Zolgensma for Spinal Muscular Atrophy

Recruiting at 128 trial locations

Overseen By Novartis Gene Therapies

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Novartis Pharmaceuticals

Must be taking: OAV-101

Disqualifiers: Interventional trial participation

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 6

700 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial focuses on understanding the long-term outcomes for individuals with spinal muscular atrophy (SMA), a serious genetic disorder affecting movement. The researchers aim to evaluate the effectiveness and safety of the gene therapy Zolgensma over time. Individuals who have received Zolgensma and have a confirmed genetic diagnosis of SMA are suitable candidates for this trial. Participants should not be enrolled in any other clinical trials for SMA treatments. As a Phase 4 trial, this study involves a treatment already approved by the FDA and proven effective, aiming to understand how it benefits a broader patient population.

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. It might be best to discuss this with the trial coordinators or your doctor.

What is the safety track record for Zolgensma?

Research has shown that Zolgensma is usually well-tolerated, but some side effects have been observed. Common reactions include increased liver enzymes and vomiting, occurring in 5% or more of patients. Other reported issues include fever and a low number of platelets, which can affect blood clotting. Most side effects appear soon after treatment. Patients and families should be aware of these possible side effects when considering participation in a clinical trial. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Zolgensma is unique because it targets the genetic root of spinal muscular atrophy (SMA) with a one-time gene therapy infusion. Unlike traditional treatments like Spinraza, which requires regular spinal injections, or Evrysdi, a daily oral medication, Zolgensma delivers a functional copy of the SMN1 gene directly to motor neuron cells. This approach has the potential to provide long-lasting effects from a single dose, which is a game-changer for patients and families seeking a more convenient and potentially more effective solution. Researchers are excited about Zolgensma because it offers the possibility of significant improvement in motor function and quality of life for patients with SMA.

What is the effectiveness track record for Zolgensma in treating spinal muscular atrophy?

Research has shown that Zolgensma, also known as onasemnogene abeparvovec, effectively treats spinal muscular atrophy (SMA). In one study, 91% of patients with SMA type 1 survived without needing long-term breathing support, marking a significant improvement. The drug enhances patients' muscle function and overall well-being, improving important health outcomes. Real-world data supports these findings, indicating that the treatment leads to better results for many patients. Zolgensma works by addressing the genetic issue causing SMA, specifically the lack of the SMN protein needed for muscle control. Overall, the evidence suggests that Zolgensma is a promising treatment for SMA. ^{6 7 8 9 10}

Who Is on the Research Team?

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?

Group I: Prospective observational registry Experimental Treatment 2 Interventions

This is a prospective, multi center, multinational, non-interventional observational registry.

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

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

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+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38250783/

Real-World Outcomes in Patients with Spinal Muscular ... These real-world outcomes support findings from the interventional trial program and demonstrate effectiveness of onasemnogene abeparvovec over a large patient ...

2. nature.com nature.com/articles/s41434-025-00535-8

Fatal outcomes following onasemnogene abeparvovec in ... That study demonstrated a significant improvement in clinical outcomes: 91% of enrolled SMA type 1 patients survived without permanent ...

3. jpccr.eu jpccr.eu/pdf-193633-119783?filename=Clinical%20outcomes%20of.pdf

Clinical outcomes of Onasemnogene Abeparvovec use in ... The drug significantly enhances key clinical outcomes and is relatively safe to use, with only a few serious adverse effects attributed to the ...

4. novartis.com novartis.com/news/media-releases/new-novartis-phase-iii-data-demonstrate-meaningful-efficacy-and-safety-results-intrathecal-onasemnogene-abeparvovec-broad-patient-population-sma

New Novartis Phase III data demonstrate meaningful ... In the Phase III STEER study, treatment with OAV101 IT led to a statistically significant 2.39-point improvement on the Hammersmith Functional ...

5. thelancet.com thelancet.com/journals/lanepe/article/PIIS2666-7762(24)00259-X/fulltext

Efficacy and safety of gene therapy with onasemnogene ... Five-year extension results of the phase 1 START trial of onasemnogene abeparvovec in spinal muscular atrophy. JAMA Neurol. 2021; 78:834-841. Crossref · Scopus ...

6. zolgensma.com zolgensma.com/safety

Safety & Side Effects The most frequently experienced adverse reactions (incidence ≥5%) were elevated liver enzymes and vomiting. Reports of thrombotic microangiopathy (TMA), ...

7. zolgensma-hcp.com zolgensma-hcp.com/clinical-trials/safety-data

Safety data | ZOLGENSMA® (onasemnogene abeparvovec ... The most common side effects that occurred in 5% or more of the children who were treated with ZOLGENSMA were elevated liver enzymes and vomiting. Safety data ...

8. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39840097/

Comprehensive analysis of adverse events associated with ... Elevated liver enzymes, fever, vomiting, and thrombocytopenia were the most common adverse reactions. Most adverse events manifested within the ...

9. medicalnewstoday.com medicalnewstoday.com/articles/drugs-zolgensma-side-effects

Zolgensma side effects: Common, mild, and serious As with other drugs, Zolgensma (onasemnogene abeparvovec-xio) can cause side effects, such as elevated liver enzymes and vomiting.

10. curesma.org curesma.org/wp-content/uploads/2021/04/Apr-2021-caregivers-guide-to-zolgensma.pdf

A Caregiver's Guide to ZOLGENSMA The most common side effects that occurred in patients treated with ZOLGENSMA were elevated liver enzymes and vomiting. The safety information provided here is ...