ClinicalTrials.gg
Not yet recruitingNCT07752043Drepamir-socUpdated Aug 7, 2026

Standard of Care Comparative Arm of Phase 1/2 Gene Therapy Trial DREPAMIR" in Severe Sickle Cell Disease Patients

An interventional study of standard of care in Sickle Cell Disease (SCD) and Vaso-occlusive Events, sponsored by Assistance Publique - Hôpitaux de Paris. Not yet recruiting at 1 site in France. Open to participants aged 12 Years to 35 Years. Per ClinicalTrials.gov, last updated 2026-08-07.

Primary conditionVaso-Occlusive CrisesRegistry: Anemia, Sickle CellReclassifiedWhy we changed it
Registry
Anemia, Sickle Cell — the first condition term the registry derived for this study.
ClinicalTrials.gg
Vaso-Occlusive Crises
Why
  1. The study's listed conditions name Vaso-Occlusive Crises, a narrower term within Anemia, Sickle Cell.
Confidence
High: the new condition is named in the study's own listed conditions. Of 351 hand-checked studies reclassified this way, 68% were exactly right and 84% in the right disease family; the registry's first term was right for 5% (measured 2026-10-21). How we classify →
Wrong?
Tell us the study's main condition →

Sponsored by Assistance Publique - Hôpitaux de Paris · Not applicable, Interventional, and Treatment

Phase
Not applicable
Study type
Interventional
Enrollment
30
Allocation
Not applicable
Ages
12 Years to 35 Years
Sex
All
01

Where this study stands

Counted across the records on this site · 2026-11-12

Among 12 studies in Vaso-Occlusive Crises by Assistance Publique - Hôpitaux de Paris — 5 open to participants now, 5 under way, 1 of them phase 3. Vaso-Occlusive Crises · Assistance Publique - Hôpitaux de Paris.

Planned enrollment
7th of 11
30 · median 40
Started
2026
with 3 others
No phase
7 of 12
1 open now
Registered
11th of 12
by date

Among the 12 studies in Vaso-Occlusive Crises by Assistance Publique - Hôpitaux de Paris, this one is the 7th largest by planned enrollment of the 11 interventional ones, is one of 4 that started in 2026, is one of 7 with no phase, and was the 11th registered.

By phase

12 studies; the count open now beside each. This study's row is marked.

No phasethis study7 · 1 open
Observational1 · 1 open
Phase 1/21 · 1 open
Phase 2/31 · 0 open
Phase 31 · 1 open
Phase 41 · 1 open

By the year they started

First in 2011; this study’s year is marked. 4 started in 2026.

Size and results

Median enrollment 40 across the 11 interventional studies, 200 across the 1 observational. Of the 6 that completed or were terminated, 0 (0%) have results posted.

Was this useful?
Watch this landscapeDownload CSVWith an account: a weekly mail of what changed among 12 studies in Vaso-Occlusive Crises by Assistance Publique - Hôpitaux de Paris, or all of them as a file.
02

Study summary

The purpose of this study is to compare the efficacy and safety of transplantation of gene modified autologous CD34+ cells in SCD patients within a therapeutic strategy that may include anti-inflammatory treatment as a pre-transplant treatment in case of severe inflammation detected at the inclusion analysis; the autologous CD34+ cell will be transduced by the bifunctional βAS3m/miR7m lentiviral vector expressing the therapeutical beta-globin, βAS3m, and the miRNA anti-HbS vs Standard Of Care (SOC).

Read the detailed description

Sickle cell disease is a severe hemoglobinopathy characterized by anemia, chronic hemolysis, and vaso-occlusive crises, leading to high morbidity and mortality.

Medical management options or Standard Of Care (SOC) currently available for SCD include supportive management of VOC, long-term RBC transfusions, and foetal haemoglobin (HbF) induction with hydroxyurea (HU).

Hematopoietic stem cell transplantation is curative but available only for a minority of patients. Gene therapy approaches (gene addition or gene editing) have shown significant reductions in vaso-occlusive events, but residual hemolysis persists and their truly curative potential remains uncertain.

