Clinical trial • Phase II • Neurology
TOMINERSEN for Huntington's disease (prodromal and early manifest)
Phase II trial of TOMINERSEN for Huntington's disease (prodromal and early manifest).
Overview
- Trial Therapeutic Area
- Neurology
- Trial Disease
- Huntington's disease (prodromal and early manifest)
- Trial Stage
- Phase II
- Drug Modality
- Oligonucleotide
Key dates
- Initial CTIS Submission Date
- 08-04-2024
- First CTIS Authorization Date
- 17-05-2024
Trial design
Randomised, placebo; ro7234292 (tominersen) 60 mg intrathecal (i.t.) q16w; ro7234292 (tominersen) 100 mg intrathecal (i.t.) q16w-controlled, adaptive Phase II trial in France, Poland, Germany and others.
- Randomised
- Yes
- Comparator
- Placebo; RO7234292 (tominersen) 60 mg intrathecal (I.T.) Q16W; RO7234292 (tominersen) 100 mg intrathecal (I.T.) Q16W
- Adaptive
- True, an independent data monitoring committee (iDMC) conducted an interim analysis and recommended discontinuation of the 60 mg Q16W dose and continuation/testing only of the 100 mg Q16W dose for the remainder of the study (participants on 60 mg were switched to 100 mg).
- Target Sample Size
- 138
- Trial Duration For Participant
- 658
Eligibility
Recruits 138 Vulnerable population selected (isVulnerablePopulationSelected = true). The protocol requires a Study Companion (inclusion criterion). Separate informed consent forms / subject information sheets are provided for participants and for companions/caregivers (multiple companion/caregiver ICF documents are listed), as well as specific ICFs for pregnant partners and infants. Consent is by the participant; companion/caregiver have separate ICFs documented..
- Pregnancy Exclusion
- Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 5 months after the final dose of study drug
- Vulnerable Population
- Vulnerable population selected (isVulnerablePopulationSelected = true). The protocol requires a Study Companion (inclusion criterion). Separate informed consent forms / subject information sheets are provided for participants and for companions/caregivers (multiple companion/caregiver ICF documents are listed), as well as specific ICFs for pregnant partners and infants. Consent is by the participant; companion/caregiver have separate ICFs documented.
Inclusion criteria
- {"criterion_text":"- Huntington’s disease (HD) gene expansion mutation carrier status with a CAP score of 400-500 inclusive\n- 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)\n- Estimated glomerular filtration rate ≥ 60 mL/min/1.73 m2 in at least one out of two maximum screening samples\n- Total body weight > 40 kg and a body mass index within the range of 18-32 kg/m2\n- Age 25- 50 years, inclusive, at the time of signing the Informed Consent Form\n- Study Companion"}
Exclusion criteria
- {"criterion_text":"- History of attempted suicide or suicidal ideation with plan that required hospital visit and/or change in level of care within 12 months prior to screening\n- Current or previous use of an ASO (including small interfering RNA) or any HTT lowering therapy (including tominersen)\n- Anti-platelet or anticoagulant therapy\n- Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 5 months after the final dose of study drug\n- History of gene therapy, cell transplantation, or brain surgery\n- Hydrocephalus"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale (DB Period)","definition_or_measurement_approach":"Incidence and severity assessed per Adverse Event Severity Grading Scale during the double-blind (DB) period"}
- {"endpoint_text":"- Change from baseline in clinical laboratory results (CSF WBC and protein) (DB Period)","definition_or_measurement_approach":"Change from baseline measured in CSF white blood cell count and protein during DB period"}
- {"endpoint_text":"- Safety MRI (DB Period)","definition_or_measurement_approach":"Safety assessed by MRI during DB period"}
- {"endpoint_text":"- Percentage change from baseline in geometric means of CSF mHTT protein levels at Month 9 (DB Period)","definition_or_measurement_approach":"Percent change from baseline in geometric mean CSF mutant huntingtin (mHTT) levels at Month 9"}
