Ireland
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Recruiting Phase 1/2

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.

NCT07432867 · tracked via the Priya Life Science France tracker
Phase
Phase 1/2
Started
2026-02-25
Last updated
2026-03-04

Condition(s) studied

Sickle Cell Disease

Investigational drug(s) / intervention(s)

DREAM01 drug productanti-inflammatory therapy

DREAM01 drug product: Each patient will receive a single IV infusion of DREAM01, autologous CD34+ stem cells transduced with βAS3m/miR7m lentiviral vector

anti-inflammatory therapy: Patient will receive anti-inflammatory therapy if necessary

Study summary

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.

Eligibility

Sex
ALL
Min age
12 Years
Max age
35 Years
Healthy volunteers
No
Inclusion 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 phlebotomy). * 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, requirement of transfusion to maintain Hb \>6.0g/dL, an episode of ACS despite adequate supportive care measures * 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 Exclusion Criteria: * 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 multidisciplinary concerted meeting * 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 procedure or possible acute viral infection) * 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 HbS\<30%) * 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 with an abnormal Brain Natriuretic Peptide dosage or an important decrease in transcutaneous Hb O2 saturation during the 6 minutes' walk test. * 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

Primary outcome measure(s)

  • Neutrophil recovery — within the 24 months following IV infusion of DREAM01
    Neutrophil recovery defined as the first of three consecutive days with an ANC of \> 500/µL
  • Platelet recovery — within the 24 months following IV infusion of DREAM01
    Platelet recovery defined as the first of three consecutive days with a platelet count of \> 20.000/µL sustained without platelet transfusion for at least seven days
  • In vivo engraftment (neutrophils and platelets) — every 3 months between 3 to 24 months following IV infusion of DREAM01
    hematopoietic reconstitution after IV infusion of the drug product
  • Adverse event — within the 24 months following IV infusion of DREAM01
    Adverse event will be measured using CTCAE
  • Transplant-related mortality (TRM) — within 100 days following IV infusion of DREAM01
    Transplant-related mortality
  • Transplant-related mortality (TRM) — within the first year following IV infusion of DREAM01
    Transplant-related mortality
  • All-cause mortality — Up to the 24 months following IV infusion of DREAM01
    Mortality
  • Efficacy of DREAM01 — between 3 and 15 months following IV infusion of DREAM01
    absence of vaso-occlusive events (VOE) in patients who have discontinued the transfusion program or/and hydroxyurea
  • Efficacy of anti-inflammatory therapy — within the 3 months following administration of anti-inflammatory therapy
    Decrease of HSPCs inflammation assessed through a reduction of the score and/or the number of inflammatory pathways (among the 6 pathways established) by transcriptomic analysis on HSPCs between inclusion and after 3 months of anti-inflammatory therapy treatment before infusion

Trial sites (1)

FacilityCityRegionStatus
Department of Biotherapy, Necker-Enfants Malades Hospital Paris Île-de-France Region Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07432867 on ClinicalTrials.gov ↗ ← All trials in France