Spinal Muscular AtrophyNCT05187260
Antisense Oligonucleotide for Spinal Muscular Atrophy
An observational study of Spinal Muscular Atrophy, run by First Affiliated Hospital of Fujian Medical University at 3 sites in China.
Trial summary
This is a longitudinal, multiple-center, observational study of patients genetically confirmed chromosome 5q SMA to monitor the efficacy, safety, tolerability of SPINRAZA® (nusinersen) for up to 24 months. Read more about this study
- Who is it for?
- Ages 1 week to 50 yearsHealthy volunteers can take part
- Conditions the study is for
- Spinal Muscular Atrophy
- What is being tested?
- Nusinersen
- How many people
- 1,000 participants
- Where
- 3 sitesChina
- Study dates
- 1 Jan 2022to 31 Jan 2024
- 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 1 week to 50 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: Patients genetically confirmed 5q SMA including types I, II and III
Who may be able to join
- Patients genetically confirmed 5q SMA including types I, II and III, who are planning to initiate treatment with SPINRAZA® (nusinersen) as part of their clinical care plan.
- Non-5q SMA patients undergoing clinical standard lumbar puncture
- Non-SMA subjects including Asymptomatic carriers of SMA, relatives of SMA patients and carriers, and patients undergoing clinical standard lumbar puncture
- Participants or Parent(s)/legal guardian(s) willing and able to complete the informed consent process
Who cannot join
- Contraindication for lumbar puncture
- Inability to access intrathecal space for nusinersen injection
Trial locations and contacts
3 sites in 1 country.
China3 sites, 3 recruiting
- Recruiting
Department of Neurology, Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100010
Yi Dai, MD,PhD, 13811177531, pumchdy@pumch.cn
Yi Dai, MD, PhD
- Recruiting
Department of Pediatrics, Fujian Medical University Union Hospital
Fuzhou, Fujian, 350001
Jun Hu, MD, PhD, 18950472106, hujun2252@126.com
Jun Hu, MD, PhD
- Not yet recruiting
Department of Neurology, First Affiliated Hospital Fujian Medical University
Fuzhou, Fujian, 350005
Yi Lin, MD, PhD, 13615039153, linyi7811@163.com
Wan-Jin Chen, MD, PhD
About this study
Official title: Antisense Oligonucleotide Therapy in Spinal Muscular Atrophy: An Observational Study in China
This is a longitudinal, multiple-center, observational study of patients genetically confirmed chromosome 5q SMA to monitor the efficacy, safety, tolerability of SPINRAZA® (nusinersen) for up to 24 months.
Read the detailed description
SPINRAZA® (nusinersen) is an antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Nusinersen, approved by the FDA for treatment of SMA in 2016, was approved by the Chinese National Medical Products Administration in 2019.
This is a prospective, longitudinal, multi-center, observational study designed to evaluate the efficacy, safety, tolerability and of nusinersen in patients genetically confirmed chromosome 5q SMA in China. Subjects with SMA I/II/III who are planning to initiate treatment with nusinersen will be enrolled in this study. All patients will be treated by their physicians according to standard clinical practice. SPINRAZA® (nusinersen) is administered as an intrathecal injection. A total of 5ml of cerebrospinal fluid (CSF) will be removed prior to administration of SPINRAZA® (nusinersen), which will be collected by the study. Neurofilament light chain (NfL) in CSF and blood will be assessed for the efficacy of nusinersen, as well as motor and pulmonary function.
There will be a total of nine visits. All the patients with 5q SMA receiving nusinersen will be visited face to face at baseline, day 14, day 28 and day 63 after treatment initiation, and then 4-month intervals through month 24/22.
