Antisense Oligonucleotide for Spinal Muscular Atrophy

Spinal Muscular Atrophy Clinical Trial

Official title:

Antisense Oligonucleotide Therapy in Spinal Muscular Atrophy: An Observational Study in China

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05187260
• Other study ID #: MRCTA,ECFAH of FMU [2021]489
• Status: Recruiting
• Start date: January 1, 2022
• Est. completion date: January 31, 2024

Study information

• Verified date: January 2022
• Source: First Affiliated Hospital of Fujian Medical University
• Contact: Yi Lin, MD, PhD
• Phone: 13615039153
• Email: linyi7811[@]163.com
• Is FDA regulated: No
• Study type: Observational

Clinical 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 1000
• Est. completion date: January 31, 2024
• Est. primary completion date: January 31, 2024
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: N/A to 50 Years

Eligibility

Inclusion Criteria:

• 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

Exclusion Criteria:

• Contraindication for lumbar puncture

• Inability to access intrathecal space for nusinersen injection

Related Conditions & MeSH terms

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

Intervention

Drug:
• Nusinersen
SPINRAZA® (nusinersen) prescribed as part of standard of care

Locations

Country	Name	City	State
China	Department of Neurology, Peking Union Medical College Hospital	Beijing	Beijing
China	Department of Neurology, First Affiliated Hospital Fujian Medical University	Fuzhou	Fujian
China	Department of Pediatrics, Fujian Medical University Union Hospital	Fuzhou	Fujian

Sponsors (3)

Lead Sponsor	Collaborator
First Affiliated Hospital of Fujian Medical University	Fujian Medical University Union Hospital, Peking Union Medical College Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in CSF Neurofilament Light Chain levels since baseline	Measured by Single-molecule Array	up to 24months
Secondary	Change in serum Neurofilament Light Chain levels since baseline	Measured by Single-molecule Array	up to 24months