Overview
Sponsor-declared trial summary
Phase
Human pharmacology (Phase I) - Other
Status
Authorised, recruiting
Participants planned
58
Countries
5
Sites
12
Spinal Muscular Atrophy
The main objective of the PIERRE-PK study is to learn how the body processes nusinersen when given by the ThecaFlex DRx system compared to a lumbar puncture. The main questions researchers want to answer are: • What is the highest amount of nusinersen found in the blood after dosing? • How much nusinersen is found i…
Key facts
- Sponsor
- Biogen Idec Research Limited
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Musculoskeletal Diseases [C05]
- Trial duration
- 21 May 2025 → ongoing
- Decision date (initial)
- 2025-05-14
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Biogen Idec Research Limited
External identifiers
- EU CT number
- 2024-514239-21-00
- ClinicalTrials.gov
- NCT06555419
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Pharmacokinetic
The main objective of the PIERRE-PK study is to learn how the body processes nusinersen when given by the ThecaFlex DRx system compared to a lumbar puncture. The main questions researchers want to answer are:
• What is the highest amount of nusinersen found in the blood after dosing?
• How much nusinersen is found in the blood over the first 24 hours after dosing?
Conditions and MedDRA coding
Spinal Muscular Atrophy
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | SOC | 10010331 | Congenital familial and genetic disorders | 21 |
Regulatory references
- Scientific advice from competent authorities
- European Medicines Agency
- Plan to share IPD
- Yes
- IPD plan description
- In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- 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.
- Participants must be enrolled in the PIERRE study to be eligible for enrolment in the PIERRE PK study.
Exclusion criteria 5
- 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]).
- Participant is naïve to nusinersen treatment.
- Participant is receiving nusinersen at a dose other than 12 mg.
- Participant has already undergone implantation of the ThecaFlex DRx system.
- Participant is pregnant, currently breastfeeding, or intending to become pregnant during the study. NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 2
- Maximum Observed Concentration (Cmax) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System.
- 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
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Spinraza 12 mg solution for injection
PRD5055458 · Product
- Active substance
- Nusinersen
- Substance synonyms
- ISIS 396443, ANTISENSE OLIGONUCLEOTIDE TARGETED TO THE SMN2 GENE, ISIS-396443, BIIB058
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRATHECAL USE
- Authorisation status
- Authorised
- ATC code
- M09AX07 — -
- Marketing authorisation
- EU/1/17/1188/001
- MA holder
- BIOGEN NETHERLANDS B.V.
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/12/976
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Biogen Idec Research Limited
- Sponsor organisation
- Biogen Idec Research Limited
- Address
- Building 5 Foundation Park, Roxborough Way Roxborough Way
- City
- Maidenhead
- Postcode
- SL6 3UD
- Country
- United Kingdom
Scientific contact point
- Organisation
- Biogen Idec Research Limited
- Contact name
- Clinical Trials Information Desk
Public contact point
- Organisation
- Biogen Idec Research Limited
- Contact name
- Clinical Trials Information Desk
Third parties 8
| Organisation | City, country | Duties |
|---|---|---|
| Suvoda LLC ORG-100043523
|
Conshohocken, United States | Interactive response technologies (IRT) |
| Almac Clinical Services Limited ORG-100017464
|
Craigavon, United Kingdom (Northern Ireland) | Other |
| Praxis Communications LLC ORG-100045170
|
Buffalo, United States | Other |
| PPD Development LP ORG-100011560
|
Richmond, United States | Other, Laboratory analysis |
| IQVIA Limited ORG-100008655
|
Reading, United Kingdom | Code 12, Code 5 |
| Azenta Germany GmbH ORG-100022621
|
Griesheim, Germany | Other |
| Greenphire LLC ORG-100041621
|
King Of Prussia, United States | Other |
| PPD Global Central Labs ORG-100046496
|
Zaventem, Belgium | Laboratory analysis |
Locations
5 EU/EEA countries · 12 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Authorised, recruitment pending | 3 | 2 |
| Germany | Authorised, recruitment pending | 3 | 2 |
| Italy | Ended | 6 | 2 |
| Poland | Authorised, recruitment pending | 7 | 2 |
| Spain | Ongoing, recruiting | 7 | 4 |
| Rest of world
United States, United Kingdom
|
— | 32 | — |
Investigational sites
Centre Hospitalier Regional De Marseille
Pédiatrie, 264 Rue Saint Pierre, 13005, Marseille
Assistance Publique Hopitaux De Paris
Pédiatrie, 104 Boulevard Raymond Poincare, 92380, Garches
Universitaetsklinikum Essen AöR
Klinik fuer Neurologie, Hufelandstrasse 55, Holsterhausen, Essen
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)
Department of Neurology, Ismaninger Strasse 22, Au-Haidhausen, Munich
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Unità di Fase I - Unità Operativa Complessa di Neuropsichiatria Infantile, Largo Francesco Vito 1, 00168, Rome
Centro Clinico Nemo
Clinical Research Center Fase I, Piazza Dell'ospedale Maggiore 3, 20162, Milan
Instytut Centrum Zdrowia Matki Polki
N/A, Ul. Rzgowska 281/289, 93-338, Lodz
Szpital Specjalistyczny Im. Ludwika Rydygiera W Krakowie Sp. z o.o.
N/A, Os. Zlotej Jesieni 1, 31-826, Cracow
Hospital Universitario La Paz
Neurologist, Paseo De La Castellana 261, 28046, Madrid
Hospital Universitario Y Politecnico La Fe
Neurologist, Avenida De Fernando Abril Martorell 106, 46026, Valencia
Hospital Universitario Regional De Malaga
Neurology, Avenida De Carlos De Haya S/N, 29010, Malaga
Hospital Sant Joan De Deu Barcelona
Neurology, Passeig De Sant Joan De Deu 2, 08950, Esplugues De Llobregat
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Italy | 2025-05-21 | 2026-02-20 | 2025-05-21 | 2026-02-20 | |
| Spain | 2025-05-29 | 2025-05-29 |
Oversight and notifications
Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77
Corrective measures 1 · Art. 77 CTR
Corrective measure CM-FR-0001
- Member state
- France
- Publication date
- 2025-05-14
- Type
- 3
- Reason
- 7
- Immediate action required
- Yes
- Justification
- In line with the version 6.4 of CTR Q&A / point 1.23, the sponsor is requested to submit a specific SM Part II only in France in order to update its CTA in line with the documentation approved during the appeal procedure within 10 days after the submission of this corrective measure.
Application history
8 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-10-08 | Italy | Acceptable 2025-02-10
|
2025-02-10 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-03-03 | Acceptable | 2025-03-20 | |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2025-05-26 | Acceptable | 2025-07-04 | |
| 4 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-10-24 | Italy | Acceptable 2026-01-14
|
2026-01-14 |
| 5 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2026-03-04 | Italy | Acceptable 2026-01-14
|
2026-03-04 |
| 6 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2026-04-28 | Italy | Acceptable 2026-01-14
|
2026-04-28 |
| 7 | SUBSTANTIAL MODIFICATION | SM-5 | 2026-05-04 | Acceptable | 2026-05-20 | |
| 8 | SUBSTANTIAL MODIFICATION | SM-7 | 2026-05-12 | Acceptable | 2026-05-29 |