Clinical trials: Sickle Cell Anaemia
Sickle Cell Disease
Study to Evaluate the Effect of Ticagrelor Versus Placebo in Reducing Vaso-Occlusive Crises Rate in Pediatric Patients With Sickle Cell Disease.
The purpose of this study is to compare the effect of ticagrelor vs placebo for the reduction of Vaso-Occlusive crises in paediatric patients with Sickle Cell Disease
- Trial name
- HESTIA5 (NCT04293172)
- Testing
- Brilinta
- Sponsor
- AstraZeneca
Chronic Pain
Effectiveness, Safety, and Tolerability Study of Oxymorphone Immediate Release (IR) Oral Liquid in Opioid Tolerant Pediatric Subjects
The purpose of this study is to evaluate the effectiveness, tolerability, and safety of oxymorphone immediate release (IR) oral liquid as an analgesic in pediatric subjects having severe to moderate chronic pain.
- Trial name
- NCT01206907
- Testing
- oxymorphone IR
- Sponsor
- Endo USA Inc., a Keenova Therapeutics Company
Anemia, Sickle Cell, Healthy Volunteers
A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease
The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with sickle cell…
- Trial name
- NCT06481306
- Testing
- BMS-986470, Famotidine, Pantoprazole
- Where
- 32 sites in United States, Canada and 3 more
- Volunteers
- 224 people, healthy volunteers welcome
- Sponsor
- Bristol-Myers Squibb
Hepatitis C Virus Infection
Ledipasvir/Sofosbuvir Fixed-Dose Combination for 12 or 24 Weeks in Genotype 1 or 4 HCV Infected Adults With Sickle Cell Disease
The primary objectives of this study are to evaluate the antiviral efficacy, safety, and tolerability of ledipasvir/sofosbuvir (LDV/SOF) fixed-dose combination (FDC) administered for 12 or 24 weeks in adults with genotype 1 or 4…
- Trial name
- NCT02301936
- Testing
- LDV/SOF
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Gilead Sciences
Sickle Cell Disease, Renal Function Disorder
Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH)
The use of hydroxyurea in sickle cell disease patients with glomerular hyperfiltration and renal failure requires a specific monitoring and dose adjustment in order to remain within the therapeutic interval while limiting the…
- Trial name
- NCT02522104
- Testing
- Siklos
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Theravia
Sickle Cell Disease
A Study of the Effect of IW-1701 (Olinciguat), a Stimulator of Soluble Guanylate Cyclase (sGC), on Patients With Sickle Cell Disease (SCD)
The primary objective of the 1701-202 STRONG SCD study is to evaluate the safety and tolerability of different dose levels of IW-1701 compared with placebo when administered daily for approximately 12 weeks to patients with…
- Trial name
- STRONG SCD (NCT03285178)
- Testing
- IW-1701
- Where
- 37 sites in United States, Lebanon and 1 more
- Volunteers
- 88 people
- Sponsor
- Cyclerion Therapeutics
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
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Sickle Cell Disease (SCD)
An Indian Multi-centric Phase IV Study to Assess the Safety of Crizanlizumab in Sickle Cell Disease Patients
Sickle cell disease (SCD) is a genetic blood disorder. Crizanlizumab is indicated to reduce the frequency of vaso-occlusive crises (VOCs) in patients with SCD aged 16 years and older.
- Trial name
- NCT04662931
- Testing
- crizanlizumab
- Where
- 7 sites in India
- Volunteers
- 140 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Anemia
Evaluation of the AMICUS RBCx System in Sickle Cell Patients
The purpose of this study is to evaluate the performance of the AMICUS Red Blood Cell Exchange (RBCx) System (Exchange and Depletion/Exchange procedures) in patients with sickle cell disease.
- Trial name
- NCT02372877
- Testing
- Amicus Red Cell Exchange in SCD patients
- Where
- 6 sites in United States
- Volunteers
- 83 people
- Sponsor
- Fenwal, Inc.
