Oncology Cardiology / Cardiovascular Respiratory / COPD / Asthma Infectious Disease Gastroenterology Diabetes / Metabolic Neurology Rheumatology Women's Health Mental Health / Psychiatry Dermatology Rare Diseases Men's Health
Clinical Trial ● Currently Recruiting Phase I NCT06555419

A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)

A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy…

📅 24 Aug 2026 ⏱ 2 min read
Currently Recruiting
This trial is actively seeking participants in the UK. Discuss eligibility with your patient before referring.
Status
Currently Recruiting
Phase
Phase I
NCT ID
NCT06555419
Start
2025-01-16
Completion
2027-09-30
ClinicaliQ Trial Snapshot
  • A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK) — Recruiting • Phase I • Dermatology • NCT06555419.
  • Study comparing how the body processes nusinersen delivered via an implantable device versus lumbar puncture in spinal muscular atrophy patients.

Verify eligibility, endpoints and current status on the original ClinicalTrials.gov registry before acting on this summary.

Use This Page For
  • Quick orientation before opening the registry record.
  • Checking recruitment status, phase and sponsor at a glance.
  • Connecting this trial to nearby guidelines, Drug Science and education.
What This Trial Is Studying

In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusinersen is given by lumbar puncture (LP). The ThecaFlex DRx system is an investigational implantable medical device developed by Alcyone Therapeutics, Inc. It consists of a catheter, which is a flexible tube, connected to a port which is placed under the skin. Alcyone Therapeutics, Inc. has an ongoing study called PIERRE to test the ThecaFlex DRx system. Participants with spinal muscular atrophy (SMA) in the PIERRE…

Full Trial Details
View this trial on the source registry
Eligibility criteria, protocol, and results when available
View Trial ↗
Share: Twitter/X LinkedIn
Related

Related Clinical Intelligence

Guidelines, Drug Science, safety briefs and education connected to this trial area.

Clinical Brief
Topical steroids: introduction of new labelling and a reminder of the possibility of severe side effects, including Topical Steroid Withdrawal Reactions
Dermatology · MHRA · 29 May 2024
Topical steroid products are safe and highly effective treatments for the management of a wide range of inflammatory skin diseases but have…
View brief →
Clinical Brief
Hundreds contact BBC about mystery skin condition ‘hell’ – but doctors can’t agree it exists
Dermatology · BBC Health · 10 Apr 2026
Doctors remain divided on whether a severe skin condition reported by hundreds of patients represents extreme eczema or a distinct entity called…
View brief →
Guideline
Subcutaneous spesolimab 1-ml formulation for preventing generalised pustular psoriasis flares in people 12 years and over (terminated appraisal)
Dermatology · 08 Apr 2026
This NICE publication is marked as a terminated appraisal, so it functions as a status notice rather than an active treatment recommendation…
View guideline →
Clinical Brief
Our skin is falling off and no-one can tell us why
Dermatology · BBC Health · 29 Mar 2026
Topical Steroid Withdrawal: Key Clinical Takeaways TSW is now subject to formal UK research: Growing social media documentation of cases has prompted…
View brief →
Drug Science
The IL-17/IL-23 Axis
Dermatology · 04 Apr 2026
The IL-17/IL-23 Axis is a clinically relevant Drug Science explainer. The IL-23/IL-17 axis is a central driver of immune-mediated inflammatory disease. IL-23,…
Explore mechanism →
Guideline
Hidradenitis Suppurativa: Assessment and Management (NICE NG73)
Dermatology · 27 Mar 2026
Diagnose hidradenitis suppurativa clinically based on recurrent painful nodules, abscesses, sinus tracts, and scars localised to intertriginous areas (axillae, groin, inframammary folds),…
View guideline →