Clinical Trials - HOT Division (Center for Bleeding and Clotting Disorders)
Please find a list of active and recent Division of Hematology, Oncology, & Transplantation clinical trials in the dropdowns below.
Currently Recruiting
Atlas Neo (Hemophilia)
Title: Atlas Neo
Enrollment Status: Open to enrollment
Contact: Diondra Howard howar709@umn.edu
About This Study: A Phase 3, single-arm, multicenter, multinational, open-label, one-way crossover study to investigate the efficacy and safety of fitusiran prophylaxis in male participants aged ≥ 12 years with hemophilia A or B with or without inhibitory antibodies to factor VIII or IX
Eligibility: Hemophilia A or B with or without inhibitors
Treatment: Fitusiran
Investigator: Dr. Jacob Cogan https://med.umn.edu/bio/jacob-cogan
Location: M Health Fairview Center for Bleeding and Clotting Disorders - Minneapolis
Protocol Number: ClinicalTrials.gov: NCT05662319
Publications: None
iTTP and (rADAMTS13) With Minimal to No Plasma Exchange (PEX)
Title: iTTP and (rADAMTS13) With Minimal to No Plasma Exchange (PEX)
Enrollment Status: Open to enrollment
Contact: Diondra Howard howar709@umn.edu
About This Study: A Phase 2b, multicenter, randomized, double-blind study of safety and efficacy of TAK-755 in adults with immune-mediated thrombotic thrombocytopenic purpura (iTTP). The main aim of this study is to determine the percentage of participants with a clinical response without plasma exchange during the study. Participants who have an acute attack of iTTP will receive TAK-755 and immunosuppressive therapy during their stay at the hospital until they achieve a clinical response. Participants will also be treated with TAK-755 for an additional time of up to 6 weeks after the acute phase. In total, participants will stay in the study for approximately 3 months
Eligibility: 18+ years of age and diagnosed with primary or secondary iTTP not yet receiving more than 2 plasma exchanges before randomization
Treatment: TAK-755 (rADAMTS13)
Investigator: Dr. Marshall Mazepa https://med.umn.edu/bio/marshall-mazepa
Location: M Health Fairview Clinics and Surgery Center - Minneapolis
Protocol Number: ClinicalTrials.gov: NCT05714969
Publications: None
ATHN10 (Hemophilia)
Title: ATHN10
Enrollment Status: Open to enrollment
Contact: Michael Nolan (michael.nolan@fairview.org or 612-365-8257) or Dr. Mark Reding (612-273-5005)
About This Study: This is a multi-center project in which the American Thrombosis & Hemostasis Network (ATHN) will offer free genetic testing to individuals with Rare Coagulation Disorders (RCD). The primary goal of this study is to document genetic data on the population of RCD patients being cared for in U.S. hemophilia treatment centers.
Eligibility: Participants must have an established RCD diagnosis, must currently receive or have received care at an ATHN affiliated hemophilia treatment center, and must have opted into the ATHNdataset.
Treatment: None. The study simply requires the collection of one blood sample.
Investigator: Dr. Mark Reding
Location: M Health Fairview Center for Bleeding and Clotting Disorders - Minneapolis
Protocol Number: Not applicable
Publications: None
ELEVATE (AHP)
Title: ELEVATE
Enrollment status: Open for enrollment
About This Study: A Global Observational Longitudinal Prospective Registry of Patients With Acute Hepatic Porphyria (AHP)
Eligibility: Patients with all types of AHP, including patients with hepatic and/or renal impairment, adolescents, elderly patients, and pregnant or lactating women. For more information, please contact the Research Coordinator, Diondra Howard, howar709@umn.edu
Treatment: Observational, No Intervention
Investigator(s): Dr. Gregory Vercellotti, Co-Investigator: Dr. Marshall Mazepa
Location: M Health Fairview Clinics and Surgery Center - Minneapolis
Protocol Number: ClinicalTrials.gov: NCT04883905
Publications: Publications are currently not available.
Longitudinal Study of the Porphyrias
Title: Longitudinal Study of the Porphyrias
Enrollment status: Open for enrollment
About This Study: The objective of this study is to conduct a longitudinal investigation of the natural history, complications and therapeutic outcomes in people with acute and cutaneous porphyria.
Eligibility: Individuals with a documented diagnosis of porphyria. For more information, please contact the Research Coordinator, Diondra Howard, howar709@umn.edu
Treatment: Observational, no intervention
Investigator: Dr. Marshall Mazepa
Location: M Health Fairview Clinics and Surgery Center - Minneapolis
Protocol Number: ClinicalTrials.gov: NCT01561157
Publications: Publications are currently not available.
Edithal/EDIT-301 Gene Therapy for Beta Thalassemia
Title: Edithal/EDIT-301 Gene Therapy for Beta Thalassemia
Enrollment status: Open for enrollment
About This Study: Phase 1/2. Autologous CRISPR-Cas12a Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells in Transfusion-Dependent Beta Thalassemia (TDT).
