Clinical Trials at Cincinnati Children's Hospital Medical Center

As of July 2026, 253 paid clinical trials are recruiting at Cincinnati Children's Hospital Medical Center, located at 3333 BURNET AVENUE, 11027, CINCINNATI, OH 45229, phone (513) 803-4738 in Cincinnati, Ohio. Compensation typically covers time, travel, and study visits — most studies also offer study-related medical care at no cost to participants.

Recruiting trial data synced daily from ClinicalTrials.gov. Last sync: July 17, 2026.

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257 clinical trials at Cincinnati Children's Hospital Medical Center

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Minima Stent System Post- Approval Study (PAS)

for Pulmonary Artery Stenosis

Sites in Cincinnati, Columbus +13 more locations

Sponsor: Renata Medical

Sex: All·Age: All ages

Code: NCT06828770·Recruiting·Listed Feb 2025

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Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy

for Cardiomyopathy

Sites in Cincinnati, Cleveland +19 more locations

Sponsor: Tenaya Therapeutics

Sex: All·Age: 0 - 18

Code: NCT05112237·Recruiting·Listed Nov 2021

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A Phase 2a Study of IV BCV in Subjects With Adenovirus Infection

for Adenovirus Infections

Sites in Cincinnati +8 more locations

Sponsor: SymBio Pharmaceuticals

Sex: All·Age: 2 Months+

Code: NCT04706923·Phase 2·Recruiting·Listed Jan 2021

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Nafamostat Efficacy in Phase 3 Registrational CRRT Study

for Acute Kidney Injury

Sites in Cincinnati +9 more locations

Sponsor: Talphera, Inc

Sex: All·Age: 18 - 80

Code: NCT06150742·Recruiting·Listed Nov 2023

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Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

for Achondroplasia

Drug: Vosoritide

Sites in Cincinnati +7 more locations

Sponsor: BioMarin Pharmaceutical

Sex: All·Age: 2 - 17

Code: NCT07441876·Phase 2, Phase 3·Recruiting·Listed Mar 2026

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Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

for Pompe Disease

Sites in Cincinnati +8 more locations

Sponsor: Shionogi

Sex: All·Age: 18+

Code: NCT07123155·Phase 2·Recruiting·Listed Aug 2025

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A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)

for Sickle Cell Disease

Sites in Cincinnati +6 more locations

Sponsor: Disc Medicine, Inc

Sex: All·Age: 18+

Code: NCT07187973·Phase 1·Recruiting·Listed Sep 2025

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AERA Pediatrics Registry

for Dysfunction of Eustachian Tube

Sites in Cincinnati +8 more locations

Sponsor: Integra LifeSciences Corporation

Sex: All·Age: Up to 18

Code: NCT07071298·Recruiting·Listed Jul 2025

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Starlight Cardiovascular Lifeline Ductus Arteriosus Stent IDE Study

for Congenital Heart Disease

Sites in Cincinnati, Columbus +6 more locations

Sponsor: Starlight Cardiovascular Inc

Sex: All·Age: 1 Minute - 6 Months

Code: NCT07114718·Recruiting·Listed Aug 2025

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A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations

for Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)

Sites in Cincinnati +8 more locations

Sponsor: Regeneron Pharmaceuticals

Sex: All·Age: All ages

Code: NCT05788536·Phase 1, Phase 2·Recruiting·Listed Mar 2023

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A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition

for X-Linked Myotubular Myopathy

Sites in Cincinnati +5 more locations

Sponsor: Astellas Gene Therapies

Sex: Male·Age: Up to 17

Code: NCT06581146·Recruiting·Listed Sep 2024

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A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)

for Heterozygous Familial Hypercholesterolemia (HeFH)

Drug: Enlicitide Decanoate

Sites in Cincinnati +5 more locations

Sponsor: Merck Sharp & Dohme LLC

Sex: All·Age: 6 - 17

Code: NCT07058077·Phase 2, Phase 3·Recruiting·Listed Jul 2025

Apply to this trial

Infant Formula in Infants and Children With Cow's Milk Allergy

for Cow's Milk Allergy

Sites in Cincinnati, Columbus +6 more locations

Sponsor: Abbott Nutrition

Sex: All·Age: 3 Months - 12

Code: NCT06456541·Recruiting·Listed Jun 2024

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A Long-term Extension (LTE) Study of Guselkumab in Pediatric Participants

for Crohns Disease

Drug: Guselkumab

Sites in Cincinnati +4 more locations

Sponsor: Janssen Research & Development, LLC

Sex: All·Age: 3+

Code: NCT06663332·Phase 3·Recruiting·Listed Oct 2024

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KiteLock 4% EDTA Lock Solution for the Prevention of Occlusions in Children With Intestinal Failure

for Pediatric Intestinal Failure

Sites in Cincinnati, Columbus +6 more locations

Sponsor: SterileCare Inc.

