Clinical trials · Pennsylvania
Spinal Muscular Atrophy clinical trials in Pennsylvania
We’re tracking 8 recruiting spinal muscular atrophy studies with a site in Pennsylvania — including Phase 1, Phase 2 and Phase 4 studies, each written for real people, not researchers.
Recruiting studies in Pennsylvania
- NCT05866419Recruiting
Study of a port and catheter system for spinal muscular atrophy
This study tests a new device called the ThecaFlex DRx System, which is a small port and catheter placed under the skin to deliver Spinraza directly into spinal fluid—instead of needing repeated spinal taps. The device aims to make receiving medication easier and safer for people with SMA.
Hershey, PennsylvaniaAges 3 years+ - NCT07287982RecruitingPhase 2
ARGX-119 for children with SMA who can walk
This study tests a new medicine called ARGX-119 in children with SMA. It is given through an IV and aims to help kids who can already walk get stronger and stay healthy.
Philadelphia, PennsylvaniaAges 5–17 - NCT04174157Recruiting
Spinal muscular atrophy patient registry for OAV-101
This is a registry that collects information from people with spinal muscular atrophy (SMA) who have been treated with OAV-101. It helps researchers understand how patients do over time after this treatment.
Hershey, PennsylvaniaAges Any age - NCT05861999RecruitingPhase 4
Risdiplam for young children with SMA after gene therapy plateau
This trial studies whether risdiplam is safe and helpful for children under 2 with spinal muscular atrophy (SMA) whose function has leveled off or declined after gene therapy. It may be an option if you saw benefits from gene therapy but then noticed a stop or setback in key abilities.
Philadelphia, PennsylvaniaAges 3 months–2 years - NCT05861986RecruitingPhase 4
Risdiplam for very young children after gene therapy for SMA
This trial studies whether risdiplam, given early after gene therapy, is safe and helps children with spinal muscular atrophy (SMA) keep improving or stay stable. It focuses on children under 2 years old who already received gene therapy and need to meet timing and health requirements.
Philadelphia, PennsylvaniaAges 3 months–2 years - NCT06555419RecruitingPhase 1
How Nusinersen works with ThecaFlex DRx system
This study looks at how a medicine called Nusinersen (Spinraza) works in the body when given through a new device called ThecaFlex DRx. It is for people with spinal muscular atrophy who are already on a steady dose of Nusinersen and are part of the main PIERRE study.
Hershey, PennsylvaniaAges 3 years+ - NCT07047144RecruitingPhase 2
Apitegromab for infants with SMA under 2 years old
This study tests a new medicine called apitegromab for babies under 2 years old with spinal muscular atrophy (SMA). It looks at whether the drug can help with muscle strength and reaching motor milestones like sitting or rolling.
Philadelphia, PennsylvaniaAges Up to 2 years - NCT05966038Recruiting
Study of ALS and related movement nerve conditions
This study collects and stores medical information from people diagnosed with ALS or certain related conditions affecting movement. It may help researchers better understand how these illnesses change over time and improve future treatments.
Philadelphia, PennsylvaniaAges 18–90
Spinal Muscular Atrophy trials by city in Pennsylvania
Where these studies are running in Pennsylvania
Institutions with a site for the recruiting spinal muscular atrophy studies listed above.
- Children's Hospital of Philadelphia · 6 studies
- Penn State Milton S Hershey Medical Center · 2 studies
- Penn State Hershey
- Temple University Hospital
- Thomas Jefferson University Hospital
- University of Pittsburgh
- University of Pittsburgh Medical Center
What taking part in a spinal muscular atrophy study involves
A screening visit first
Before anything else, the study team checks whether you fit — usually a visit with some tests. You can stop at any point, and screening is typically free.
Care at a nearby site
Study visits happen at a clinic or hospital taking part. Many studies cover the cost of the study treatment and related visits, and some reimburse travel.
You stay in control
Taking part is voluntary and you can leave a study at any time, for any reason, without affecting your regular care.
The team decides eligibility
Our fit check is a helpful first read, not a decision. The study team makes the final call after reviewing your health history.
Spinal Muscular Atrophy trials in other states
Common questions
- Are there spinal muscular atrophy clinical trials in Pennsylvania?
- Yes. We're currently tracking 8 recruiting spinal muscular atrophy studies with a site in Pennsylvania, each rewritten in plain language so you can see what it's testing and who it's for.
- How do I find out if I qualify for a spinal muscular atrophy study in Pennsylvania?
- Each study lists its eligibility rules — age, diagnosis, prior treatments. On every trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Do I have to live in Pennsylvania to take part?
- Not necessarily. These studies have a site in Pennsylvania, but eligibility is about your health, not your address — some people travel to take part, and a few studies reimburse travel. The study team can tell you what's required.
- Does it cost anything to join a spinal muscular atrophy trial?
- Using Clin2 is always free. Many trials cover the study treatment and related visits; some reimburse travel. The study team explains exactly what's covered before you decide.
Not the right time?
New spinal muscular atrophy studies open in Pennsylvania regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.