Clinical Research Directory

Lenalidomide With or Without Epoetin Alfa in Treating Patients With Myelodysplastic Syndrome and Anemia

This randomized phase III trial studies lenalidomide to see how well it works with or without epoetin alfa in treating patients with myelodysplastic syndrome and anemia. Lenalidomide may stop the growth of myelodysplastic syndrome by blocking blood flow to the cells. Colony stimulating factors, such as epoetin alfa, may increase the number of immune cells found in bone marrow or peripheral blood. It is not yet known whether lenalidomide is more effective with or without epoetin alfa in treating patients with myelodysplastic syndrome and anemia.

Dapagliflozin for Anemia in Lower-Risk Myelodysplastic Syndromes

This study is a prospective, single-arm, phase II clinical trial designed to evaluate the efficacy and safety of dapagliflozin in improving anemia in patients with lower-risk myelodysplastic syndromes (MDS). Anemia is the most common clinical problem in patients with lower-risk MDS and often leads to fatigue, reduced quality of life, and the need for repeated blood transfusions. Current treatment options, including erythropoiesis-stimulating agents and other therapies, are not effective in all patients, and additional treatment options are needed. Dapagliflozin is a sodium-glucose cotransporter-2 (SGLT2) inhibitor that is widely used for the treatment of diabetes, heart failure, and chronic kidney disease. Previous studies have shown that SGLT2 inhibitors can increase hemoglobin levels, possibly by stimulating erythropoiesis. In this study, eligible patients will receive dapagliflozin 10 mg orally once daily for 24 weeks. The primary objective is to evaluate the hemoglobin response rate during the study period. Secondary objectives include changes in hemoglobin levels, transfusion requirements, and safety outcomes. This study aims to explore whether dapagliflozin can serve as a potential treatment option for anemia in patients with lower-risk MDS.

Levonorgestrel Intrauterine System Effects on Hemoglobin and Serum Ferritin Among Anemic Women in Kenya

Women with mild/moderate anemia who are seeking contraception will be randomized to a levonorgestrel (LNG) intrauterine system (IUS) or an LNG/ethinyl estradiol (EE)/ferrous fumarate combined oral contraceptive (COC) regimen and followed prospectively for 12-18 months. Approximately 400 participants will be enrolled. The primary hypothesis that the mean change in hemoglobin in the group assigned to the LNG IUS will be superior to the COC/ferrous fumarate (control) group after 12-months of product use.

Registry Platform Myelofibrosis and Anemia

The purpose of the project is to set up a national, prospective, longitudinal, multicenter cohort study, a tumor registry platform, to document uniform data on characteristics, molecular diagnostics, treatment and course of disease and to collect patient-reported outcomes for patients with primary and secondary myelofibrosis and anemia in Germany.

Study of DISC-0974-201 in Participants With IBD and Anemia

This is a Phase 2, multicenter, randomized, double-blind placebo-controlled study of DISC-0974 to evaluate safety, tolerability, and efficacy in participants with IBD and anemia of inflammation.

Multiple Micronutrient Supplementation With Digital Layering Among Adolescents in Tanzania

This study is a three-arm, individually randomized controlled trial evaluating the impact of digitally delivered nutrition education, layered onto multiple micronutrient supplementation (MMS), on anemia and related health behaviors among adolescents in Dar es Salaam, Tanzania. A total of 1,200 adolescents aged 15-19 years with access to a phone (own or shared) will be enrolled from the Dar es Salaam Health and Demographic Surveillance System and followed for 9 months, with assessments at baseline, 4 months, and 9 months. All participants will receive a brief in-person nutrition education session, printed brochures on adolescent nutrition and anemia, and a 2-month supply of daily MMS tablets with instructions and access to refills (Control arm). In Intervention Arm I, participants will receive the same package plus weekly one-way SMS/WhatsApp messages reinforcing key nutrition content and adherence to MMS and refills. In Intervention Arm II, participants will receive all components of Arm I plus fortnightly, in-person group digital nutrition education sessions that include interactive content and opportunities to co-create and share digital nutrition messages with peers. All participants will receive information on replenishing the tablets. Participants in Intervention Arm I and Intervention Arm II will receive additional nutrition, diet, and physical activity-related messages along with reminders and motivational encouragement to replenish their supplement stocks. The primary outcome is anemia prevalence, assessed using hemoglobin concentration and WHO age- and sex-specific cutoffs. Secondary outcomes include moderate/severe anemia, hemoglobin levels, adherence to MMS pick-up and consumption, nutrition literacy, dietary diversity, fruit and vegetable intake, physical activity, underweight/overweight/obesity, and digital literacy. The trial also includes a mixed-methods process evaluation of feasibility, acceptability, reach, engagement with the digital components, and a cost estimation of the digital strategies.

