Phase 1 Sickle Cell Disease Clinical Trials
21 recruitingPhase 1
What is a Phase 1 trial?
Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.
Showing 1–20 of 21 trials
Recruiting
Phase 1Phase 2
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
Sickle Cell Disease
Novartis Pharmaceuticals161 enrolled3 locationsNCT06546670
Recruiting
Phase 1
SCD Stem Cell Mobilization and Apheresis Using Motixafortide
Sickle Cell Disease
St. Jude Children's Research Hospital15 enrolled2 locationsNCT06442761
Recruiting
Phase 1
Methylphenidate to Address Attention and Executive Deficits Among Children With Sickle Cell Disease
Cognitive ImpairmentSickle Cell DiseaseExecutive Dysfunction
St. Jude Children's Research Hospital72 enrolled1 locationNCT07226219
Recruiting
Phase 1Phase 2
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
Sickle Cell Disease
Mark Walters, MD9 enrolled2 locationsNCT04774536
Recruiting
Phase 1
Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease
Sickle Cell Disease
Fulcrum Therapeutics24 enrolled6 locationsNCT07431398
Recruiting
Phase 1
A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)
Sickle Cell Disease
Disc Medicine, Inc24 enrolled3 locationsNCT07187973
Recruiting
Phase 1Phase 2
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
Sickle Cell Disease
Kamau Therapeutics15 enrolled4 locationsNCT04819841
Recruiting
Phase 1Phase 2
Efficacy Safety Study of Gene Therapy for Sickle Cell DiseaseSCD Using Autologous CD34+ Cells Transduced ex Vivo, Carrying a Corrected Globin Gene and a Silencing RNA.
Sickle Cell Disease
Assistance Publique - Hôpitaux de Paris15 enrolled1 locationNCT07432867
Recruiting
Phase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell DiseaseHb-SS DiseaseHemoglobin S+3 more
National Heart, Lung, and Blood Institute (NHLBI)25 enrolled1 locationNCT05904093
Recruiting
Phase 1
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
Beta-ThalassemiaSickle Cell DiseaseSickle Cell Anemia (HbSS, or HbSβ-thalassemia0)+1 more
Daniel Bauer10 enrolled1 locationNCT06647979
Recruiting
Phase 1
Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
Sickle Cell Disease
Abdullah Kutlar18 enrolled1 locationNCT01245179
Recruiting
Phase 1Phase 2
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
HemoglobinopathiesBone Marrow Failure SyndromesMetabolic Disorders+3 more
Washington University School of Medicine29 enrolled4 locationsNCT03128996
Recruiting
Phase 1
Gene Editing For Sickle Cell Disease
Sickle Cell Disease
St. Jude Children's Research Hospital25 enrolled1 locationNCT06506461
Recruiting
Phase 1Phase 2
Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease
Sickle Cell Disease
Emory University21 enrolled2 locationsNCT02447874
Recruiting
Phase 1Phase 2
Cannabidiol in Sickle Cell Disease
Sickle Cell Disease
Icahn School of Medicine at Mount Sinai52 enrolled1 locationNCT06930703
Recruiting
Phase 1
A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
Sickle Cell Disease
EpiDestiny, Inc.20 enrolled1 locationNCT04055818
Recruiting
Phase 1
PET Imaging of Vaso-Occlusive Crisis (VOC) in SCD
Sickle Cell Disease
Enrico Novelli20 enrolled1 locationNCT04925492
Recruiting
Phase 1Phase 2
Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease
Sickle Cell DiseaseSickle-Cell Disease With Crisis
Essen Biotech85 enrolled1 locationNCT06399107
Recruiting
Phase 1Phase 2
LEARNER- Low dosE AspiRiN prEterm tRial (Angola)
Sickle Cell DiseasePregnancy ComplicationsPre-Eclampsia+1 more
Instituto Nacional de Investigacao em Saude, Angola450 enrolled1 locationNCT06417411
Completed
Phase 1
A Single and Multiple Dose Study to Determine the Safety and Tolerability of GBT021601 in Healthy Adult Volunteers - Part A
Sickle Cell Disease
Linear Clinical Research Ltd64 enrolled1 locationACTRN12620001324909