- {"endpoint_text":"- Change from baseline in cUHDRS (non-U.S.) and TFC (U.S.) at 16 months (DB Period)","definition_or_measurement_approach":"Change from baseline in composite Unified Huntington’s Disease Rating Scale (cUHDRS) for non-US and Total Functional Capacity (TFC) for US at 16 months"}
- {"endpoint_text":"- Incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale (OLE Period)","definition_or_measurement_approach":"Incidence and severity assessed per Adverse Event Severity Grading Scale during the open-label extension (OLE) period"}
- {"endpoint_text":"- Change over time in clinical laboratory results (CSF, WBC, and protein) (OLE period)","definition_or_measurement_approach":"Longitudinal change in CSF white blood cell count and protein over time during OLE"}
- {"endpoint_text":"- Safety MRI (OLE period)","definition_or_measurement_approach":"Safety assessed by MRI during OLE period"}
Secondary endpoints
- {"endpoint_text":"- Change from baseline in vital signs (DB period)","definition_or_measurement_approach":"Change in vital signs from baseline measured at visits during DB period"}
- {"endpoint_text":"- Change from baseline in ECG parameters (DB period)","definition_or_measurement_approach":"Change in ECG parameters from baseline during DB period"}
- {"endpoint_text":"- Change from baseline in plasma clinical laboratory results (DB period)","definition_or_measurement_approach":"Change from baseline in plasma clinical laboratory values during DB period"}
- {"endpoint_text":"- Change from baseline in MoCA (DB period)","definition_or_measurement_approach":"Change from baseline in Montreal Cognitive Assessment score during DB period"}
- {"endpoint_text":"- Proportion 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 (DB period)","definition_or_measurement_approach":"Proportion assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) at each visit (DB period); detailed case focus for severe cases"}
- {"endpoint_text":"- Change from baseline at 16 months for the assessments of TFC (non-U.S.)/cUHDRS (U.S.), SDMT, TMS, and SWR (DB period)","definition_or_measurement_approach":"Change from baseline at 16 months for functional and cognitive endpoints: TFC/cUHDRS, Symbol Digit Modalities Test (SDMT), Total Motor Score (TMS), Stroop Word Reading (SWR)"}
- {"endpoint_text":"- Change from baseline in CSF NfL levels at 16 months (DB period)","definition_or_measurement_approach":"Change from baseline in CSF neurofilament light chain (NfL) at 16 months"}
- {"endpoint_text":"- Incidence of anti-drug antibodies (ADAs) at specified timepoints relative to the prevalence of ADAs at baseline (DB period)","definition_or_measurement_approach":"Incidence of ADAs measured at prespecified timepoints compared to baseline prevalence"}
- {"endpoint_text":"- Titers will be determined if ADAs are identified (DB period)","definition_or_measurement_approach":"ADA titers to be measured if ADAs are detected"}
- {"endpoint_text":"- Change over time in cUHDRS, TFC, SDMT, TMS, SWR (OLE period)","definition_or_measurement_approach":"Longitudinal change in clinical and cognitive scales during OLE"}
- {"endpoint_text":"- Change over time in vital signs (OLE period)","definition_or_measurement_approach":"Longitudinal change in vital signs during OLE"}
- {"endpoint_text":"- Change over time in ECG parameters (OLE period)","definition_or_measurement_approach":"Longitudinal change in ECG parameters during OLE"}
- {"endpoint_text":"- Change over time in plasma clinical laboratory results (OLE period)","definition_or_measurement_approach":"Longitudinal change in plasma lab values during OLE"}
- {"endpoint_text":"- Change over time in MoCA (OLE period)","definition_or_measurement_approach":"Longitudinal change in MoCA during OLE"}
- {"endpoint_text":"- Proportion 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 (OLE period)","definition_or_measurement_approach":"Proportion assessed by C-SSRS at each visit during OLE; detailed case focus for severe cases"}
- {"endpoint_text":"- Incidence of ADAs at specified timepoints (OLE period)","definition_or_measurement_approach":"Incidence of anti-drug antibodies assessed at specified timepoints during OLE"}
Recruitment
- Digital Remote Recruitment
- True, recruitment materials include a lumbar puncture video script and digital brochures/flyers; ePRO and IxRS systems are used for electronic data capture and patient interactions.