Sex: All·Age: 4 Weeks - 18

Code: NCT05879835·Recruiting·Listed May 2023

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A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors

for ALK Fusion-positive Solid or CNS Tumors

Sites in Cincinnati +5 more locations

Sponsor: Hoffmann-La Roche

Sex: All·Age: Up to 17

Code: NCT04774718·Phase 1, Phase 2·Recruiting·Listed Mar 2021

Apply to this trial

Prospective Clinical Assessment Study in Children With Hypochondroplasia

for Hypochondroplasia

Sites in Cincinnati +7 more locations

Sponsor: QED Therapeutics, a BridgeBio company

Sex: All·Age: 30 Months - 16

Code: NCT06410976·Recruiting·Listed May 2024

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Study of Oral Administration of LP-118 in Patients With Relapsed or Refractory CLL, SLL, MDS, MDS/MPN, AML, CMML-2, MPN-BP, ALL, MF, NHL, RT, MM or T-PLL.

for Non Hodgkin Lymphoma

Sites in Cincinnati, Columbus +6 more locations

Sponsor: Newave Pharmaceutical Inc

Sex: All·Age: 13+

Code: NCT04771572·Phase 1·Recruiting·Listed Feb 2021

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Study Evaluating the Safety and Efficacy of Neffy or Intramuscular Adrenalin in Patients With Allergic Reactions After Oral Food Challenge or Allergen Immunotherapy

for Allergic Reactions

Sites in Cincinnati +5 more locations

Sponsor: ARS Pharmaceuticals, Inc.

Sex: All·Age: 4+

Code: NCT06834165·Phase 4·Recruiting·Listed Feb 2025

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A Safety and Immunogenicity Study of CHIKV VLP Vaccine in Children.

for Chikungunya Virus

Sites in Cincinnati +7 more locations

Sponsor: Bavarian Nordic

Sex: All·Age: 1 - 11·Accepts healthy volunteers

Code: NCT07003984·Phase 3·Recruiting·Listed Jun 2025

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A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

for Fabry Disease

Sites in Cincinnati +5 more locations

Sponsor: Amicus Therapeutics

Sex: All·Age: 2 - 11

Code: NCT06904261·Phase 3·Recruiting·Listed Apr 2025

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A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease

for Fabry Disease

Sites in Cincinnati +5 more locations

Sponsor: Chiesi Farmaceutici S.p.A.

Sex: All·Age: 2 - 17

Code: NCT06328608·Phase 2, Phase 3·Recruiting·Listed Mar 2024

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A Study Comparing KB803 and Matched Placebo in Patients With Dystrophic Epidermolysis Bullosa

for Dystrophic Epidermolysis Bullosa

Sites in Cincinnati +4 more locations

Sponsor: Krystal Biotech, Inc.

Sex: All·Age: 6 Months+

Code: NCT07016750·Phase 3·Recruiting·Listed Jun 2025

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Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

for Sensorineural Hearing Loss, Bilateral

Sites in Cincinnati +5 more locations

Sponsor: Akouos, Inc.

Sex: All·Age: All ages

Code: NCT05821959·Phase 1, Phase 2·Recruiting·Listed Apr 2023

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Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).

for IGF1 Deficiency

Sites in Cincinnati +4 more locations

Sponsor: Esteve Pharmaceuticals, S.A.

Sex: All·Age: 2 - 18

Code: NCT00903110·Recruiting·Listed May 2009

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Otoferlin Gene-mediated Hearing Loss Natural History Study

for Sensorineural Hearing Loss, Bilateral

Sites in Cincinnati +3 more locations

Sponsor: Akouos, Inc.

