RecruitingPhase 1interventionalRules rewrite

Phase 1: testing autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein for Sickle Cell Disease

Q: Is NCT07708350 still recruiting?

The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.

Q: What phase is NCT07708350?

Phase 1. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

Q: Can PlainTrials tell me if I qualify for NCT07708350?

No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.

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 deoxyribonucleic acid (DNA)) from the patient to treat his or her own disease.

Official registry: https://clinicaltrials.gov/study/NCT07708350 · Daniel Bauer · enrollment listed as 5

What this phase means

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.

What the registry says happens

If you join, the listed intervention(s) are autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, or Sequencing Assay for Variant rs114518452. The registry lists allocation as na. A promising approach for the treatment of genetic diseases is called gene therapy.

They plan to enroll about 5 people at Boston. Taking part typically means an infusion or injection of a biologic, using or being fitted with a device plus check-ins.

Official title (unrewritten)

A Pilot Trial of Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease

Interventions: autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452

Primary outcome (registry wording): Primary engraftment

Who it is for (from inclusion criteria)

Ages 13 Years to 35 Years
Diagnosis of either a) sickle cell disease with genotype HbSS, HbS/B0 thalassemia, HbSD, or HbSO
Adequate hematologic parameters including:
a. White blood cell (WBC) count within the range of 2.5 - 25.0 x 109 /L b. Platelet count within the range of 150 - 700 x 109 /L
Adequate organ function and performance status:
Karnofsky performance status ≥70%
Serum creatinine \</=1.5 times the upper limit of normal for age, and calculated creatinine clearance or Glomerular Filtration Rate (GFR) \</= 60 mL/min/1.73 m2
Direct bilirubin ≤ 2.0 mg/dL

Who the registry excludes

Subjects who have concomitant condition or illness including, but not limited to:
Uncontrolled infection, such as current febrile illness, infection requiring parenteral antibiotics, or systemic fungal infection
Active complication of underlying hemoglobinopathy that would place the patient at unacceptable risk for participation, in the judgment of the Investigators
Major surgery in the past 30 days
Medical/psychiatric illness/social situations that would limit compliance with study requirements as determined by the treating physician
Contraindication to administration of conditioning medication (busulfan)
3\. Subjects who have undergone allogeneic or autologous hematopoietic stem cell transplant previously

Public study contact (from the registry)

Emily Morris

631-355-8724

gene.therapy@childrens.harvard.edu

Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.

Sites listed on ClinicalTrials.gov

1 listed

Boston Children's Hospital — Boston, Massachusetts (recruiting)

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FAQ about this NCT record

Is NCT07708350 still recruiting?

The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.

What phase is NCT07708350?

Phase 1. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

Can PlainTrials tell me if I qualify for NCT07708350?

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