- Planned Sample Size
- 138
- Recruitment Window Months
- 49
- Consent Approach
- Informed consent is taken from the participant. The study requires a Study Companion and separate companion/caregiver informed consent forms are provided (multiple companion/caregiver ICFs listed). There are also ICFs for pregnant partners and infant health information. ICFs and study materials are available in multiple languages as evidenced by protocol/public title translations and country-specific ICF documents (English plus translations including French, German, Spanish, Polish, Portuguese, Italian and country-specific ICFs).
Methods
- Patient recruitment and advertising/promotion services provided by Publicis Healthcare Communications Group (patient-facing materials and advertising).
- Study flyers and clinical trial brochures (K2 recruitment material / study flyers listed among documents).
- Lumbar puncture patient video script / educational video materials used in recruitment materials.
- Patient reimbursement managed by Greenphire LLC.
- Electronic data capture/ePRO and IXRS services (ePRO provider Ersearchtechnology Inc./Clario; Signant Health listed as IxRS provider) used in study operations (supporting remote data collection).
Geography
- Total Number Of Sites
- 33
- Total Number Of Participants
- 162
France
- Earliest CTIS Part Ii Submission Date
- 24-04-2024
- Latest Decision Or Authorization Date
- 24-05-2024
- Processing Time Days
- 30
- Number Of Sites
- 9
- Number Of Participants
- 27
Sites
- Site Name
- Les Hopitaux Universitaires De Strasbourg
- Department Name
- Neurologie
- Principal Investigator Name
- Christine Tranchant
- Principal Investigator Email
- Christine.Tranchant@chru-strasbourg.fr
- Contact Person Name
- Christine Tranchant
- Contact Person Email
- Christine.Tranchant@chru-strasbourg.fr
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Neurologie
- Principal Investigator Name
- Clémence Simonin
- Principal Investigator Email
- clemence.simonin@chu-lille.fr
- Contact Person Name
- Clémence Simonin
- Contact Person Email
- clemence.simonin@chu-lille.fr
- Site Name
- Centre Hospitalier Universitaire De Toulouse
- Department Name
- Neurologie
- Principal Investigator Name
- Jérémie Pariente
- Principal Investigator Email
- jeremie.pariente@inserm.fr
- Contact Person Name
- Jérémie Pariente
- Contact Person Email
- jeremie.pariente@inserm.fr
- Site Name
- Assistance Publique Hopitaux De Marseille
- Department Name
- Neurologie
- Principal Investigator Name
- Jean-Philippe Azulay
- Principal Investigator Email
- Jean-philippe.AZULAY@ap-hm.fr
- Contact Person Name
- Jean-Philippe Azulay
- Contact Person Email
- Jean-philippe.AZULAY@ap-hm.fr
- Site Name
- Centre Hospitalier Universitaire D'Angers
- Department Name
- Neurologie
- Principal Investigator Name
- Christophe Verny
- Principal Investigator Email
- chverny@chu-angers.fr
- Contact Person Name
- Christophe Verny
- Contact Person Email
- chverny@chu-angers.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Neurologie
- Principal Investigator Name
- Katia Youssov
- Principal Investigator Email
- katia.youssov@aphp.fr
- Contact Person Name
- Katia Youssov
- Contact Person Email
- katia.youssov@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Bordeaux
- Department Name
- Service de Génétique Médicale
- Principal Investigator Name
- Cyril Goizet
- Principal Investigator Email
- cyril.goizet@chu-bordeaux.fr
- Contact Person Name
- Cyril Goizet
- Contact Person Email
- cyril.goizet@chu-bordeaux.fr
- Site Name
- Centre Hospitalier Universitaire De Montpellier
- Department Name
- Neurologie
- Principal Investigator Name
- Cecilia Marelli
- Principal Investigator Email
- c-marelli@chu-montpellier.fr
- Contact Person Name
- Cecilia Marelli
- Contact Person Email
- c-marelli@chu-montpellier.fr
- Site Name
- Centre Hospitalier Universitaire De Strasbourg (duplicate entry if present)
- Department Name
- Neurologie
Poland
- Earliest CTIS Part Ii Submission Date
- 24-04-2024
- Latest Decision Or Authorization Date
- 27-05-2024
- Processing Time Days
- 33
- Number Of Sites
- 3
- Number Of Participants
- 20
Sites
- Site Name
- Wojskowy Instytut Medycyny Lotniczej
- Department Name
- Klinika Neurologii
- Principal Investigator Name
- Grzegorz Witkowski
- Principal Investigator Email
- gwitkowski@wiml.waw.pl
- Contact Person Name
- Grzegorz Witkowski
- Contact Person Email
- gwitkowski@wiml.waw.pl
- Site Name
- Krakowska Akademia Neurologii Sp. z o.o.