In France, the absence of a control arm in clinical trials has limited regulatory approval, supporting the development of the DREPAMIR protocol, which incorporates a Standard Of Care control group to better assess the efficacy clinical value and cost effectiveness of gene therapy in severe sickle cell disease.

The aim of this study is to compare the efficacy and safety of transplantation of gene modified autologous CD34+ cells in SCD patients within a therapeutic strategy that may include anti-inflammatory treatment as a pre-transplant treatment in case of severe inflammation detected at the inclusion analysis; the autologous CD34+ cell will be transduced by the bifunctional βAS3m/miR7m lentiviral vector expressing the therapeutical beta-globin, βAS3m, and the miRNA anti-HbS vs Standard Of Care (SOC).

Drepamir-SOC is the control arm of the main study DREPAMIR (NCT07432867)

03

Conditions studied

  • Sickle Cell Disease (SCD)
  • Vaso-occlusive Events

Browse trials for

Keywords

  • Sickle cell disease
  • Vaso-occlusive events
04

In context

Vaso-Occlusive Crises Reclassified

104 studies on the registry are indexed under Vaso-Occlusive Crises; 31 are open to participants now.

This study's planned enrollment of 30 is below the median of 81 across 86 interventional studies indexed under Vaso-Occlusive Crises.

Browse Vaso-Occlusive Crises studies →

Lead sponsor

Assistance Publique - Hôpitaux de Paris is the lead sponsor of 3,592 studies on the registry; 1,033 are open to participants now.

Compared within Vaso-Occlusive Crises, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Anemia, Sickle Cell. The counts are of registry records indexed under each term, refreshed daily. How we classify →

05

Who can participate

Ages eligible
12 Years to 35 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • Age 12 - 35 years
  • Diagnosis of HbSS by 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 enrollment 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, were unable to tolerate 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 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

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
  • Patients who have already been treated with gene therapy or BMT
  • Hematologic evaluation: Leukopenia (WBC \<3,000/µL) or neutropenia (ANC \<1,000/µL) or thrombocytopenia ( count \<100,000/µL) within 90 days prior to mobilization or harvest (not due to an erytrapheresis procedure or possible acute viral infection)
  • or PTT >1.5 times the (ULN) or bleeding disorder
  • Two alpha deletions (risk of alpha-thalassemia after gene therapy)

Evaluations within 6 months prior to screening visit:

  • 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 \<60ml/min/1.73 m²
  • Cardiac evaluation: \<40% by cardiac echocardiogram or by MUGA scan or clinically significant 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 O2 saturation during the 6 minutes' walk test.
  • Seropositivity for (Human Immunodeficiency Virus), (Hepatitis C Virus), HTLV-1 (Human T-Lymphotropic Virus), or active 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
  • Any clinically significant active infection
  • Participation in another clinical study with an 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.
Terms in these criteria, in plain language(15)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Hemoglobin (Hb)

The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

INR, PT and aPTT (clotting tests)

Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.

Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

GFR and eGFR (kidney filtering)

GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².

Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.

LVEF (left ventricular ejection fraction)

The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.

ECG (electrocardiogram)

A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Hepatitis C tests

Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.

After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.

Hepatitis B tests

Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.

Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.

Allogeneic and autologous transplant

Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

06

Study design

Phase
Not applicable
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
30 participants (estimated)

Study arms

  • Other
    Standard of care

    Standard of care

    Drug: standard of care

Interventions

  • Drugstandard of care

    Supportive management of VOC, long-term RBC transfusions, and foetal haemoglobin (HbF) induction with hydroxyurea (HU) Myocardic MRI (evaluation of function and fibrosis) + hematocrit measurement QoL questionnaires Imaging (brain MRI, transcranial and cervical ultrasound) Cardiac US, ECG Cardiac and Liver MRI (Fe assessment) Physical ability assessments Neuropsychological assessments Fertility

