Clinical trial • Phase III • Neurology
VATIQUINONE for Friedreich ataxia
Phase III trial of VATIQUINONE for Friedreich ataxia. open-label. 100 participants.
Overview
- Trial Therapeutic Area
- Neurology
- Trial Disease
- Friedreich ataxia
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 27-09-2024
- First CTIS Authorization Date
- 24-10-2024
Trial design
open-label Phase III trial across 4 sites in Germany, France, Italy and others.
- Open Label
- Yes
- Target Sample Size
- 100
Eligibility
Recruits 100 paediatric patients.
- Pregnancy Exclusion
- Pregnancy or breastfeeding
- Vulnerable Population
- Children/minors are included. Assent and consent handling provided: parent/guardian consent (parents ICF) and age-appropriate assent forms for minors (documents for Assent 7-11 and Assent 12-17 are listed). Subject provides consent where applicable. Subject information and consent/assent documents are provided for multiple countries/languages (documents available in English, French, Italian, Spanish and German as indicated).
Inclusion criteria
- {"criterion_text":"- Subjects with FA who completed and directly rolled over from a previous vatiquinone clinical study.\n- Males and females of childbearing potential must be willing to use an effective method of contraception (eg, implants, injectables, transdermal patch, combined oral contraceptives, barrier methods, and intrauterine devices) from the time consent is signed until 30 days after last dose of study drug or Early Termination Visit. Male subjects must agree not to donate sperm during the study and for at least 30 days after the last dose of study drug or Early Termination Visit. Note: Double-barrier method (ie, condom with spermicide) is required if no other methods of contraception are in use.\n- Subject provides consent to participate."}
Exclusion criteria
- {"criterion_text":"- Current participation in any other interventional study\n- Pregnancy or breastfeeding"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Adverse events (AEs)/serious adverse events (SAEs), laboratory abnormalities, vital signs, and electrocardiograms (ECGs)","definition_or_measurement_approach":""}
Secondary endpoints
- {"endpoint_text":"- Change from baseline in the mFARS and its 4 subscales (Upright Stability, Upper Limb, Lower Limb, Bulbar)","definition_or_measurement_approach":"Change from baseline measured using the Modified Friedreich Ataxia Rating Scale (mFARS) and its four subscales"}
Recruitment
- Planned Sample Size
- 100
- Recruitment Window Months
- 60
- Consent Approach
- Informed consent required from subjects. For minors, parent/guardian consent is provided and age-appropriate assent is obtained (assent forms for ages 7-11 and 12-17 are included). Separate parent/guardian ICFs and pregnant partner ICFs are available. Subject information, ICF and assent documents are available in multiple languages (English, French, Italian, Spanish and German as indicated in the submitted documents).
Geography
- Total Number Of Sites
- 4
- Total Number Of Participants
- 34
Germany
- Latest Decision Or Authorization Date
- 24-10-2024
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- Universitaetsklinikum Tuebingen AöR
- Department Name
- Neurology
- Contact Person Name
- Ludger,Johannes Schöls
- Contact Person Email
- service@med.uni-tuebingen.de
France
- Latest Decision Or Authorization Date
- 25-10-2024
- Number Of Sites
- 1
- Number Of Participants
- 10
Sites
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Neurology
- Contact Person Name
- Alexandra Durr
- Contact Person Email
- elodie.petit@icm-institute.org
Italy
- Latest Decision Or Authorization Date
- 25-11-2024
- Number Of Sites
- 1
- Number Of Participants
- 10
Sites
- Site Name
- Ospedale Pediatrico Bambino Gesu
- Department Name
- Pediatric
- Contact Person Name
- Enrico Bertini
- Contact Person Email
- centromalattieneuromuscolari@opbg.net
Spain
- Latest Decision Or Authorization Date
- 31-10-2024
- Number Of Sites
- 1
- Number Of Participants
- 9
Sites
- Site Name
- Hospital Sant Joan De Deu Barcelona
- Department Name
- Neurology
- Contact Person Name
- Alejandra Darling
- Contact Person Email
- info@fsjd.org
Sponsor
Primary sponsor
- Full Name
- PTC Therapeutics Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Suvoda LLC
- Responsibilities
- Trial Supply Managment
- Name
- PCI Pharma Services Germany GmbH
- Responsibilities
- Drug Packaging and distribution
- Name
- Pharmaceutical Product Development LLC
- Name
- Medidata Solutions Inc.
- Responsibilities
- eCRF Database and site payments
- Name
- QPS LLC
- Responsibilities
- PK sample analysis
- Name
- CTI Clinical Trial and Consulting Services Europe GmbH
- Name
- Iqvia Rds Ireland Limited
- Responsibilities
- Pharmacovigilance/safety managment and oversight
- Name
- Mde Services Group Limited
- Responsibilities
- Patient travel and reimbursement
Third parties
- {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"Trial Supply Managment","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Germany","full_name":"PCI Pharma Services Germany GmbH","duties_or_roles":"Drug Packaging and distribution","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"eCRF Database and site payments","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"QPS LLC","duties_or_roles":"PK sample analysis","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"CTI Clinical Trial and Consulting Services Europe GmbH","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Iqvia Rds Ireland Limited","duties_or_roles":"Pharmacovigilance/safety managment and oversight","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Mde Services Group Limited","duties_or_roles":"Patient travel and reimbursement","organisation_type":"Non-Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Vatiquinone
- Active Substance
- VATIQUINONE
- Modality
- Small molecule
- Routes Of Administration
- Oral
- Route
- Oral
- Orphan Designation
- Yes
- Maximum Dose
- 200 mg/day
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