Phase 1 Spinal Muscular Atrophy Trial, Recruiting (NCT06555419)

Phase 1 Spinal Muscular Atrophy Trial, Recruiting NCT06555419 Sponsor: Biogen Condition: Spinal Muscular Atrophy

Printed from Voxsanity (voxsanity.com.au) — NCT06555419 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

Back to Spinal Muscular Atrophy

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

You are already taking nusinersen (a medication for SMA) at a dose of 12 mg every 4 months as a regular ongoing treatment.
You must already be enrolled in a related study called the PIERRE study in order to take part in this trial.

Who may not be able to join:

You are currently taking part in, or have recently taken part in (within the last 6 months), another clinical trial for SMA treatment — with some exceptions, such as the PIERRE study or trials testing certain muscle-related treatments like apitegromab or taldefgrobep alfa.
You have never received nusinersen treatment before.
You are currently taking nusinersen at a dose other than 12 mg.
You have already had a device called the ThecaFlex DRx system surgically implanted in your body.
You are pregnant, currently breastfeeding, or planning to become pregnant during the study.

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.

Last synced 28 July 2026

Phase 1: approximately ~10% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.

Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Principal Investigator: Medical Director, Biogen

Phone: 866-633-4636

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Voxsanity

Clinical trials and treatments, explained plainly · voxsanity.com.au

Date:

To: My Doctor

Re: Clinical trial enquiry

I am writing to enquire about the following clinical trial which may be relevant to my care:

Trial: NCT06555419

Sponsor: Biogen

Phase: Phase 1

Status: Recruiting

Condition: Spinal Muscular Atrophy

Trial contact: 866-633-4636

More information: https://clinicaltrials.gov/study/NCT06555419

Eligibility summary (AI generated)

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

You are already taking nusinersen (a medication for SMA) at a dose of 12 mg every 4 months as a regular ongoing treatment.
You must already be enrolled in a related study called the PIERRE study in order to take part in this trial.

Who may not be able to join:

You are currently taking part in, or have recently taken part in (within the last 6 months), another clinical trial for SMA treatment — with some exceptions, such as the PIERRE study or trials testing certain muscle-related treatments like apitegromab or taldefgrobep alfa.
You have never received nusinersen treatment before.
You are currently taking nusinersen at a dose other than 12 mg.
You have already had a device called the ThecaFlex DRx system surgically implanted in your body.
You are pregnant, currently breastfeeding, or planning to become pregnant during the study.

Important: Always verify eligibility with the trial site directly before applying.

Access in Australia: A treatment being studied in a clinical trial is generally not yet listed on the PBS. Where a medicine is not approved or not available through a trial, the TGA Special Access Scheme may allow a doctor to access unapproved therapeutic goods for individual patients. See voxsanity.com.au/sas-navigator/ for a plain English explainer.

This information was sourced from ClinicalTrials.gov via Voxsanity (data last synced 28 July 2026). It is not medical advice. Please verify the trial's current status directly with the trial site before acting.

Trial details

Status

Recruiting

Phase

Phase 1

Sponsor

Biogen

Registry

ClinicalTrials.gov

Start date

16 January 2025

Est. completion

25 June 2027

Where this trial is recruiting

🇫🇷 France 🇩🇪 Germany 🇮🇹 Italy 🇵🇱 Poland 🇪🇸 Spain 🇬🇧 United Kingdom 🇺🇸 United States

Primary endpoints

Maximum Observed Concentration (Cmax) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System; Area Under the Plasma Concentration-Time Curve From Zero Time to 24 Hours After Intrathecal Administration (AUC0-24h) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov