Clinical trial • Phase I • Musculoskeletal
NUSINERSEN for Spinal Muscular Atrophy
Phase I trial of NUSINERSEN for Spinal Muscular Atrophy. open-label. 32 participants.
Overview
- Trial Therapeutic Area
- Musculoskeletal
- Trial Disease
- Spinal Muscular Atrophy
- Trial Stage
- Phase I
- Drug Modality
- Oligonucleotide
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 08-10-2024
- First CTIS Authorization Date
- 10-02-2025
Trial design
open-label Phase I trial in Spain, Poland, France and others.
- Open Label
- Yes
- Target Sample Size
- 32
- Trial Duration For Participant
- 410
Eligibility
Recruits 32 paediatric patients.
- Pregnancy Exclusion
- Participant is pregnant, currently breastfeeding, or intending to become pregnant during the study. NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
- Vulnerable Population
- Vulnerable population selected; the trial includes pediatric participants (public title and population age ranges indicate adult and pediatric participants). No details on consent or assent handling (age-specific consent forms, assent procedures or languages) are provided in the source JSON.
Inclusion criteria
- {"criterion_text":"- Participant is on regular maintenance dosing of nusinersen (12 milligrams [mg] dose) every 4 months, with 4 months (± 2 weeks) between the LP-delivered study dose and the last nusinersen dose prior to study enrollment.\n- Participants must be enrolled in the PIERRE study to be eligible for enrolment in the PIERRE PK study."}
Exclusion criteria
- {"criterion_text":"- Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrolment in other interventional clinical trials for the treatment of SMA (except for the PIERRE study or interventional clinical trials of myostatin inhibitors [e.g., apitegromab and taldefgrobep alfa]).\n- Participant is naïve to nusinersen treatment.\n- Participant is receiving nusinersen at a dose other than 12 mg.\n- Participant has already undergone implantation of the ThecaFlex DRx system.\n- Participant is pregnant, currently breastfeeding, or intending to become pregnant during the study. NOTE: Other protocol defined Inclusion/Exclusion criteria may apply."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Maximum Observed Concentration (Cmax) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System.","definition_or_measurement_approach":"Measure the highest amount of nusinersen found in the blood after dosing (Cmax) following intrathecal administration via standard lumbar puncture (LP) and via ThecaFlex DRx System; aligns with main objective asking 'What is the highest amount of nusinersen found in the blood after dosing?'"}
- {"endpoint_text":"- Area Under the Plasma Concentration-Time Curve From Zero Time to 24 Hours After Intrathecal Administration (AUC0-24h) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System","definition_or_measurement_approach":"Measure area under the plasma concentration-time curve from time zero to 24 hours after intrathecal administration (AUC0-24h) for nusinersen administered via standard LP and via ThecaFlex DRx System; aligns with main objective asking 'How much nusinersen is found in the blood over the first 24 hours after dosing?'"}
Recruitment
- Planned Sample Size
- 32
- Recruitment Window Months
- 13
- Consent Approach
- Not specified in source JSON (no detailed informed consent/assent process, age-specific consent documents, or languages available are provided).
Methods
- Recruitment and retention materials developed/provided by Praxis Communications LLC (as listed under sponsor third parties: 'Recruitment and retention materials')
- Patient travel and reimbursement support provided by Greenphire LLC (listed under sponsor third parties: 'Patient travel and reimbursement')
Geography
- Total Number Of Sites
- 9
- Total Number Of Participants
- 26
Spain
- Earliest CTIS Part Ii Submission Date
- 25-11-2024
- Latest Decision Or Authorization Date
- 19-01-2026
- Processing Time Days
- 420
- Number Of Sites
- 2
- Number Of Participants
- 7
Sites
- Site Name
- Hospital Universitario La Paz
- Department Name
- Neurologist
- Principal Investigator Name
- Maria del Mar Garcia Romero
- Principal Investigator Email
- yambee@hotmail.com
- Contact Person Name
- Maria del Mar Garcia Romero
- Contact Person Email
- yambee@hotmail.com
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Neurologist
- Principal Investigator Name
- Juan Francisco Vazquez Costa
- Principal Investigator Email
- vazquez_juacos@gva.es
- Contact Person Name
- Juan Francisco Vazquez Costa
- Contact Person Email
- vazquez_juacos@gva.es
Poland
- Earliest CTIS Part Ii Submission Date
- 08-01-2025
- Latest Decision Or Authorization Date
- 19-01-2026
- Processing Time Days
- 376
- Number Of Sites
- 2
- Number Of Participants
- 7
Sites
- Site Name
- Szpital Specjalistyczny Im. Ludwika Rydygiera W Krakowie Sp. z o.o.
