Clinical trials: Sickle Cell Disorders
Sickle Cell Disease
Preventing Acute Chest Syndrome by Transfusion Feasibility Study
Acute chest syndrome (ACS) is similar to severe pneumonia and is a common cause of hospitalizations for people with sickle cell disease (SCD). Blood transfusions are one treatment option for ACS.
- Trial name
- PROACTIVE (NCT00951808)
- Testing
- Single blood transfusion, Standard care
- Where
- 24 sites in United States
- Volunteers
- 237 people
- Sponsor
- Carelon Research
Sickle Cell Disease, Leg Ulcers
Resolution of Sickle Cell Leg Ulcers With Voxelotor
This study is a Phase 3, multicenter, randomized, placebo-controlled study to evaluate the efficacy of voxelotor and standard of care for the treatment of leg ulcers in participants with sickle cell disease.
- Trial name
- RESOLVE (NCT05561140)
- Testing
- Voxelotor Oral Tablet
- Where
- 23 sites in Brazil, Kenya and 1 more
- Volunteers
- 88 people
- Sponsor
- Pfizer
Sickle Cell Disease
High Dose Vitamin D for Sickle Cell Disease
Vitamin D deficiency (VDD) is very common among African American adolescents and adults in the US, ten times higher than is seen in Caucasians. VDD is also quite common in sickle cell disease (SCD).
- Trial name
- SCD-VitD (NCT01331148)
- Testing
- Vitamin D
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Wake Forest University Health Sciences
Infections, Streptococcal
Immunization of Children Between 8 Weeks and 2 Years of Age With GSK Pneumococcal Vaccine GSK1024850A
The aim of the study is to evaluate the immunogenicity, safety and reactogenicity of GSK Biologicals' pneumococcal conjugate vaccine GSK1024850A.
- Trial name
- NCT01175083
- Testing
- GSK1024850A (Synflorix), Tritanrix-HepB/Hib, Polio Sabin
- Where
- Burkina Faso
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Sickle Cell Disease
Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031
Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials
- Trial name
- 034OLE (NCT03573882)
- Testing
- Voxelotor
- Where
- 60 sites in United States, Canada and 9 more
- Volunteers
- 179 people
- Sponsor
- Pfizer
Sickle Cell Disease
Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO).
The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis.
- Trial name
- NCT01090323
- Testing
- ICL670
- Where
- 39 sites in United States, Canada and 3 more
- Volunteers
- 185 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Vaso-occlusive Crisis
Treatment of Sickle Cell Patients Hospitalized in Pain Crisis With Prophylactic Dose Low-molecular-weight Heparin (LMWH) Versus Placebo
Sickle cell disease (SCD) is one of the most common inherited diseases worldwide and exhibits highest frequency in people of African descent.
- Trial name
- NCT01419977
- Testing
- Dalteparin
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Duke University
Investigation of Platelet Aggregation in Paediatric Patients With Sickle Cell Disease
A Pharmacokinetic (PK) and Pharmacodynamic (PD) Dose-ranging Phase II Study of Ticagrelor in Paediatric Patients With Sickle Cell Disease
The purpose of this Phase II dose-ranging study is to investigate pharmacokinetic (PK) and pharmacodynamic (PD) properties of various doses of ticagrelor followed by 4 weeks of twice-daily treatment in paediatric patients with…
- Trial name
- HESTIA 1 (NCT02214121)
- Testing
- Ticagrelor Dose 1a + Dose 2a, Ticagrelor Dose 1b + Dose 2b
- Where
- 18 sites in United States, Canada and 4 more
- Volunteers
- 46 people
- Sponsor
- AstraZeneca
Sickle Cell Disease, Vaso-Occlusive Crisis
Adjuvant Low-dose Ketamine in Pediatric Sickle Cell Vaso-occlusive Crisis
Acute vaso-occlusive episodes (VOEs) in sickle cell disease (SCD) are primarily managed with opioids. Tolerance and hyperalgesia to opioids develops due to N-methyl-D-aspartate (NMDA)-receptor mediated activation of the…
- Trial name
- AKTSS (NCT03296345)
- Testing
- Ketamine
- Where
- United States
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Sickle Cell Disease
Dose Escalation Study to Evaluate the Safety, Tolerability, PK and PD of Voxelotor in Patients With SCD
Study participants will undergo up to four periods of voxelotor administered orally at progressively higher dose levels from 1500 mg until either a maximum tolerated dose (MTD) or 3000 mg/day dose is reached, whichever occurs…
- Trial name
- NCT04247594
- Testing
- Voxelotor
- Where
- 6 sites in United Kingdom
- Volunteers
- 6 people
- Sponsor
- Pfizer
Sickle Cell Disease
Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease
This Phase 1/Phase 2 study will evaluate GMI-1070, a pan-selectin inhibitor, in adults with stable sickle cell disease. The study will assess safety, pharmacokinetics, and microvascular effects of intravenous GMI-1070 in the…
- Trial name
- NCT00911495
- Testing
- GMI-1070
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- GlycoMimetics Incorporated
Sickle Cell Disease
Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease
The objective of this study is to evaluate the pharmacokinetics of deferiprone and its 3-O-glucuronide metabolite following administration of a single 1500 mg dose of Ferriprox in patients with sickle cell disease.