07

What researchers measure

Primary outcomes

  1. Disease-related mortality

    Time frame: Up to the 24 months follow-up

  2. All-cause mortality

    Time frame: Up to the 24 months follow-up

  3. Occurrence of vaso-occlusive events

    Time frame: between 3 and 15 months following IV infusion of DREAM01 of the corresponding matched DREPAMIR patient

Secondary outcomes

  1. Percentage of HbS

    Measure of HbS

    Time frame: Up to the 24 months follow-up

  2. Percentage of HbF

    Measure of HbF

    Time frame: Up to the 24 months follow-up

  3. Rate of Hemolysis

    Biological parameters that reflect hemolysis : Total hemoglobin, Reticulocytes, lactate dehydrogenase LDH, circulating erythroblasts, haptoglobin, free plasmatic heme, no conjugated bilirubin, erythropoietin EPO

    Time frame: Up to the 24 months follow-up

  4. Rate of Anemia

    Biological parameters that reflect anemia : Total hemoglobin, Reticulocytes, lactate dehydrogenase LDH, circulating erythroblasts, haptoglobin, free plasmatic heme, no conjugated bilirubin, erythropoietin EPO

    Time frame: Up to the 24 months follow-up

  5. Transfusion requirement

    absence of vaso-occlusive events (VOE) in patients who have discontinued the transfusion program or/and hydroxyurea

    Time frame: Up to the 24 months follow-up

  6. Change in number of units of RBCs transfused

    Time frame: Up to the 24 months follow-up

  7. Changes in brain function

    Occurrence of ischemic lesions, vascular stenosis, aneurysm assessed using cervical doppler ultrasound and cerebral MRI

    Time frame: Up to the 24 months follow-up

  8. Changes in brain function

    Occurrence of pathological flow acceleration or vascular stenosis using carotid and transcranial Doppler ultrasound

    Time frame: Up to the 24 months follow-up

  9. Changes in ocular function

    Assessed Using Fundus examination

    Time frame: Up to the 24 months follow-up

  10. Changes in ocular function

    Assessed Using angiography

    Time frame: Up to the 24 months follow-up

  11. Changes in cardiac function

    Evaluated through electrocardiographic (ECG) assessment : overall interpretation with 3 categories such as Normal/ Abnormal, not clinically significant/ Abnormal, clinically significant

    Time frame: Up to the 24 months follow-up

  12. Changes in the occurrence of left ventricular ejection fraction [LVEF] right and left atrial

    Occurrence of left ventricular ejection fraction \[LVEF\], right and left atrial, left assessed using cardiac ultrasound.

    Time frame: Up to the 24 months follow-up

  13. Changes in the occurrence of left ventricular ejection fraction [LVEF] right and left atrial

    Occurrence of left ventricular ejection fraction \[LVEF\], right and left atrial, left assessed using cardiac MRI (including myocardial imaging)

    Time frame: Up to the 24 months follow-up

  14. Changes in the occurrence of left ventricular ejection fraction [LVEF] right and left atrial

    Occurrence of left ventricular ejection fraction \[LVEF\], right and left atrial, left assessed using Doppler echocardiography.

    Time frame: Up to the 24 months follow-up

  15. Changes in the occurrence of left ventricular ejection fraction [LVEF] right and left atrial

    Occurrence of left ventricular ejection fraction \[LVEF\], right and left atrial, left assessed using transthoracic echocardiography

    Time frame: Up to the 24 months follow-up

  16. Changes in left ventricular size

    Changes in left ventricular size assessed using cardiac ultrasound.

    Time frame: Up to the 24 months follow-up

  17. Changes in left ventricular size

    Changes in left ventricular size assessed using cardiac MRI (including myocardial imaging)

    Time frame: Up to the 24 months follow-up

  18. Changes in left ventricular size

    Changes in left ventricular size assessed using Doppler echocardiography

    Time frame: Up to the 24 months follow-up

  19. Changes in left ventricular size

    Changes in left ventricular size assessed using transthoracic echocardiography

    Time frame: Up to the 24 months follow-up

  20. Changes in left ventricular wall thickness

    Changes in the left ventricular wall thickness assessed using cardiac ultrasound.