- Department Name
- Centrum Neurologii Klinicznej
- Principal Investigator Name
- Monika Rudzińska-Bar
- Principal Investigator Email
- mrudzinska@afm.edu.pl
- Contact Person Name
- Monika Rudzińska-Bar
- Contact Person Email
- mrudzinska@afm.edu.pl
- Site Name
- Copernicus Podmiot Leczniczy Sp. z o.o.
- Department Name
- Szpital Św. Wojciecha, Oddział Neurologiczny
- Principal Investigator Name
- Jarosław Sławek
- Principal Investigator Email
- jaroslawek@gumed.edu.pl
- Contact Person Name
- Jarosław Sławek
- Contact Person Email
- jaroslawek@gumed.edu.pl
Germany
- Earliest CTIS Part Ii Submission Date
- 24-04-2024
- Latest Decision Or Authorization Date
- 22-05-2024
- Processing Time Days
- 28
- Number Of Sites
- 8
- Number Of Participants
- 40
Sites
- Site Name
- Universitaetsklinikum Ulm AöR
- Department Name
- Department of Neurology
- Principal Investigator Name
- Bernhard Landwehrmeyer
- Principal Investigator Email
- bernhard.landwehrmeyer@uni-ulm.de
- Contact Person Name
- Bernhard Landwehrmeyer
- Contact Person Email
- bernhard.landwehrmeyer@uni-ulm.de
- Site Name
- Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.
- Department Name
- Klinik für Neurodegenerative Erkrankungen
- Principal Investigator Name
- Patrick Weydt
- Principal Investigator Email
- Patrick.Weydt@ukbonn.de
- Contact Person Name
- Patrick Weydt
- Contact Person Email
- Patrick.Weydt@ukbonn.de
- Site Name
- Katholisches Klinikum Bochum gGmbH
- Department Name
- Neurologische Klinik der Ruhr-Universitaet Bochum
- Principal Investigator Name
- Carsten Saft
- Principal Investigator Email
- Carsten.Saft@ruhr-uni-bochum.de
- Contact Person Name
- Carsten Saft
- Contact Person Email
- Carsten.Saft@ruhr-uni-bochum.de
- Site Name
- Universitaetsklinikum Erlangen AöR
- Department Name
- Molekulare Neurologie
- Principal Investigator Name
- Martin Regensburger
- Principal Investigator Email
- martin.regensburger@uk-erlangen.de
- Contact Person Name
- Martin Regensburger
- Contact Person Email
- martin.regensburger@uk-erlangen.de
- Site Name
- Kbo Isar-Amper-Klinikum Taufkirchen (Vils)
- Department Name
- Huntington-Zentrum-Sued
- Principal Investigator Name
- Alzbeta Muehlbaeck
- Principal Investigator Email
- alzbeta.muehlbaeck@kbo.de
- Contact Person Name
- Alzbeta Muehlbaeck
- Contact Person Email
- alzbeta.muehlbaeck@kbo.de
- Site Name
- Universitaetsklinikum Schleswig-Holstein AöR
- Department Name
- Zentrum für Seltene Erkrankungen
- Principal Investigator Name
- Alexander Münchau
- Principal Investigator Email
- alexander.muenchau@uni-luebeck.de
- Contact Person Name
- Alexander Münchau
- Contact Person Email
- alexander.muenchau@uni-luebeck.de
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Klinik für Psychiatrie und Psychotherapie, CCM
- Principal Investigator Name
- Josef Priller
- Principal Investigator Email
- Josef.Priller@charite.de
- Contact Person Name
- Josef Priller
- Contact Person Email
- Josef.Priller@charite.de
- Site Name
- Universitaetsklinikum Aachen AöR
- Department Name
- Klinik für Neurologie
- Principal Investigator Name
- Kathrin Reetz
- Principal Investigator Email
- kreetz@ukaachen.de
- Contact Person Name
- Kathrin Reetz
- Contact Person Email
- kreetz@ukaachen.de
Italy
- Earliest CTIS Part Ii Submission Date
- 24-04-2024
- Latest Decision Or Authorization Date
- 22-05-2024
- Processing Time Days
- 28
- Number Of Sites
- 2
- Number Of Participants
- 14
Sites
- Site Name
- IRCCS Foundation Istituto Neurologico Carlo Besta
- Department Name
- UOC Genetica Medica - Neurogenetica
- Principal Investigator Name
- Caterina Mariotti
- Principal Investigator Email
