EUCTR2018-001968-33-FR进行中（未招募）1 期
Étude ouverte de phase 1/2 évaluant la sécurité et l’efficacité de l’injection de cellules CD34 + autologues transduites ex vivo par le vecteur lentiviral GLOBE1 qui exprime le gène de la globine ßAS3 chez des patients atteints de drépanocytose. - DREPAGLOBE
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 10
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •iIndividuals eligible to participate in this study must meet all of the following criteria:
- •Age 5 - 35 years
- •Diagnosis of HbSS by Hb electrophoresis or genetic analysis
- •Clinical history or ongoing evidence of severe sickle cell anemia with one OR more of the following clinical complications demonstrating disease severity:
- •1.At least 3 vaso occlusive crises requiring hospitalization, under hydroxyurea or transfusion, within 2 years prior to enrollment
- •2.One severe acute chest syndrome (ACS) hospitalized in intensive care unit
- •3.At least 2 episodes of ACS within the prior 3 years), including one under HU.
- •4.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).
- •5.Cerebral vasculopathy confirmed by MRA (magnetic resonance angiography) without Moya-moya
- •6.Presence of sickle cell cardiomyopathy documented by Doppler echocardiography (left ventricular ejection fraction (LVEF) <55% AND tricuspid regurgitation velocity >2.5m/s on cardiac echocardiograph),
- •7.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, if 18 years of age or older, have actively made the choice to not take the recommended daily HU regimen. 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 crises requiring hospitalization, no rise in Hb >1.5 g/dl from pre-HU baseline or requires transfusion to maintain Hb > 6.0 g/dL, Has 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 of a social security regime (or exemption),
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 6
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 4
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Individuals who meet any of the following exclusion criteria will not be eligible to participate in the study:Existence of a matched sibling donor Patients who have started new treatment for SCD within 6months of enrollment
- •Hematologic evaluation: Leukopenia (WBC < 3000 uL) or neutropenia (ANC < 1000 uL) or thrombocytopenia (platelet count < 100,000 uL) within 90 days prior to mobilization or harvest ( not due to an erythrapheresis procedure)
- •PT/INR or PTT > 1.5 times upper limit of normal (ULN) or clinically significant bleeding disorder
- •Evaluations within 6 months prior to screening visit:ALT or AST > 1.5 times ULN
- •Liver Cirrhosis suspicion on echography, CT scan or MRI AND confirmed by histology Liver iron > 125 µmol/g by MRIMeasured GFR < 60 ml/min/1.73 m 2 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;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>25mmHg). 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 impeded the ability to participate in the study;Patients who failed previous HSCT and are severely ill ;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;Each patient will receive a single intravenous infusion of the DREPAGLOBE drug product at dose range of 3 to 20 million CD34+ cells/kg post-transduction.Source of CD34+ will be :
- •1)Bone- Marrow 2)CD34 + mobilized with Plerixafor after a single or multiple harvests3)The DREPAGLOBE drug product consists in autologous CD34+ hematopoietic stem cells transduced with Self-inactivating lentiviral vector (GLOBE1 AS3) encoding the human ?AS3-globin gene and suspended in HSA (5% Albunorm™) in the final immediate container for the intended medical use. All subjects are to receive the DREPAGLOBE drug product on Day 1 via IV infusion according to applicable SOPs, with vital signs being monitored concurrently. The minimum dose to be administered is 3.0×106 CD34+ cells/kg.Exchange transfusion before the mobilization procedure ;Mobilization by Plerixaf
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