    Time frame: Up to the 24 months follow-up

  21. Changes in left ventricular wall thickness

    Changes in the left ventricular wall thickness assessed using cardiac MRI (including myocardial imaging)

    Time frame: Up to the 24 months follow-up

  22. Changes in left ventricular wall thickness

    Changes in the left ventricular wall thickness assessed using Doppler echocardiography

    Time frame: Up to the 24 months follow-up

  23. Changes in left ventricular wall thickness

    Changes in the left ventricular wall thickness assessed using transthoracic echocardiography

    Time frame: Up to the 24 months follow-up

  24. Changes in systolic pulmonary artery pressure [sPAP ]

    Changes in systolic pulmonary artery pressure \[sPAP\], assessed using cardiac ultrasound.

    Time frame: Up to the 24 months follow-up

  25. Changes in systolic pulmonary artery pressure [sPAP ]

    Changes in systolic pulmonary artery pressure \[sPAP\], assessed using cardiac MRI (including myocardial imaging)

    Time frame: Up to the 24 months follow-up

  26. Changes in systolic pulmonary artery pressure [sPAP ]

    Changes in systolic pulmonary artery pressure \[sPAP\], assessed using Doppler echocardiography

    Time frame: Up to the 24 months follow-up

  27. Changes in systolic pulmonary artery pressure [sPAP ]

    Changes in systolic pulmonary artery pressure \[sPAP\], assessed using transthoracic echocardiography

    Time frame: Up to the 24 months follow-up

  28. Changes in tricuspid regurgitation velocity [TRV ]

    Changes in tricuspid regurgitation velocity \[TRV\] assessed using cardiac ultrasound

    Time frame: Up to the 24 months follow-up

  29. Changes in tricuspid regurgitation velocity [TRV ]

    Changes in tricuspid regurgitation velocity \[TRV\] assessed using cardiac MRI (including myocardial imaging)

    Time frame: Up to the 24 months follow-up

  30. Title : Changes in tricuspid regurgitation velocity [TRV]

    Changes in tricuspid regurgitation velocity \[TRV\] assessed using Doppler echocardiography

    Time frame: Up to the 24 months follow-up

  31. Title : Changes in tricuspid regurgitation velocity [TRV]

    Changes in tricuspid regurgitation velocity \[TRV\] assessed using transthoracic echocardiography

    Time frame: Up to the 24 months follow-up

  32. Changes in E/A ratio

    Changes in of E/A ratio assessed using cardiac ultrasound, cardiac MRI (including myocardial imaging), Doppler echocardiography and transthoracic echocardiography

    Time frame: Up to the 24 months follow-up

  33. Change in serum electrolyte panel

    Time frame: Up to the 24 months follow-up

  34. Change in serum creatinine

    Time frame: Up to the 24 months follow-up

  35. Change in estimated glomerular filtration rate (eGFR )

    Renal function assessed through estimated glomerular filtration rate (eGFR) calculated using CKD-EPI equation

    Time frame: Up to the 24 months follow-up

  36. Change in urinary microalbumin

    Time frame: Up to the 24 months follow-up

  37. Change in protein excretion

    Time frame: Up to the 24 months follow-up

  38. Change in urinary creatinine

    Time frame: Up to the 24 months follow-up

  39. Changes in creatinine clearence

    Time frame: Up to the 24 months follow-up

  40. Change in liver enzyme AST

    Time frame: Up to the 24 months follow-up

  41. Change in liver enzyme ALT

    Time frame: Up to the 24 months follow-up

  42. Change in liver enzyme GGT

    Time frame: Up to the 24 months follow-up

  43. Changes in in liver enzyme ALP

    Time frame: Up to the 24 months follow-up

  44. Change in total bilirubin

    Time frame: Up to the 24 months follow-up

  45. Change in unconjugated (free) bilirubin

    Time frame: Up to the 24 months follow-up

  46. Changes in hepatic function

    overall interpretation with 3 categories such as Normal/ Abnormal, not clinically significant/ Abnormal, clinically significant

