Effect of Nusinersen on Adults With Spinal Muscular Atrophy

Adult Spinal Muscular Atrophy Clinical Trial

Official title:

Effect of Nusinersen on Motor Function in Adult Patients With Spinal Muscular Atrophy Types 2 and 3

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03878030
• Other study ID #: 18-0149
• Status: Completed
• Start date: April 1, 2019
• Est. completion date: July 13, 2021

Study information

• Verified date: March 2023
• Source: Northwell Health
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Observational study of adult patients with spinal muscular atrophy types 2 and 3 receiving nusinersen

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: July 13, 2021
• Est. primary completion date: July 13, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 60 Years

Eligibility

Inclusion Criteria:

• Genetically confirmed 5q SMA

• ability to access intrathecal space for nusinersen injection

Exclusion Criteria:

• Renal impairment

• thrombocytopenia

• inability to access intrathecal space by CT or flouro guided injection

Related Conditions & MeSH terms

• Adult Spinal Muscular Atrophy
• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal

Intervention

Drug:
• nusinersen
Subjects will receive nusinersen and be observed with motor assessments for 24 months

Locations

Country	Name	City	State
United States	Northwell Health Neuroscience	Great Neck	New York

Sponsors (1)

Lead Sponsor	Collaborator
Northwell Health

Country where clinical trial is conducted

United States, 

References & Publications (2)

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 G — View Citation

Mercuri 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 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Two years
Primary	Change in upper limb motor function	Change from baseline to end of study in upper limb module score	Two years
Primary	Change in 6 minute walk test in ambulatory patients	Change in 6 minute walk test from baseline to end of study	Two years
Secondary	Change in pulmonary function by spirometry forced vital capacity (FVC)	Change in FVC from baseline to end of study	Two years
Secondary	Change in 10 meter walk test in ambulatory patients	Change from baseline to end of study in the 10 meter walk test	Two years
Secondary	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	Two years
Secondary	Change in pulmonary function by spirometry forced expiratory volume (FEV)	Change from baseline to end of study in FEV	Two years