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ClinicalTrials.gov public records Last synced Oct 7, 2026, 2:30 PM EDT

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Showing 49–72 of 85 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Pediatric Hemolytic Anemia Clear all
Recruiting Phase 2 Interventional

Sub-dissociative Dose Ketamine in Treatment of Vaso-occlusive Pain Event in Children and Young Adults

NCT07369024
View directory record Official record
Conditions
Sickle Cell Disease, Vaso-Occlusive Pain Episode in Sickle Cell Disease, Ketamine Infusion, Pain Management, Vaso-Occlusive Crises
Interventions
Sub-dissociative Ketamine Infusion, Normal Saline Placebo Infusion
Drug
Lead sponsor
Mohsen Saidinejad
Other
Eligibility
5 Years to 20 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 7, 2026, 2:30 PM EDT
Completed Phase 2 Interventional Results available

Arginine Therapy for Sickle Cell Disease Pain

NCT02536170
View directory record Official record
Conditions
Sickle Cell Disease, Vaso-occlusive Pain Episode
Interventions
L-arginine, L-arginine Loading Dose, Placebo
Drug · Other
Lead sponsor
Emory University
Other
Eligibility
3 Years to 21 Years
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
3
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 6, 2023 · Synced Oct 7, 2026, 2:30 PM EDT
Completed Phase 1 Interventional

Pharmacokinetics (PK) of Liquid Hydroxyurea in Pediatric Patients With Sickle Cell Anemia

NCT01506544
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Hydroxyurea
Drug
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
2 Years to 17 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
6
States / cities
Birmingham, Alabama • Chicago, Illinois • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 12, 2014 · Synced Oct 7, 2026, 2:30 PM EDT
Completed No phase listed Observational

Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)

NCT03609814
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
Interventions
Clofarabine, Fludarabine Injection
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 4, 2021 · Synced Oct 7, 2026, 2:30 PM EDT
Completed Not applicable Interventional

Preliminary Feasibility and Efficacy of Behavioral Intervention to Reduce Pain-Related Disability in Pediatric SCD

NCT04388241
View directory record Official record
Conditions
Sickle Cell Disease, Pain
Interventions
The Balance Program
Behavioral
Lead sponsor
Children's National Research Institute
Other
Eligibility
8 Years to 17 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Sep 6, 2022 · Synced Oct 7, 2026, 2:30 PM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 7, 2026, 2:30 PM EDT
Completed No phase listed Observational Results available

Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study)

NCT00890396
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
Hydroxyurea
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 7 Years
Enrollment
163 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
14
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Miami, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2020 · Synced Oct 7, 2026, 2:30 PM EDT
Completed Not applicable Interventional Results available

Development and Adaptation of I-STRONG for SCD

NCT06110754
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
I-STRONG for SCD
Behavioral
Lead sponsor
Emory University
Other
Eligibility
12 Years to 18 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
2
States / cities
Atlanta, Georgia • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 19, 2025 · Synced Oct 7, 2026, 2:30 PM EDT
Completed Phase 1 Interventional

Sickle Cell Pain: Intervention With Capsaicin Exposure

NCT03899246
View directory record Official record
Conditions
Neuropathic Pain, Sickle Cell Disease
Interventions
Eight Percent Topical Capsaicin
Drug
Lead sponsor
Children's Hospital of Michigan
Other
Eligibility
14 Years to 21 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Jul 29, 2020 · Synced Oct 7, 2026, 2:30 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Adjuvant Low-dose Ketamine in Pediatric Sickle Cell Vaso-occlusive Crisis

NCT03296345
View directory record Official record
Conditions
Sickle Cell Disease, Vaso-Occlusive Crisis
Interventions
Ketamine
Drug
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
10 Years to 25 Years
Enrollment
62 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2018
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Apr 20, 2021 · Synced Oct 7, 2026, 2:30 PM EDT
Recruiting Phase 1Phase 2 Interventional

Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease

NCT07252050
View directory record Official record
Conditions
Sickle Cell Disease, Hematopoetic Stem Cell Transplant, Haploidentical Hematopoietic Stem Cell Transplant, Haploidentical Stem Cell Transplantation, Graft Failure
Interventions
Ruxolitinib
Drug
Lead sponsor
Arkansas Children's Hospital Research Institute
Other
Eligibility
12 Years to 45 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
4
States / cities
Aurora, Colorado • Atlanta, Georgia • New Orleans, Louisiana + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 7, 2026, 2:30 PM EDT
Not listed Not applicable Interventional

