BEACON: A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease and Severe Vaso-Occlusive Crises

Date Added
June 13th, 2023
PRO Number
Pro00121176
Researcher
Jennifer Jaroscak

List of Studies


Keywords
Transplant
Summary

This is a research study to find out if the study drug called BEAM-101 is safe and effective in the treatment of patients with severe Sickle cell disease (SCD). The study drug, BEAM-101 is a new investigational (experimental) therapy that is the first in human use of this drug.

The study medication uses patients' own stem cells that are harvested through apheresis (process where blood is removed, stem cells collected and blood is returned), changed by genetic modification, and transplanted back into the individual through intravenous infusion to treat severe SCD.

Participation in this study is expected to last approximately 24 months, starting at time of screening, through the collection of cells, transplantation of study drug, and 15-month follow-up period. Study visits during the screen and mobilization period will vary depending on the cell collection process, in other words, the mobilization and collection period could be 3 separate visits to harvest adequate stem cells. Subjects are then hospitalized for the conditioning period, transplantation of study drug and engraftment period (when blood counts return to normal). The follow up visits after discharge from the hospital will be monthly for the first 6 months, then every other month until 24 months post transplant period.

At the end of the 24 months, all participants will be asked to enroll in the long-term extension study for a duration of 13 years making the total follow-up period of 15 years.

Institution
MUSC
Recruitment Contact
Brandi Day
843-792.3379
dayb@musc.edu

Novel Cellular Therapy for the Treatment of Pain Associated with Chronic Pancreatitis

Date Added
July 18th, 2023
PRO Number
Pro00127525
Researcher
Hongjun Wang

List of Studies


Keywords
Pain
Summary

Patients with chronic pancreatitis often suffer from severe abdominal pain that reduce their quality of life. The major purpose of this study is evaluate the safety and efficacy of an infusion of the patient's own stem cells to relieve chronic pain. The stem cells will be isolated from the patient's bone marrow and grown in the clean lab to be used for treatment. After infusion into the vein, the participant will be followed for 12 months to evaluate their pain and other outcomes.

Institution
MUSC
Recruitment Contact
Leah Benn
843-792-2813
bennle@musc.edu

A 52-Week Open-Label Extension Study of Pimavanserin in Children and Adolescents with Irritability Associated with Autism Spectrum Disorder (ASD)

Date Added
August 28th, 2023
PRO Number
Pro00127713
Researcher
McLeod Gwynette

List of Studies


Keywords
Autism
Summary

Pimavanserin is an antipsychotic may have some beneficial effects on core autism symptoms and co-morbid conditions such as irritability, anxiety, sleep disorders, mood instability due to epilepsy, etc.). These potential benefits stem from pimavanserin's impact on the serotonin system in the body. This study will investigate pimavanserin in the treatment of irritability, core autism symptoms, and co-morbid conditions in children ages 6-17 years old.

Institution
MUSC
Recruitment Contact
McLeod Gwynette
843-792-4113
gwynette@musc.edu

Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles for Hospitalized Patients with Moderate-to-Severe ARDS: A Phase III Clinical Trial

Date Added
October 19th, 2023
PRO Number
Pro00132554
Researcher
Andrew Goodwin

List of Studies


Keywords
Critical Care
Summary

This study is a multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the safety and efficacy of ExoFlo for the treatment of moderate-to-severe ARDS (Acute Respiratory Distress Syndrome). The purpose of this study is to research and evaluate the safety and efficacy of intravenous (IV) administration of bone marrow mesenchymal stem cell derived extracellular vesicles, ExoFlo, as treatment for Moderate-to-Severe ARDS. Bone Marrow Mesenchymal Stem Cell (bmMSC)-Derived Extracellular Vesicles is an investigational drug created from human bone marrow being studied for the treatment of moderate-to-severe ARDS. This is a research study that will involve monitoring oxygen and inflammation levels after taking the investigational product and assessing the safety of the investigational product. The experimental treatment is a biologic product called Bone Marrow Mesenchymal Stem Cell (bmMSC)-Derived Extracellular Vesicles Allograft Product ExoFlo, which is purified from the bone marrow of a healthy well-screened individual. Participants will receive either 15mL of the investigational product with 85 mL of normal saline or 100 mL of normal saline only (placebo). The expected duration of participation in the study is a maximum of 61 days, which includes 1-day screening prior to treatment and 60 days following the first treatment.

Institution
MUSC
Recruitment Contact
Zerlinna Teague
8437920965
recruitment@musc.edu

STEMCAP-1: Safety and Efficacy of Mesenchymal Stem Cells Associated with Chronic Pancreatitis Pain

Date Added
December 19th, 2023
PRO Number
Pro00132905
Researcher
Hongjun Wang

List of Studies


Keywords
Pain
Summary

Patients with chronic pancreatitis often suffer from severe abdominal pain that reduce their quality of life. The major purpose of this study is evaluate the safety and efficacy of an infusion of donor derived mesenchymal stem cells to relieve chronic pain. After cell infusion into the vein, the participant will be followed for 6 months to evaluate their pain and other outcomes. There are a total of 5 clinic visits with a total study participation of up to 7 months.

Institution
MUSC
Recruitment Contact
Leah Benn
843-792-2813
bennle@musc.edu



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