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Home › Conditions › Sickle Cell Disease

Sickle Cell Disease

Part of Anemia

17 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Sickle Cell Disease

116 trials tracked for Sickle Cell Disease: 15 in phase 3 or 4 and 5 with published results. The most-cited published study has 624 citations.

116Trials tracked
15Phase 3 & 4
0Recruiting
5With published results
Phase distribution
Phase 4 5 Phase 3 10 Phase 2 49 Phase 1 15 Other / NA 37
  1. Phase 3 Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE) Completed · 624 cited
  2. Phase 3 Novel Use Of Hydroxyurea in an African Region With Malaria Completed · 148 cited
  3. Phase 4 Pilot Study for Patients With Poor Response to Deferasirox Completed · 93 cited
  4. Phase 3 Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial Completed · 13 cited
  5. Phase 3 Age of Blood in Sickle Cell Transfusion Completed · 5 cited
  6. Phase 4 Comparing Acute Pain Management Protocols for Patients With Sickle Cell Disease Completed
Show 44 more trials
  1. Phase 4 Rejuvesol® Washed RBC in Sickle Cell Patients Requiring Frequent Transfusions Completed
  2. Phase 4 Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH) Completed
  3. Phase 4 Actigraphy Improvement With Voxelotor (ActIVe) Study Completed
  4. Phase 3 Stem Cell Transplant for Hemoglobinopathy Completed
  5. Phase 3 Stem Cell Transplantation for Sickle Cell Anemia Completed
  6. Phase 3 A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises Completed
  7. Phase 3 Intravenous Magnesium for Sickle Cell Vasoocclusive Crisis Completed
  8. Phase 3 Choosing Opioid Management for Pain and Analyzing Acute Chest Syndrome (ACS) Rates Equally Completed
  9. Phase 3 Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload Completed
  10. Phase 2 Pharmacokinetics of Oral Hydroxyurea Solution Completed
  11. Phase 2 Hydroxyurea and EPO in Sickle Cell Disease Completed
  12. Phase 2 Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs Completed
  13. Phase 2 Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension Completed
  14. Phase 2 Effect of Simvastatin Treatment on Vaso-occlusive Pain in Sickle Cell Disease Completed
  15. Phase 2 A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia) Completed
  16. Phase 2 Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease Completed
  17. Phase 2 Vaporized Cannabis for Chronic Pain Associated With Sickle Cell Disease Completed
  18. Phase 2 An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001 Completed
  19. Phase 2 SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinical Trial) Completed
  20. Phase 2 Simvastatin (Zocor) Therapy in Sickle Cell Disease Completed
  21. Phase 2 High Dose Vitamin D for Sickle Cell Disease Completed
  22. Phase 2 A Multi-Center Study of Riociguat in Patients With Sickle Cell Diseases Completed
  23. Phase 2 A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease Completed
  24. Phase 2 A Study to Assess the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell Disease Completed
  25. Phase 2 Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease Completed
  26. Phase 2 Daily Vitamin D for Sickle-cell Respiratory Complications Completed
  27. Phase 2 Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies Completed
  28. Phase 2 Study to Assess Safety and Impact of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Pain Crises Completed
  29. Phase 2 Treatment of Sickle Cell Patients Hospitalized in Pain Crisis With Prophylactic Dose Low-molecular-weight Heparin (LMWH) Versus Placebo Completed
  30. Phase 2 Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients Completed
  31. Phase 2 Nitrous Oxide Analgesia Vaso-occlusive Crisis Completed
  32. Phase 2 Inhaled Mometasone to Reduce Painful Episodes in Patients With Sickle Cell Disease Completed
  33. Phase 2 Fixed Dose Flavonoid Isoquercetin on Thrombo-Inflammatory Biomarkers in Subjects With Stable Sickle Cell Disease Completed
  34. Phase 2 Effect of Atorvastatin on Endothelial Dysfunction and Albuminuria in Sickle Cell Disease Completed
  35. Phase 2 Transplantation for Patients With Sickle Cell Disease From Mismatched Family Donors of Bone Marrow Completed
  36. Phase 2 HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity Completed
  37. Phase 2 Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation Completed
  38. Phase 2 Vitamin D for Sickle-cell Respiratory Complications Completed
  39. Phase 2 A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease Completed
  40. Phase 2 Arginine Therapy for Sickle Cell Disease Pain Completed
  41. Phase 2 Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO). Completed
  42. Phase 2 Decitabine for High-Risk Sickle Cell Disease Completed
  43. Phase 2 Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease Completed
  44. Phase 2 Assessing the Safety of Buprenorphine in People With Sickle Cell Disease Completed

