Adult Spinal Muscular AtrophyNCT03878030
Effect of Nusinersen on Adults With Spinal Muscular Atrophy
An observational study of Adult Spinal Muscular Atrophy, run by Northwell Health at 1 site in United States.
Trial summary
Observational study of adult patients with spinal muscular atrophy types 2 and 3 receiving nusinersen Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Adult Spinal Muscular Atrophy
- What is being tested?
- nusinersen
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Apr 2019to 13 Jul 2021
- 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 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Study population: The population to be studied are a homogenous group of adult subjects with 5q SMA seeking treatment with nusinersen. The subjects will have a broad phenotype spectrum of motor weakness including ability to ambulate, non-ambulatory and varying degrees of upper limb motor function and respiratory and swallow abilities, with some more affected and requiring invasive ventilation or NIV, and/or PEG tubes for nutrition.
Who may be able to join
- Genetically confirmed 5q SMA
- ability to access intrathecal space for nusinersen injection
Who cannot join
- Renal impairment
- thrombocytopenia
- inability to access intrathecal space by CT or flouro guided injection
Trial locations and contacts
1 site in 1 country.
United States1 site
Northwell Health Neuroscience
Great Neck, New York, 11021
About this study
Official title: Effect of Nusinersen on Motor Function in Adult Patients With Spinal Muscular Atrophy Types 2 and 3
Observational study of adult patients with spinal muscular atrophy types 2 and 3 receiving nusinersen
Read the detailed description
Observational study to assess effects of nusinersen on motor function in adult patients with spinal muscular atrophy who are both ambulatory and non-ambulatory. Subjects will receive standard of care with nusinersen intrathecal injection and undergo baseline and every 6 month motor assessments and pulmonary function testing during the first two years of treatment with nusinersen.
- Conditions
- Adult Spinal Muscular Atrophy
- Being tested
- nusinersen
- 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.
nusinersenDrug
Also called Spinraza
Subjects will receive nusinersen and be observed with motor assessments for 24 months
| Study group | Role | What they receive |
|---|---|---|
| Subjects with spinal muscular atrophy types 2 and 3 | Intrathecal nusinersen will be administered to all subjects per FDA approved label. Receives: Drug: nusinersen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in quantitative motor function
Primary Endpoint: Change from baseline to end of study in quantitative motor strength by dynamometry of upper limb muscles - shoulder abduction and elbow flexion/extension.
Measured: Two years
Change in upper limb motor function
Change from baseline to end of study in upper limb module score
Measured: Two years
Change in 6 minute walk test in ambulatory patients
Change in 6 minute walk test from baseline to end of study
Measured: Two years
Secondary outcomes (4)
Change in pulmonary function by spirometry forced vital capacity (FVC)
Change in FVC from baseline to end of study
Measured: Two years
Change in 10 meter walk test in ambulatory patients
Change from baseline to end of study in the 10 meter walk test
Measured: Two years
Change in compound muscle action potential (CMAP) amplitude by nerve conduction velocity (NCV) criteria
Change from baseline to end of study in CMAP amplitude of responses from median, ulnar and peroneal motor nerves
Measured: Two years
Change in pulmonary function by spirometry forced expiratory volume (FEV)
Change from baseline to end of study in FEV
Measured: Two years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2019
- Study start1 Apr 2019
- Primary completion13 Jul 2021
- Study completion13 Jul 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Adult Spinal Muscular AtrophyStudies of the same condition, open and under way.
- More trials run by Northwell HealthThe 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northwell Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Anthony Geraci, MD, Principal investigator, Northwell Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03878030
- Sponsor's protocol ID
- 18-0149
- Status last verified
- Mar 2023
- First submitted
- 10 Mar 2019
- Last updated
- 20 Mar 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, Chiriboga CA, Saito K, Servais L, Tizzano E, Topaloglu H, Tulinius M, Montes J, Glanzman AM, Bishop K, Zhong ZJ, Gheuens S, Bennett CF, Schneider E, Farwell W, De Vivo DC; ENDEAR Study Group. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. N Engl J Med. 2017 Nov 2;377(18):1723-1732. doi: 10.1056/NEJMoa1702752.
PubMed 29091570Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, Connolly AM, Iannaccone ST, Kirschner J, Kuntz NL, Saito K, Shieh PB, Tulinius M, Mazzone ES, Montes J, Bishop KM, Yang Q, Foster R, Gheuens S, Bennett CF, Farwell W, Schneider E, De Vivo DC, Finkel RS; CHERISH Study Group. Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy. N Engl J Med. 2018 Feb 15;378(7):625-635. doi: 10.1056/NEJMoa1710504.