Luspatercept for Clonal Cytopenias of Uncertain Significance

The purpose of this clinical trial is to test how well the drug luspatercept works in improving low blood cell counts in people with clonal cytopenias of uncertain significance (CCUS). The main questions the study seeks to answer include: * How many patients experience improvements in their low blood counts (red cells, platelets, or white cells) within 24 weeks, based on specific criteria for blood conditions like myelodysplastic syndromes (MDS)? * How long these improvements last before the condition worsens or changes. * The percentage of participants showing improvements at 12, 24, and 48 weeks. * How long it takes for the condition to progress to more severe diseases like myeloid disorders. * How long red blood cell responses last and how quickly these responses are seen. * The average change in hemoglobin levels over 24 weeks. * How many patients need blood transfusions during the study and how soon transfusions are required. * Changes in participants' well-being and energy levels based on a standardized questionnaire. * Monitoring for any side effects, including progression to MDS or leukemia, heart-related issues, or sudden increases in hemoglobin. Participants will: * Receive luspatercept as an injection every three weeks. * Visit the clinic every three weeks for treatment and monitoring.

A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia

The purpose of the study is to evaluate the efficacy and safety of luspatercept plus best supportive care (BSC) vs placebo plus BSC on anemia in adult participants with α-thalassemia hemoglobin H (HbH) disease and determine the safety and drug levels in adolescent participants.

Efficacy and Tolerability of Sucrosomial® Iron vs Ferric Maltol in Iron Deficient Women

This study aims to compare two oral iron treatments, Sucrosomial® Iron and Ferric Maltol, in women with mild to moderate iron deficiency anemia. Many patients experience gastrointestinal side effects or poor tolerability with traditional iron supplements, which may limit adherence. Participants will be randomly assigned to receive either Sucrosomial® Iron or Ferric Maltol for 12 weeks. The primary objective is to determine whether Sucrosomial® Iron is as effective as Ferric Maltol in normalizing hemoglobin levels.

INTERCEPT Blood System for RBCs Study in Regions at Potential Risk for Zika Virus Transfusion-Transmitted Infections

To evaluate the safety and efficacy of red blood cells (RBCs) prepared with the INTERCEPT Blood System for Red Blood Cells Pathogen Reduction Treatment (PRT) in comparison to conventional RBCs in patients who require RBC transfusion support.

Effects of Pazopanib on Hereditary Hemorrhagic Telangiectasia Related Epistaxis and Anemia (Paz)

During the Efficacy Study (Part B), the investigators will study whether Pazopanib, taken daily for 24 weeks, will reduce the severity of nose bleeds in patients with hereditary hemorrhagic telangiectasia (HHT). Patients will either be provided active drug or a placebo \[sugar - inactive pill\], and be tested for nose bleed severity throughout the trial, including particularly nose bleed duration. Investigators will also test for blood loss, as well as for safety. This study is funded by the US Department of Defense USAMRAA and FDA/OOPD.

Effects of SGLT2 Inhibitors on Erythropoiesis in Heart Failure

This retrospective cohort study investigates the effects of SGLT2 inhibitor therapy on erythropoiesis and its association with cardiac and renal outcomes in patients with chronic heart failure. The study will compare hematological parameters, echocardiographic measures, and clinical outcomes between patients who initiated SGLT2 inhibitor therapy and a matched control group receiving guideline-directed medical therapy alone over a 12-month follow-up period.

