Clinical trials: Severe Anaemia
Crohn Disease
Study to Evaluate the Efficacy and Safety of PF-06480605 in Adult Participants With Moderate to Severe Crohn's Disease
This Phase 2a, multicenter, randomized, double-blind, placebo-controlled study examines subcutaneous dose of PF-06480605 150 mg administered every 4 weeks in participants with moderate to severe active Crohn's Disease to…
- Trial name
- NCT05471492
- Testing
- Investigational Product
- Sponsor
- Telavant, Inc.
Anemia
A Study to Evaluate the Safety and Efficacy of Epoetin Alfa Versus Placebo to Reduce the Need for Blood Transfusions and Reduce the Occurrence of Severe Anemia During the Time Period Surrounding Total Hip Replacement Surgery
The purpose of this study is to evaluate the safety and effectiveness of two different dosing schedules of epoetin alfa versus placebo for decreasing the need for blood transfusions and preventing the occurrence of severe anemia…
- Trial name
- NCT00269958
- Testing
- epoetin alfa
- Volunteers
- 208 people
- Sponsor
- Janssen-Ortho Inc., Canada
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
A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease
This is an open label, multicenter, Phase 1/2 study in approximately eight adults with severe Sickle Cell Disease (SCD). The study will evaluate the safety, tolerability, and efficacy of autologous hematopoietic stem cell…
- Trial name
- NCT03653247
- Testing
- Plerixafor, Busulfan, BIVV003
- Where
- 5 sites in United States
- Volunteers
- 7 people
- Sponsor
- Sangamo Therapeutics
Beta Thalassemia Major Anemia, Myelodysplastic Syndrome
A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
The goal of this clinical trial is to learn about SP-420 ability to remove iron from organs in subjects with transfusion-dependent β-thalassemia or transfusion-dependent low-risk myelodysplastic syndrome.
- Trial name
- NCT05693909
- Testing
- SP-420
- Where
- Denmark
- Volunteers
- 90 people
- Sponsor
- Pharmacosmos A/S
Acute Anemia
A Phase 1 Safety Study of SANGUINATE™ In Patients With Acute Severe Anemia.
In this open-label study, eligible and consenting adult patients admitted to the hospital for critical care that have acute severe anemia, defined as a blood hemoglobin level ≤ 5 g/dL, but who are unwilling or unable to receive…
- Trial name
- NCT02754999
- Testing
- SANGUINATE™
- Where
- 32 sites in United States
- Volunteers
- 103 people
- Sponsor
- Prolong Pharmaceuticals
Transfusion Dependent Beta-thalassemia
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age.
- Trial name
- NCT03432364
- Testing
- ST-400 Investigational product
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Sangamo Therapeutics
Beta Thalassemia Major Anemia, Iron Overload
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on…
- Trial name
- START (NCT03591575)
- Testing
- Deferiprone oral solution
- Where
- 4 sites in Egypt, Indonesia
- Volunteers
- 64 people
- Sponsor
- Chiesi Canada Corp
Beta Thalassemia Major
Phase 2a Pilot Study of NBMI Treatment in Patients With Beta Thalassemia Major, Requiring Iron Chelation
A pilot study to explore safety and efficacy of NBMI treatment in patients with Beta Thalassemia Major requiring iron chelation Investigational product: NBMI (N1,N3-bis(2-mercaptoethyl) isophthalamide), INN: Emeramide Indication…
- Trial name
- EMERA007 (NCT04092205)
- Testing
- Emeramide
- Where
- Albania
- Volunteers
- 13 people
- Sponsor
- EmeraMed
Beta Thalassemia Major, Beta Thalassemia Intermedia
Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.
Dose finding study to determine the safety and tolerability of Sotatercept (ACE-011) in adults with Beta (β)-Thalassemia
- Trial name
- NCT01571635
- Testing
- SOTATERCEPT (ACE-011)
- Where
- 18 sites in France, Greece and 2 more
- Volunteers
- 46 people
- Sponsor
- Celgene
Beta-thalassemia Major, Hemosiderosis
Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)
To allow patients treated with deferasirox in the core study to continue iron chelation therapy for 2 years or until the drug became locally commercially available.