PubMed 29443664
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Nusinersen on Motor Function in Adult Patients With Spinal Muscular Atrophy Types 2 and 3
- ClinicalTrials.gov ID
- NCT03878030
- Acronym / sponsor ID
- 18-0149
- Conditions
- Adult Spinal Muscular Atrophy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Northwell HealthUniversity, hospital or other
- Study start
- 1 Apr 2019 (actual)
- Primary completion
- 13 Jul 2021 (actual)
- Study completion
- 13 Jul 2021 (actual)
- First posted
- 18 Mar 2019 (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.
Observational study of adult patients with spinal muscular atrophy types 2 and 3 receiving nusinersen
Detailed description
Observational study to assess effects of nusinersen on motor function in adult patients with spinal muscular atrophy who are both ambulatory and non-ambulatory. Subjects will receive standard of care with nusinersen intrathecal injection and undergo baseline and every 6 month motor assessments and pulmonary function testing during the first two years of treatment with nusinersen.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Subjects with spinal muscular atrophy types 2 and 3 | – | Drug: nusinersenIntrathecal nusinersen will be administered to all subjects per FDA approved label. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- The population to be studied are a homogenous group of adult subjects with 5q SMA seeking treatment with nusinersen. The subjects will have a broad phenotype spectrum of motor weakness including ability to ambulate, non-ambulatory and varying degrees of upper limb motor function and respiratory and swallow abilities, with some more affected and requiring invasive ventilation or NIV, and/or PEG tubes for nutrition.
Key inclusion criteria (2 listed)
- Genetically confirmed 5q SMA
- ability to access intrathecal space for nusinersen injection
Key exclusion criteria (3 listed)
- Renal impairment
- thrombocytopenia
- inability to access intrathecal space by CT or flouro guided 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 |
|---|---|---|
| nusinersenAlso: SpinrazaSubjects will receive nusinersen and be observed with motor assessments for 24 months | Drug | Subjects with spinal muscular atrophy types 2 and 3 |
Primary outcome
Change in quantitative motor function
Time frame: Two years
Primary Endpoint: Change from baseline to end of study in quantitative motor strength by dynamometry of upper limb muscles - shoulder abduction and elbow flexion/extension.
Change in upper limb motor function
Time frame: Two years
Change from baseline to end of study in upper limb module score
Change in 6 minute walk test in ambulatory patients
Time frame: Two years
Change in 6 minute walk test from baseline to end of study
4 secondary outcomes
Change in pulmonary function by spirometry forced vital capacity (FVC)
Time frame: Two years
Change in 10 meter walk test in ambulatory patients
Time frame: Two years
Change in compound muscle action potential (CMAP) amplitude by nerve conduction velocity (NCV) criteria
Time frame: Two years
Change in pulmonary function by spirometry forced expiratory volume (FEV)
Time frame: Two 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 20 Mar 2023.
- Results publication: PubMed 29091570Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, Chiriboga CA, Saito K, Servais L, Tizzano E, Topaloglu H, Tulinius M, Montes J, Glanzman AM, Bishop K, Zhong ZJ, Gheuens S, Bennett CF, Schneider E, Farwell W, De Vivo DC; ENDEAR Study Group. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. N Engl J Med. 2017 Nov 2;377(18):1723-1732. doi: 10.1056/NEJMoa1702752.
- Results publication: PubMed 29443664Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, Connolly AM, Iannaccone ST, Kirschner J, Kuntz NL, Saito K, Shieh PB, Tulinius M, Mazzone ES, Montes J, Bishop KM, Yang Q, Foster R, Gheuens S, Bennett CF, Farwell W, Schneider E, De Vivo DC, Finkel RS; CHERISH Study Group. Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy. N Engl J Med. 2018 Feb 15;378(7):625-635. doi: 10.1056/NEJMoa1710504.
- Individual participant dataSharing planned: No
Related trials for Adult Spinal Muscular Atrophy
Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Recruiting
SMAdult - Monitoring of Movement on Spinal Muscular Atrophy
Spinal Amyotrophy
Active, not recruitingPhase 1
A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec
Healthy Volunteer, Muscular Atrophy, Spinal
RecruitingObservational study
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
Spinal Muscular Atrophy
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.
Anthony Geraci, MD principal investigator
Northwell Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Adult Spinal Muscular Atrophy, run by Northwell Health at 1 site in United States.
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 3 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 20 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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?
Change in quantitative motor function (time frame: Two years). The study lists 3 primary outcomes in all. 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 NCT03878030, which also lists 2 linked publications.
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 20 Mar 2023.