Clinical Trials - HOT Division Classical Hematology

Please find a list of active and recent Division of Hematology, Oncology, & Transplantation clinical trials in the dropdowns below.

Currently Recruiting

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Currently Recruiting

Atlas Neo (Hemophilia)

Title: Atlas Neo

Enrollment Status: Open to enrollment       

Contact: Zoe Wright [email protected]

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

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: [email protected] 

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.

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: [email protected] 

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.

Ianalumab (VAY736) - ITP

Title: Ianalumab (VAY736) - ITP

Enrollment status: Recruiting but not yet enrolling (in startup)

About This Study: A phase 3 randomized, double-blind study of ianalumab (VAY736) versus placebo in addition to eltrombopag in patients with primary immune thrombocytopenia (ITP) who had an insufficient response or relapsed after first line steroid treatment (VAYHIT2)

Eligibility: A diagnosis of primary ITP, with insufficient response to, or relapsed after a first line corticosteroid therapy +/- IVIG.

Treatment: Ianalumab

Investigator:  Dr. Marshall Mazepa
Contact: Zoe Wright [email protected]

Location: M Health Fairview Clinics and Surgery Center - Minneapolis

Protocol Number: NCT05653349

Publications: Publications are currently not available.

Ianalumab (VAY736) - wAIHA

Title: Ianalumab (VAY736) - wAIHA

Enrollment status: Currently enrolling

About This Study:  A phase 3, randomized, double-blind, study to assess efficacy and safety of ianalumab (VAY736) versus placebo in warm autoimmune hemolytic anemia (wAIHA) patients who failed at least one line of treatment (VAYHIA)

Eligibility:  Patients with primary or secondary warm autoimmune hemolytic anemia (wAIHA) who had insufficient response to, or relapsed after at least one line of treatment

Conctact: Zoe Wright [email protected]

Treatment: Ianalumab

Investigator: Marshall Mazepa

Location: M Health Fairview Clinical Research Unit

Protocol Number: NCT05648968

Publications: Publications are currently not available.

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 [email protected] 

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

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, Zoe Wright [email protected]

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.

MAYARI (TTP)

Title: MAYARI (TTP)

Enrollment status: Currently enrolling

About This Study: An open-label, single-arm, multicenter study to evaluate the efficacy and safety of caplacizumab and immunosuppressive therapy without first-line therapeutic plasma exchange in adults with immune-mediated thrombotic thrombocytopenic purpura

Eligibility: Immune-mediated TTP

Treatment: Caplacizumab

Investigator:  Dr. Marshall Mazepa
Contact: Zoe Wright [email protected]

Protocol Number: NCT05468320

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, [email protected]

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.

Pregnancy AND Anticoagulation (PANDA)

Title: Pregnancy AND Anticoagulation (PANDA)

Enrollment status: Currently enrolling

About This Study:  Pregnancy AND Anticoagulation (PANDA) study: a VENUS Prospective Cohort Study of Management of Anticoagulation Around Delivery

Eligibility:  Pregnant patients requiring anticoagulation medication

Treatment: Observational, no intervention

Investigator: Jacob Cogan

Publications: Publications are currently not available.

SCOPE HIM (Hemophilia and Surgery)

Title: SCOPE HIM (Hemophilia and Surgery)

Enrollment status: Currently enrolling

About This Study: A Phase 3 Study of the Safety and Efficacy of Coagulation Factor VIIa (Recombinant) for the Prevention of Excessive Bleeding in Patients with Congenital Hemophilia A or B with Inhibitors to Factor VIII or IX Undergoing Elective Major Surgical Procedures

Eligibility: Congenital Hem A or B diagonsis with inhibitors and undergoing a major elective surgery

Treatment: LR769 (Factor VIIa product)

Investigator:  Dr. Marshall Mazepa
Contact: Zoe Wright [email protected]

Protocol Number: NCT05695391

Publications: Publications are currently not available.

SerpinPC (Hemophila)

Title: SerpinPC (Hemophila)

Enrollment status: Currently enrolling

About This Study: A Global, Open-label, Adaptive Design Study to Investigate the Efficacy and Safety of SerpinPC in Subjects With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

Eligibility: Diagnosis of severe Hem A or moderately severe to severe Hem B

Treatment: SerpinPC (serine protease inhibitor)

Investigator:  Mark Reding
Contact: Zoe Wright [email protected]

Protocol Number: NCT05468320
Location: M Health Fairview Center for Bleeding and Clotting Disorders

Publications: Publications are currently not 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, [email protected]

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

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Closed for Enrollment

ATHN10 (Hemophilia)

Title: ATHN10

Enrollment Status: Open to enrollment 

Contact: Michael Nolan ([email protected] 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

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: [email protected] 

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.

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.

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, [email protected] 

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.

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.

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, [email protected]

Treatment: CSL889 

Investigator: Dr. Alex Boucher

Location: M Health Fairview Clinical Research Unit

Protocol Number: ClinicalTrials.gov: NCT04285827

Publications: Abstract available 

ELEVATE

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, [email protected]

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.