Healthy, Sickle Cell Anemia
Dose Escalation Study of PF-07209326 in Healthy Participants and Participants With Sickle Cell Disease
This Phase 1 first-in-human, first-in-patient, single ascending dose and multiple dose study will be a randomized, double-blind, placebo-controlled investigation of the safety, tolerability, and pharmacokinetics of PF-07209326 in…
- Trial name
- NCT04255875
- Testing
- PF-07209326
- Where
- 16 sites in United States
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Pfizer
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Sickle Cell Disease, Thalassemia
Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. The overall study objectives are to evaluate the safety and efficacy of CordIn™.
- Trial name
- NCT02504619
- Testing
- CordIn
- Where
- 3 sites in United States, France
- Volunteers
- 1 people
- Sponsor
- Gamida Cell ltd
Sickle Cell Disease
Safety and Pharmacokinetics of SANGUINATE™ in Sickle Cell Disease (SCD) Patients
Prolong proposes to test safety, tolerability and pharmacokinetics of SANGUINATE™ in sickle cell disease (SCD) patients. Prolong's preclinical studies showed that SANGUINATE™ was safe in a number of different animal models and…
- Trial name
- NCT01374165
- Where
- Israel
- Sponsor
- Prolong Pharmaceuticals
Sickle Cell Disease, Pharmacokinetics
Pharmacokinetics and Safety of Endari (L-glutamine) in Sickle Cell Disease Patients
L-glutamine has been approved in the US to reduce the acute complications of sickle cell disease (SCD) in adult and pediatric patients 5 years of age and older.
- Trial name
- NCT04684381
- Testing
- L-glutamine
- Where
- United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Emmaus Medical, Inc.
Sickle Cell Disease, Thalassemia
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.
- Trial name
- FLORAL (NCT06609226)
- Testing
- Etavopivat A, Etavopivat B, Etavopivat C
- Where
- 107 sites in United States, Canada and 15 more
- Volunteers
- 480 people
- Sponsor
- Novo Nordisk A/S
Sickle Cell Disease
Gaming Technology to Engage Adolescent Sickle Cell Patients in Pain Management
Sickle cell disease (SCD) is a common genetic disorder characterized by episodes of pain, yet assessments to identify type, intensity, frequency, and phase of pain among SCD adolescents is lacking.
- Trial name
- NCT03291613
- Testing
- Pinpoint App
- Where
- 2 sites in United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Klein Buendel, Inc.
Sickle Cell Anemia
Study of Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patients With Sickle Cell Anemia
The study assesses the efficacy, safety and tolerability of ACZ885 (canakinumab) in pediatric and young adult patients with sickle cell anemia (SCA).
- Trial name
- NCT02961218
- Testing
- ACZ885
- Where
- 15 sites in United States, Canada and 5 more
- Volunteers
- 49 people
- Sponsor
- Novartis Pharmaceuticals
Healthy Adult Subjects, Sickle Cell Disease
Safety, Tolerability and Pharmacokinetics of FTX-6058
This is a study to evaluate the safety, tolerability and pharmacokinetics of FTX-6058 in healthy adult subjects and adult subjects with sickle cell disease (SCD).
- Trial name
- NCT04586985
- Testing
- FTX-6058 - Two Dosing Periods, FTX-6058 / Midazolam Syrup
- Where
- 2 sites in United States
- Volunteers
- 109 people, healthy volunteers welcome
- Sponsor
- Fulcrum Therapeutics
Sickle Cell Disease
Effect of NUV001 Supplementation in Patients Suffering From Sickle Cell Disease (SCD)
This is a pilot study of daily dosing of NUV001 as a dietary supplement in 12 sickle cell disease patients with 3 months of follow-up plus 1 month after supplementation.The present study is designed to evaluate, first, the safety…
- Trial name
- NCT05789355
- Testing
- NUV001
- Where
- France
- Volunteers
- 12 people
- Sponsor
- LGD