Genomic of CONgenital Sideroblastic Anemias

Congenital sideroblastic anemias (CSA) are a group of rare disorders characterized by abnormal iron utilization during erythropoiesis, leading to mitochondrial iron overload, the formation of ring sideroblasts, and ineffective erythropoiesis resulting in anemia. Ring sideroblasts are erythroid precursors that contain non-heme iron deposits in their mitochondria, forming a distinctive ring-like pattern around the nucleus. Mitochondria are double membrane organelle provide a large amount of energy for cellular activities, by the process of oxidative phosphorylation (OXPHOS). The role of mitochondria has been well described in erythropoiesis. CSA exhibits clinical heterogeneity, affecting only the erythroid system in some cases, while in others presenting as part of broader syndromic conditions. Their molecular basis remains imperfectly known, although the development of next- generation sequencing technology brought tremendous advances in the understanding of their genetic features. More than 20 genes have been identified as causative of CSA, with all modes of inheritance observed: X-linked recessive, autosomal dominant, autosomal recessive, pseudo- dominant, and mitochondrial. These genes are typically involved in one of four key mitochondrial pathways: i) Heme biosynthesis (e.g., ALAS2, SLC25A38); ii) Iron-sulfur cluster biosynthesis and transport (e.g., GLRX5, HSPA9, HSCB); iii) tRNA synthesis and maturation (e.g., PUS1, YARS2, LARS2, IARS2, SARS2, MARS1, TRNT1); iv) Mitochondrial respiratory chain synthesis (e.g., NDUFB11). However, in nearly 30% of cases within the French CSA cohort, the underlying genetic cause remains unknown. In these patients with molecularly unexplained whole genome or exome sequencing approaches focusing on genes involved in mitochondrial function and iron metabolism identified several possibly pathogenic variants in CSA patients. These genes were not clearly described as playing a role in erythropoiesis or heme or iron metabolism. We hope to confirm their role in CSA. However, in nearly 30% of cases within the French CSA cohort , the underlying genetic cause remains unknown. The investigators hope to confirm the role in CSA of gene identified with exome sequencing approaches.

The Influence of Anemia on HbA1c Readings in Non-Diabetic Patients

The goal of this observational study is to learn about the effects of different types of anemia on HbA1c test results in non-diabetic adults who have anemia but do not have diabetes. The main question it aims to answer is: \- Do different types of anemia (iron deficiency, vitamin B12/folate deficiency, thalassemia, and anemia of chronic illness) cause HbA1c levels to be falsely high or low in people without diabetes?

Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia

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.

A Study to Compare Elritercept With Epoetin Alfa to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions

The main aim of this study is to assess how elritercept works in lowering the need for RBC (red blood cell) transfusions and how safe elritercept is when compared with epoetin alfa. Other aims are to learn if elritercept improves tiredness as reported by participants without needing RBC transfusion compared with epoetin alfa, the RBC transfusion burden and quality of life compared with epoetin alfa. The study also aims to find out the extent of the immune response to elritercept. The study will also check on the medical problems (safety) of elritercept.

Guideline Adherence for Preoperative Anemia Management in a PAC

Anemia in surgical patients is a common seriously problem; around 40 % of patients presenting for major surgery are anemic problem. Patients with major surgery have significantly higher rates of acute blood loss. Whereas , patients with pre-operative anemia prone to be transfused blood component in pre-operative or intraoperative and postoperative periods that associated with worse outcomes , prolonged hospital stays , increased risk of morbidity and mortality . Therefore, patients undergoing major surgery should be optimization for pre-operative anemia. In November 2021, Siriraj Preanesthesia Assessment Center (SIPAC) has developed and implemented a preoperative anemia management guideline which is one pillar of perioperative patient blood management. The objective of this guideline is to optimize red blood cell mass before patients having operation. The investigators are realize the important of pre-operative anemia of patients who undergoing elective surgery. The investigators will aim to evaluate adherence to the preoperative anemia management guideline protocol and perioperative outcomes and use the data of this study to setting guidelines for preoperative anemia evaluation and management in SIPAC of department of anesthesiology in Siriraj hospital for improving workflow and optimization before elective surgery, supporting to a reduction in blood transfusion, hospital stay, morbidity and health care costs of public health of Thailand.