- caterina.mariotti@istituto-besta.it
- Contact Person Name
- Caterina Mariotti
- Contact Person Email
- caterina.mariotti@istituto-besta.it
- Site Name
- Azienda Unita Sanitaria Locale Di Bologna
- Department Name
- UOC Clinica Neurologica Metropolitana (NeuroMet)
- Principal Investigator Name
- Cesa Lorella Maria Scaglione
- Principal Investigator Email
- cesa.scaglione@isnb.it
- Contact Person Name
- Cesa Lorella Maria Scaglione
- Contact Person Email
- cesa.scaglione@isnb.it
Denmark
- Earliest CTIS Part Ii Submission Date
- 24-04-2024
- Latest Decision Or Authorization Date
- 20-05-2024
- Processing Time Days
- 26
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Rigshospitalet
- Department Name
- Hukommelsesklinikken
- Principal Investigator Name
- Lena Hjermind
- Principal Investigator Email
- lena.elisabeth.hjermind.01@regionh.dk
- Contact Person Name
- Lena Hjermind
- Contact Person Email
- lena.elisabeth.hjermind.01@regionh.dk
Portugal
- Earliest CTIS Part Ii Submission Date
- 24-04-2024
- Latest Decision Or Authorization Date
- 17-05-2024
- Processing Time Days
- 23
- Number Of Sites
- 2
- Number Of Participants
- 10
Sites
- Site Name
- Hospital De Santa Maria E.P.E.
- Department Name
- Neurociências e Saúde Mental
- Principal Investigator Name
- Leonor Guedes
- Principal Investigator Email
- leonor.guedes@ulssm.min-saude.pt
- Contact Person Name
- Leonor Guedes
- Contact Person Email
- leonor.guedes@ulssm.min-saude.pt
- Site Name
- CNS Saude Lda.
- Department Name
- Clínica Médica
- Principal Investigator Name
- Joaquim Ferreira
- Principal Investigator Email
- science@cnscampus.com
- Contact Person Name
- Joaquim Ferreira
- Contact Person Email
- science@cnscampus.com
Austria
- Earliest CTIS Part Ii Submission Date
- 24-04-2024
- Latest Decision Or Authorization Date
- 22-05-2024
- Processing Time Days
- 28
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- Medizinische Universitaet Innsbruck
- Department Name
- University clinic for neurology
- Principal Investigator Name
- Klaus Seppi
- Principal Investigator Email
- lki.ne.parkinson@tirol-kliniken.at
- Contact Person Name
- Klaus Seppi
- Contact Person Email
- lki.ne.parkinson@tirol-kliniken.at
Spain
- Earliest CTIS Part Ii Submission Date
- 24-04-2024
- Latest Decision Or Authorization Date
- 22-05-2024
- Processing Time Days
- 28
- Number Of Sites
- 7
- Number Of Participants
- 43
Sites
- Site Name
- Hospital Universitario De Burgos
- Department Name
- Neurología
- Principal Investigator Name
- Esther Cubo Delgado
- Principal Investigator Email
- mcubo@saludcastillayleon.es
- Contact Person Name
- Esther Cubo Delgado
- Contact Person Email
- mcubo@saludcastillayleon.es
- Site Name
- Hospital Universitario Virgen De La Macarena
- Department Name
- Neurología
- Principal Investigator Name
- Rafael Perez Noguera
- Principal Investigator Email
- rafapereznoguera@gmail.com
- Contact Person Name
- Rafael Perez Noguera
- Contact Person Email
- rafapereznoguera@gmail.com
- Site Name
- Hospital Universitario De Badajoz
- Department Name
- Neurología
- Principal Investigator Name
- David Jesus Ceberino Muñoz
- Principal Investigator Email
- david.ceberino@salud-juntaex.es
- Contact Person Name
- David Jesus Ceberino Muñoz
- Contact Person Email
- david.ceberino@salud-juntaex.es
- Site Name
- Hospital Universitario Ramon Y Cajal
- Department Name
- Neurología
- Principal Investigator Name
- Jose Luis López-Sendón Moreno
- Principal Investigator Email
- jlsendonmoreno@salud.madrid.org
- Contact Person Name
- Jose Luis López-Sendón Moreno
- Contact Person Email
- jlsendonmoreno@salud.madrid.org
