What side effects are associated with this type of intervention?

Common treatments for Sickle Cell Disease include hydroxyurea, red blood cell transfusions, and investigational therapies like gene therapy. Hydroxyurea can cause side effects such as bone marrow suppression, leading to low blood cell counts, and potential infertility in men. Red blood cell transfusions carry risks of alloimmunization, iron overload, and transfusion reactions. Gene therapy, specifically autologous transplant of lentiviral vector modified peripheral blood, is still under investigation, but potential side effects may include insertional mutagenesis, immune reactions, and long-term effects on hematopoiesis. Patients should discuss these risks with their healthcare provider to make informed decisions.

What prior FDA approvals exist?

Hydroxyurea is a well-established treatment for Sickle Cell Disease (SCD) with significant evidence supporting its efficacy in reducing acute painful episodes and other complications. While gene therapy, such as the autologous transplant of lentiviral vector modified peripheral blood, is being studied for its potential to correct the hemoglobin defect in SCD, it remains in experimental stages with no randomized clinical trials reported to date. Thus, hydroxyurea remains the primary FDA-approved treatment for SCD, while gene therapy continues to be explored in clinical trials.

What other types of research exist?

Promising novel alternatives to autologous transplant of lentiviral vector modified peripheral blood for SCD patients include: 1) Hematopoietic Stem Cell Transplantation (HCT) from a matched related donor, which has shown success in preventing stroke and stabilizing neurologic abnormalities. 2) Genome editing tools such as CRISPR/Cas9, which aim to correct the sickle cell mutation or induce fetal globin expression. These alternatives offer potential curative options but come with considerations regarding donor availability, patient eligibility, and the risks associated with transplantation and gene editing.

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Gene Therapy

Stem Cell Gene Therapy for Sickle Cell Disease

Phase 1 & 2

Waitlist Available

Led By Gary Schiller, MD

Research Sponsored by Donald B. Kohn, M.D.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Inadequate clinical response to hydroxyurea (HU), defined as any one of the following outcomes, while on HU for at least 3 months:

- 2 or more acute sickle pain crises requiring hospitalization

Must not have

Patient must not have any known cancer or other malignant disease or active infection by CT or MRI of head, chest or ultrasound of abdomen

Poorly controlled hypertension as determined by BP with systolic >135 or diastolic >95 mmHg despite treatment.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 24 months

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new treatment for adults with severe sickle cell disease using their own modified blood cells. The treatment aims to fix the genetic problem causing sickle cell disease and help patients produce healthy red blood cells. This approach could be better than current treatments and avoid complications from donor transplants. The new therapy involves modifying the patient's own blood cells to correct the genetic issue.

Who is the study for?

Adults with severe sickle cell disease who have had complications like pain crises, stroke, or acute chest syndrome despite treatment can join this trial. They must not be pregnant, have HIV/HCV/HTLV-1/hepatitis B/Cancer, and should not have a suitable sibling donor for transplant.

What is being tested?

The trial is testing a gene therapy where patients' own blood stem cells are modified with the βAS3-FB vector to potentially treat sickle cell disease. It's in Phase I to check safety and initial effectiveness.

What are the potential side effects?

Potential side effects may include reactions at the infusion site, immune response against the modified cells, or other unforeseen issues due to genetic modification of blood cells.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My condition did not improve after taking hydroxyurea for 3 months.

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I have been hospitalized for sickle cell pain crises at least twice.

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I can care for myself but may need occasional help.

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I am 18 years old or older.

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I've had a severe lung issue not caused by asthma in the last 2 years despite treatment.

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I have had a stroke or a lasting brain problem for more than a day.

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I had episodes of swollen fingers or toes in my childhood.

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I have severe bone tissue death.

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I have been receiving regular blood transfusions for over a year to manage my sickle cell disease.

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My hemoglobin levels have not increased by more than 1.5 gm/dl from the baseline, or I need transfusions to keep my hemoglobin above 6.0 gm/dL.

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My sickle cell disease is confirmed by genetic testing.

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I was hospitalized for a severe chest problem in the last 2 years.

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I have been hospitalized for sickle cell pain crises at least twice in the last 2 years.

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I have had priapism at least twice.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I don't have any other cancer or active infections.

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My blood pressure is high (>135/95) despite taking medication.

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I do not have active infections like HIV, Hepatitis B/C, HTLV-1, CMV, or parvovirus B19.

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My lung function is significantly reduced.

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My liver tests are much higher than normal.

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My kidney function tests are not within the normal range.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Evaluation of Safety

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: βAS3-FB vector transduced peripheral blood CD34+ cellsExperimental Treatment1 Intervention

This is a single arm study without randomization. All subjects will receive the intervention of BetaAS3 lentiviral vector-modified autologous peripheral blood stem cell transplant.

0 / 20Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Sickle Cell Disease (SCD) include hydroxyurea, red blood cell transfusions, and investigational gene therapies. Hydroxyurea increases fetal hemoglobin (HbF) production, reducing red blood cell sickling and pain episodes. Red blood cell transfusions increase the number of normal red blood cells, improving oxygen delivery and reducing sickled cells. Gene therapy, such as autologous transplant of lentiviral vector modified peripheral blood, aims to correct the hemoglobin defect at the genetic level, potentially offering a long-term solution by enabling the production of normal hemoglobin. These treatments are crucial for SCD patients as they can reduce complications, improve quality of life, and increase survival rates.

Gene therapy for sickle cell disease.Gene therapy for sickle cell disease.Gene therapy for sickle cell disease.