Nusinersen for Spinal Muscular Atrophy

(NURTURE Trial)

No longer recruiting at 46 trial locations

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Biogen

Must not be taking: Investigational drugs

Disqualifiers: Hypoxemia, Clinical SMA signs, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 2891 days

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines how well the medicine Nusinersen works for babies with spinal muscular atrophy (SMA) before symptoms appear. The main goal is to determine if Nusinersen can delay or prevent serious breathing problems or death. Eligible participants are babies under six weeks old with a genetic diagnosis of SMA but no symptoms. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial information does not specify whether participants must stop taking their current medications. It's best to discuss this with the study team or your doctor.

Is there any evidence suggesting that Nusinersen is likely to be safe for humans?

Research has shown that nusinersen has been studied for its safety in treating spinal muscular atrophy (SMA). In long-term studies, patients generally handled the treatment well. Common side effects include headache, back pain, and nausea, but these were not severe for most people.

Nusinersen is approved in over 71 countries, suggesting it is safe for treating SMA. This approval indicates widespread use, offering more information on its safety. However, like any treatment, risks exist, so discussing any concerns with the trial team or your doctor is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for SMA?

Unlike the standard treatments for Spinal Muscular Atrophy (SMA) that often target symptoms or require frequent administration, Nusinersen works by enhancing the production of a critical protein called SMN, which is vital for motor neuron survival. This drug is administered directly into the spinal fluid via an intrathecal injection, allowing it to specifically target the central nervous system more effectively. Researchers are excited about Nusinersen because it offers a new approach to addressing the underlying cause of SMA, potentially leading to more significant and lasting improvements for patients.

What evidence suggests that Nusinersen might be an effective treatment for spinal muscular atrophy?

Research has shown that Nusinersen, which participants in this trial will receive, effectively treats spinal muscular atrophy (SMA). Studies have found that Nusinersen improves muscle strength and movement abilities in people with SMA. Evidence from earlier research also suggests that Nusinersen can delay or prevent the need for breathing support in babies with SMA. Long-term studies indicate that the treatment remains effective and safe over time. Overall, Nusinersen has proven to improve health outcomes for those with SMA. ^{5 6 7 8 9}

Who Is on the Research Team?

Medical Director

Principal Investigator

Biogen

Are You a Good Fit for This Trial?

This trial is for infants aged ≤ 6 weeks with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA). They must have a specific genetic makeup of SMA, an ulnar nerve test result ≥ 1 mV, and be born within certain gestational age ranges. Infants cannot participate if they've had previous treatments for SMA or show signs of low oxygen levels or abnormal blood tests.

Inclusion Criteria

I have a confirmed genetic form of SMA.

My genetic test shows I have 2 or 3 copies of the SMN2 gene.

Your ulnar nerve's electrical activity is normal at the start of the study.

Exclusion Criteria

I have not had gene therapy, ASO treatment, or cell transplantation for SMA.

I show signs that my doctor thinks could be SMA.

You have important problems with your blood or other body chemicals.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive multiple doses of Nusinersen administered intrathecally

Up to 2891 days

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 2891 days

What Are the Treatments Tested in This Trial?

Interventions

• Nusinersen

Trial Overview

The study is testing the effectiveness of multiple doses of Nusinersen given through spinal injection in preventing or delaying respiratory intervention or death in infants with SMA. It aims to understand how this treatment can affect these children before symptoms appear.

How Is the Trial Designed?

Group I: Nusinersen Experimental Treatment 1 Intervention

Nusinersen administered as an intrathecal injection

Nusinersen is already approved in United States, European Union for the following indications:

Approved in United States as Spinraza for:

• Spinal muscular atrophy in pediatric and adult patients

Approved in European Union as Spinraza for:

• Spinal muscular atrophy

Who Is Running the Clinical Trial?

Biogen

Lead Sponsor

Trials

655

Recruited

468,000+

Daniel Quirk

Biogen

Chief Medical Officer

MD

Christopher A. Viehbacher

Biogen

Chief Executive Officer since 2022

Graduated from Queen's University, Kingston, Ontario, Canada

Citations

1.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/38361750/

Long-term efficacy and safety of nusinersen in adults with ...

We investigated the efficacy and safety of nusinersen in adults with SMA over 38 months, the longest time period to date in a large cohort of patients from ...

2.

clinicaltrials.gov

clinicaltrials.gov/study/NCT02292537

NCT02292537 | A Study to Assess the Efficacy and Safety ...

The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset ...

3.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC4782111/

Results from a phase 1 study of nusinersen (ISIS-SMNRx) in ...

This study provides Class IV evidence that in children with SMA, intrathecal nusinersen is not associated with safety or tolerability concerns. Spinal muscular ...

4.

ir.ionis.com

ir.ionis.com/news-releases/news-release-details/new-england-journal-medicine-publishes-first-phase-3-study

The New England Journal of Medicine Publishes First ...

The end of study results from ENDEAR, the Phase 3 study of SPINRAZA (nusinersen) for the treatment of spinal muscular atrophy (SMA), were published today.

5.

investors.biogen.com

investors.biogen.com/news-releases/news-release-details/new-data-nusinersen-underscore-biogens-commitment-advancing

New Data for Nusinersen Underscore Biogen's ...

SPINRAZA has shown sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 10 years.

6.

ncbi.nlm.nih.gov

ncbi.nlm.nih.gov/books/NBK533983/

Summary of Safety Data from Long-Term Studies - NCBI - NIH

To provide a summary of safety data from long-term studies of nusinersen in patients with spinal muscular atrophy (SMA).

7.

spinrazahcp.com

spinrazahcp.com/en_us/home/why-spinraza/safety.html

SPINRAZA® (nusinersen) Side Effects and Safety Profile | HCP

View the SPINRAZA® safety profile, including warnings and precautions, and side effects. See full Safety & Prescribing Info.

8.

clinicaltrials.gov

clinicaltrials.gov/study/NCT01703988

Study Details | NCT01703988 | An Open-label Safety, ...

This study will test the safety, tolerability, and pharmacokinetics of escalating doses of nusinersen (ISIS 396443) administered into the spinal fluid ...

9.

investors.biogen.com

investors.biogen.com/news-releases/news-release-details/new-higher-dose-nusinersen-efficacy-and-safety-data-presented

New Higher Dose Nusinersen Efficacy and Safety Data ...

SPINRAZA is approved in more than 71 countries to treat infants, children and adults with spinal muscular atrophy (SMA). As a foundation of care ...