Clinical trials: Sickle Cells Disease
Diagnoses Disease
Evaluation of HemoTypeSC as a Novel Rapid Test for Point-of-Care Screening for Sickle-Cell Disease, Hemoglobin C Disease, and Carrier Status in Low-Resource Settings
Sickle cell disease is a life-threatening genetic disorder that can be effectively treated following early diagnosis via newborn screening.
- Trial name
- NCT03619798
- Testing
- HemoTypeSC
- Where
- United States
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- Silver Lake Research Corporation
Sickle Cell Disease, Sickle Cell Anemia
Actigraphy Improvement With Voxelotor (ActIVe) Study
This is a study to evaluate the effect of voxelotor on daily physical activity and sleep quality, as measured by a wrist-worn device in participants with sickle cell disease (SCD) and chronic moderate anemia.
- Trial name
- ActIVe (NCT04400487)
- Testing
- Voxelotor
- Where
- 10 sites in United States
- Volunteers
- 25 people
- Sponsor
- Pfizer
Sickle Cell Disease
Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease
The purpose of the study is to Evaluate the Effect of Ticagrelor versus Placebo in Reducing the Rate of Vaso-Occlusive Crises in Paediatric Patients with Sickle Cell Disease
- Trial name
- HESTIA3 (NCT03615924)
- Testing
- Ticagrelor
- Where
- 55 sites in United States, Belgium and 14 more
- Volunteers
- 193 people
- Sponsor
- AstraZeneca
Cancer, Breakthrough Pain
Efficacy/Safety Study of ACTIQ® for Opioid-Tolerant Children and Adolescents With Breakthrough Pain (BTP)
The primary objective of the study is to evaluate the efficacy of ACTIQ treatment for the management of breakthrough pain (BTP) compared to placebo treatment in children with cancer and non-cancer pain who are receiving…
- Trial name
- NCT00236041
- Testing
- ACTIQ®
- Where
- 31 sites in United States, Puerto Rico
- Volunteers
- 38 people
- Sponsor
- Cephalon
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Sickle Cell Disease
An Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia
This is an open-label extension study of IMR-687 in adult patients who completed Imara's blinded Phase 2a study (IMR-SCD-102). The open-label extension study will evaluate long-term safety and tolerability.
- Trial name
- NCT04053803
- Testing
- IMR-687
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 30 people
- Sponsor
- Imara, Inc.
Sickle Cell Disease
Plasma DNA and Vascular Remodelling in Patients With Sickle Cell Disease
The purpose of this study is to evaluate the relationship between plasma DNA levels and micro- and macro-circulatory vascular remodelling in patients with sickle cell disease
- Trial name
- PADRE (NCT02721472)
- Testing
- micro- and macro-circulatory vascular remodelling measures not practice in routine care, Biological measures not practice in routine care
- Where
- France
- Volunteers
- 44 people
- Sponsor
- Theravia
Healthy Volunteers, Anemia, Sickle Cell
A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease
The purpose of the study is to assess the safety and tolerability of AG-946 in healthy volunteers after oral administration of single ascending doses (SAD) and multiple ascending doses (MAD) of AG-946 over 14 or up to 28 days of…
- Trial name
- NCT04536792
- Testing
- AG-946
- Where
- 10 sites in United States, Denmark and 1 more
- Volunteers
- 122 people, healthy volunteers welcome
- Sponsor
- Agios Pharmaceuticals, Inc.
Sickle Cell Disease Vaso-occlusive Crisis
Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis
This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with…
- Trial name
- NCT06699849
- Testing
- CSL889
- Where
- 30 sites in United States, Turkey (Türkiye) and 1 more
- Volunteers
- 70 people
- Sponsor
- CSL Behring
Sickle Cell Disease
Clinical Study on the Safety and Efficacy of BRL-101 in the Treatment of Sickle Cell Disease
This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem…
- Trial name
- NCT06287086
- Testing
- BRL-101 autologous hematopoietic stem and progenitor cells injection
- Volunteers
- 1 people
- Sponsor
- Bioray Laboratories
Sickle Cell Disease (SCD)
Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of ALXN1820 in Adult Participants With Sickle Cell Disease
The primary objective of this study is to assess the safety and tolerability of ALXN1820 SC (subcutaneous) in participants with SCD (Sickle Cell Disease).
