Treatment Trials

1,260 Clinical Trials for Various Conditions

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The Efficacy and Safety of Elritercept in Adult Participants with Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) with Anemia (RENEW)
Description

This study (KER-050-D301) is evaluating the efficacy and safety of elritercept (KER-050) versus placebo in adult participants with transfusion-dependent anemia with very low, low, or intermediate risk MDS, or more recently defined as myelodysplastic neoplasms, with or without ring sideroblasts. The study is divided into the Screening Period, Double-blind Treatment Period, Safety Follow-Up Period and Long-term Follow-up Period. Approximately 255 participants will be enrolled, randomized 2:1 to receive either elritercept or placebo.

Conditions

Myelodysplastic Syndromes

Intravenous Iron Versus Oral Iron for the Treatment of Iron Deficiency Anemia
Description

This study aims to assess whether administering intravenous iron early in pregnancy, compared to standard oral iron treatment, can enhance hemoglobin levels before delivery and reduce the need for blood transfusions in patients with iron deficiency anemia. Patients diagnosed with iron deficiency anemia were randomly assigned to receive either oral or intravenous iron. Before treatment initiation, patients completed a symptom questionnaire baseline hemoglobin, and ferritin levels were measured. Follow-up visits occurred four weeks later and at 24 to 28 weeks gestation, involving reassessment of symptoms, laboratory testing, and monitoring of treatment adherence. Final hemoglobin levels were determined before delivery, and data on the need for blood transfusion at delivery were recorded.

Conditions

Iron Deficiency Anemia of Pregnancy

Pilot Feasibility Study of a Novel Non-invasive Device for Diagnosis of Anemia
Description

In low-resource areas of the world, anemia screening relies on analyzing a blood sample and is generally carried out in health facilities. Current anemia screening approaches have not yielded satisfactory results due to critical limitations including lack of a) reliable access to laboratory facilities, b) reliable non-invasive out-of-hospital screening tools for community health-workers, c) integration of anemia data across health systems and d) distinction between hemolytic and nutritional causes. Currently available non-invasive tools have unacceptably low accuracy and cannot distinguish between nutritional and hemolytic etiologies. Prototype Anemia Diagnostic Assistant (ADA) We have developed a prototype Anemia Diagnostic Assistant for non-invasive, simultaneous detection of two markers of anemia, blood hemoglobin and the End-Tidal carbon monoxide levels. The device comprises an optical sensor module and ETCO breath sampling module. The unique and significant advantage of the instrument is its ability to detect, independently, two orthogonal variables that are required for differential diagnosis of the nutritional and hemolytic anemia: 1. Hemoglobin concentration using a non-invasive diffuse reflectance spectroscopy. Using high-fidelity, 11 wave-length spectral sensor, the device will provide optical quantification of hemoglobin levels. Optical hemoglobin sensing using diffused reflectance spectroscopy is well known, however; using traditional spectrophotometry instrumentation is very costly and thus impractical for this project. As a solution, we propose to use a validated commercially-available high accuracy 11-wavelength sensor within the visual and near-infrared (IR) range of the spectrum. The 11-wavelength spectrum will allow for sufficient accuracy in measuring the reflectance AND transmittance at isosbestic wavelengths on hemoglobin extinction curve as well as to compensate for the presence of melanin, which is a major interferant in optical determination of hemoglobin concentration. \[9\] The sensor was originally designed to collect data on the earlobe and/or the fingertip. Additional iterations include a sensor that straps around the wrist (similar to a smartwatch). Both versions of the device may be used in the study, and both feature standard USB or Bluetooth connectivity to ubiquitous mobile Android an iOS-based mobile platform . 2. End-tidal carbon monoxide. Using a non-invasive probe placed in proximity to the nostrils, the device will measure ETCO as a proxy measure of hemolysis. ETCO measurement as a measure for the presence of hemolysis is well documented in the medical literature and is commonly used in newborns units as a screening method for the presence of hemolysis \[7,8,10\]. A positive finding (presence of elevated levels of ETCO) will then prompt a referral for further hospital testing. The objective of this study is to determine the hemoglobin and ETCO concentration in healthy volunteers using the prototype device and compare the results with the hemoglobin and ETCO concentrations obtained using standard of care devices and the CoSense system for the (ETCO measurement).

Conditions

Anemia

A Prospective Interventional Study Assessing the Clinical and Operational Effectiveness of Transitioning From Mircera to Daprodustat for the Treatment of Anemia in End Stage Kidney Disease
Description

Investigator-initiated, interventional, prospective study to assess the clinical and operational effectiveness of daprodustat in adult patients receiving in center hemodialysis or peritoneal home dialysis who are transitioning from Mircera to daprodustat.

