Phase 1 · Missouri
Phase 1 clinical trials in Missouri
54 studies have at least one recruiting site in Missouri. 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.
54 recruiting studiesSource: ClinicalTrials.gov, refreshed October 26, 2026How we list studies
Studies
- RecruitingPhase 1/Phase 2
An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
Affinia Therapeutics
- Location
- Kansas City, MO + 3 other US sites
- Eligibility basics
- Ages 18–70
- RecruitingPhase 1/Phase 2
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
Precision BioSciences, Inc.
- Location
- St Louis, MO + 1 other US site
- Eligibility basics
- Ages 2–7 · Men only
- RecruitingPhase 1/Phase 2
A Study to Learn About the Safety of BIIB147 and Its Effects in Adults With Amyotrophic Lateral Sclerosis (ALS)
Biogen
- Location
- St Louis, MO + 1 other US site
- Eligibility basics
- Ages 18+
- RecruitingPhase 1
A Phase 1 Study of LY5830966 in Participants With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (PPV-MF), or Post-Essential Thrombocythemia Myelofibrosis (PET-MF) Who Have Been Failed by a Type I…
Ajax Therapeutics, Inc., a wholly owned subsidiary of Eli Lilly and Company
- Location
- St Louis, MO + 12 other US sites
- Eligibility basics
- Ages 18+
- RecruitingPhase 1
Study to Evaluate the Maximal Use of Ruxolitinib Cream in Adult and Adolescent Participants With Hidradenitis Suppurativa
Incyte Corporation
- Location
- Bolivar, MO + 15 other US sites
- Eligibility basics
- Ages 12–75
- RecruitingPhase 1/Phase 2
A Study of Oral Nuvisertib (TP-3654) in Patients With Myelofibrosis
Sumitomo Pharma America, Inc.
- Location
- St Louis, MO + 29 other US sites
- Eligibility basics
- Ages 18+
- RecruitingPhase 1/Phase 2
HS235 Study in Overweight and Obese Participants With Combined Pre and Post-Capillary Pulmonary Hypertension Due to Heart Failure With Preserved Ejection Fraction
35Pharma Inc
- Location
- Kansas City, MO + 1 other US site
- 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
- Columbia, MO + 6 other US sites
- Eligibility basics
- Ages 18–79
- RecruitingPhase 1/Phase 2
Treatment of Participants With Primary or Secondary Progressive Multiple Sclerosis
Tr1X, Inc.
- Location
- St Louis, MO + 7 other US sites
- Eligibility basics
- Ages 18–65
- RecruitingPhase 1/Phase 2
ATR 1072 in Participants With PRKAG2 Syndrome
Atrium Therapeutics
- Location
- Kansas City, MO
- Eligibility basics
- Ages 18–65
- RecruitingPhase 1
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort
Sarepta Therapeutics, Inc.
- Location
- St Louis, MO + 10 other US sites
- Eligibility basics
- Ages 2+ · Men only
- RecruitingPhase 1/Phase 2
CNP-103 in Adolescent and Adult Subjects Ages 12-35 With Recently Diagnosed (Within 6 Months) Stage 3 Type 1 Diabetes (T1D)
COUR Pharmaceutical Development Company, Inc.
- Location
- Kansas City, MO + 44 other US sites
- Eligibility basics
- Ages 12–35
- RecruitingPhase 1/Phase 2
A Study to Investigate the Safety, Tolerability, and Efficacy of AZD0120 in Adults With Refractory SLE
AstraZeneca
- Location
- St Louis, MO + 8 other US sites
- Eligibility basics
- Ages 18–70
- RecruitingPhase 1
An Open-label Study of AZD0120 in Adults With Multiple Sclerosis
AstraZeneca
- Location
- St Louis, MO + 4 other US sites
- Eligibility basics
- Ages 18–60
- 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
- Saint Joseph, MO + 78 other US sites
- Eligibility basics
- Ages 18–45
- RecruitingPhase 1
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
Denali Therapeutics Inc.
- Location
- St Louis, MO + 3 other US sites
- Eligibility basics
- Ages 18–75
- RecruitingPhase 1
A Study of Amicidin-β Topical Solution for Patients With Surgical or Traumatic Wound Infections
Macro Biologics, Inc.
- Location
- St Louis, MO + 4 other US sites
- Eligibility basics
- Ages 18–79
- RecruitingPhase 1/Phase 2
Descartes-08 for Children, Adolescents, and Young Adults With Autoimmune Disorders
Cartesian Therapeutics
- Location
- St Louis, MO + 2 other US sites
- Eligibility basics
- Ages 12+
- RecruitingPhase 1/Phase 2
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Solid Biosciences Inc.
- Location
- St Louis, MO + 11 other US sites
- Eligibility basics
- Ages 0 mo–17 · Men only
- RecruitingPhase 1/Phase 2
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
Kamau Therapeutics
- Location
- St Louis, MO + 5 other US sites
- Eligibility basics
- Ages 12–50
- RecruitingPhase 1/Phase 2
A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.
Alexion Pharmaceuticals, Inc.
- Location
- St Louis, MO + 9 other US sites
- Eligibility basics
- Ages 18+
- RecruitingPhase 1
Study of BHV-1400 in IgA Nephropathy
Biohaven Therapeutics Ltd.
- Location
- Chesterfield, MO + 13 other US sites
- Eligibility basics
- Ages 18–65
- RecruitingPhase 1
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of MBX 4291 in Adult Participants With Obesity
MBX Biosciences
- Location
- Kansas City, MO + 3 other US sites
- Eligibility basics
- Ages 18–65
- 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
- St Louis, MO + 14 other US sites
- Eligibility basics
- Ages 17–30
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.