Eligibility: Patients 18-35 years with beta-thalassemia who have needed >=10 units or 100 ml/kg pRBCs/year for 2 years. No available matched related donor. More Information: Research Coordinator - Alli Travis, RN: atravis@umn.edu
Treatment: EDIT 301 gene therapy
Investigator(s): Dr. Roy Kao
Location: M Health Fairview University of Minnesota Medical Center
Protocol Number: ClinicalTrials.gov: NCT05444894
Publications: Publications are currently not available.
HGB210/Lovotibeglogene Gene Therapy for Sickle Cell Disease
Title: HGB210/Lovotibeglogene Gene Therapy for Sickle Cell Disease
Enrollment status: Open for enrollment
About This Study: Phase 3. Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Sickle Cell Disease
Eligibility: Patients 5-17 years with SS, S-beta0, and S-beta+ sickle cell disease, who have had >=4 severe VOEs in the last 2 years (painful VOEs, acute chest syndrome, priapism, hepatic/splenic sequestration requiring >=24 hr hospitalization or >=2 outpt infusion visits). No history of overt stroke. More Information: Research Coordinator - Lauren Matzke, RN: matzk042@umn.edu
Treatment: Lovotibeglogene autotemcel gene therapy
Investigator(s): Dr. Ashish Gupta
Location: M Health Fairview University of Minnesota Medical Center
Protocol Number: ClinicalTrials.gov: NCT04293185
Publications: Publications are currently not available.
BEAM-101 Gene Therapy for Sickle Cell Disease
Title: BEAM-101 Gene Therapy for Sickle Cell Disease
Enrollment status: Open for enrollment
About This Study: Phase 1/2. Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) to Increase Fetal Hemoglobin (HbF) Production in Patients with Severe Sickle Cell Disease
Eligibility: Patients 18-35 years with SS, S-beta0, and S-beta+ sickle cell disease, who have had 4 severe VOEs in the last 2 years (painful VOEs needing any inpatient/outpatient medical facility visit, acute chest syndrome, priapism, or splenic sequestration). No history of overt stroke, moyamoya on MRA. More Information: Research Coordinator - Lauren Matzke, RN: matzk042@umn.edu
Treatment: BEAM-101 autologous gene therapy
Investigator(s): Dr. Ashish Gupta and Dr. Roy Kao
Location: M Health Fairview Clinics and Surgery Center - Minneapolis
Protocol Number: ClinicalTrials.gov: NCT05456880
Publications: Publications are currently not available.
Pomalidomide for the Treatment of Bleeding in HHT (PATH-HHT)
Title: Pomalidomide for the Treatment of Bleeding in HHT (PATH-HHT)
Enrollment status: Open for enrollment
About This Study: The PATH study is exploring the use of an oral medication called pomalidomide for the treatment of nosebleeds. Adults suffering from HHT (hereditary hemorrhagic telangiectasia) with moderate to severe nosebleeds (epistaxis) who require iron infusions or blood transfusions are eligible. During the 6-month study, patients might receive either pomalidomide or a matching placebo (sugar pill) in addition to their usual care
Eligibility: Patients >18 years of age with a clinical diagnosis of HHT and Epistaxis severity score ≥ 3 measured over the preceding three months, measured at the screening visit. More Information: Research Coordinator - Ashley Ramaker, ramak037@umn.edu
Treatment: pomalidomide
Investigator(s): Dr. Holly Boyer (ENT) , Co-Investigator: Dr. Marshall Mazepa
Location: M Health Fairview Clinics and Surgery Center - Minneapolis
Protocol Number: ClinicalTrials.gov: NCT03910244
Publications: Publications are currently not available.
Polycythemia Vera and Essential Thrombocythemia (MPN)
Title: Polycythemia Vera and Essential Thrombocythemia
Enrollment status: Open for enrollment
About This Study: Role of endothelial cells in MPN in bleeding and clotting. Non-interventional, observation study to investigate mechanisms of bleeding and clotting in MPN patients. The study collects extra blood samples during routine visits at the Masonic Cancer Center. Blood samples are used for research related to thrombosis.
Eligibility: Individuals with a documented diagnosis of Myeloproliferative Neoplasms (MPN) For more information, please contact the Research Coordinator, Diondra Howard, howar709@umn.edu
Treatment: Observational, no intervention
Investigator: Dr. Joan Beckman
Location: M Health Fairview Clinics and Surgery Center - Minneapolis
Protocol Number: Identifier not available.
Publications: 1. Reeves BN, Beckman JD. Novel Pathophysiological Mechanisms of Thrombosis in Myeloproliferative Neoplasms. Current Hematologic Malignancy Reports. 2021. 2. Beckman JD, DaSilva A, Aronovich E, Nguyen A, Nguyen J, Hargis G, et al. JAK-STAT inhibition reduces endothelial prothrombotic activation and leukocyte-endothelial proadhesive interactions. J Thromb Haemost. 2023.
