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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05686551
Registration number
NCT05686551
Ethics application status
Date submitted
16/12/2022
Date registered
17/01/2023
Date last updated
10/06/2024
Titles & IDs
Public title
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.
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Scientific 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
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Secondary ID [1]
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Other
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Secondary ID [2]
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BN42489
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Huntington Disease
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Condition category
Condition code
Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Tominersen 60 mg
Treatment: Drugs - Placebo
Treatment: Drugs - Tominersen 100 mg
Experimental: Tominersen 60 mg -
Placebo Comparator: Placebo -
Experimental: Tominersen 100 mg -
Treatment: Drugs: Tominersen 60 mg
60 mg tominersen administered intrathecally every 16 weeks
Treatment: Drugs: Placebo
Matching placebo administered intrathecally every 16 weeks
Treatment: Drugs: Tominersen 100 mg
100 mg tominersen administered intrathecally every 16 weeks
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale
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Assessment method [1]
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Timepoint [1]
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Up to Approximately 24 Months
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Primary outcome [2]
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Change from baseline in clinical laboratory results - Cerebrospinal fluid (CSF) White Blood Cell (WBC) (1/uL)
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Assessment method [2]
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Timepoint [2]
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From Baseline Visit (Day 1), and Months 4, 8, 9, 12, 16
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Primary outcome [3]
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Change from baseline in clinical laboratory results Cerebrospinal fluid (CSF) protein (g/L)
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Assessment method [3]
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Timepoint [3]
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From Baseline Visit (Day 1), and Months 4, 8, 9, 12, 16
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Primary outcome [4]
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Change in baseline in structural MRI assessing any new abnormalities including radiographic features consistent with hydrocephalus and other relevant MRI safety findings
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Assessment method [4]
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Timepoint [4]
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From Baseline, Months 4, 8, 12, 16 and Up to Approximately Month 24
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Primary outcome [5]
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Percentage change from baseline in geometric means of CSF mHTT protein levels at Month 9
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Assessment method [5]
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Timepoint [5]
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Baseline and Month 9
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Primary outcome [6]
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Change from baseline in composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores (non-U.S. sites) at 16 months
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Assessment method [6]
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Change in scores on the scale
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Timepoint [6]
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Baseline to 16 Months
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Primary outcome [7]
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Change from baseline in Total Functional Capacity (TFC) Scores (U.S. sites) at 16 months
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Assessment method [7]
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Change in scores on the scale
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Timepoint [7]
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Baseline to 16 Months
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Secondary outcome [1]
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Change from baseline in MoCA Scores
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Assessment method [1]
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Timepoint [1]
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From Baseline, Months 4, 8, 12, 16 and up to approximately Month 24
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Secondary outcome [2]
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Percentage of participants with suicidal ideation or behavior as assessed by C-SSRS score at each visit, including detailed focus on any individual cases identified as having severe ideation or behavior during the study conduct
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Assessment method [2]
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Timepoint [2]
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Up to Approximately 24 Months
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Secondary outcome [3]
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Change from baseline at 16 months for the assessments of TFC (non-U.S. sites) Scores
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Assessment method [3]
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Timepoint [3]
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Baseline to 16 Months
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Secondary outcome [4]
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Change from baseline at 16 months for the assessments of cUHDRS (U.S. sites) Scores
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Assessment method [4]
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Timepoint [4]
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Baseline to 16 Months
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Secondary outcome [5]
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Change from baseline at 16 months for the assessments of Symbol Digit Modalities Test (SDMT) Scores
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Assessment method [5]
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Timepoint [5]
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Baseline to 16 Months
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Secondary outcome [6]
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Change from Baseline at 16 Months for the Assessments of Stroop Word Reading (SWR) Scores
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Assessment method [6]
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Timepoint [6]
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Baseline to 16 Months
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Secondary outcome [7]
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Change from baseline at 16 months for the assessments of Total Motor Score (TMS)
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Assessment method [7]
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Timepoint [7]
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Baseline to 16 Months
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Secondary outcome [8]
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Change from baseline in CSF Neurofilament light Chain (NfL) levels at 16 months
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Assessment method [8]
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Timepoint [8]
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Baseline to 16 Months
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Secondary outcome [9]
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Incidence of anti-drug antibodies (ADAs) at specified timepoints relative to the prevalence of ADAs at baseline
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Assessment method [9]
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Timepoint [9]
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From Baseline, Months 4, 8, 12, 16 and Up to Approximately Month 24
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Secondary outcome [10]
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Titers determined if ADAs are identified
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Assessment method [10]
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Timepoint [10]
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From Baseline, Months 4, 8, 12, 16 and Up to Approximately Month 24
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Eligibility
Key inclusion criteria
Key Inclusion Criteria
-Huntington's disease (HD) gene expansion mutation carrier status with a CAP score of
400-500 inclusive
Either:
- Prodromal HD (defined as DCL 2 to 3, Independence Scale (IS) ?70, and ?TFC8); or
- Early manifest HD (defined as DCL 4, Independence Scale (IS) ?70, and ?TFC8);
- Total body weight > 40 kg and a body mass index within the range of 18-32 kg/m2
- Study Companion
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Minimum age
25
Years
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Maximum age
50
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
Exclusion Criteria
- Current or previous use of an ASO (including small interfering 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 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
3/02/2023
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/04/2027
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Actual
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Sample size
Target
300
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,VIC,WA
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Recruitment hospital [1]
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WESTMEAD HOSPITAL; Deparment of Neurology - Westmead
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Recruitment hospital [2]
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Monash Medical Centre - Clayton
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Recruitment hospital [3]
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Royal Melbourne Hospital; Department of Neurology - Parkville
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Recruitment hospital [4]
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Perron Institute for Neurological and Translational Science - Nedlands
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Recruitment postcode(s) [1]
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2145 - Westmead
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Recruitment postcode(s) [2]
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3168 - Clayton
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Recruitment postcode(s) [3]
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3050 - Parkville
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Recruitment postcode(s) [4]
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6009 - Nedlands
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Recruitment outside Australia
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Alabama
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Caba
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Ciudad Autonoma Buenos Aires
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Innsbruck
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Gümligen
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Leeds
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London
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Southampton
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Hoffmann-La Roche
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Address
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Ethics approval
Ethics application status
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Summary
Brief 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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05686551
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Contact person for public queries
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Reference Study ID Number: BN42489 https://forpatients.roche.com/
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Address
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Phone
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888-662-6728 (U.S. Only)
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05686551
Download to PDF