Muscular Atrophy, SpinalPANDANCT04419233
Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection
An observational study of Muscular Atrophy, Spinal, run by Biogen at 9 sites in China.
Trial summary
The primary objective of this study is to evaluate the safety of nusinersen sodium injection in the postmarketing setting in China.The secondary objectives are to collect data on the efficacy and the pharmacokinetics of nusinersen sodium injection in the post-marketing setting in China. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Muscular Atrophy, Spinal
- What is being tested?
- Nusinersen Sodium Injection
- Type of treatment
- Injection or infusion
- How many people
- 50 participants
- Where
- 9 sitesChina
- Study dates
- 18 Nov 2020to 21 Nov 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants who are prescribed nusinersen sodium injection in China according to the local marketing authorization (including documentation of 5q SMA diagnosis), agree to the collection and use of data as specified in the protocol, and meet the eligibility criteria.
Who may be able to join
- Ability of the participant or his/her legally authorized representative (e.g., parent or legal guardian), as appropriate and applicable, to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local subject privacy regulations.
- The decision to newly prescribe nusinersen sodium injection according to the local marketing authorization, including documentation of 5q SMA diagnosis, has been made by the Investigator and agreed to by the participant (or legally authorized representative)
Who cannot join
- Hypersensitivity to the active substance or any of the excipients of nusinersen sodium injection.
- Current or past administration of nusinersen sodium injection, in either a commercial or a clinical study setting.
- Other unspecified reasons that, in the opinion of the Investigator or the Sponsor, make the participant unsuitable for enrollment.
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
China9 sites
Research Site
Beijing, Xicheng, 100045
Research Site
Changchun, Jilin, 130021
Research Site
Chengdu, Sichuan, 610041
Research Site
Fuzhou, Fujian, UNK
Research Site
Guangzhou, Guangdong, UNK
Research Site
Hangzhou, Zhejiang, 310052
Research Site
Minhang, Shanghai Municipality, 201103
Research Site
Suzhou, Jiangsu, UNK
Research Site
Xicheng, Beijing Municipality, 100045
About this study
Official title: A Multicenter, Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection When Used in Routine Medical Practice in China
The primary objective of this study is to evaluate the safety of nusinersen sodium injection in the postmarketing setting in China.The secondary objectives are to collect data on the efficacy and the pharmacokinetics of nusinersen sodium injection in the post-marketing setting in China.
- Conditions
- Muscular Atrophy, Spinal
- Being tested
- Nusinersen Sodium Injection
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nusinersen Sodium InjectionDrug
Also called BIIB058, ISIS 396443, Spinraza
Administered as specified in the treatment arm.
| Study group | Role | What they receive |
|---|---|---|
| All Participants | Participants with 5q SMA and who were prescribed with nusinersen sodium injection in China according to the local marketing authorization. Receives: Drug: Nusinersen Sodium Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose results in death, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect, or is a medically important event.
Measured: Day 0 up to End of Treatment (2 Years)
Secondary outcomes (5)
Percentage of Participants who Achieved World Health Organization (WHO) Motor Milestone
WHO Multicentre Growth Reference Study (MGRS), a component to assess gross motor development and is usually assessed in terms of age of achievement of motor milestones. Six distinct gross motor milestones criteria are as follows: sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone.
Measured: Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
Hammersmith Infant Neurological Examination (HINE) Section 2 Scores
Section 2 of the HINE is used to assess motor milestones of the participants. It is composed of 8 motor milestone categories: voluntary grasp, ability to kick in supine position, head control, rolling, sitting, crawling, standing, and walking.
Measured: Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
Number of Participants with Ventilatory Support
Measured: Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
Plasma Concentrations of Nusinersen Sodium Injection
Measured: Pre-dose and multiple time points post-dose on Day 0, post-dose on Day 28 and pre-dose every 4 months up to end of treatment (2 years)
Cerebrospinal Fluid (CSF) Concentrations of Nusinersen Sodium Injection
Measured: pre-dose on Day 0, Day 63, and Every 4 Months Up to End of Treatment (2 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2020
- Study start18 Nov 2020
- Primary completion21 Nov 2023
- Study completion21 Nov 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Muscular Atrophy, SpinalStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04419233
- Sponsor's protocol ID
- 232SM402
- Acronym
- PANDA
- Status last verified
- Dec 2023
- First submitted
- 3 Jun 2020
- Last updated
- 7 Dec 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Publications
Jiang Y, Wang Y, Xiong H, Li W, Luo R, Chen W, Yin F, Lu J, Liang J, Chen WJ, Lu X, Wang H, Tang J, Monine M, Makepeace C, Jin X, Foster R, Chin R, Berger Z. A Post-Marketing Surveillance Study of Nusinersen for Spinal Muscular Atrophy in Routine Medical Practice in China: Interim Results. Adv Ther. 2024 Jul;41(7):2743-2756. doi: 10.1007/s12325-024-02852-7. Epub 2024 May 9.