Assessing Health System Readiness for Scaling Antenatal MMS in Cambodia

Malnutrition among pregnant women in low- and middle-income countries (LMICs) can cause micronutrient deficiencies that result in adverse maternal and neonatal health outcomes. The World Health Organization has recently recommended antenatal multiple micronutrient supplementation (MMS) that includes iron and folic acid (IFA) to improve maternal and neonatal health outcomes. MMS likely provides additional antenatal benefits over IFA supplementation alone. The Cambodian government, in partnership with Helen Keller International, is piloting MMS implementation in Takeo Province, which will inform nationwide scale-up of MMS. Study Purpose: The Takeo implementation must be evaluated to understand context-specific implementation of MMS and health system readiness for scale-up. This study assesses system readiness through four domains from the Intervention Scalability Assessment Tool (ISAT): (1) fidelity and adaptation, (2) reach and acceptability, (3) delivery setting and workforce capacity, and (4) implementation infrastructure. Population: The pilot involves transitioning to MMS from IFA across all 86 health centers, all 6 referral hospitals, and the provincial hospital in Takeo province. This study includes pregnant women receiving antenatal care, antenatal healthcare providers and facilities, and hospital managers in Takeo. The study also includes national governing bodies for MMS delivery. Methods: This mixed-methods study uses ISAT as a framework for developing data collection methods. Sampling strategies emphasize urban and rural representation across all operational districts in Takeo and diverse stakeholder perspectives at multiple levels of the health system. Data collection includes: * 12 focus group discussions (FGDs) with health center providers to assess provider adherence to MMS delivery guidelines, perspectives on the transition to MMS, and additional resources needed for MMS delivery * Workload assessment surveys at FGDs to quantify any burden on providers and resource gaps to deliver MMS * MMS stockout monitoring at 18 health centers and 7 hospitals to assess supply chain reliability * 15 key informant interviews with hospital managers to assess perspectives on the integration of MMS into antenatal services and facility readiness for MMS delivery * Phone surveys with 630 pregnant women to assess acceptability and adherence to MMS at 90- and 180-days after MMS distribution * One FGD with national-level stakeholders to assess national-level readiness, economic planning, and resource planning for sustainable MMS implementation and scale-up * Rapid economic evaluation to estimate the cost of nationwide scale-up Potential Impact: This study will generate insights into real-world implementation of MMS in Cambodia. It will identify context-specific adaptations, implementation gaps, and resource planning frameworks needed for nationwide scale-up of MMS. Successful scale-up is anticipated to improve maternal and neonatal health in Cambodia. Findings can also be used to guide other LMICs seeking to transition to and sustainably implement MMS.

Quality of Life-Guided Transfusion in Refractory MDS or AML

Patients with refractory myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) in exclusive palliative care frequently receive red blood cell transfusions based on hemoglobin thresholds, despite limited evidence of clinical benefit in this setting. This prospective randomized study compares a standard hemoglobin-based transfusion strategy to a quality-of-life-guided strategy using the EQ-5D-5L questionnaire, with the aim of reducing transfusion burden while maintaining patient safety and quality of life.

RBC Irradiation, Anemia and Gut Injury

The purpose of this trial is to study the effect that anemia and red blood cell (RBC) transfusions have on oxygen levels in the digestive tracts of extremely low birth weight (ELBW) infants and to look for possible markers in a baby's blood, urine and/or stool that may lead to a better understanding of what makes an ELBW infant at risk for digestive tract problems such as necrotizing enterocolitis.

129Xe MRI Cardiopulmonary

The goal of this NIH-sponsored study is to characterize three biomarkers derived from 129Xe gas exchange MRI and to understand how they change in response to interventions.

Early Percutaneous Transluminal Angioplasty in Diabetic Foot Syndrome (PTA-DFS)