Sex: All·Age: Up to 44

Code: NCT05572073·Recruiting·Listed Oct 2022

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A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

for Glycogen Storage Disease Type II Infantile Onset

Sites in Cincinnati +5 more locations

Sponsor: Amicus Therapeutics

Sex: All·Age: Up to 17

Code: NCT04808505·Phase 3·Recruiting·Listed Mar 2021

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A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

for Mucopolysaccharidosis II

Sites in Cincinnati +7 more locations

Sponsor: Denali Therapeutics Inc.

Sex: All·Age: 2 - 25

Code: NCT05371613·Phase 2, Phase 3·Recruiting·Listed May 2022

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Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy

for DMD-Associated Dilated Cardiomyopathy

Sites in Cincinnati, Columbus +1 more location

Sponsor: Sardocor Corp.

Sex: Male·Age: 18+

Code: NCT06224660·Phase 1·Recruiting·Listed Jan 2024

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64Cu-LNTH-1363S in Patients With Sarcoma or Gastrointestinal Tract Cancer

for Metastatic Sarcoma

Sites in Cincinnati +4 more locations

Sponsor: Lantheus Medical Imaging

Sex: All·Age: 15+

Code: NCT06298916·Phase 1, Phase 2·Recruiting·Listed Mar 2024

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Early Signals of the Transition From Immune Quiescence to Activation in the Liver Allograft Microenvironment and in the Circulation

for Liver Transplant

Sites in Cincinnati +9 more locations

Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

Sex: All·Age: 3 - 13

Code: NCT07549503·Phase 2·Recruiting·Listed Apr 2026

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A Pragmatic Clinical Trial of the WE BEAT Well-Being Education Program in Adolescent Congenital Heart Disease: WE BEAT CHD Study

for Patient-Reported Outcome Measures (PROMs)

Sites in Cincinnati +13 more locations

Sponsor: Carelon Research

Sex: All·Age: 12 - 17

Code: NCT07525843·Recruiting·Listed Apr 2026

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A Study of Uveitis in Children <18 Years of Age

for Uveitis

Sites in Cincinnati, Columbus +2 more locations

Sponsor: Jaeb Center for Health Research

Sex: All·Age: Up to 17

Code: NCT07518134·Recruiting·Listed Apr 2026

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Bowel Continence Across the Lifespan in People With Spina Bifida

for Spina Bifida

Sites in Cincinnati, Columbus +22 more locations

Sponsor: David Chu

Sex: All·Age: 5+

Code: NCT07390318·Recruiting·Listed Feb 2026

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Quercetin in Patients With XIAP (X-linked Inhibitor of Apoptosis) Deficiency

for XIAP Deficiency

1 location in Cincinnati

Sponsor: Children's Hospital Medical Center, Cincinnati

Sex: Male·Age: 2+

Code: NCT07433621·Phase 1·Recruiting·Listed Feb 2026

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Quantifying Motor Network Dynamics to Predict and Enhance Outcomes in Pediatric Dystonia

for Dystonia

1 location in Cincinnati

Sponsor: Children's Hospital Medical Center, Cincinnati

Sex: All·Age: 6 - 21·Accepts healthy volunteers

Code: NCT07325175·Recruiting·Listed Jan 2026

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Effects of Exercise on Lipids and Endothelial Function in Youth

for Obesity & Overweight

1 location in Cincinnati

Sponsor: Children's Hospital Medical Center, Cincinnati

Sex: All·Age: 10 - 20

Code: NCT07409064·Recruiting·Listed Feb 2026

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Zoledronate to Prevent Bone Health Complications in Pediatric Hematopoietic Stem Cell Transplant Survivors

for Hematopoietic Stem Cell Transplantation

1 location in Cincinnati

Sponsor: Children's Hospital Medical Center, Cincinnati

Sex: All·Age: 5 - 18

Code: NCT07375290·Phase 1·Recruiting·Listed Jan 2026

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Verifying Antibodies After Live Immunization Delivery (VALID): A Study of Measles Vaccine Immunogenicity in Children With Sickle Cell Disease

for Sickle Cell Disease

1 location in Cincinnati

Sponsor: Children's Hospital Medical Center, Cincinnati

Sex: All·Age: 6 Months - 6

Code: NCT07356050·Recruiting·Listed Jan 2026

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Collection of Research Data and Samples From Patients Who Experience Immunotherapy Side Effects

for Malignancy

Sites in Cincinnati, Franklin +403 more locations

Sponsor: Alliance for Clinical Trials in Oncology

Sex: All·Age: All ages

Code: NCT04242095·Recruiting·Listed Jan 2020

Apply to this trial