FOCUS for Pediatric Sickle Cell Disease and Cancer

NCT04075877
View directory record Official record
Conditions
Pediatric Cancer, Sickle Cell Disease, Quality of Life, Depressive Symptoms, Coping Skills
Interventions
FOCUS
Behavioral
Lead sponsor
Georgia State University
Other
Eligibility
12 Years to 18 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
2
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Nov 13, 2020 · Synced Oct 7, 2026, 2:30 PM EDT
Recruiting No phase listed Observational

Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease

NCT06941389
View directory record Official record
Conditions
Sickle Cell Disease (SCD)
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
3 Years to 20 Years
Enrollment
480 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
31
States / cities
Birmingham, Alabama • Wilmington, Delaware • Washington D.C., District of Columbia + 19 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 7, 2026, 2:30 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Utilizing A Single Session Problem-Solving Intervention With Caregivers of Pediatric Patients Receiving Chronic Transfusion to Treat Sickle Cell Disease

NCT04606160
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Single Session Problem-Solving Intervention
Behavioral
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
7 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 21, 2022 · Synced Oct 7, 2026, 2:30 PM EDT
Active, not recruiting Phase 3 Interventional

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

NCT05356195
View directory record Official record
Conditions
Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years to 11 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Oct 7, 2026, 2:30 PM EDT
Terminated Phase 4 Interventional Results available

Desmopressin for Bedwetting in Children With SCD

NCT04420585
View directory record Official record
Conditions
Nocturnal Enuresis, Anemia, Sickle Cell
Interventions
Desmopressin
Drug
Lead sponsor
Montefiore Medical Center
Other
Eligibility
8 Years to 21 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
The Bronx, New York
Source: ClinicalTrials.gov public record
Updated May 7, 2024 · Synced Oct 7, 2026, 2:30 PM EDT
Completed Phase 4 Interventional Results available

Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT00867932
View directory record Official record
Conditions
Hemoglobinuria, Paroxysmal
Interventions
Eculizumab
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 17 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
3
States / cities
Orange, California • Pensacola, Florida • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 31, 2018 · Synced Oct 7, 2026, 2:30 PM EDT
Completed No phase listed Observational

Pain Management Protocol for Pediatric Sickle Cell Disease

NCT00386048
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Brief Cognitive-Behavioral Pain Management Training
Behavioral
Lead sponsor
University of South Carolina
Other
Eligibility
8 Years to 18 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
1
States / cities
Columbia, South Carolina
Source: ClinicalTrials.gov public record
Updated Apr 28, 2021 · Synced Oct 7, 2026, 2:30 PM EDT
Completed No phase listed Observational

Evaluation of Nocturnal Enuresis and Barriers to Treatment Among Pediatric Patients With Sickle Cell Disease

NCT01959958
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Questionnaire/Interview
Other
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
6 Years to 17 Years
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 11, 2016 · Synced Oct 7, 2026, 2:30 PM EDT
Completed Phase 2 Interventional Results available

An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome

NCT01193348
View directory record Official record
Conditions
Atypical Hemolytic-Uremic Syndrome
Interventions
Eculizumab
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
1 Month to 18 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
4
States / cities
Atlanta, Georgia • Hackensack, New Jersey • Corpus Christi, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 29, 2015 · Synced Oct 7, 2026, 2:30 PM EDT
Completed Phase 1 Interventional Results available

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

NCT00744692
View directory record Official record
Conditions
Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
Interventions
Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
Biological · Drug
Lead sponsor
Duke University
Other
Eligibility
Up to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 13, 2014 · Synced Oct 7, 2026, 2:30 PM EDT
Terminated Phase 2 Interventional Results available

Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere

NCT03825341
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Hydroxyurea, Hydroxyurea Oral Capsule
Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
9 Months to 18 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 18, 2022 · Synced Oct 7, 2026, 2:30 PM EDT
Completed No phase listed Observational Results available

Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol

NCT01783990
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Hydroxyurea
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
24 Months to 18 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
14
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Miami, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2020 · Synced Oct 7, 2026, 2:30 PM EDT
Completed No phase listed Observational

Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

NCT03609827
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
Interventions
Melphalan
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 4, 2021 · Synced Oct 7, 2026, 2:30 PM EDT