NCT03878030

Brief Summary

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Apr 2019

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 10, 2019

Completed
8 days until next milestone

First Posted

Study publicly available on registry

March 18, 2019

Completed
14 days until next milestone

Study Start

First participant enrolled

April 1, 2019

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 13, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 13, 2021

Completed
Last Updated

March 20, 2023

Status Verified

March 1, 2023

Enrollment Period

2.3 years

First QC Date

March 10, 2019

Last Update Submit

March 15, 2023

Conditions

Adult Spinal Muscular Atrophy

Outcome Measures

Primary Outcomes (3)

  • 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

  • Change in upper limb motor function

    Change from baseline to end of study in upper limb module score

    Two years

  • Change in 6 minute walk test in ambulatory patients

    Change in 6 minute walk test from baseline to end of study

    Two years

Secondary Outcomes (4)

  • Change in pulmonary function by spirometry forced vital capacity (FVC)

    Two years

  • Change in 10 meter walk test in ambulatory patients

    Two years

  • Change in compound muscle action potential (CMAP) amplitude by nerve conduction velocity (NCV) criteria

    Two years

  • Change in pulmonary function by spirometry forced expiratory volume (FEV)

    Two years

Study Arms (1)

Subjects with spinal muscular atrophy types 2 and 3

Intrathecal nusinersen will be administered to all subjects per FDA approved label.

Drug: nusinersen

Interventions

nusinersenDRUG

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

Also known as: Spinraza
Subjects with spinal muscular atrophy types 2 and 3

Eligibility Criteria

Age18 Years - 60 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)
Sampling MethodProbability Sample
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.

You may qualify if:

  • Genetically confirmed 5q SMA
  • ability to access intrathecal space for nusinersen injection

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Northwell Health Neuroscience

Great Neck, New York, 11021, United States

Location

Related 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 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.

    PMID: 29091570RESULT

  • 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 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.

    PMID: 29443664RESULT

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Interventions

nusinersen

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular Diseases

Study Officials

  • Anthony Geraci, MD

    Northwell Health

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 10, 2019

First Posted

March 18, 2019

Study Start

April 1, 2019

Primary Completion

July 13, 2021

Study Completion

July 13, 2021

Last Updated

March 20, 2023

Record last verified: 2023-03

Data Sharing

IPD Sharing
Will not share

Locations

US(1)