- Trial name
- PHOENIX (NCT05565092)
- Testing
- ALXN1820
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Sickle Cell Disease
Safety of Single Doses of CSL889 in Adult Patients With Sickle Cell Disease
This is a phase 1, first-in-human, multi-center, open-label, single dose cohort study to evaluate the safety and tolerability, pharmacokinetics (PK), exploratory pharmacodynamics (PD), and biomarkers of target engagement of…
- Trial name
- NCT04285827
- Testing
- CSL889
- Where
- 15 sites in United States, Netherlands and 1 more
- Volunteers
- 28 people
- Sponsor
- CSL Behring
Sickle Cell Disease
Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
This study consists of four parts, Parts A, B, C, and D. Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease ages 6 to 17 years.
- Trial name
- HOPE-KIDS (NCT02850406)
- Testing
- Voxelotor
- Where
- 20 sites in United States, Lebanon and 1 more
- Volunteers
- 147 people
- Sponsor
- Pfizer
Sickle Cell Disease
TRF-1101 Assessment in Sickle Cell Disease
This study is designed to assess the safety, tolerability, and activity of TRF-1101 on microvascular blood flow, vascular endothelial injury, and vasoocclusive pain associated with sickle cell disease.
- Trial name
- NCT00773890
- Testing
- TRF-1101
- Where
- 6 sites in United States
- Volunteers
- 80 people
- Sponsor
- TRF Pharma, Inc
Sickle Cell Disease, Sickle Cell Anemia
A Study of HQK-1001 in Patients With Sickle Cell Disease
The purpose of this study is to evaluate the safety and tolerability of three dose levels of HQK-1001 administered once daily for 26 weeks in subjects with sickle cell disease.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin S Disease
- Trial name
- NCT01322269
- Testing
- HQK-1001
- Where
- 16 sites in United States, Canada and 3 more
- Volunteers
- 52 people
- Sponsor
- HemaQuest Pharmaceuticals Inc.
Sickle Cell Disease
Drug Interaction Study of GBT440 With Caffeine, S-warfarin, Omeprazole, and Midazolam in Healthy Subjects
The purpose of this study to evaluate the effect of concomitant administration of GBT440 on caffeine (a CYP1A2 probe substrate), S warfarin (a CYP2C9 probe substrate), omeprazole (a CYP2C19 probe substrate), and midazolam (a…
- Trial name
- NCT02567682
- Testing
- GBT440
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Global Blood Therapeutics
Sickle Cell Disease
Evaluation of Different Dose Regimens of Aes-103 Given for 28 Days to Subjects With Stable Sickle Cell Disease
Sickle cell disease (SCD) is a genetic blood disorder characterized by the presence of sickle-shaped red blood cells. In the U.S. and the U.K. this occurs primarily in persons of African origin.
- Trial name
- NCT01987908
- Testing
- Aes-103
- Where
- United Kingdom
- Volunteers
- 35 people
- Sponsor
- Baxalta now part of Shire
Sickle Cell Disease
A Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell Disease
The purpose of this study was to evaluate the safety and tolerability, and the pharmacokinetic and biologic activity of INCB059872 in participants with sickle cell disease.
- Trial name
- NCT03132324
- Testing
- INCB059872
- Where
- 7 sites in United States
- Volunteers
- 12 people
- Sponsor
- Incyte Corporation
Sickle Cell Disease
Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with…
- Trial name
- NCT07224360
- Testing
- Anumigilimab
- Where
- 38 sites in United States, Belgium and 5 more
- Volunteers
- 63 people
- Sponsor
- CSL Behring
Anemia, Sickle Cell
A Study of the Absorption, Metabolism, and Excretion of GBT440 in Healthy Male Subjects
This study will provide information regarding the metabolic pathway of GBT440, the need for evaluation of potential drug-drug interactions, and the need for studies in special populations.
- Trial name
- NCT02497924
- Testing
- GBT440
- Where
- United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Global Blood Therapeutics