Trials Sponsors Conditions Drugs Pricing

Conditions

Spinal Muscular Atrophy Spinal Muscular Atrophy Type 3 Spinal Muscular Atrophy Type II

Outcome Measures

Primary Outcomes (2)

Change from baseline in the 6-Minute Walk Test (6MWT) for ambulatory SMA patients
Assess effectiveness of SPINRAZA® (nusinersen) treatment on mobility and ambulation in ambulatory adult SMA patients, comparing changes in total distance walked in in six minutes from baseline until end of treatment at 30 months..
30 months
Change from baseline in Revised Upper Limb Module (RULM) for weak ambulatory and non-ambulatory SMA patients
Assess the effectiveness of SPINRAZA® (nusinersen) treatment on upper extremity function in ambulatory and non-ambulatory adult SMA patients, comparing change in RULM score from baseline until end of treatment at 30 months.
30 months

Secondary Outcomes (1)

To describe the demographic and clinical characteristics, disease burden, treatment preferences, and subjective assessments of disease progression
6 months to 1 year

Study Arms (2)

Cohort 1

This is a prospective, longitudinal, multi-center, observational study designed to evaluate the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) in ambulatory and non-ambulatory adult patients with SMA. Subjects with SMA II/III that are 18 years to 70 years of age who are planning to initiate treatment with SPINRAZA® (nusinersen) as part of their clinical care plan will be enrolled in this study. This study does not provide SPINRAZA® (nusinersen) or cover costs associated with standard clinical care.These patients will be treated by their respective physicians according to standard clinical practice. Study visits, some of which including standardized assessments of strength and function, will occur at baseline, day 15 after treatment initiation, day 30, day 60, and then 4-month intervals through month 30.

Drug: Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care

Cohort 2

The cohort 2 is a one time survey to gain a better understanding of this adult population and their treatment preferences.

Other: One time survey

Interventions

Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of careDrug

This is an observational study of adult patients with SMA to examine the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) for up to 30 months.

Cohort 1

One time surveyOther

One time survey

Cohort 2

Eligibility Criteria

Age18 Years - 70 Years

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

Sampling MethodNon-Probability Sample

Study Population

Subjects with SMA that are 18 years to 70 years of age who are planning to initiate treatment with SPINRAZA® (nusinersen) as part of their clinical care plan. For Cohort 2 - Subjects with SMA that are 18 years to 70 years with SMA type II/III.

You may qualify if:

Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.

Sponsors & Collaborators

Washington University School of Medicine
Otherlead

Study Sites (11)

Barrow Neurological Institute

Phoenix, Arizona, 85013, United States

Georgetown University

Washington D.C., District of Columbia, 20007, United States

Johns Hopkins

Baltimore, Maryland, 21287, United States

Massachusetts General Hospital-Harvard University

Boston, Massachusetts, 02114, United States

Memorial Healthcare

Owosso, Michigan, 48867, United States

Washington University School of Medicine

St Louis, Missouri, 63110, United States

New York University School of Medicine

New York, New York, 10003, United States

Related Publications (3)

De Vivo DC, Bertini E, Swoboda KJ, Hwu WL, Crawford TO, Finkel RS, Kirschner J, Kuntz NL, Parsons JA, Ryan MM, Butterfield RJ, Topaloglu H, Ben-Omran T, Sansone VA, Jong YJ, Shu F, Staropoli JF, Kerr D, Sandrock AW, Stebbins C, Petrillo M, Braley G, Johnson K, Foster R, Gheuens S, Bhan I, Reyna SP, Fradette S, Farwell W; NURTURE Study Group. Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study. Neuromuscul Disord. 2019 Nov;29(11):842-856. doi: 10.1016/j.nmd.2019.09.007. Epub 2019 Sep 12.
PMID: 31704158BACKGROUND
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: 29091570

Biospecimen

Retention: SAMPLES WITH DNA

5cc of Cerebral Spinal Fluid (CSF) is collected and typically discarded as part of the typical clinical encounter when SPINRAZA® (nusinersen) is administered. This 5cc of CSF will be collected for research purposes at each dosing visit. Blood samples, including whole blood, serum, and plasma (3 teaspoons total) will be collected within seven days of standard of care SPINRAZA® (nusinersen) administration at dose #1, 2, 4, 5, 7, 9 and 11.

MeSH Terms

Conditions

Muscular Atrophy, SpinalSpinal Muscular Atrophies of Childhood

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular DiseasesHeredodegenerative Disorders, Nervous SystemGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

Craig Zaidman, MD
Washington University School of Medicine
PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

First Submitted

October 15, 2018

First Posted

October 17, 2018

Study Start

August 16, 2018

Actual (reported)

Primary Completion

December 31, 2024

Estimated (date passed)

This estimate has passed. The registry has not confirmed an actual date for this milestone.

You may not qualify if:

Revised upper limb score ≤ 3.
Respiratory insufficiency, defined by the medical necessity for invasive or noninvasive ventilation for \>16 hours during a 24-hour period, at screening.
Presence of a symptomatic severe active infection or illness during the screening period that is likely to impact the performance on the clinical assessments.
Prior exposure to SPINRAZA® (nusinersen).
Prior disorder, injury (e.g., upper or lower limb fracture) or surgical procedure which impacts the subject's ability to perform any of the outcome measure testing required in the protocol and from which the subject has not fully recovered or achieved a stable baseline.
Treatment with an investigational drug (e.g., oral albuterol/salbutamol, riluzole, carnitine, creatine, sodium phenylbutyrate, etc.), biological agent, or device within 1-month of Screening or 5 half-lives of study agent, whichever is longer. Patients using intrathecal drug delivery devices, including investigational devices with an active IDE designation in the United States, may be eligible but require Study PI approval prior to enrollment.
Any history of exposure to gene therapy, antisense oligonucleotide therapy, or cell transplantation that was intended for the treatment of SMA.
Ongoing medical condition that according to the Clinical Center Investigator would interfere with the conduct and assessments of the study. Examples are medical disability (e.g., wasting or cachexia, severe anemia, etc.) that would interfere with the assessment of safety or would compromise the ability of the subject to undergo study procedures.
Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.
Males and females with SMA type II or type III, aged 18 to 70 years at the time of enrollment.
Genetic documentation of 5Q SMA homozygous gene deletion, mutation, or compound heterozygote.
Believed to be able to complete the structured interview.
\. Ongoing medical condition that according to the Clinical Center Investigator would interfere with the conduct and assessments of the study.

Spinraza in Adult Spinal Muscular Atrophy

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (1)

Study Arms (2)

Cohort 1

Cohort 2

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (11)

Related Publications (3)

Biospecimen

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

First Submitted

First Posted

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (1)

Study Arms (2)

Cohort 1

Cohort 2

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (11)

Related Publications (3)

Biospecimen

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations