Phase 1 · Pennsylvania
Phase 1 clinical trials in Pennsylvania
102 studies have at least one recruiting site in Pennsylvania. Phase 1 (and early Phase 1) trials are usually the first time a medicine is studied in people. They are typically small, closely monitored, and many are run in dedicated research units, sometimes with overnight stays.
102 recruiting studiesSource: ClinicalTrials.gov, refreshed October 26, 2026How we list studies
Studies
- RecruitingPhase 1/Phase 2
AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy
UniQure Biopharma B.V.
- Location
- Philadelphia, PA + 15 other US sites
- Eligibility basics
- Ages 18–75
- RecruitingPhase 1/Phase 2
4D-150 in Patients With Neovascular (Wet) Age-Related Macular Degeneration
4D Molecular Therapeutics
- Location
- Bethlehem, PA + 19 other US sites
- Eligibility basics
- Ages 50+
- RecruitingPhase 1/Phase 2
Study to Evaluate Safety, Efficacy and Immunogenicity of Acne mRNA Vaccine in Adults With Moderate to Severe Acne
Sanofi Pasteur, a Sanofi Company
- Location
- Camp Hill, PA + 78 other US sites
- Eligibility basics
- Ages 18–45
- RecruitingPhase 1/Phase 2
AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
NGGT INC.
- Location
- Pittsburgh, PA + 4 other US sites
- Eligibility basics
- Ages 18–55
- RecruitingPhase 1/Phase 2
A Study to Evaluate KRIYA-825 (VV-14295) in Adults With Geographic Atrophy Secondary to Age-related Macular Degeneration
Kriya Therapeutics, Inc.
- Location
- Erie, PA
- Eligibility basics
- Ages 55–80
- RecruitingPhase 1/Phase 2
A Study to Assess the Safety and Efficacy of IPN10200 in Adult Participants With Upper Limb Spasticity.
Ipsen
- Location
- Elkins Park, PA + 3 other US sites
- Eligibility basics
- Ages 18–70
- RecruitingPhase 1
Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)
Chugai Pharmaceutical
- Location
- Philadelphia, PA + 6 other US sites
- Eligibility basics
- Ages 18–85
- RecruitingPhase 1/Phase 2
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Solid Biosciences Inc.
- Location
- Philadelphia, PA + 11 other US sites
- Eligibility basics
- Ages 0 mo–17 · Men only
- RecruitingPhase 1
A Study to Learn if Bimekizumab Given in Different Ways is Safe and Moves Similarly Throughout the Body Over Time in Adults
UCB Biopharma SRL
- Location
- Duncansville, PA + 29 other US sites
- Eligibility basics
- Ages 18+
- RecruitingPhase 1
A Study of CDX-622 in Participants With Mild to Moderate Asthma
Celldex Therapeutics
- Location
- Pittsburgh, PA + 3 other US sites
- Eligibility basics
- Ages 18+
- RecruitingPhase 1
R-2487 in Patients With Rheumatoid Arthritis
Rise Therapeutics LLC
- Location
- Duncansville, PA + 5 other US sites
- Eligibility basics
- Ages 18–75
- RecruitingPhase 1/Phase 2
A Study Exploring Changes in a Variety of Biomarkers Following Dosing With MT1988 in Participants at Clinical High Risk for Psychosis
Monument Therapeutics Limited
- Location
- Philadelphia, PA + 14 other US sites
- Eligibility basics
- Ages 17–30
- RecruitingPhase 1/Phase 2
Evaluate REC-4881 in Participants With Familial Adenomatous Polyposis (FAP)
Recursion Pharmaceuticals Inc.
- Location
- Philadelphia, PA + 6 other US sites
- Eligibility basics
- Ages 18+
- RecruitingPhase 1/Phase 2
Study of EXE-346 Live Biotherapeutic to Reduce High Bowel Movement Frequency in Subjects With an IPAA (PROF)
Exegi Pharma, LLC
- Location
- Hershey, PA + 7 other US sites
- Eligibility basics
- Ages 18+
- RecruitingPhase 1
A Phase 1 Study of FT819 in B-cell Mediated Autoimmune Disease
Fate Therapeutics
- Location
- Philadelphia, PA + 15 other US sites
- Eligibility basics
- Ages 12–70
- RecruitingPhase 1/Phase 2
A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
Hoffmann-La Roche
- Location
- Philadelphia, PA + 4 other US sites
- Eligibility basics
- Ages 25–65
- RecruitingPhase 1/Phase 2
Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease
Ray Therapeutics, Inc.
- Location
- Pittsburgh, PA + 2 other US sites
- Eligibility basics
- Ages 16+
- RecruitingPhase 1
AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis
Amylyx Pharmaceuticals Inc.
- Location
- Philadelphia, PA + 9 other US sites
- Eligibility basics
- Ages 18+
- RecruitingPhase 1/Phase 2
Study to Assess the Safety, Tolerability, and Preliminary Efficacy of ST266 in Infants With Necrotizing Enterocolitis (NEC)
Noveome Biotherapeutics, formerly Stemnion
- Location
- Hershey, PA + 7 other US sites
- Eligibility basics
- Ages 0 mo–2 mo
- RecruitingPhase 1/Phase 2
A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
ModernaTX, Inc.
- Location
- Philadelphia, PA + 5 other US sites
- Eligibility basics
- Ages 1+
- RecruitingPhase 1/Phase 2
Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients
Opus Genetics, Inc
- Location
- Philadelphia, PA + 1 other US site
- Eligibility basics
- Ages 4+
- RecruitingPhase 1/Phase 2
Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
ModernaTX, Inc.
- Location
- Philadelphia, PA + 7 other US sites
- Eligibility basics
- Any age listed
- RecruitingPhase 1
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
Ionis Pharmaceuticals, Inc.
- Location
- Philadelphia, PA + 2 other US sites
- Eligibility basics
- Ages 2–17 · Men only
- RecruitingPhase 1/Phase 2
A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease
GC Biopharma Corp
- Location
- Pittsburgh, PA + 5 other US sites
- Eligibility basics
- Ages 18+
Who these studies look for
Phase 1 studies often have narrow eligibility: specific age and weight ranges, no recent participation in another study, no nicotine, and limits on medications. Some Phase 1 studies recruit patients rather than healthy volunteers, so check each listing.
Matching a page like this one never means you qualify. Each research team decides eligibility at screening.
Questions
Are Phase 1 trials riskier?
Every study carries risk. Phase 1 studies involve less human data about the product, which is why they are closely monitored. The informed consent document explains the known risks; read it carefully and ask questions before signing.
Why do some Phase 1 studies include overnight stays?
Staying in the unit lets staff dose on a precise schedule and collect samples around the clock. Where a record mentions an inpatient stay we flag it on the listing.