Clinical Trials & Studies

Division of Diabetes, Endocrinology and Metabolism
Clinical Trials & Studies (listed below)

Epidemiology of Diabetes Intervention and Complications (EDIC)

The EDIC study is a multi-center, observational study designed to follow the type 1 diabetic volunteers who participated in the Diabetes Control and Complications Trial (DCCT). EDIC has been following subjects since 1994. This study is currently following participants.

Principal Investigator
John Bantle, MD

More Information
John Bantle, MD
bantl001@umn.edu
(612) 626-0462

CLINICAL TRIALS

Hypoglycemia Studies

Naloxone, Hypoglycemia & Exercise

Preventing hypoglycemia after exercise can be difficult. The purpose of this study is to learn if naloxone can be used to treat exercise-related hypoglycemia symptoms. Naloxone is currently used as a substance abuse treatment medication but research studies have shown it to possibly be effective in preventing next-day hypoglycemia.

This study is looking for people who:

• Are between the age of 18 – 65
• Have type 1 diabetes for 2 - 30 years
• Are willing to avoid exercise for the week before the study

This study involves one (1) screening visit where you will ride an exercise bike to determine your V02 max and wear a continuous glucose sensor (CGM). About 1 week later, you will return to the University of Minnesota for one (1) full day and one half (1/2) day visit. At the full day visit, you will ride an exercise bike and be given either naloxone or a placebo. The following day, we will lower your blood sugar to make you hypoglycemic and ask you about your symptoms. You will return about 8 weeks later to repeat the full day and half day parts of the study. If all parts of the study are completed, compensation is up to $675.

Recurrent Hypoglycemia in Type 1 Diabetes

This study has two aims that are currently enrolling subjects

Aim 1 involves one (1) screening visit, wearing a continuous glucose sensor (CGM) and an activity/sleep tracker, and two (2) full day visits to the University of Minnesota Center for Magnetic Resonance Research (CMRR). On each of the full day visits, you will have your blood sugar lowered 2 times and we will take 1 MRI scan of your brain. If all parts of the study are completed, compensation is up to $1,025.

Aim 2 involves one (1) screening visit, wearing a continuous glucose sensor (CGM) and an activity/sleep tracker, and three (3) visits to the University of Minnesota Center for Magnetic Resonance Research (CMRR) or Clinical Research Unit (CRU). On day one visit (CMRR), you will have your blood sugar increased and we will take an MRI scan of your brain. On day 2 (CRU) your blood sugar lowered 2 times and blood samples will be taken. On day 3 (CMRR) your blood sugar will lowered (similar to day 2) and then your blood sugar will be increased and an MRI taken of your brain (similar to day 1). If all parts of the study are completed, compensation is up to $1,300

Nidufexor (LBM763)

Type 2 diabetes is the most common type of diabetes, affecting almost 10% of the population. Patients with diabetes are at increased risk of kidney disease and make up almost 50% of the new cases of end-stage kidney disease in the US. Nidufexor is a new agent that acts on a bile acid receptor in the liver, potentially reducing fibrosis and inflammation.  This may have the potential to delay diabetic kidney disease in patients with diabetes. The aim of this study is to test if Nidufexor is safe, well tolerated and efficacious in patients with type 2 diabetes.  This study is sponsored by Novartis.

Eligibility to participate

You may be eligible to participate in the Nidufexor study if you:

• Are between the ages of 18 – 75 years
• Have type 2 diabetes
• Are concerned you may have or may develop kidney problems related to diabetes

This study does not accept healthy participants. This study is currently recruiting study participants.

What is involved in the Study?

• Participants will be randomly assigned to take Nidufexor or a placebo (inactive pill) for 32 weeks.
• During this time, participants will periodically have follow up visits at the University of Minnesota.

Principal Investigator
Luiza Caramori, MD, PhD, MSc

More Information
(612) 301-6685

CLINICALTRIALS

Preventing Early Renal Function Loss in Diabetes (PERL)

Patients with type 1 diabetes remain at risk for end-stage renal disease despite improvements in blood pressure and blood glucose control. The PERL study was a three-year multi-center study funded by the National Institutes of Health (NIH) aiming to determine whether allopurinol could reduce loss of renal function in people with type 1 diabetes.  This study is now completed, and patient who enrolled in PERL are currently participating in PERLage, PERL follow-up study, and/or in the PERL Kidney Biopsy study, a PERL Ancillary study (see below).

Ancillary Studies

Kidney Biopsy

The purpose of this study is to obtain kidney tissue which, in combination with the data obtained in the main PERL study, will be studied to identify new ways to predict who will develop more serious diabetic kidney disease and identify potential targets for new therapies.  Only patients who participate in PERL are eligible for this study.Measuring GFR using Filter Paper

Follow-up Study

PERLage is the non-interventional (no study medication) follow-up of the PERL trial.  Patients who were recruited in PERL have an annual follow-up evaluation to determine the course of kidney, heart and eye disease as it relates to diabetes.  Only patients who participated in PERL are eligible for this study.

Principal Investigator
Luiza Caramori, MD, PhD, MSc

More Information
(612) 301-6685

STUDY WEBSITE

CLINICAL TRIALS

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.

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.

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.

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.

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.

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.

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.

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 

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.

ONGOING TRIALS (RECRUITING CLOSED)

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.

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.

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.

Division of Pulmonary, Allergy, Critical Care and Sleep Medicine

Clinical Trials & Studies