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
A Clinical Study of Letermovir (MK-8228) in Children and Adolescents Who Receive a Kidney Transplant (KT) (MK-8228-077)
Merck Sharp & Dohme LLC
- Location
- Philadelphia, PA + 20 other US sites
- Eligibility basics
- Up to age 17
- RecruitingPhase 1/Phase 2
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
AskBio Inc
- Location
- Philadelphia, PA + 4 other US sites
- Eligibility basics
- Ages 18–65
- RecruitingPhase 1/Phase 2
Phase 1b/2 Study of IV Sarilumab in Adult With RA
Sanofi
- Location
- Duncansville, PA + 11 other US sites
- Eligibility basics
- Ages 18+
- RecruitingPhase 1
A Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of RO7823653 in Participants With Diabetic Macular Edema (DME)
Genentech, Inc.
- Location
- Erie, PA + 11 other US sites
- Eligibility basics
- Ages 18+
- RecruitingPhase 1/Phase 2
ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
Ionis Pharmaceuticals, Inc.
- Location
- Philadelphia, PA + 7 other US sites
- Eligibility basics
- Ages 2–65 · Men only
- RecruitingPhase 1/Phase 2
Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)
Ascidian Therapeutics, Inc
- Location
- Philadelphia, PA + 8 other US sites
- Eligibility basics
- Ages 12+
- RecruitingPhase 1/Phase 2
RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus
Cabaletta Bio
- Location
- Philadelphia, PA + 17 other US sites
- Eligibility basics
- Ages 12–65
- RecruitingPhase 1
A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)
Disc Medicine, Inc
- Location
- Pittsburgh, PA + 10 other US sites
- Eligibility basics
- Ages 18+
- RecruitingPhase 1
NEXUS Study: A Study to Test Single and Multiple Doses of MER511 Given to Adults With Graves' Disease
Merida Biosciences
- Location
- Philadelphia, PA + 10 other US sites
- Eligibility basics
- Ages 18–65
- RecruitingPhase 1
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Solid Biosciences Inc.
- Location
- Philadelphia, PA + 2 other US sites
- Eligibility basics
- Ages 18–40
- RecruitingPhase 1
A Study to Learn About the Study Medicine Called Zavegepant (PF-07930207) in Children With a History of Migraine
Pfizer
- Location
- Philadelphia, PA + 10 other US sites
- Eligibility basics
- Ages 6–11
- RecruitingPhase 1
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
Agios Pharmaceuticals, Inc.
- Location
- Pittsburgh, PA + 2 other US sites
- Eligibility basics
- Ages 18–69
- RecruitingPhase 1/Phase 2
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
UniQure Biopharma B.V.
- Location
- Pittsburgh, PA + 9 other US sites
- Eligibility basics
- Ages 18–65 · Men only
- RecruitingPhase 1/Phase 2
Study of Circular RNA Treatment in Patients With Radiation Induced Xerostomia-1
RiboX Therapeutics Ltd.
- Location
- Philadelphia, PA + 3 other US sites
- Eligibility basics
- Ages 18+
- RecruitingPhase 1
A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
Insmed Gene Therapy LLC
- Location
- Philadelphia, PA + 6 other US sites
- Eligibility basics
- Ages 18–79
- RecruitingPhase 1/Phase 2
A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1
Sanofi
- Location
- Philadelphia, PA + 4 other US sites
- Eligibility basics
- Ages 10–55
- RecruitingPhase 1
A Study of the Safety and Tolerability of ASP7317 in Senior Adults Who Are Losing Their Clear, Sharp Central Vision Due to Geographic Atrophy Secondary to Dry Age-related Macular Degeneration
Astellas Institute for Regenerative Medicine
- Location
- Philadelphia, PA + 12 other US sites
- Eligibility basics
- Ages 50+
- RecruitingPhase 1
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)
Biogen
- Location
- Hershey, PA + 11 other US sites
- Eligibility basics
- Ages 3+
- RecruitingPhase 1/Phase 2
Dose Escalation Study of EG110A, Administered by Intradetrusor Injections to Adults With Neurogenic Detrusor Overactivity-related Incontinence Following Spinal Cord Injury Who Regularly Perform Clean Intermittent…
Cyllene Therapeutics
- Location
- Philadelphia, PA + 3 other US sites
- Eligibility basics
- Ages 18–75
- RecruitingPhase 1
A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease
Verve Therapeutics, Inc.
- Location
- Philadelphia, PA + 9 other US sites
- Eligibility basics
- Ages 18–70
- RecruitingPhase 1/Phase 2
Study of CTO1681 for the Prevention and Treatment of CRS in Patients Receiving CAR T-Cell Therapy
CytoAgents, Inc.
- Location
- Pittsburgh, PA + 5 other US sites
- Eligibility basics
- Ages 18+
- RecruitingPhase 1/Phase 2
ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy
Alexion Pharmaceuticals, Inc.
- Location
- Philadelphia, PA + 6 other US sites
- Eligibility basics
- Ages 18–70
- RecruitingPhase 1/Phase 2
A Study of VV-14305 for the Treatment of Thyroid Eye Disease
Kriya Therapeutics, Inc.
- Location
- Erie, PA + 2 other US sites
- Eligibility basics
- Ages 18–80
- RecruitingPhase 1/Phase 2
Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss
Skylark Bio Inc.
- Location
- Allentown, PA + 3 other US sites
- Eligibility basics
- Ages 10 mo–7
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.