PubMed 38722537
Study at a glance
From the registry record, last updated by the sponsor on 7 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection When Used in Routine Medical Practice in China
- ClinicalTrials.gov ID
- NCT04419233
- Acronym / sponsor ID
- PANDA / 232SM402
- Conditions
- Muscular Atrophy, Spinal
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 18 Nov 2020 (actual)
- Primary completion
- 21 Nov 2023 (actual)
- Study completion
- 21 Nov 2023 (actual)
- First posted
- 5 Jun 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to evaluate the safety of nusinersen sodium injection in the postmarketing setting in China.The secondary objectives are to collect data on the efficacy and the pharmacokinetics of nusinersen sodium injection in the post-marketing setting in China.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All Participants | – | Drug: Nusinersen Sodium InjectionParticipants with 5q SMA and who were prescribed with nusinersen sodium injection in China according to the local marketing authorization. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants who are prescribed nusinersen sodium injection in China according to the local marketing authorization (including documentation of 5q SMA diagnosis), agree to the collection and use of data as specified in the protocol, and meet the eligibility criteria.
Key inclusion criteria (2 listed)
- Ability of the participant or his/her legally authorized representative (e.g., parent or legal guardian), as appropriate and applicable, to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local subject privacy regulations.
- The decision to newly prescribe nusinersen sodium injection according to the local marketing authorization, including documentation of 5q SMA diagnosis, has been made by the Investigator and agreed to by the participant (or legally authorized representative)
Key exclusion criteria (4 listed)
- Hypersensitivity to the active substance or any of the excipients of nusinersen sodium injection.
- Current or past administration of nusinersen sodium injection, in either a commercial or a clinical study setting.
- Other unspecified reasons that, in the opinion of the Investigator or the Sponsor, make the participant unsuitable for enrollment.
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nusinersen Sodium InjectionAlso: BIIB058, ISIS 396443, SpinrazaAdministered as specified in the treatment arm. | Drug | All Participants |
Primary outcome
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Day 0 up to End of Treatment (2 Years)
An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose results in death, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect, or is a medically important event.
5 secondary outcomes
Percentage of Participants who Achieved World Health Organization (WHO) Motor Milestone
Time frame: Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
Hammersmith Infant Neurological Examination (HINE) Section 2 Scores
Time frame: Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
Number of Participants with Ventilatory Support
Time frame: Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
Plasma Concentrations of Nusinersen Sodium Injection
Time frame: Pre-dose and multiple time points post-dose on Day 0, post-dose on Day 28 and pre-dose every 4 months up to end of treatment (2 years)
Cerebrospinal Fluid (CSF) Concentrations of Nusinersen Sodium Injection
Time frame: pre-dose on Day 0, Day 63, and Every 4 Months Up to End of Treatment (2 years)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Dec 2023.
- Derived from this study: PubMed 38722537Jiang Y, Wang Y, Xiong H, Li W, Luo R, Chen W, Yin F, Lu J, Liang J, Chen WJ, Lu X, Wang H, Tang J, Monine M, Makepeace C, Jin X, Foster R, Chin R, Berger Z. A Post-Marketing Surveillance Study of Nusinersen for Spinal Muscular Atrophy in Routine Medical Practice in China: Interim Results. Adv Ther. 2024 Jul;41(7):2743-2756. doi: 10.1007/s12325-024-02852-7. Epub 2024 May 9.
- Individual participant dataSharing planned: Yes. In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Biogen
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Muscular Atrophy, Spinal, run by Biogen at 9 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Day 0 up to End of Treatment (2 Years)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04419233, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Dec 2023.