- Principal Investigator Name
- Ryszard Nowak
- Principal Investigator Email
- badaniakliniczne@rydygierkrakow.pl
- Contact Person Name
- Ryszard Nowak
- Contact Person Email
- badaniakliniczne@rydygierkrakow.pl
- Site Name
- Instytut Centrum Zdrowia Matki Polki
- Principal Investigator Name
- Łukasz Przysło
- Principal Investigator Email
- lukasz.przyslo@iczmp.edu.pl
- Contact Person Name
- Łukasz Przysło
- Contact Person Email
- lukasz.przyslo@iczmp.edu.pl
France
- Earliest CTIS Part Ii Submission Date
- 06-01-2025
- Latest Decision Or Authorization Date
- 14-01-2026
- Processing Time Days
- 373
- Number Of Sites
- 2
- Number Of Participants
- 3
Sites
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- Pédiatrie
- Principal Investigator Name
- Cécile Halbert
- Principal Investigator Email
- cecile.halbert@ap-hm.fr
- Contact Person Name
- Cécile Halbert
- Contact Person Email
- cecile.halbert@ap-hm.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Pédiatrie
- Principal Investigator Name
- Marta Gomez
- Principal Investigator Email
- marta.gomezgarciadelabanda@aphp.fr
- Contact Person Name
- Marta Gomez
- Contact Person Email
- marta.gomezgarciadelabanda@aphp.fr
Germany
- Earliest CTIS Part Ii Submission Date
- 30-01-2025
- Latest Decision Or Authorization Date
- 15-01-2026
- Processing Time Days
- 350
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Universitaetsklinikum Essen AöR
- Department Name
- Klinik fuer Neurologie
- Principal Investigator Name
- Tim Hagenacker
- Principal Investigator Email
- tim.hagenacker@uk-essen.de
- Contact Person Name
- Tim Hagenacker
- Contact Person Email
- tim.hagenacker@uk-essen.de
Italy
- Earliest CTIS Part Ii Submission Date
- 21-10-2024
- Latest Decision Or Authorization Date
- 12-02-2026
- Processing Time Days
- 479
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- Unità di Fase I - Unità Operativa Complessa di Neuropsichiatria Infantile
- Principal Investigator Name
- Mercuri Eugenio Maria
- Principal Investigator Email
- eugeniomaria.mercuri@policlinicogemelli.it
- Contact Person Name
- Mercuri Eugenio Maria
- Contact Person Email
- eugeniomaria.mercuri@policlinicogemelli.it
- Site Name
- Centro Clinico Nemo
- Department Name
- Clinical Research Center Fase I
- Principal Investigator Name
- Sansone Valeria
- Principal Investigator Email
- valeria.sansone@centrocliniconemo.it
- Contact Person Name
- Sansone Valeria
- Contact Person Email
- valeria.sansone@centrocliniconemo.it
Sponsor
Primary sponsor
- Full Name
- Biogen Idec Research Limited
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United Kingdom
Contract research organisations
- Name
- Suvoda LLC
- Responsibilities
- Role code 3 (as listed in sponsor third parties)
- Name
- Azenta Germany GmbH
- Responsibilities
- Pharmacokinetic and CSF long term storage Europe
- Name
- Greenphire LLC
- Responsibilities
- Patient travel and reimbursement
- Name
- Almac Clinical Services Limited
- Responsibilities
- Drug labelling, packaging, shipping & import
- Name
- PPD Development LP
- Responsibilities
- Pharmacokinetic and CSF sample testing; other role code 4
- Name
- PPD Global Central Labs
- Responsibilities
- Laboratory services (role code 4)
- Name
- Praxis Communications LLC
- Responsibilities
- Recruitment and retention materials
- Name
- IQVIA Limited
- Responsibilities
- Clinical trial services (roles listed as codes 12 and 5)
Third parties
- {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"[{\"id\":1006077,\"code\":\"3\"}]","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Germany","full_name":"Azenta Germany GmbH","duties_or_roles":"Pharmacokinetic and CSF long term storage Europe","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient travel and reimbursement","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Clinical Services Limited","duties_or_roles":"Drug labelling, packaging, shipping & import","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"Pharmacokinetic and CSF sample testing; additional role code 4","organisation_type":"Pharmaceutical company"}
- {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"Role code 4","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Praxis Communications LLC","duties_or_roles":"Recruitment and retention materials","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Roles with codes 12 and 5 (as listed)","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Spinraza 12 mg solution for injection
- Active Substance
- NUSINERSEN
- Modality
- Oligonucleotide
- Routes Of Administration
- Intrathecal
- Route
- Intrathecal
- Authorisation Status
- Marketing authorisation EU/1/17/1188/001 (product entry present in JSON)
- Orphan Designation
- Yes
- Starting Dose
- 12 mg
- Dose Levels
- 12 mg (maintenance dosing)
- Frequency
- Every 4 months (maintenance dosing)
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