- Conditions
- Beta-thalassemia Major, Hemosiderosis, Iron Overload, Rare Anemia
- Trial name
- ESCALATOR (NCT00171301)
- Testing
- Deferasirox
- Where
- 5 sites in Egypt, Lebanon and 3 more
- Volunteers
- 233 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype
The purpose of the study is to evaluate the efficacy and safety of CTX001 (exa-cel) in adolescent and adult participants with severe sickle cell disease (SCD), βS/βC genotype (HbSC).
- Trial name
- NCT05951205
- Testing
- Exa-cel
- Sponsor
- Vertex Pharmaceuticals Incorporated
Sickle Cell Disease, Hemoglobinopathies
A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with severe sickle cell disease (SCD).
- Trial name
- NCT04853576
- Testing
- EDIT-301
- Where
- 24 sites in United States, Canada
- Volunteers
- 45 people
- Sponsor
- Editas Medicine, Inc.
Advanced Solid Tumors or Non-Hodgkin's Lymphoma (NHL)
A Phase 1/2 Trial of SRA737 in Subjects With Advanced Cancer
The purpose of this clinical study is to establish the safety profile, determine the maximum tolerated dose (MTD) and recommend a Phase 2 dose and schedule of SRA737; and to evaluate the efficacy of SRA737 in…
- Trial name
- NCT02797964
- Testing
- SRA737
- Where
- 15 sites in United Kingdom
- Volunteers
- 107 people
- Sponsor
- Sierra Oncology LLC - a GSK company
Pain, Cancer
Extension Study of ACTIQ Treatment for Children and Adolescents With Breakthrough Pain
The objective of the study is to monitor the safety (adverse event data) of longer-term use of ACTIQ (Oral Transmucosal Fentanyl Citrate [OTFC]) treatment in children with pain associated with cancer, sickle cell disease, or…
- Conditions
- Pain, Cancer, Sickle Cell Anemia, Severe Burns
- Trial name
- NCT00236093
- Testing
- ACTIQ (Oral Transmucosal Fentanyl Citrate [OTFC])
- Where
- 28 sites in United States, Canada and 1 more
- Sponsor
- Cephalon
Severe Aplastic Anemia
Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia
This is a multicenter, randomized, placebo-control, phase III study to investigate hetrombopag in subjects with severe AA who are treated naive. 180 treated naive patients with SAA will be enrolled in the study.
- Trial name
- NCT03825744
- Testing
- Hetrombopag Olamine+Standard Therapy
- Where
- China
- Volunteers
- 240 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Sickle Cell Disease
CS-206 in Patients With Sickle Cell Disease
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 injection in treating sickle cell disease.
- Trial name
- NCT06565026
- Testing
- CS-206
- Where
- China
- Volunteers
- 5 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Cytopaenia
A Study to Assess the Safety and Efficacy of Eltrombopag in Japanese Subjects With Refractory, Moderate or More Severe Aplastic Anemia
This was a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Japanese moderate or more severe aplastic anemia (AA) subjects with a platelet count <30,000/microliter who were refractory…
- Trial name
- NCT02148133
- Testing
- Eltrombopag 12.5 mg, Eltrombopag 25 mg
- Where
- 12 sites in Japan
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis
A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis
This study (study ID PAC203 North America; PAC303 ex-North America) is evaluating 200 mg BID of pacritinib compared to physician's choice (P/C) therapy in patients with MF and severe thrombocytopenia (platelet count <50,000/μL).
- Conditions
- Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis
- Trial name
- PACIFICA (NCT03165734)
- Testing
- Pacritinib, Physician's Choice medications
- Where
- 207 sites in United States, Australia and 22 more
- Volunteers
- 407 people
- Sponsor
- Swedish Orphan Biovitrum
GVHD, Adult Acute Myeloid Leukemia
Study of US-ATG-F to Prevent Chronic Graft Versus Host Disease (GVHD)
The study objective is to compare the efficacy and safety of US-ATG-F as a supplement to standard of care prophylaxis versus standard of care prophylaxis alone in moderate to severe chronic GVHD-free survival.
- Conditions
- GVHD, Adult Acute Myeloid Leukemia, Adult Acute Lymphoid Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT01295710
- Testing
- US-ATG-F
- Where
- 28 sites in United States, Australia
- Volunteers
- 260 people
- Sponsor
- Neovii Biotech