Clinical trials: Newborn; Anemia
Sickle Cell Disease
A GBT021601 ADME Microtracer Study in Healthy Volunteers
An Open-label Study of GBT021601 in 8 to 10 healthy male or female participants to evaluate the absorption, distribution, metabolism, and excretion (ADME) of GBT021601.
- Trial name
- NCT05718687
- Testing
- GBT021601
- Where
- Netherlands
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Pfizer
Transfusional Iron Overload, Transfusional Hemosiderosis
Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload
This is a clinical research study in patients who have iron overload in the heart due to chronic blood transfusions. The study will have 2 treatment groups and will compare the safety and efficacy of chelation therapy with a…
- Trial name
- CORDELIA (NCT00600938)
- Testing
- Core Study: Deferasirox, Core Study: Deferoxamine, Extension: deferoxamine to deferasirox
- Where
- 21 sites in Canada, China and 9 more
- Volunteers
- 197 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)
An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease
- Trial name
- NCT05632354
- Testing
- Osivelotor
- Where
- 9 sites in United States, Nigeria
- Volunteers
- 47 people
- Sponsor
- Pfizer
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
Transfusion-dependent Iron Overload
A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload
This study uses a single arm, multi-center, open-label trial design. The study will assess the efficacy and safety of 52 weeks of treatment with deferasirox (ICL670) in patients with evidence of transfusion induced iron overload.
- Trial name
- NCT00171821
- Testing
- Deferasirox
- Where
- 97 sites in Australia, Austria and 21 more
- Volunteers
- 1,784 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease
A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises
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)…
- Conditions
- Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease, Vaso-occlusive Crisis
- Trial name
- NCT04935879
- Testing
- Inclacumab
- Where
- 58 sites in United States, Brazil and 10 more
- Volunteers
- 241 people
- Sponsor
- Pfizer
Myelodysplastic Syndrome, Beta Thalassemia
A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemia
The purpose of this observational study is to assess the real-world safety of luspatercept in Korean participants with myelodysplastic syndrome (MDS) or beta thalassemia.
- Trial name
- NCT06073860
- Testing
- Luspatercept
- Where
- 2 sites in South Korea
- Volunteers
- 104 people
- Sponsor
- Bristol-Myers Squibb
Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.
- Conditions
- Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies
- Trial name
- NCT06363760
- Testing
- Safety and efficacy assessments
- Where
- 18 sites in United States, Canada
- Volunteers
- 54 people
- Sponsor
- Editas Medicine, Inc.
Sickle Cell Disease, Nephropathy
A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy
The primary purpose of this study is to evaluate the effect of mitapivat on albumin creatinine ratio (ACR) response in participants with sickle cell disease (SCD) and nephropathy.
- Trial name
- NCT06286046
- Testing
- Mitapivat
- Sponsor
- Agios Pharmaceuticals, Inc.
Sickle Cell Disease
A Study of SHP655 (rADAMTS13) in Sickle Cell Disease
TAK-755 (previously known as SHP655) is a medicine used to treat sickle cell disease (SCD). The main aim of the study is to measure the safety and tolerability of TAK-755 in SCD participants.
- Trial name
- RAISE (NCT03997760)
- Testing
- TAK-755
- Where
- 14 sites in United States
- Volunteers
- 19 people
- Sponsor
- Shire
Sickle Cell Disease, Vaso-occlusive Crisis
IMPACTS Trial: Investigation of the Modulation of Phospholipase in Acute Chest Syndrome
The study will be conducted at 15-20 US centers in a randomized, placebo-controlled, double-blind fashion. Enrollees will be hospitalized sickle cell disease (SCD) patients at-risk for acute chest syndrome (ACS) based on the…
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Acute Chest Syndrome
- Trial name
- NCT00434473
- Testing
- A-001
- Where
- 6 sites in United States
- Volunteers
- 30 people
- Sponsor
- Anthera Pharmaceuticals
Sickle Cell Disease
A Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) in Participants With Sickle Cell Disease (SCD).
The purpose of this study is to evaluate crovalimab for the treatment of a sickle cell pain crisis (also known as a VOE) that requires hospitalisation in adult and adolescent participants with SCD.
- Trial name
- CROSSWALK-a (NCT04912869)
- Testing
- Crovalimab
- Where
- 16 sites in United States, Brazil and 8 more
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
Blood and Lymphatic Diseases
Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: Long-term efficacy of the…
- Trial name
- NCT05145062
- Testing
- BIVV003, ST-400
- Where
- 7 sites in United States
- Volunteers
- 8 people
- Sponsor
- Sangamo Therapeutics
Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Post Hematopoietic Stem Cell Transplantation
This was a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study was to examine the safety and efficacy of deferasirox to decrease iron overload (IOL) in the posttransplant…
- Trial name
- NCT01610297
- Testing
- ICL670
- Where
- 6 sites in Turkey (Türkiye)
- Volunteers
- 27 people
- Sponsor
- Novartis Pharmaceuticals
Pregnancy; Fetomaternal Hemorrhage
Clinical Efficacy of Postpartum Immunization of Rh and Coombs Negative Women With Sensitization Risk.
This a open, prospective, multicenter, single-arm, phase III study for clinical assessment of anti-Rh immunoglobulin (KamRho-D®) in Rh and Coombs negative women with risk sensitization.
- Trial name
- NCT01666249
- Testing
- Immunoglobulin Anti-RhD
- Where
- 5 sites in Brazil
- Volunteers
- 347 people, healthy volunteers welcome
- Sponsor
- Azidus Brasil
Plasmodium Falciparum Malaria, Malaria
Effect of Plasmodium Falciparum Exposure and Sickle Cell Trait on Infection Rates and Kinetics After IV Administration of PfSPZ Challenge
The study is designed to establish infectivity of Plasmodium falciparum sporozoites (PfSPZ) via intravenous (IV) administration in three groups with different malaria immunity-status: 1.
- Trial name
- NCT02237586
- Testing
- PfSPZ Challenge
- Where
- Gabon
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Sanaria Inc.
Sickle Cell Disease
Open-Label Extension Study to Pioneer Study 6058-SCD-101
This is an open-label study to evaluate the safety and tolerability of long-term treatment with pociredir without a comparator in participants with SCD who have previously been treated and shown benefit with pociredir in feeder…
- Trial name
- NCT07401823
- Testing
- Pociredir
- Where
- 8 sites in United States
- Volunteers
- 1 people
- Sponsor
- Fulcrum Therapeutics
Crohn's Disease
A Study to Test the Effect of CDP870 in the Treatment of Crohn's Disease Over 26 Weeks, Comparing CDP870 to a Dummy Drug (Placebo)
A 26 week study to examine the efficacy, safety and pharmacokinetics of CDP870 in Crohn's disease
- Trial name
- NCT00152490
- Testing
- Certolizumab Pegol (CDP870)
- Volunteers
- 604 people
- Sponsor
- UCB Pharma
Fanconi Anemia Complementation Group A
A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A
The objective of this study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with a lentiviral vector carrying the…
- Trial name
- NCT03814408
- Testing
- RP-L102
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Sickle Cell Disease, Sickle-Cell; Hb-SC
A Study of IMR-687 in Healthy Adult Volunteers
The purpose of this Phase 1a, first in human, randomized, double-blind, placebo-controlled study is to evaluate the safety, tolerability, PK and PD profile of the orally administered IMR-687 in healthy adult subjects.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hb-SC, Sickle Beta 0 Thalassemia
- Trial name
- NCT02998450
- Testing
- IMR-687
- Where
- United States
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- Cardurion Pharmaceuticals, Inc.