    Time frame: Up to the 24 months follow-up

  47. Change in diffusing capacity for carbon monoxide (DLCO )

    Time frame: Up to the 24 months follow-up

  48. Change in vital capacity (VC )

    Time frame: Up to the 24 months follow-up

  49. Change in residual volume (RV )

    Time frame: Up to the 24 months follow-up

  50. Change in FEV1/FVC ratio (Tiffeneau index )

    Time frame: Up to the 24 months follow-up

  51. Changes in bone metabolism

    Assessed through osteodensitometry

    Time frame: Up to the 24 months follow-up

  52. Changes in muscular function

    Overall interpretation with 3 categories such as Normal/ Abnormal, not clinically significant/ Abnormal, clinically significant

    Time frame: Up to the 24 months follow-up

  53. Occurrence of iron overload

    Time frame: Up to the 24 months follow-up

  54. Fertility evaluation

    Assessed through hormonal testing

    Time frame: Up to the 24 months follow-up

  55. Quality of life Evaluation: patient health

    Medical Outcomes Study Short Form 36 SF-36 The Short Form (36) Health Survey is a 36-item, patient-reported survey of patient health. 0-100 scale so that a higher score indicates a better outcome.

    Time frame: Up to the 24 months follow-up

  56. Quality of life Evaluation : fatigue

    FACIT-Fatigue (Functional Assessment of Chronic Illness Therapy-Fatigue Scale) FACIT-Fatigue is a 13-item self-report scale that assesses fatigue and its effect on daily activities and function. 0-52 scale so that a higher score indicates a better outcome.

    Time frame: Up to the 24 months follow-up

  57. Quality of life Evaluation: physical , mental, and social health

    PROMIS® (Patient-Reported Outcomes Measurement Information System) is a set of person-centered measures that evaluates and monitors physical, mental, and social health in adults and children. PROMIS domain scores are reported as T-scores, with a mean of 50 and a standard deviation of 10 in the reference population. Higher scores indicate better health for physical function and ability to participate in social roles and activities, whereas higher scores indicate worse health for fatigue, pain interference, depressive symptoms, anxiety, and sleep disturbance.

    Time frame: Up to the 24 months follow-up

  58. Health-realted Quality of life Evaluation

    Pediatric Quality of Life Inventory (PedsQL ) Generic Core Scales PedsQL is a modular system that assesses health-related quality of life in healthy and ill children and adolescents. It combines generic core scales and disease-specific modules into one measurement system. 0-100 scale, so that higher scores indicate better outcome.

    Time frame: Up to the 24 months follow-up

  59. Walk ability

    6-minute walk-test

    Time frame: Up to the 24 months follow-up

  60. jump

    Vertical jump test : The jump height will be recorded using video analysis software. The average height of the 3 jumps will be calculated

    Time frame: Up to the 24 months follow-up

  61. Physical ability

    physical ability questionnaire, using the Global physical activity questionnaire (GPAQ) (16 items) developed by WHO. Minimum value is 0 with no theoretical maximum value. Higher scores indicate better outcome.

    Time frame: Up to the 24 months follow-up

  62. Cardiopulmonary capacity

    Cardiopulmonary exercise test, using the Cardio Pulmonary Exercise Test

    Time frame: Up to the 24 months follow-up

  63. Costs

    Costs of treatment calculated yearly and cost per event (VOC and ATS)

    Time frame: Up to the 24 months follow-up

  64. Cost effectiveness

    Model-based incremental cost effectiveness ratio of gene therapy with inputs for SoC

    Time frame: Up to the 24 months follow-up

  65. Changes in cardiac global function

    Assessed through cardiac magnetic resonance imaging, using ventricular function - Overall interpretation with 3 categories such as Normal/ Abnormal, not clinically significant/ Abnormal, clinically significant

