This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD.
Official registry: https://clinicaltrials.gov/study/NCT06506461 · St. Jude Children's Research Hospital · enrollment listed as 25
Phase 1: the first tests in people. The main question is safety and dose, not whether it cures the condition. Groups are small. Side effects are watched closely.
Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
If you join, the listed intervention(s) are Plerixafor, Busulfan, or Gene-modified CD34+ cells. The registry lists allocation as na. This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD.
They plan to enroll about 25 people at Memphis. Taking part typically means taking a study drug (or dummy pill), an infusion or injection of a biologic plus check-ins.
St. Jude Autologous Genome Edited Stem Cells For Sickle Cell Disease-1
Interventions: Plerixafor, Busulfan, Gene-modified CD34+ cells, Motixafortide
Primary outcome (registry wording): Incidence of neutrophil engraftment by day +42 after infusion of the CRISPR/Cas9-edited CD34+ HSPCs.
Akshay Sharma, MBBS, MSc
Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.
This study is not a treatment protocol and no experimental treatments are involved. Study participants may be seen as needed for clinical, translational and basic research studies, or as medically indicated.
NCT00081523 · Official ClinicalTrials.gov record · National Heart, Lung, and Blood Institute (NHLBI)
This study will collect representative blood samples from healthy children and adults and from children and adults who have unique red blood cell features that are related to sickle cell disease. Sickle cell disease is a blood disease that limits the ability of red blood cells to carry oxygen throughout the body.
NCT00542230 · Official ClinicalTrials.gov record · National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Background: Some people with the same disorder on a genetic level have more complications than others. Researchers want to look for a link between the PKLR gene and sickle cell disease (SCD) symptoms.
NCT03685721 · Official ClinicalTrials.gov record · National Heart, Lung, and Blood Institute (NHLBI)
The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.
Phase 1. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.
How to read a ClinicalTrials.gov record · Questions to ask the coordinator · Glossary