Conditions

Anemia, Renal Insufficiency, Chronic, Renal Anemia

ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions
Description

The purpose of the study is to compare the efficacy and safety of Luspatercept vs epoetin alfa in the treatment of anemia in adults due to IPSS-R very low, low, intermediate-risk MDS in ESA-naïve participants who are non-transfusion dependent (NTD).

Conditions

Myelodysplastic Syndromes

Iron Deficiency Anemia (IDA) and the Brain
Description

This is a trial with an observational and an interventional arm, in patients with moderate to severe anemia and control subjects. The main purposes of this study is to phenotype the scope of neurocognitive deficits from iron deficiency anemia (IDA) in adult women, determine derangements in cerebral perfusion, vascular reactivity, functional connectivity, and blood brain barrier permeability in adult-onset IDA and relate them to neurocognitive deficits, as well as determine the reversibility and durability of both the physiologic and neurocognitive derangements by iron replacement therapy. All eligible subjects will be asked to provide informed consent before participating in the study.

Conditions

IDA - Iron Deficiency Anemia, Anemia, Iron Deficiency Anemia, Anemia, Iron Deficiency

Iron Replacement to Reduce Anemia During Neoadjuvant Chemotherapy
Description

This is an open-label, single institution study evaluating the efficacy of intravenous iron sucrose monotherapy in patients with thoracic, breast, GU and GI malignancies undergoing neoadjuvant chemotherapy with the goal of undergoing surgical resection.

Conditions

Anemia

Effectiveness of Small Phlebotomy Tubes in Reducing Blood Transfusions in Adult Medical Intensive Care Unit and Intermediate Care Unit Patients With Anemia
Description

The purpose of this study is to evaluate the effectiveness of small phlebotomy tubes to reduce RBC transfusions in medical intensive care unit (ICU) and Intermediate care unit (IMU) patients with low hemoglobin compared with standard size tubes, to compare the intervention and the control groups in regards to: ICU length of stay (LOS), ICU mortality, hospital LOS, and hospital mortality and to assess the acceptability of small phlebotomy tubes in adult ICU and IMU patients.

Conditions

Anemia

Study of DISC-0974 to Assess the Safety, Tolerability, PK and PD of DISC-0974 in Participants with CKD and Anemia
Description

This Phase 1b study of DISC-0974 will assess the safety, tolerability, pharmacokinetics (PK) and Pharmacodynamics (PD) of DISC-0974 in adult participants with Non-Dialysis Dependent Chronic Kidney Disease and Anemia.

Conditions

Chronic Kidney Diseases, Anemia of Chronic Kidney Disease

Luspatercept for Anemia in Lower Risk MDS or Non-proliferative MDS/MPN Neoplasms
Description

The purpose of the study is to see if participants with anemia due to their type of MDS or MDS/MPN will experience a more decreased need for regular blood transfusions if they take luspatercept plus best supportive care, and what effect, good and/or bad, luspatercept has on them and their anemia due to MDS or MDS/MPN. The safety and tolerability of luspatercept will also be evaluated in this study.

Conditions

Myelodysplastic Syndromes, Myeloproliferative Neoplasm, Anemia

Anemia Studies in CKD: Erythropoiesis Via a Novel PHI Daprodustat - Pediatric (ASCEND-P)
Description

This is an international, multicenter trial, evaluating pharmacokinetics (PK) (4 weeks), safety (52 weeks), and hemoglobin (Hgb) response (52 weeks) to daprodustat in children and adolescent participants with anemia associated with chronic kidney disease (CKD) incorporating 2 independent sub-trials (Non dialysis \[ND\] and Dialysis \[D\]). This study will enroll participants with anemia associated with CKD, in 2 distinct sub-populations differing only by their CKD stage and dialysis requirement (ND: CKD stage 3 to 5 not yet receiving dialysis and D: CKD stage 5d undergoing peritoneal dialysis \[PD\] or hemodialysis \[HD\]). The maximum duration of the study will be approximately 60 weeks, including Screening period (up to 4 weeks), treatment period (52 weeks), and follow-up period (4 weeks). Outcome measures are identical for the ND and D sub-trials, but will be separately assessed in each sub- trials, overall and within each age subgroups (12 to less than \[\<\] 18 years, 6 to \<12 years, 2 to \<6 years, and 3 months to \<2 years). Except for PK and dose change, which is within each age group only.

Conditions

Anaemia

A Study of Etavopivat for the Treatment of Anemia in Patients With Myelodysplastic Syndromes (MDS)
Description

The purpose of this study is to evaluate the safety and efficacy of etavopivat (FT-4202) for the treatment of anemia in adult patients with very low risk, low risk, or intermediate risk MDS.

Conditions

Very Low Risk, Low Risk, or Intermediate Risk MDS Per IPSS-R

A Study of Tebapivat (AG-946) in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes (LR-MDS)
Description

This purpose of this study is to establish proof of concept of tebapivat in participants with LR-MDS in Phase 2a and to evaluate the effect of tebapivat on transfusion independence (TI) in participants with LR-MDS in phase 2b.

Conditions

Myelodysplastic Syndromes

Study of DISC-0974 in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia
Description

This phase 1b/2a open-label study will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of DISC-0974 as well as categorize the effects on anemia response in subjects with myelofibrosis or myelodysplastic syndrome and anemia.

Conditions

Myelofibrosis; Anemia, Anemia, Myelofibrosis, Myelofibrosis Due to and Following Polycythemia Vera, Primary Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis, Myelodysplastic Syndromes

Single-dose Intravenous Iron Therapy for Anemia After Orthopaedic Trauma
Description

Acute blood loss in orthopaedic trauma and operative fracture care contributes substantially to perioperative anemia, which places participants at increased risk for complications including surgical site infection, cardiovascular complications, and even death. Anemia has further clinical implications in quality of life measures and is associated with fatigue, impaired physical performance, decreased exercise capacity, and mood disturbances. Thus, evaluation and treatment of perioperative anemia is critical in risk mitigation within orthopaedic surgery. The current standard of care for anemia is transfusion of packed red blood cells in only cases of severe anemia due to the substantial associated risks. A safer alternative is desirable because a critical number of participants do not meet the restrictive transfusion threshold and may suffer negative effects from anemia during recovery from the acute insult. The focus of this project is to pilot an investigation of the benefits of intravenous iron therapy (IVIT) in traumatically injured patients. Specifically, Aim I will determine feasibility of study design, recruitment, randomization, intervention implementation, blinded procedures, and retention. In Aim II, time to return to normal hemoglobin following traumatic orthopaedic injury will be assessed. With Aim III, the investigators will measure IVIT effect on participant-reported fatigue, physical function, and depression, and further determine if resolution of anemia is correlated to improvements in these measures in traumatically injured orthopaedic patients. Aim IV will focus on evaluating the role of IVIT on immune cells through a variety of novel laboratory assessments. The investigators expect this study to provide a better understanding of IVIT, which has the potential to alter providers' treatment approach of anemia in participants who sustain traumatic orthopaedic injury, thereby leading to decreased risks and improved recovery.

Conditions

Acute Blood Loss Anemia, Fracture

Practical Anemia Bundle for SusTained Blood Recovery
Description

The purpose of this study is to test a multi-faceted anemia treatment plan to reduce the severity of anemia and to promote hemoglobin and functional recovery in adults who have been in the intensive care unit (ICU).

Conditions

Anemia, Critical Illness

Evaluate the Safety and Efficacy of Ferric Maltol Oral Suspension vs. Ferrous Sulfate Oral Liquid in Children and Adolescents Aged 2 to 17 Years With Iron-deficiency Anaemia, With a Single Arm Study in Infants Aged 1 Month to Less Than 2 Years
Description

The objective of the study is to compare the safety and gastrointestinal tolerability of ferric maltol oral suspension and ferrous sulfate oral liquid in children and adolescents aged 2 years to 17 years, and assess the safety and tolerability of ferric maltol oral suspension in children 1 month to less than 2 years, in the treatment of iron deficiency anaemia during the 12 weeks treatment period.

Conditions

Anemia, Iron-deficiency

Study to Evaluate the Safety and Efficacy of Oral Vadadustat in Pediatric Participants With Anemia of Chronic Kidney Disease Naive to Erythropoiesis-Stimulating Agents
Description

This study will assess the safety and efficacy of once daily dosing of vadadustat for the treatment of pediatric participants with anemia of chronic kidney disease (CKD) naive to erythropoiesis-stimulating agent (ESA) treatment.

Conditions

Anemia of Chronic Kidney Disease

Study to Evaluate the Safety and Efficacy of Oral Vadadustat in Pediatric Participants With Anemia of Chronic Kidney Disease
Description

This study will assess the safety and efficacy of once daily dosing of vadadustat for the treatment of pediatric participants with anemia of Chronic Kidney Disease (CKD) after conversion from an Erythropoiesis Stimulating Agent (ESA).

Conditions

Anemia of Chronic Kidney Disease

Study Evaluating the Efficacy and Safety of Dose Conversion From a Long-acting Erythropoiesis-stimulating Agent (Mircera®) to Three Times Weekly Oral Vadadustat for the Maintenance Treatment of Anemia in Hemodialysis Subjects
Description

This study will be conducted to demonstrate the efficacy and safety of vadadustat administered three times weekly (TIW) compared to a long-acting erythropoiesis-stimulating agent (ESA) (Mircera®) for the maintenance treatment of anemia in hemodialysis participants.

