A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Genetix Biotherapeutics Inc.
Study ID
NCT02140554
Phase
PHASE1/PHASE2
Status
Completed

Conditions

Eligibility Criteria

Sex
ALL
Age
12 Years - 50 Years
Healthy Volunteers
Not accepted

Interventions

  • lovo-cel — GENETIC
    lovo-cel is administered by IV infusion following myeloablative conditioning with busulfan.

Study Details

This is a non-randomized, open label, multi-site, single dose, Phase 1/2 study in approximately 50 adults and adolescents with severe SCD. The study will evaluate hematopoietic stem cell and progenitor stem cell (collectively referred to as hematopoietic stem and progenitor cells or HSPCs) transplantation using lovo-cel.

Key Dates

Start date
Feb 2, 2015
Status verified
Mar 2025
Primary completion
Jul 31, 2023
Completion
Jan 30, 2024

Study Design

Enrollment
54 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Group A
    Participants who had rescue cells that were collected by bone marrow harvest method and had received treatment with lovo-cel which consists of autologous CD34+ hematopoietic stem cells (HSCs) and progenitor stem cells (PSCs) (collectively referred to as hematopoietic stem and progenitor cells or HSPCs) collected from participants with sickle cell disease (SCD) by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene (the original drug product manufacturing process for this study).
  • Experimental: Group B
    Group B1 participant had rescue cells and drug product cells that were collected by bone marrow harvest method and drug product was manufactured with autologous CD34+ HSPCs collected by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene. This participant's drug product was produced in 2 lots each using two different manufacturing processes (the original drug product manufacturing process and a refined drug product manufacturing process). Group B2 Plerixafor mobilization and apheresis were used for collection of rescue cells and exploratory manufacturing development. A single Group B2 participant received treatment of lovo-cel manufactured with autologous CD34+ HSPCs collected by bone marrow harvest transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene (using only the refined drug product manufacturing process). Note: Groups B1 and B2 are combined as "Group B" for results reporting purposes.
  • Experimental: Group C
    Plerixafor mobilization and apheresis were used for collection of rescue cells, and drug product. Participants received treatment of lovo-cel manufactured with autologous CD34+ HSPCs collected by plerixafor mobilization and apheresis transduced with BB305 lentiviral vector encoding the human beta-A-T87Q globin gene using a further refined manufacturing process similar to commercial manufacturing.

Primary Outcome Measure

Percentage of Group C Participants Who Achieved Complete Resolution of Vaso-occlusive Events (VOE-CR) [ Time Frame: From 6 months to 18 months post lovo-cel infusion ]

Locations (11)

FacilityCityStateZIPSite coordinators
-BirminghamAlabama--
-OaklandCalifornia--
-AtlantaGeorgia30322-
-ChicagoIllinois--
-BethesdaMaryland--
-HackensackNew Jersey--
-New Hyde ParkNew York--
-New YorkNew York--
-Chapel HillNorth Carolina--
-PhiladelphiaPennsylvania--
-CharlestonSouth Carolina--

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By condition
Sickle cell disease

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