Showing the 50 most-cited and recently-updated of 116 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found Updated — new results For clinicians

Sickle Cell Disease: what the trials found

Hydroxyurea is associated with significant clinical outcomes in sickle cell disease. In a study involving post-transplant patients, hydroxyurea was linked to an 88.0% two-year event-free survival rate and high rates of neutrophil recovery (92.9% to 100.0%) 1. Additionally, hydroxyurea demonstrated a 15% hemoglobin (Hb) response rate in another cohort 2.

Voxelotor has been evaluated for its impact on hemoglobin levels; results showed that 41.7% of participants achieved an increase in Hb of more than 1 g/dL at week 12, and 26.1% achieved this same increase at week 24 6. A larger Phase 3 trial also reported a significant number of participants achieving an Hb increase greater than 1 g/dL from baseline to week 24 10.

Pain management strategies show varied results in clinical trials. Standardized, weight-based hydromorphone was associated with a statistically significant reduction in pain scores (p=0.0311) and showed consistent improvements in visual analogue scale (VAS) scores over time (p=0.0049), while also showing lower rates of nausea during emergency department visits compared to other groups (p=0.0001) 9. L-arginine hydrochloride was associated with a reduction in pain scores and PROMIS pain interference scores 12.

Other established interventions include deferasirox 19, which showed significant results in pharmacokinetic measures 5; alemtuzumab 15; siklos 7; rejuvesol 8; and inclusion of busulfan, fludarabine, ATG, TLI 16. Inclacumab was evaluated for vaso-occlusive crises (VOCs), but did not show a statistically significant difference in the rate or time to first VOC through week 48 14.

Intravenous magnesium sulfate was studied for hospital stay duration and morphine equivalents used, though these findings did not reach statistical significance 17. Transfusion studies also assessed the proportion of biochemically old red cell units 13.

Recent results — preliminary, needs further review

  • Biotin labeled red blood cells showed varying percentages of presence (97.3%, 80%, and 21%) in a Phase 1 trial 3.
  • Daily oral vitamin D3 (3,333 IU) was evaluated regarding the ratio of residual lung volume to total lung capacity 20.
  • CliniMACS was investigated in a small cohort 4.

For the clinician treating this condition

  • Hydroxyurea is associated with high rates of neutrophil recovery and significant event-free survival post-transplant, as well as measurable hemoglobin responses.
  • Voxelotor has demonstrated the ability to increase hemoglobin levels by more than 1 g/dL in a subset of patients at weeks 12 and 24.
  • Standardized, weight-based hydromorphone is associated with significant reductions in pain scores and lower rates of nausea during emergency visits compared to alternative protocols.
  • Inclacumab did not show statistically significant differences in the rate or timing of vaso-occlusive crises over a 48-week period.

