients With an Unchanged or Improved Score on the Clinical Global Impression, Change Scale (CGI-C) Score [ Time Frame: Baseline to Week 101 ]
  8. Percentage of Participants with Adverse Events [ Time Frame: Up to 117 Weeks (29 months) ]
  9. Change From Baseline in Montreal Cognitive Assessment (MoCA) [ Time Frame: Screening, Baseline, Weeks 5, 21, 37, 53, 69, 85 and 101 and early treatment termination ]
  10. Percentage of Participants With Suicidal Ideation or Behavior, as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) Score [ Time Frame: Screening, Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, end of treatment for early treatment termination and at early treatment termination ]
  11. Concentration of RO7234292 in Plasma [ Time Frame: Baseline, Weeks 13, 21, 37, 53, 69, 85, and 101 and early treatment termination ]
  12. Trough Concentration of RO7234292 in CSF [ Time Frame: Baseline, Weeks 13, 21, 37, 53, 69, 85, and 101 and early treatment termination ]
  13. Incidence of Anti-Drug Antibodies (ADAs) [ Time Frame: Baseline, Weeks 13, 21, 37, 53, 69, 85, and 101 and early treatment termination and safety follow-up visit ]
  14. Titer and Antibody Subtype, determined if ADAs are Identified [ Time Frame: Baseline, Weeks 13, 21, 37, 53, 69, 85, and 101 and early treatment termination and safety follow-up visit ]
  15. Change From Baseline in CSF mHTT Protein Level [ Time Frame: Baseline, Week 101 ]
  16. Change From Baseline in Whole and Regional Brain Volumes, as detrmined by structural magnetic resonance imaging (MRI) [ Time Frame: Baseline, Week 101 ]
  17. Change From Baseline in CSF Neurofilament Light Chain (NfL) Proteint Level [ Time Frame: Baseline, Week 101 ]


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Ages Eligible for Study:   25 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Manifest HD diagnosis, defined as a DCL score of 4
  • Independence Scale (IS) score >= 70
  • Genetically confirmed disease by direct DNA testing with a CAP score >400
  • Clinical assessment to ensure individual has intact functional independence at baseline to maintain self-care and core activities of daily living (ADLs).

Exclusion Criteria:

  • Any serious medical condition or clinically significant laboratory, or vital sign abnormality at screening that, in the investigator's judgment, precludes the patient's safe participation in and completion of the study
  • Pregnant or breastfeeding, or intending to become pregnant during the study or within 5 months after the final dose of study drug

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03761849


Contacts
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Contact: Reference Study ID Number: BN40423 www.roche.com/about_roche/roche_worldwide.htm 888-662-6728 (U.S. and Canada) global-roche-genentech-trials@gene.com