- Conditions
- Spinal Muscular Atrophy
- Being tested
- Nusinersen
- Keywords
- Spinal Muscular Atrophy, Antisense oligonucleotide, Neurofilament light chain
- Medical subject headings
- Muscular Atrophy, Spinal, Charcot-Marie-Tooth disease, Type 1F
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NusinersenDrug
SPINRAZA® (nusinersen) prescribed as part of standard of care
| Study group | Role | What they receive |
|---|---|---|
| 5q SMA type I | Not described Receives: Drug: Nusinersen | |
| 5q SMA type II | Not described Receives: Drug: Nusinersen | |
| 5q SMA type III | Not described Receives: Drug: Nusinersen | |
| Non-5q SMA | Not described | |
| Non-SMA subjects | Including asymptomatic carriers of SMA, relatives of SMA patients and carriers, and patients undergoing clinical standard lumbar puncture |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in CSF Neurofilament Light Chain levels since baseline
Measured by Single-molecule Array
Measured: up to 24months
Secondary outcomes (1)
Change in serum Neurofilament Light Chain levels since baseline
Measured by Single-molecule Array
Measured: up to 24months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jan 2022
- Study start1 Jan 2022
- Primary completion31 Jan 2024 (estimated)
- Study completion31 Jan 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular AtrophyStudies of the same condition, open and under way.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- First Affiliated Hospital of Fujian Medical University
- Sponsor type
- University, hospital or other
- Collaborators
- Fujian Medical University Union Hospital, Peking Union Medical College Hospital
- Responsible party
- Principal investigator, Wan-Jin Chen, First Affiliated Hospital of Fujian Medical University
- Investigators
- Wan-Jin Chen, Principal investigator, First Affiliated Hospital Fujian Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05187260
- Sponsor's protocol ID
- MRCTA,ECFAH of FMU [2021]489
- Status last verified
- Jan 2022
- First submitted
- 25 Dec 2021
- Last updated
- 26 Jan 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Antisense Oligonucleotide Therapy in Spinal Muscular Atrophy: An Observational Study in China
- ClinicalTrials.gov ID
- NCT05187260
- Acronym / sponsor ID
- MRCTA,ECFAH of FMU [2021]489
- Conditions
- Spinal Muscular Atrophy
- Keywords
- Spinal Muscular Atrophy, Antisense oligonucleotide, Neurofilament light chain
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 1,000 participants (estimated)
- Lead sponsor
- First Affiliated Hospital of Fujian Medical UniversityUniversity, hospital or other
- Collaborators
- Fujian Medical University Union Hospital, Peking Union Medical College Hospital
- Study start
- 1 Jan 2022 (actual)
- Primary completion
- 31 Jan 2024 (estimated)
- Study completion
- 31 Jan 2024 (estimated)
- First posted
- 11 Jan 2022 (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.
This is a longitudinal, multiple-center, observational study of patients genetically confirmed chromosome 5q SMA to monitor the efficacy, safety, tolerability of SPINRAZA® (nusinersen) for up to 24 months.
Detailed description
SPINRAZA® (nusinersen) is an antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Nusinersen, approved by the FDA for treatment of SMA in 2016, was approved by the Chinese National Medical Products Administration in 2019.
This is a prospective, longitudinal, multi-center, observational study designed to evaluate the efficacy, safety, tolerability and of nusinersen in patients genetically confirmed chromosome 5q SMA in China. Subjects with SMA I/II/III who are planning to initiate treatment with nusinersen will be enrolled in this study. All patients will be treated by their physicians according to standard clinical practice. SPINRAZA® (nusinersen) is administered as an intrathecal injection. A total of 5ml of cerebrospinal fluid (CSF) will be removed prior to administration of SPINRAZA® (nusinersen), which will be collected by the study. Neurofilament light chain (NfL) in CSF and blood will be assessed for the efficacy of nusinersen, as well as motor and pulmonary function.
There will be a total of nine visits. All the patients with 5q SMA receiving nusinersen will be visited face to face at baseline, day 14, day 28 and day 63 after treatment initiation, and then 4-month intervals through month 24/22.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| 5q SMA type I | – | Drug: Nusinersen |
| 5q SMA type II | – | Drug: Nusinersen |
| 5q SMA type III | – | Drug: Nusinersen |
| Non-5q SMA | – | – |
| Non-SMA subjects | – | –Including asymptomatic carriers of SMA, relatives of SMA patients and carriers, and patients undergoing clinical standard lumbar puncture |
Who may qualify
- Ages
- 1 week to 50 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- Patients genetically confirmed 5q SMA including types I, II and III
Key inclusion criteria (4 listed)
- Patients genetically confirmed 5q SMA including types I, II and III, who are planning to initiate treatment with SPINRAZA® (nusinersen) as part of their clinical care plan.
- Non-5q SMA patients undergoing clinical standard lumbar puncture
- Non-SMA subjects including Asymptomatic carriers of SMA, relatives of SMA patients and carriers, and patients undergoing clinical standard lumbar puncture
- Participants or Parent(s)/legal guardian(s) willing and able to complete the informed consent process
Key exclusion criteria (2 listed)
- Contraindication for lumbar puncture
- Inability to access intrathecal space for nusinersen injection
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 |
|---|---|---|
| NusinersenSPINRAZA® (nusinersen) prescribed as part of standard of care | Drug | 5q SMA type I, 5q SMA type II, 5q SMA type III |
Primary outcome
Change in CSF Neurofilament Light Chain levels since baseline
Time frame: up to 24months
Measured by Single-molecule Array
1 secondary outcome
Change in serum Neurofilament Light Chain levels since baseline
Time frame: up to 24months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jan 2022.
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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.
Wan-Jin Chen principal investigator
First Affiliated Hospital Fujian Medical University
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Spinal Muscular Atrophy, run by First Affiliated Hospital of Fujian Medical University at 3 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 4 inclusion and 2 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: status not confirmed, as of the sponsor's last update on 26 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Yi Lin, MD, PhD (13615039153, linyi7811@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in CSF Neurofilament Light Chain levels since baseline (time frame: up to 24months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 26 Jan 2022.