Phase 1 Sickle Cell Clinical Trials

27 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 120 of 27 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

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

Sickle Cell Disease
Disc Medicine, Inc24 enrolled5 locationsNCT07187973
Recruiting
Phase 1

Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease

Sickle Cell Disease
City of Hope Medical Center11 enrolled1 locationNCT03279094
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

Gene Editing For Sickle Cell Disease

Sickle Cell Disease
St. Jude Children's Research Hospital25 enrolled1 locationNCT06506461
Recruiting
Phase 1Phase 2

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

Sickle Cell DiseaseGraft FailureHematopoetic Stem Cell Transplant+2 more
University of Colorado, Denver24 enrolled4 locationsNCT07252050
Recruiting
Phase 1Phase 2

RH Genotype Matched RBC Transfusions

Sickle Cells Disease
Children's Hospital of Philadelphia35 enrolled1 locationNCT04156893
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 DiseaseAttention Deficit Hyperactivity Disorder (ADHD)+1 more
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 1Phase 2

A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease

Healthy VolunteersAnemia, Sickle Cell
Bristol-Myers Squibb184 enrolled19 locationsNCT06481306
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

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

A Safety, Efficacy, and Pharmacokinetic (PK) Study of HBI-002, an Oral Carbon Monoxide (CO) Therapeutic, in Subjects With Sickle Cell Disease (SCD)

Anemia, Sickle Cell
Hillhurst Biopharmaceuticals, Inc.9 enrolled2 locationsNCT06144749
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 1Phase 2

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Diamond-Blackfan AnemiaSickle Cell Anemiabeta thalassemia major
Paul Szabolcs5 enrolled1 locationNCT03653338
Recruiting
Phase 1Phase 2

Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease

Sickle Cell Disease
Emory University21 enrolled2 locationsNCT02447874