Clinical Research Directory

Buprenorphine for the Management of Pain in Children With Sickle Cell Disease

The goal of this clinical trial is to learn whether a single dose of buccal buprenorphine (BELBUCA) is safe when used in place and/or with full agonist opioids that would otherwise be started during a sickle cell pain crisis admission, in adolescents and young adults (ages 12-22) with sickle cell disease (SCD) who are hospitalized for acute pain. The main questions it aims to answer are: * Is a single dose of buccal buprenorphine, given at doses between 75 and 450 micrograms, safe in this population? * Does buccal buprenorphine reduce the amount of full agonist opioid pain medicine participants need through their patient-controlled analgesia (PCA) pump, and does it change pain scores? * Are participants and their care team satisfied with pain management when buccal buprenorphine is used? * What are the pharmacokinetic properties (how the body absorbs, distributes, and clears the drug) of buccal buprenorphine in this age group? This is a single-arm, open-label, dose-escalation study: every participant receives buccal buprenorphine, and there is no placebo or separate comparison group. Instead, researchers will compare each participant's own opioid use and pain scores in the 8 hours before receiving buccal buprenorphine to the 8 hours afterward, to see whether the dose reduces the need for additional opioid pain medicine. Participants will: * Be treated first with standard-of-care patient-controlled analgesia (PCA) pain medicine (hydromorphone) during the early, acute-stabilization phase of their hospital stay. * Once their pain is stable and they meet criteria to transition to oral opioids, as would happen under usual care, they will receive a single dose of buccal buprenorphine instead, at a dose (75, 150, 300, or 450 micrograms) assigned according to a dose-escalation design. * Continue to have access to demand-only PCA pain medicine for breakthrough pain after the study dose. * Undergo continuous monitoring of heart rate, oxygen level, and carbon dioxide level, plus pain and sedation checks every 4 hours, for 48 hours after the dose. * Have blood samples collected, only when blood is already being drawn for clinical care, to help study how the body processes the drug. * Complete a brief satisfaction survey about their pain management after the dose. * Be contacted by phone or telehealth, and have their medical record reviewed, 30 days after the dose to check for any safety concerns.

Hypoxic Red Blood Cells in Sickle Cell Anemia

The overall objective of this study is to evaluate the effectiveness and safety of transfusing hypoxic red blood cells manufactured with the Hemanext ONE system in patients with sickle cell anemia. The Hemanext ONE device was cleared through the De Novo process in September 2023.

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease

A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order to fix or replace a diseased gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of Graft-Versus-Host Disease (GVHD), reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. The method used to fix or replace a diseased gene is called gene editing. A person's own cells are edited using a specialized biological medicine that has been formulated for use in human beings. Fetal hemoglobin (HbF) is a healthy, non-sickling kind of hemoglobin. Investigators have recently discovered a gene called BCL11A that is very important in the control of fetal hemoglobin expression. Increasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, and therefore potentially cure the condition.

Targeting the Pathophysiology of Sickle Cell-Related Kidney Disease Using the SGLT2 Inhibitors, Empagliflozin

Sickle cell anemia (SCA) is an inherited red blood disorder. The kidneys are among the most commonly affected organ systems in SCA. The Food and Drug Administration (FDA) has approved empagliflozin as a treatment to reduce the decline of kidney function in those with kidney disease. The proposed research study aims to determine whether empagliflozin can prevent the progression of kidney dysfunction in patients with sickle cell anemia (SCA) who are at high risk of developing advanced chronic kidney disease (CKD).

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order to fix or replace a diseased gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of Graft-Versus-Host Disease (GVHD), reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. The method used to fix or replace a diseased gene is called gene editing. A person's own cells are edited using a specialized biological medicine that has been formulated for use in human beings. Fetal hemoglobin (HbF) is a healthy, non-sickling kind of hemoglobin. Investigators have recently discovered a gene called BCL11A that is very important in the control of fetal hemoglobin expression. Increasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, and therefore potentially cure the condition.