Actively Recruiting
Efficacy Safety Study of Gene Therapy for Sickle Cell DiseaseSCD Using Autologous CD34+ Cells Transduced ex Vivo, Carrying a Corrected Globin Gene and a Silencing RNA.
Led by Assistance Publique - Hôpitaux de Paris · Updated on 2026-03-04
15
Participants Needed
1
Research Sites
361 weeks
Total Duration
On this page
Sponsors
A
Assistance Publique - Hôpitaux de Paris
Lead Sponsor
I
Imagine Institute
Collaborating Sponsor
AI-Summary
What this Trial Is About
The purpose of this study is to evaluate the Safety and Efficacy of DREAM01, a gene therapy for Sickle Cell Disease (SCD). The therapy consists of transplanting autologous CD34+ cells transduced ex vivo with a bifunctional lentiviral vector expressing βAS3m-globin and an anti-βS miRNA. It aims to reduce or eliminate vaso-occlusive events and long-term organ damage in severe SCD patients lacking a Human Leukocyte Antigen (HLA) identical sibling donor.
CONDITIONS
Official Title
Efficacy Safety Study of Gene Therapy for Sickle Cell DiseaseSCD Using Autologous CD34+ Cells Transduced ex Vivo, Carrying a Corrected Globin Gene and a Silencing RNA.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 12 and 35 years
- Willingness to undergo bone marrow aspiration (myelogram)
- Confirmed diagnosis of HbSS by hemoglobin electrophoresis and genetic analysis
- History or current evidence of severe sickle cell anemia with at least one of the following: three or more vaso-occlusive crises requiring hospitalization in past 2 years, one severe acute chest syndrome in ICU, two or more episodes of acute chest syndrome including one under hydroxyurea treatment, or specific priapism conditions
- Failed or inadequate response to hydroxyurea therapy despite at least 3 months of treatment
- Karnovsky/Lansky performance score of 60% or higher
- Sexually active patients must agree to use double-barrier contraception for at least 12 months post-infusion
- Ability to provide informed consent and social security affiliation
You will not qualify if you...
- Presence of a matched sibling donor
- Chromosomal or molecular abnormalities identified by bone marrow analysis deemed dangerous by specialists
- Leukopenia (WBC <3,000/μL), neutropenia (ANC <1,000/μL), or thrombocytopenia (platelets <100,000/μL) within 90 days before stem cell collection
- Abnormal clotting tests or bleeding disorders
- Two alpha globin gene deletions (risk of alpha-thalassemia after gene therapy)
- Allergies to study drugs or their components
- Prior gene therapy treatment
- Elevated liver enzymes (ALT or AST >3 times normal)
- Severe liver iron overload or signs of liver cirrhosis
- Reduced kidney function (GFR <60 ml/min/1.73 m²)
- Significant heart abnormalities or reduced ejection fraction (<40%)
- Stroke with major neurological deficits
- Certain cerebral blood vessel diseases requiring chronic transfusion
- Lung disease with reduced function
- Confirmed pulmonary hypertension
- Active infections with HIV, hepatitis B or C, HTLV-1, CMV, or parvovirus B19
- Pregnancy or breastfeeding
- Current or past cancer except treated non-melanoma skin cancer
- Family history of familial cancer syndrome
- Significant psychiatric disorders affecting participation
- Failed previous hematopoietic stem cell transplant
- Active infection
- Participation in another investigational drug study within 30 days
- Any condition increasing safety risk or preventing protocol compliance
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Department of Biotherapy, Necker-Enfants Malades Hospital
Paris, Île-de-France Region, France, 75015
Actively Recruiting
Research Team
M
Marina CAVAZZANA, MD, PhD
CONTACT
N
Nelly BRIAND, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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