Conditions

Anemia Associated With Chronic Kidney Disease (CKD)

A Study of TP-0184 to Treat Anemia in Adults With IPSS-R Low or Intermediate Risk MDS
Description

This study will evaluate preliminary safety and efficacy of TP-0184 to treat anemia when administered to adult patients with Revised International Prognostic Scoring System (IPSS-R) low or intermediate risk MDS. The recommended Phase 2 dose (RP2D) will be determined by the maximum tolerated dose (MTD) or maximum administered dose (MAD) in the Phase 1 portion of the study.

Conditions

Anemia in Myelodysplastic Syndromes

Daily Versus Alternate Day Iron Supplementation for the Treatment of Iron Deficiency Anemia in Pregnancy
Description

The target population for our study is pregnant women in the first or second trimester with a diagnosis of iron deficiency anemia. If a subject is eligible, written consent will be obtained by person to person contact. Eligible participants will be randomized to receive either daily oral iron supplementation or every other day oral iron supplementation.

Conditions

Pregnancy Related, Iron Deficiency Anemia of Pregnancy

Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment
Description

This study was a multi-center, randomized, open-label, active comparator-controlled, parallel group study. The purpose of this Phase 3 study in PNH patients presenting with residual anemia despite treatment with anti-C5 antibody, was to determine whether iptacopan is efficacious and safe for the treatment of PNH through demonstration of superiority of iptacopan compared to anti-C5 antibody treatment.

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

INCB000928 Administered as a Monotherapy or in Combination With Ruxolitinib in Participants With Anemia Due to Myeloproliferative Disorders
Description

This Phase 1/2, open-label, dose-finding study is intended to evaluate the safety and tolerability, PK, PD, and efficacy of INCB000928 administered as monotherapy or in combination with ruxolitinib in participants with MF who are transfusion-dependent or presenting with symptomatic anemia. This study will consist of 2 parts: dose escalation and expansion.

Conditions

Anemia, Post-essential Thrombocythemia Myelofibrosis, Post-polycythemia Vera Myelofibrosis

Neurovascular Complications and White Matter Damage in Acquired Anemias
Description

This is an observational trial, in patients with moderate to severe anemia and control subjects. The main purpose of this study is to understand whether normal brain blood flow, oxygen extraction reserve, white matter volumes, and brain functional connectivity are affected by acquired anemia. The investigators will perform baseline MRI monitoring for all subjects. All eligible subjects will be asked to provide informed consent before participating in the study.

Conditions

Anemia, Iron-deficiency Anemia, Healthy Controls

Trial Evaluating the Efficacy and Safety of Oral Vadadustat Once Daily (QD) and Three Times Weekly (TIW) for the Maintenance Treatment of Anemia in Hemodialysis Subjects Converting From Erythropoiesis-Stimulating Agents (ESAs)
Description

This trial will be conducted to demonstrate the efficacy and safety of vadadustat compared to darbepoetin alfa for the maintenance treatment of anemia in hemodialysis participants after conversion from current erythropoiesis-stimulating agent (ESA) therapy.

Conditions

Anemia

Gene Therapy for Fanconi Anemia, Complementation Group A
Description

The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A). Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the FANCA gene. After transduction, the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure.

Conditions

Fanconi Anemia Complementation Group A

Evaluation of Efficacy and Safety of Roxadustat for the Treatment of Chemotherapy Induced Anemia
Description

The purpose of this study is to find out if roxadustat (also known as FG-4592) is safe and effective for the treatment of anemia in participants receiving chemotherapy treatment for cancer.

Conditions

Chemotherapy Induced Anemia

Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Vadadustat in Hemodialysis Subjects With Anemia Associated With Chronic Kidney Disease
Description

This study will be conducted to assess the pharmacokinetics of vadadustat 600, 750, and 900 milligrams daily, and intravenous erythropoiesis-stimulating agent (darbepoetin alfa or epoetin alfa), in hemodialysis participants with anemia associated with chronic kidney disease.

Conditions

Anemia Associated With Chronic Kidney Disease

A Study to Evaluate Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects
Description

This is a Phase 3, randomized, open-label, multicenter, study in male and female pediatric subjects (2 years to \<18 years of age) with IDA, or felt by their clinician to be at risk of developing IDA. This study allows for enrollment of subjects with IDA regardless of etiology, except for CKD subjects (pediatric CKD subjects are being studied in a separate ferumoxytol protocol).

Conditions

Iron Deficiency Anemia