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Locations
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United States, Alabama
Uab Medicine Recruiting
Birmingham, Alabama, United States, 35294
United States, Arizona
Barrow Neurological Institute Recruiting
Phoenix, Arizona, United States, 85013
United States, California
University of California San Diego Recruiting
La Jolla, California, United States, 92093
Stanford Univ Medical Center Recruiting
Palo Alto, California, United States, 94304
SC3 Research Group, Inc Recruiting
Pasadena, California, United States, 91105
University of California Davis Medical System Not yet recruiting
Sacramento, California, United States, 95817
United States, Colorado
Rocky Mountain Movement Disorders Center Active, not recruiting
Englewood, Colorado, United States, 80113
United States, District of Columbia
Georgetown University; Research Division, Psychiatry Not yet recruiting
Washington, District of Columbia, United States, 20007
United States, Florida
University of South Florida Recruiting
Tampa, Florida, United States, 33613
United States, Illinois
Northwestern University Recruiting
Evanston, Illinois, United States, 60208
United States, Maryland
John Hopkins University School of Medicine Recruiting
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Beth Israel Deaconess Medical Center Recruiting
Boston, Massachusetts, United States, 02215
United States, Missouri
Washington University School of Medicine Recruiting
Saint Louis, Missouri, United States, 63110
United States, New York
Columbia University Recruiting
New York, New York, United States, 10032
Dent Neurological Institute Recruiting
Williamsville, New York, United States, 14221
United States, Pennsylvania
University of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15213
United States, Tennessee
Vanderbilt University Medical Center Recruiting
Nashville, Tennessee, United States, 37232
United States, Texas
The University of Texas Health Science Center at Houston; McGovern Medical School Recruiting
Houston, Texas, United States, 77030
United States, Utah
University of Utah Clinical Neurosciences Center Recruiting
Salt Lake City, Utah, United States, 84132
United States, Washington
Evergreen Health Care Center Recruiting
Kirkland, Washington, United States, 98034
Canada, Alberta
University of Alberta Hospital Not yet recruiting
Edmonton, Alberta, Canada, T6G 1Z1
Canada, British Columbia
University of British Columbia Hospital; Division of Neurology Recruiting
Vancouver, British Columbia, Canada, V6T 2B5
Canada, Nova Scotia
True North Clinical Research-Halifax Not yet recruiting
Halifax, Nova Scotia, Canada, B3S 1M7
Canada, Ontario
Centre for Movement Disorders Recruiting
North York, Ontario, Canada, M3B 2S7
Ottawa Hospital Research Institute Recruiting
Ottawa, Ontario, Canada, K1Y 4E9
Canada, Quebec
Centre Hospitalier de l'Université de Montréal (CHUM) Recruiting
Montreal, Quebec, Canada, H2X 0C2
Spain
Hospital de Cruces; Servicio de Neurologia Recruiting
Barakaldo, Vizcaya, Spain, 48903
Hospital Universitario de Badajoz; Servicio de Neurología Recruiting
Badajoz, Spain, 06080
Hospital de la Santa Creu i Sant Pau; Servicio de Neurologia Recruiting
Barcelona, Spain, 08025
Hospital Clinic Servicio de Neurologia Recruiting
Barcelona, Spain, 08036
Hospital Universitario de Burgos. Servicio de Neurología Recruiting
Burgos, Spain, 09006
Hospital Ramon y Cajal; Servicio de Neurologia Recruiting
Madrid, Spain, 28034
Fundacion Jimenez Diaz; Servicio de Neurología Recruiting
Madrid, Spain, 28040
Hospital Universitario Virgen Macarena; Servicio de Neurologia Recruiting
Sevilla, Spain, 41009
Hospital Universitario la Fe; Servicio de Neurologia Active, not recruiting
Valencia, Spain, 46026
United Kingdom
Aberdeen Royal Infirmary; Medical Genectics Not yet recruiting
Aberdeen, United Kingdom, AB25 2ZD
Birmingham and Solihull Mental Health Foundation Trust; Institute of Clinical Sciences Not yet recruiting
Birmingham, United Kingdom, B15 2TT
Cambridge Centre for Brain Repair; Department of Clinical Nuerosciences, Addenbrookes Hospital Not yet recruiting
Cambridge, United Kingdom, CB2 0SP
University Hospital of Wales; Division of Psychological Medicine and Clinical Neurosciences Not yet recruiting
Cardiff, United Kingdom, CF14 4XW
Queen Elizabeth University Hospital Glasgow Not yet recruiting
Glasgow, United Kingdom, G51 4TF
Leeds General Infirmary Recruiting
Leeds, United Kingdom, LS1 3EX
National Hospital For Neurology and Neurosurgery Not yet recruiting
London, United Kingdom, WC1N 3BG
Central Manchester University Hospitals NHS Foundation Trust; Manchester Centre for Genomic Medicine Not yet recruiting
Manchester, United Kingdom, M13 9WL
John Radcliffe Hospital Recruiting
Oxford, United Kingdom, OX3 9DU
Sheffield Childrens NHS Trust; Genectics Department Not yet recruiting
Sheffield, United Kingdom, S10 2TH
University Hospital Southampton NHS Foundation Trust Not yet recruiting
Southhampton, United Kingdom, SO16 6YD
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
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Study Director: Clinical Trials Hoffmann-La Roche

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Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT03761849     History of Changes
Other Study ID Numbers: BN40423
GENERATION HD1 ( Other Identifier: Hoffmann-La Roche )
First Posted: December 3, 2018    Key Record Dates
Last Update Posted: May 22, 2019
Last Verified: May 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Hoffmann-La Roche:
HD, RG6042, intrathecal, ASO, antisense oligonucleotide

Additional relevant MeSH terms:
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Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Dementia
Chorea
Dyskinesias
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Neurocognitive Disorders
Mental Disorders