Sickle cell disease (SCD) is a group of inherited blood disorders that result in rigid, sickle-shaped red blood cells which may result in complications such as anemia, multi-organ injury, and increased susceptibility to infections. During Sickle Cell Awareness Month (held every September), clinicians should take some time to consider the disease. By remaining alert for early signs of SCD in patients, clinicians can identify the disease early, which is crucial in helping prevent stroke or other long-term effects. 

Completed clinical trials for SCD represent some of the latest research on SCD therapies, improved disease understanding, and they open doors to additional clinical trials, which provide patients with additional treatment options. Today’s clinical trials rundown features the most recently completed, phase 3, clinical trials for SCD.

Comparison of the Five Most Recently Completed Clinical Trials on Sickle Cell Disease
Study TitleResults?EnrollmentStart DateCompletion Date
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell DiseaseNo Results Posted63November 27, 2018July 7, 2025
Hydroxyurea Optimization Through Precision StudyNo Results Posted104January 17, 2019March 19, 2025
Sickle Cell Disease Treatment With Arginine Therapy (STArT) TrialYes271June 21, 2021July 11, 2024
A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive CrisesNo Results Posted243October 14, 2021June 6, 2024
Age of Blood in Sickle Cell TransfusionYes26October 4, 2017January 11, 2024

Breakdown of the Five Most Recently Completed Sickle Cell Disease Clinical Trials

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

  • Sponsor: Vertex Pharmaceuticals Incorporated
  • Goal: The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.
    Interventions: CTX001
  • Primary Outcome Measures / Timeframe: 
    • Proportion of subjects who have not experienced any severe vaso-occlusive crisis (VOC) for at least 12 consecutive months (VF12) / From 60 days after last RBC transfusion up to 2 years after CTX001 infusion.
    • Proportion of subjects with engraftment (first day of three consecutive measurements of absolute neutrophil count [ANC] ≥500/µL on three different days) / Within 42 days after CTX001 infusion.
    • Time to engraftment / From CTX001 infusion up to two years after CTX001 infusion.
    • Frequency and severity of collected adverse events (AEs) / From screening to 2 years after CTX001 infusion.
    • Incidence of transplant-related mortality (TRM) within 100 days after CTX001 infusion / Within 100 days after CTX001 infusion.
    • Incidence of TRM within 1 year after CTX001 infusion / Within 1 year after CTX001 infusion.
    • All-cause mortality / 2 years after mobilization.
  • Study Details
  • Source

Hydroxyurea Optimization Through Precision Study

  • Sponsor: Lifespan
  • Goal: The primary objective of the study is to evaluate whether a pharmacokinetics-based starting hydroxyurea dose thieves superior fetal hemoglobin response to standard weight-based initial dosing.
  • Interventions: Hydroxyurea
  • Primary Outcome Measures: Fetal Hemoglobin (HbF) Response Following Six Months of Hydroxyurea Therapy
  • Timeframe: Six months after starting daily hydroxyurea therapy
  • Study Details
  • Source

Sickle Cell Disease Treatment with Arginine Therapy (STArT) Trial

  • Sponsor: Claudia R. Morris
  • Goal: The trial is designed to test intravenous (IV) arginine therapy in children with sickle cell disease (SCD) and vaso-occlusive painful episodes (VOE) to further knowledge on efficacy and safety of this orphan drug.
  • Interventions: L-Arginine Hydrochloride
  • Primary Outcome Measures: Time-to-crisis Resolution
  • Timeframe: From study drug delivery to last IV opioid treatment (up to 1,724.1 hours)
  • Study Details
  • Source

A Study to Assess the Safety and Efficacy of Inclacumab in Participants with Sickle Cell Disease Experiencing Vaso-occlusive Crises

  • Sponsor: Pfizer
  • Goal: This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs) in approximately 240 adult and adolescent participants (≥ 12 years of age) with sickle cell disease (SCD).
  • Interventions: Inclacumab, placebo
  • Primary Outcome Measures: Rate of VOCs during the 48-week treatment period.
  • Timeframe: Day 1 – Week 48
  • Study Details
  • Source

Age of Blood in Sickle Cell Transfusion

  • Sponsor: University of North Carolina, Chapel Hill
  • Goal: In aim 1, the study staff will determine the biochemical differences between ≥30 day-old versus ≤10 day-old units. In aim 2, the study staff will determine the physiologic effects of the transfused blood in a patient with SCD. Lastly, in aim 3, the study staff will explore the clinical implications of receiving older red cells over a 3 month period.
  • Interventions: Transfusion
  • Primary Outcome Measures: Proportion of Biochemically Old Red Cell Units
  • Timeframe: Through third transfusion, an average of 18 weeks.
  • Study Details
  • Source
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