AI synthesis of 18 cited trials, updated Jun 20, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Local ancestry-aware GWAS finds 38 European-tract and 12 African-tract loci for eGFR in sickle cell disease
Hematology Meta-analysis
Local ancestry-aware GWAS finds 38 European-tract and 12 African-tract loci for eGFR in sickle cell disease New genetic markers may help identify kidney damage in sickle cell disease
This genome-wide association meta-analysis used local ancestry-aware analysis to identify genomic regions linked to estimated glomerular fil…
Researchers identified specific genetic regions that may help doctors spot and treat kidney issues in people with sickle cell disease earlie…
Oct 27, 2026
Mitapivat Improves Haemoglobin Concentration in Patients with Chronic Sickle Cell Disease
Hematology RCT
Mitapivat Improves Haemoglobin Concentration in Patients with Chronic Sickle Cell Disease Trial shows mitapivat improves hemoglobin in sickle cell disease
Mitapivat demonstrated a significant increase in haemoglobin levels for patients with sickle cell disease in this Phase 3 trial.
A new clinical trial shows that mitapivat significantly improves hemoglobin levels for adults and older teens with sickle cell disease.
Oct 24, 2026
Forty independent genetic loci associated with acute chest syndrome occurrence in sickle cell disease patients
Hematology Meta-analysis
Forty independent genetic loci associated with acute chest syndrome occurrence in sickle cell disease patients Identifying Genetic Markers Linked to Chest Problems in Sickle Cell Disease
This meta-analysis of genetic variants in patients with sickle cell disease identified 40 independent genetic loci associated with acute che…
New research identifies specific genetic markers that may help predict and manage serious chest complications in patients with sickle cell d…
medRxiv Oct 19, 2026
RBC alloimmunization affects 8.76% of transfused sickle cell patients in resource-limited settings
Hematology Meta-analysis
RBC alloimmunization affects 8.76% of transfused sickle cell patients in resource-limited settings New data shows common risks for sickle cell disease patients
This meta-analysis pooled data from 3128 patients with sickle cell disease who received red blood cell transfusions in resource-limited coun…
New data reveals that nearly 9% of sickle cell patients who receive blood transfusions develop a common immune reaction.
Oct 1, 2026
Integrated haemotherapeutic systems improve transfusion safety and sustainability for sickle cell disease patients in Africa
Rheumatology Sys. Review
Integrated haemotherapeutic systems improve transfusion safety and sustainability for sickle cell disease patients in Africa New strategies aim to improve blood safety for sickle cell disease
This narrative review synthesizes current challenges and proposed solutions for sickle cell disease haemotherapy in Africa. The authors argu…
Newer strategies for blood management aim to solve common problems like blood shortages and infection risks for people with sickle cell dise…
Frontiers Sep 25, 2026
Chronic pain in aging adults with sickle cell disease shifts toward persistent, multifactorial syndromes
Cardiology Sys. Review
Chronic pain in aging adults with sickle cell disease shifts toward persistent, multifactorial syndromes Sickle Cell Pain Changes With Age, Review Finds
This narrative review examines the pathophysiology and management of chronic pain in aging adults with sickle cell disease. The authors conc…
Sickle cell pain isn't just crises anymore. As patients age, it becomes persistent and driven by organ damage, a review finds.
Frontiers Sep 23, 2026
Surgery achieves 70% single-surgery anatomical success in sickle cell retinopathy
Ophthalmology Meta-analysis
Surgery achieves 70% single-surgery anatomical success in sickle cell retinopathy Surgery for Sickle Cell Retinopathy Shows 70% Success Rate
This systematic review and meta-analysis of 7 studies (408 eyes) found that surgical treatment for sickle cell retinopathy yields a pooled s…
A review of 7 studies finds surgery for sickle cell retinopathy has a 70% single-surgery success rate and improves vision.
Frontiers Jul 24, 2026
AI-augmented screening improves hereditary hemolytic anemia detection with 92.8% pooled sensitivity
Hematology Meta-analysis
AI-augmented screening improves hereditary hemolytic anemia detection with 92.8% pooled sensitivity AI tools improve accuracy for screening hereditary blood disorders
This meta-analysis of 133,498 individuals screened for hereditary hemolytic anemia (HHA) carriers found that AI-augmented complete blood cou…
New data shows that adding artificial intelligence to blood tests can better identify people carrying genes for conditions like sickle cell …
Jun 29, 2026
Systematic review reveals high prevalence of sickle cell disease across infants and youth in sub-Saharan Africa
Hematology Meta-analysis
Systematic review reveals high prevalence of sickle cell disease across infants and youth in sub-Saharan Africa Sickle cell disease affects nearly 2 percent of kids under 15 in sub-Saharan Africa
A comprehensive meta-analysis of 40 studies from 22 nations highlights the burden of sickle cell disease in sub-Saharan Africa. Prevalence r…
New analysis shows sickle cell disease is more common than thought in infants and children across sub-Saharan Africa, highlighting a critica…
Jun 1, 2026
Narrative review of CRISPR-Cas9 and Fanzor gene therapies for sickle cell disease
Genetics & Precision Medicine Sys. Review
Narrative review of CRISPR-Cas9 and Fanzor gene therapies for sickle cell disease New CRISPR gene therapies for sickle cell disease face lingering technology limits
This narrative review synthesizes evidence on CRISPR-Cas9-based gene therapies and the Fanzor system for sickle cell disease. The authors no…
A review of CRISPR-Cas9 gene therapies for sickle cell disease notes that limitations in the technology still persist.
Frontiers May 26, 2026
Cross-Sectional Analysis of Cardiac Structure and Function in 57 Adults With Sickle Cell Disease
Cardiology Sys. Review
Cross-Sectional Analysis of Cardiac Structure and Function in 57 Adults With Sickle Cell Disease Heart Risk Found in Young Sickle Cell Patients
This observational, cross-sectional study assessed echocardiographic profiles in 57 adults with confirmed Sickle Cell Disease at a tertiary …
Young adults with sickle cell disease often show early signs of heart strain, explaining why some feel tired or short of breath even when ot…
medRxiv Apr 20, 2026
POCUS-guided assessments and serial exchange transfusions improved outcomes in a 20-year-old male with sickle cell disease and acute chest syndrome
Cardiology Cohort
POCUS-guided assessments and serial exchange transfusions improved outcomes in a 20-year-old male with sickle cell disease and acute chest syndrome Bedside Ultrasound Saves Sickle Cell Lives
This single case report describes a 20-year-old male with HbSS, acute chest syndrome, and acute pulmonary hypertension managed in the ICU.
A simple ultrasound at the bedside can tell doctors exactly when a sickle cell patient needs another blood transfusion to save their life.
Frontiers Apr 16, 2026