The planned study is a Randomized Controlled Monocentric Trial, which will provide evidence on whether early angiography in percutaneous transluminal angioplasty (PTA) readiness ("immediate" treatment, within 48h) has advantages over the "standard of care", i.e., an elective procedure ("elective PTA") for the treatment of diabetic foot ulcer (DFU). The primary study endpoint is to investigate the impact of the "early PTA" within 48 hours on wound-healing assessed by wound area changes after PTA using a 3D-camera with artificial intelligence (AI)-based wound-analysis-system. The secondary endpoint is the effect of early PTA on the combined occurrence of major adverse limb (MALE) and cardiac events (MACE) over 12 months post-angioplasty using time-to-event analysis. Data will be collected at baseline, 24 hours, 1, 2, 3, 6, and 12 months after PTA. Diabetic kidney disease, distal symmetric polyneuropathy, retinopathy, cardiomyopathy, laboratory analyses, clinical scores, AI-based fundus photography, echocardiography, duplex sonography, and pulse oscillography will be assessed. Explanatory variables for wound healing are wound microbiome changes using whole-genome sequencing and oxygen saturation of the wound environment measured using near-infrared spectroscopy. Altered microbiome composition in ulcers can lead to severe local and systemic infections and complications, including major amputations. Nevertheless, the specific significance of the wound microbiome composition in chronic ischaemic ulcers in type 2 diabetes and the impact of PTA on the wound microbiome in type 2 diabetes is unclear. The exact timing for treating peripheral arterial disease (PAD) by revascularization in DFU after initial diagnosis is unknown and has yet to be fully understood.

Ferric Citrate in Iron Deficiency Anemia During Pregnancy

Maternal anemia remains a significant public health concern in low and middle income countries including Bangladesh, contributing to adverse maternal and neonatal outcomes. Despite the widespread use of oral ferrous fumarate, gastrointestinal intolerance and suboptimal adherence remain persistent challenges, indicating the need for alternative iron formulations with improved efficacy and safety profiles. Hence, this study aims to evaluate the efficacy and safety of oral ferric citrate compared to oral ferrous fumarate in the management of maternal iron deficiency anemia during second trimester of pregnancy. This phase 3, open-label, two-arm, parallel-group randomized controlled trial will be conducted among pregnant women aged 18-35 years, with gestational age between 13 and 26 weeks, diagnosed with moderate to severe iron deficiency anemia (hemoglobin 7-9.9 g/dL and serum ferritin \<30 μg/L). Exclusion criteria are non-iron deficiency anemia, multiple pregnancy, severe comorbidities, and recent intravenous iron therapy. Eligible participants will be randomized in a 1:1 ratio to receive either oral ferric citrate 210 mg (210 mg elemental iron) once daily for 8 weeks or oral ferrous fumarate 200 mg (66 mg elemental iron) twice daily for 12 weeks or until delivery, whichever occurs earlier. The primary endpoint is the change in maternal hemoglobin concentration from baseline to week 4 of randomization. Secondary endpoints include maternal outcome such as prevalence of anemia and iron deficiency at 36 weeks gestation and at 6 weeks postpartum, changes in maternal health-related quality of life (assessed by EQ-5D-5L), incidence of severe anemia requiring transfusion, and serious maternal medical events and neonatal outcomes such as birth weight, placental weight, birth weight percentiles, rates of low birth weight, abortion, stillbirth, preterm birth, and cord blood hemoglobin and ferritin levels, as well as infant hemoglobin and iron indices at 6 weeks of age. Safety endpoints will include incidence of adverse events, serious adverse events, maternal sick visits during the follow-up period. Ethical approval for this trial was obtained from the institutional review boards of Dhaka Medical College, and written informed consent will be secured from all participants prior to enrollment. The findings from this trial are expected to inform clinical guidelines by establishing whether ferric citrate offers a clinically advantageous alternative to Ferrous fumarate for managing iron deficiency anemia during pregnancy.

Acceptability, Adherence, and Impact on the Bioavailability of Iron and Zinc of the Dietary Supplements Goodphyte IB Defense and Goodphyte Immunity in Adults With Chronic Diseases.

The primary objective of the present postdoctoral research is to evaluate the acceptability and adherence of two dietary supplements containing microbial phytase, Goodphyte IB Defense and Goodphyte Immunity, in adult individuals with chronic diseases, namely Idiopathic Inflammatory Bowel Diseases (IIBD)-that is, Crohn's disease (CD) or Ulcerative Colitis (UC)-Arterial Hypertension (AH), Anemia (AN), or Multiple Sclerosis (MS). Secondarily, this study will investigate potential changes in iron and zinc absorption following phytase supplementation in these individuals and, consequently, possible changes in their quality of life.