    Time frame: Up to the 24 months follow-up

  66. Changes in myocardial tissular

    Using late gadolinium myocardial enhancement

    Time frame: Up to the 24 months follow-up

  67. Changes in myocardial tissular

    Using native T1 mapping

    Time frame: Up to the 24 months follow-up

  68. Changes in myocardial tissular

    Using extracellular volume

    Time frame: Up to the 24 months follow-up

  69. Changes in myocardial tissular

    Using diffusion tensor imaging (DTI)

    Time frame: Up to the 24 months follow-up

08

Study locations

1 site
  • Necker-Enfants Malades Hospital, Apheresis Unit
    Paris, Île-de-France Region 75015, France
09

References and documents

Publications

  • Magrin E, Semeraro M, Hebert N, Joseph L, Magnani A, Chalumeau A, Gabrion A, Roudaut C, Marouene J, Lefrere F, Diana JS, Denis A, Neven B, Funck-Brentano I, Negre O, Renolleau S, Brousse V, Kiger L, Touzot F, Poirot C, Bourget P, El Nemer W, Blanche S, Treluyer JM, Asmal M, Walls C, Beuzard Y, Schmidt M, Hacein-Bey-Abina S, Asnafi V, Guichard I, Poiree M, Monpoux F, Touraine P, Brouzes C, de Montalembert M, Payen E, Six E, Ribeil JA, Miccio A, Bartolucci P, Leboulch P, Cavazzana M. Long-term outcomes of lentiviral gene therapy for the beta-hemoglobinopathies: the HGB-205 trial. Nat Med. 2022 Jan;28(1):81-88. doi: 10.1038/s41591-021-01650-w. Epub 2022 Jan 24. PubMed 35075288 ↗
  • Brusson M, Chalumeau A, Martinucci P, Romano O, Felix T, Poletti V, Scaramuzza S, Ramadier S, Masson C, Ferrari G, Mavilio F, Cavazzana M, Amendola M, Miccio A. Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies. Mol Ther Nucleic Acids. 2023 Mar 22;32:229-246. doi: 10.1016/j.omtn.2023.03.012. eCollection 2023 Jun 13. PubMed 37090420 ↗
  • Sobrino S, Joseph L, Magrin E, Chalumeau A, Hebert N, Corsia A, Denis A, Roudaut C, Aussel C, Leblanc O, Brusson M, Felix T, Diana JS, Petrichenko A, El Etri J, Godard A, Tibi E, Manceau S, Treluyer JM, Mavilio F, Bushman FD, Marcais A, Castelle M, Neven B, Hermine O, Renolleau S, Magnani A, Asnafi V, El Nemer W, Bartolucci P, Six E, Semeraro M, Miccio A, Cavazzana M. Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease. Nat Commun. 2025 Apr 1;16(1):3137. doi: 10.1038/s41467-025-58321-4. PubMed 40169559 ↗
10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Start is estimated for Sep 2026; primary completion for Mar 2030, 42 months later.

How these figures are worked out, and which studies are left out of them.

Registered
Jul 9, 2026
First posted
Aug 7, 2026
StartEst.
Sep 2026
Primary completionEst.
Mar 2030
CompletionEst.
Mar 2030
Last update
Aug 7, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Aug 7, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT07752043
Collaborators
URC-CIC Paris Descartes Necker Cochin, Imagine Institute, Association Française contre les Myopathies (AFM), Paris, Marie Lannelongue Hospital
Responsible party
Sponsor
First posted
Aug 7, 2026
Start date
Sep 2026 (estimated)
Primary completion
Mar 2030 (estimated)
Completion
Mar 2030 (estimated)
Last update
Aug 7, 2026

Study contacts

Joseph LAURE, MD, PhD
Contact
laure.joseph@aphp.fr
01 44 49 55 91 ext. +33
Nelly BRIAND, PHD
Contact
nelly.briand@aphp.fr
01 44 38 18 62 ext. +33
Marina CAVAZZANA, MD, PhD
study director · Department of Biotherapy, Necker-Enfants Malades Hospital

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is not yet recruiting, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.

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