Safety of Single Doses of CSL889 in Adult Patients with Sickle Cell Disease
Title: Safety of Single Doses of CSL889 in Adult Patients with Sickle Cell Disease
Enrollment status: Open for enrollment
About This Study: This is a phase 1, first-in-human, multi-center, open-label, single dose cohort study to evaluate the safety and tolerability, pharmacokinetics (PK), exploratory pharmacodynamics (PD), and biomarkers of target engagement of CSL889 following single intravenous (IV) doses in subjects with sickle cell disease (SCD). The study involves sequential dose escalation of cohorts with between-group assessments of key safety and PK variables.
Eligibility: Patients 18 to 60 years of age with diagnosed sickle cell disease stable for at least 30 days prior to enrollment. For more information, please contact the Research Coordinator, Diondra Howard, howar709@umn.edu
Treatment: CSL889
Investigator: Dr. Alex Boucher
Location: M Health Fairview Clinical Research Unit
Protocol Number: ClinicalTrials.gov: NCT04285827
Publications: Abstract available
USTMA P200 (TTP)
Title: USTMA P200
Enrollment status: Open for enrollment
About This Study: United States Thrombotic Microangiopathy (USTMA) Thrombotic Thrombocytopenic Purpura (TTP) Clinical Data and Biologic Sample Repository. The primary objective of this study is to establish a tissue and clinical data repository of patients diagnosed with thrombotic thrombocytopenic purpura (TTP) for patients that are treated at member institutions of the United States Thrombotic Microangiopathy (USTMA) consortium.
Eligibility: Recently diagnosed with acquired TTP or may be in remission from a previous diagnosis of acquired TTP. For more information, please contact the Research Coordinator, Diondra Howard, howar709@umn.edu
Treatment: Observational, No Intervention
Investigator: Dr. Marshall Mazepa
Location: M Health Fairview Clinics and Surgery Center - Minneapolis
Protocol Number: Identifier not available.
Publications: Publications are currently not available.
Closed for Enrollment
GeneR8-1 (Hemophilia)
Title: GeneR8-1
Enrollment status: Closed for enrollment
About This Study: A Phase III open-label, single-arm study to evaluate the efficacy and safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene transfer of Human Factor VIII in Hemophilia A patients with residual FVIII levels ≤ 1 IU/dL receiving prophylactic FVIII infusions (BMN 270-301).
Eligibility: Males ≥ 18 years of age with hemophilia A and residual FVIII levels ≤ 1 IU/dL
Treatment: BMN270 gene Therapy
Investigator: Dr. Mark Reding
Location: M Health Fairview Center for Bleeding and Clotting Disorders - Minneapolis
Protocol Number: ClinicalTrials.gov: NCT03370913
Publications: Publications are currently not available.
HEM-POWR (Hemophilia)
Title: HEM-POWR
Enrollment status: Closed for Enrollment
About This Study: Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A (HEM-POWR)
Eligibility: Previously treated patients with hemophilia A receiving damoctocog alfa pegol with any kind of treatment modality (on-demand, prophylaxis, or intermittent prophylaxis).
Treatment: Damoctocog alfa pegol (Jivi)
Investigator: Dr. Joan Beckman
Location: M Health Fairview Center for Bleeding and Clotting Disorders
Protocol Number: ClinicalTrials.gov: NCT03932201
Publications: Publications are currently not available.
USTMA P201 (TTP)
Title: USTMA P201
Enrollment status: Closed for enrollment
About This Study: A Pilot Study of Acquired Thrombocytopenic Purpura and Neuropsychologic Complications: A multi-center, prospective, and longitudinal study of neuropsychological complications in patients with acquired TTP in remission.
Eligibility: Patients with a prior history of acquired TTP mediated by documented severe (<10%) ADAMTS13 deficiency and a documented anti-ADAMTS13 antibody. Patients must be at least 3 months from their last acute TTP episode at the time of enrollment
Treatment: Observational, No Intervention
Investigator: Dr. Marshall Mazepa
Location: M Health Fairview Clinics and Surgery Center - Minneapolis
Protocol Number: Publications are currently not available.
Publications: Publications are currently not available.
Predict (Hemophilia)
Title: Predict
Enrollment Status: Closed for enrollment
About This Study:A multicenter, prospective, open-label, clinical study to assess the effect of using a new risk score approach to select the most appropriate prophylaxis regimen for reaching a favorable outcome, when hemophilia A patients switch from standard half-life products to damoctocog alfa pegol (Jivi)
Eligibility: Hemophilia A prophylaxis with any SHL FVIII product on a stable dose/regimen for at least 12 months before entering the study.
Treatment: Damoctocog alfa pegol
Investigator: Dr. Jacob Cogan
Location: M Health Fairview Center for Bleeding and Clotting Disorders - Minneapolis
Protocol Number: ClinicalTrials.gov: NCT05036278
Publications: None