Research across Anemia

Related studies from across the Anemia family.

Systematic review finds first FDA-approved CRISPR therapy and broader clinical trial exploration
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Systematic review finds first FDA-approved CRISPR therapy and broader clinical trial exploration First CRISPR gene therapy approved for blood disorders, with broader research underway
A systematic review of clinical trials registered at clinicaltrials.gov found the first CRISPR-Cas9 therapy obtained FDA approval in late 20…
The first CRISPR gene therapy is approved for sickle-cell anemia and β-thalassemia, with ongoing trials exploring its use for cancer and inf…
Frontiers Apr 3, 2026
Report examines use of recommended health care measures for sickle cell anemia complications in youth
Hematology
Report examines use of recommended health care measures for sickle cell anemia complications in youth Report examines use of preventive care for children with sickle cell anemia
A report describes the use of recommended health care measures to prevent complications of sickle cell anemia in children and adolescents in…
A new report looked at how children and adolescents with sickle cell anemia in the U.S.
CDC Apr 2, 2026

Questions about Sickle Cell Disease

How does delayed treatment affect children with sickle cell and osteomyelitis?

Delayed treatment in children with sickle cell disease and osteomyelitis increases risks of bone damage, infection spread, and severe complications like sepsis.

Full answer →
Does sickle cell disease make pediatric osteomyelitis harder to treat?

Yes, sickle cell disease makes pediatric osteomyelitis harder to treat due to increased risk of atypical infections like Salmonella, diagnostic challenges from overlapping symptoms, and higher complication rates.

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Did the Renizgamglogene autotemcel infusion increase hemoglobin in severe Sickle Cell Disease patients?

Yes, the Renizgamglogene autotemcel infusion increased hemoglobin in severe Sickle Cell Disease patients, with most treated individuals showing engraftment and higher hemoglobin levels within the first year of follow-up.

Full answer →
What echocardiographic profiles are seen in adults with Sickle Cell Disease?

Adults with sickle cell disease show three main echocardiographic profiles: high cardiac output with enlarged chambers, mild remodeling with higher pulmonary pressure, or near-normal structure; these patterns predict different clinical outcomes.

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