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Tests treatment safety and results for Sickle Cell Disease
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.
ClinicalTrials.gov ID: NCT07432867
What this study is testing
What is DREAM01 drug product?
DREAM01 drug product is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for sickle cell disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 35
You may be able to join if
- Age 12 - 35 years
- Acceptation of myelogram (bone marrow aspiration)
- Diagnosis of HbSS by Hb electrophoresis and genetic analysis to analyse the alpha locus
- Clinical history or ongoing evidence of severe sickle cell anemia with one OR more of the following clinical complications demonstrating disease...
- At least 3 vaso-occlusive crises requiring hospitalization, under hydroxyurea or transfusion, within 2 years prior to enrolment
You likely can't join if
- Existence of a matched sibling donor
- Based on myelogram, the presence of chromosomal (detected by karyotyping) or molecular abnormalities (detected by NGS) and retained dangerous by the...
- Hematologic evaluation: Leukopenia (WBC \<3,000/µL) or neutropenia (ANC \<1,000/µL) or thrombocytopenia (platelet count \<100,000/µL) within 90 days...
- PT/INR or PTT \>1.5 times the upper limit of normal (ULN) or clinically significant bleeding disorder
- Two alpha deletions (risk of alpha-thalassemia after gene therapy)
- Hypersensitivity to the active substances of the administered drugs (plerixafor, busulfan, anti-inflammatory therapy) or to any of their excipients
See the full eligibility criteria
- Age 12 - 35 years
- Acceptation of myelogram (bone marrow aspiration)
- Diagnosis of HbSS by Hb electrophoresis and genetic analysis to analyse the alpha locus
- Clinical history or ongoing evidence of severe sickle cell anemia with one OR more of the following clinical complications demonstrating disease severity:
- At least 3 vaso-occlusive crises requiring hospitalization, under hydroxyurea or transfusion, within 2 years prior to enrolment
- One severe acute chest syndrome (ACS) hospitalized in the intensive care unit
- At least 2 episodes of ACS, including one under HU.
- Acute priapism (at least 2 episodes \>3h in the preceding year or in the year prior to the start of a regular transfusion program), OR stuttering priapism ≥ 1 by week under sickle cell treatment (HU, transfusion or...
- Tricuspid regurgitation velocity \>2.8m/s on cardiac echocardiograph without pulmonary hypertension confirmed by right heart catheterization (mPAP\>\<25mmHg)
- Failed hydroxyurea (HU) therapy, OR Inadequate clinical response to HU, defined as any one of the following outcomes, while on HU for at least 3 months: 2 or more acute sickle pain crisis requiring hospitalization...
- Karnovsky/Lansky performance score ≥ 60%
- Sexually active patients must be willing to use an acceptable method of double-barrier contraception for at least 12 months post-infusion (beyond 12 months at the discretion of the investigator)
- Procedure for obtaining consent (adults, dependent minors, to give their consent)
- Affiliation to social security
- Existence of a matched sibling donor
- Based on myelogram, the presence of chromosomal (detected by karyotyping) or molecular abnormalities (detected by NGS) and retained dangerous by the Hemato-Oncology referent and validated during a specific...
- Hematologic evaluation: Leukopenia (WBC \<3,000/µL) or neutropenia (ANC \<1,000/µL) or thrombocytopenia (platelet count \<100,000/µL) within 90 days prior to mobilization or harvest (not due to an erythrapheresis...
- PT/INR or PTT \>1.5 times the upper limit of normal (ULN) or clinically significant bleeding disorder
- Two alpha deletions (risk of alpha-thalassemia after gene therapy)
- Hypersensitivity to the active substances of the administered drugs (plerixafor, busulfan, anti-inflammatory therapy) or to any of their excipients
- Patients who have already been treated with gene therapy Evaluations within 6 months prior to screening visit:
- ALT or AST \>3 times ULN
- Severe liver iron overload evaluated by MRI (\>15mg Fe/g dry weight or \>270umol Fe/g dry weight) or liver cirrhosis suspicion on echography or elastometry or CT scan or MRI AND confirmed by histology
- Measured GFR \<60ml/min/1.73 m²
- Cardiac evaluation: LVEF \<40% by cardiac echocardiogram or by MUGA scan or clinically significant ECG abnormalities
- Stroke with significant CNS sequelae i.e., Rankin \>2
- Specific sickle cell disease cerebral vasculopathy confirmed by MRA (magnetic resonance angiography) OR transcranial doppler ultrasound with or without Moya-moya WITH an indication of chronic transfusion program (target...
- Lung interstitial infiltrate AND Forced Vital Capacity less than 70% AND DLCO less than 60% at steady state
- Confirmed pulmonary hypertension defined by a right heart catheterization (PAPm \>25 mmHg). Right heart catheterization is required if tricuspid regurgitation velocity \>2.8m/s on cardiac echocardiograph OR \>2.5m/s...
- Seropositivity for HIV (Human Immunodeficiency Virus), HCV (Hepatitis C Virus), HTLV-1 (Human T-Lymphotropic Virus), or active Hepatitis B Virus, or active infection by CMV or parvovirus B19, based on positive blood PCR.
- Pregnancy or breastfeeding in a postpartum female
- Any current cancer or prior history of a malignant disease, with the exception of curatively treated non-melanoma skin cancer
- Immediate family member with an established or suspected Familial Cancer Syndrome
- Diagnosis of significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study
- Patients who failed previous HSCT
- Any clinically significant active infection
- Participation in another clinical study with an investigational drug within 30 days of screening
- Any condition, based on perspective of the medical monitor and treating investigator, which may lead to increased safety risk or inability to comply with the protocol
The study team makes the final eligibility decision.
Where it's taking place
- Paris, Île-de-France Region, France
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years to 35 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Paris, Île-de-France Region, France. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.