- Trial name
- NCT01835496
- Testing
- single 1500 mg dose of Ferriprox
- Where
- Canada
- Volunteers
- 8 people
- Sponsor
- ApoPharma
Pneumococcal Infections
A Study to Evaluate the Safety, Tolerability, and Immunogenicity of V114 in Children With Sickle Cell Disease (V114-023/PNEU-SICKLE)
This study is designed to describe the safety, tolerability, and immunogenicity of V114 in children with sickle cell disease.
- Trial name
- NCT03731182
- Testing
- V114, Prevnar 13™
- Where
- 19 sites in United States, Brazil and 5 more
- Volunteers
- 104 people
- Sponsor
- Merck Sharp & Dohme LLC
Sickle Cell Disease, Thalassemia
Effects of Transfusion of Older Stored Red Cells
The purpose of this study is to determine the effects of transfusion of fresh and stored blood on patients. The investigators hope to test: whether a similar effect (older stored blood is associated with worse outcomes) is seen…
- Trial name
- NCT01534676
- Testing
- Transfusion, Blood, Chelation therapy
- Where
- United States
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- Columbia University
Sickle Cell Disease
Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)
This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD).
- Trial name
- NCT03263559
- Testing
- Haploidentical Bone Marrow Transplantation, Hydroxyurea, Rabbit-ATG
- Where
- 32 sites in United States
- Volunteers
- 95 people
- Sponsor
- Medical College of Wisconsin
Sickle Cell Disease
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease
The purpose of this study is to determine the correct prasugrel dosage to be given to children with sickle cell disease (SCD).
- Trial name
- Crescent (NCT01476696)
- Testing
- Prasugrel
- Where
- 9 sites in United States
- Volunteers
- 33 people
- Sponsor
- Eli Lilly and Company
Sickle Cell Disease, Vaso-occlusive Pain Episode
Arginine Therapy for Sickle Cell Disease Pain
The aim of this study is to determine whether giving extra arginine, a simple amino acid, to patients with sickle cell disease seeking treatment for a pain crisis (vaso-occlusive painful events (VOE) will decrease pain scores…
- Trial name
- NCT02536170
- Testing
- L-arginine, L-arginine Loading Dose
- Where
- 3 sites in United States
- Volunteers
- 108 people
- Sponsor
- Emory University
Anemia, Sickle Cell
Antioxidant Therapy to Reduce Inflammation in Sickle Cell Disease
The purpose of this study is to determine whether alpha-lipoic acid and acetyl-L-carnitine will lower systemic inflammation in patients with Sickle Cell Disease by reducing oxidative stress, which will result in a decrease in the…
- Trial name
- NCT01054768
- Testing
- alpha-lipoic acid and acetyl-L-carnitine, Control
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Sickle Cell Disease
A Study Aimed to Assess the Needs of Subjects With Sickle Cell Disease and Healthcare Professionals Managing Sickle Cell Disease Patients in Selected Nigerian Centres
This pilot research is aimed to assess the needs of patients and health workers involved in Sickle Cell Disease (SCD) management in Nigeria.
- Trial name
- NCT04505969
- Testing
- Questionnaire to assess needs
- Where
- 4 sites in Nigeria
- Volunteers
- 192 people
- Sponsor
- Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus
Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia
Pharmacokinetics of Oral Hydroxyurea Solution
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
- Trial name
- HUPK (NCT03763656)
- Testing
- Oral Hydroxyurea (100 mg/mL) Solution
- Where
- 6 sites in Jamaica, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Nova Laboratories Limited