700 Participants Needed

Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

Recruiting at 105 trial locations
NG
NG
NG
Overseen ByNovartis Gene Therapies
Age: Any Age
Sex: Any
Trial Phase: Academic
Sponsor: Novartis Pharmaceuticals
Must be taking: OAV-101
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an autosomal recessive, early childhood disease with an incidence of 1:10,000 live births. SMA is the leading cause of infant mortality due to genetic diseases.The purpose of this registry is to assess the long term outcomes of patients with SMA in the context of advances in treatment options and also to characterize and assess long-term safety and effectiveness of OAV-101.

Who Is on the Research Team?

NP

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Are You a Good Fit for This Trial?

Inclusion Criteria

Patients with SMA, genetically confirmed on or after 24 May 2018.
Appropriate consent/assent has been obtained for participation in the registry

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Registry Enrollment

Participants are enrolled in a long-term observational registry to assess outcomes and safety of SMA treatments

15 years
Visits as per normal clinical practice

Follow-up

Participants are monitored for safety and effectiveness after enrollment

15 years
Every 6 months for 2 years, then annually

What Are the Treatments Tested in This Trial?

Interventions

  • Prospective observational registry
  • Zolgensma
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Prospective observational registryExperimental Treatment2 Interventions
This is a prospective, multi center, multinational, non-interventional observational registry.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials
2,963
Recruited
4,275,000+
Founded
1996
Headquarters
Basel, Switzerland
Known For
Precision medicine
Top Products
Gleevec, Cosentyx, Entresto, Kisqali
Dr. Vas Narasimhan profile image

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye profile image

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

AveXis, Inc.

Lead Sponsor

Trials
3
Recruited
137,000+

United BioSource, LLC

Industry Sponsor

Trials
15
Recruited
11,800+
Unbiased ResultsWe believe in providing patients with all the options.
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