- Site Name
- Hospital Universitario De Cruces
- Department Name
- Neurología
- Principal Investigator Name
- Tamara Fernández Valle
- Principal Investigator Email
- Tamara.fernandezvalle@osakidetza.eus
- Contact Person Name
- Tamara Fernández Valle
- Contact Person Email
- Tamara.fernandezvalle@osakidetza.eus
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Neurología
- Principal Investigator Name
- Carmen Peiró Vilaplana
- Principal Investigator Email
- peiro_marvil@gva.es
- Contact Person Name
- Carmen Peiró Vilaplana
- Contact Person Email
- peiro_marvil@gva.es
- Site Name
- Hospital De La Santa Creu I Sant Pau
- Department Name
- Neurología
- Principal Investigator Name
- Jaime Kulisevsky Bojarski
- Principal Investigator Email
- jkulisevsky@santpau.cat
- Contact Person Name
- Jaime Kulisevsky Bojarski
- Contact Person Email
- jkulisevsky@santpau.cat
Sponsor
Primary sponsor
- Full Name
- F. Hoffmann-La Roche AG
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Switzerland
Contract research organisations
- Name
- Pharmaceutical Research Associates Group B.V.
- Responsibilities
- Legacy Specialty Laboratory; laboratory services
- Name
- PPD Development LP
- Responsibilities
- Bioanalytical QA Management / laboratory duties
- Name
- Iqvia Laboratories Limited
- Responsibilities
- Legacy Specialty Laboratory; laboratory services
- Name
- Labcorp Central Laboratory Services SARL
- Responsibilities
- Central laboratory services
- Name
- Charles River Laboratories International
- Responsibilities
- Legacy Specialty Laboratory; laboratory services
Third parties
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient reimbursement","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ePRO","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"MicroCoat Biotechnologie GmbH","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"Pharmaceutical Research Associates Group B.V.","duties_or_roles":"Legacy Specialty Laboratory; other laboratory duties","organisation_type":"Pharmaceutical company"}
- {"country":"Canada","full_name":"Charles River Laboratories International","duties_or_roles":"Legacy Specialty Laboratory; other laboratory duties","organisation_type":"Health care"}
- {"country":"United Kingdom","full_name":"Iqvia Laboratories Limited","duties_or_roles":"Legacy Specialty Laboratory; other laboratory duties","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Q2q Communications Limited","duties_or_roles":"Meeting Organization","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Publicis Healthcare Communications Group Limited","duties_or_roles":"Patient Recruitment and Advertising / Promotion Services","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Signant Health Global LLC","duties_or_roles":"IxRS Provider","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Ixico Technologies Limited","duties_or_roles":"Imaging","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"Clinical laboratory services (listed duties)","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- RO7234292
- Active Substance
- TOMINERSEN
- Modality
- Oligonucleotide
- Routes Of Administration
- I.T. BOLUS INJECTION TO THE INTRATHECAL SPACE
- Route
- Intrathecal (I.T.) bolus injection to the intrathecal space
- Starting Dose
- 60 mg
- Dose Levels
- 60 mg; 100 mg
- Frequency
- Q16W for 16 months
- Maximum Dose
- 100 mg
- Dose Escalation Increase
- 60 mg -> 100 mg (per iDMC recommendation participants on 60 mg were switched to 100 mg)
- Investigational Product Name
- RO7234292 Placebo
- Modality
- Other
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