Overview
Sponsor-declared trial summary
Sickle-cell disease
- Evaluate long-term safety of treatment with lovocel (lovotibeglogene autotemcel, also known as bb1111 or LentiGlobin BB305 Drug Product for Sickle Cell Disease) in subjects with sickle cell disease (SCD) - Evaluate long-term efficacy of treatment with bb1111 in subjects with SCD
Key facts
- Sponsor
- Genetix Biotherapeutics Inc.
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Trial duration
- 3 Nov 2020 → ongoing
- Decision date (initial)
- 2024-06-25
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- bluebird bio, Inc.
External identifiers
- EU CT number
- 2024-513901-30-00
- EudraCT number
- 2019-004266-18
- ClinicalTrials.gov
- NCT04628585
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Safety, Pharmacodynamic
- Evaluate long-term safety of treatment with lovocel (lovotibeglogene autotemcel, also known as bb1111 or LentiGlobin BB305 Drug Product for Sickle Cell Disease) in subjects with sickle cell disease (SCD)
- Evaluate long-term efficacy of treatment with bb1111 in subjects with SCD
Conditions and MedDRA coding
Sickle-cell disease
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.0 | PT | 10040644 | Sickle cell disease | 100000004850 |
Regulatory references
- Plan to share IPD
- Yes
| EU CT number | Title | Sponsor |
|---|---|---|
| 2012-000695-42 | A Phase I/II Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the β-Hemoglobinopathies (Sickle Cell Anemia and β-Thalassemia Major) by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with a Lentiviral beta-A-T87Q-Globin Vector (LentiGlobin® BB305 Drug Product) |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/ legal guardian(s)
- Treated with drug product for therapy of SCD in a bluebird bio-sponsored clinical study
Exclusion criteria 1
- There are no exclusion criteria for this study.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 4
- The number of subjects with immune-related AEs (e.g., autoimmune disorders, graft-versus-host disease, opportunistic infections, HIV)
- The number of subjects with new or worsening hematologic disorders
- The number of subjects with new or worsening neurologic disorders
- The number of subjects with malignancies
Secondary endpoints 8
- Vaso-Occlusive Events Endpoints: The proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15
- The proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15
- Annualized number of severe VOEs over time through Year 15
- Annualized number of VOEs over time through Year 15
- Change from parent study baseline in annualized number of severe VOEs over time through Year 15
- Hematologic Endpoints: Assessment of the following over time post-drug product infusion through Year 15: − total Hb − non-transfused total Hb − HbS percentage of non-transfused total Hb − HbAT87Q percentage of non-transfused total Hb − non-HbS percentage of non-transfused total Hb
- Change from parent study baseline through Year 15 in the following hemolysis markers: absolute reticulocyte count, % reticulocytes/erythrocytes, total bilirubin, indirect bilirubin, haptoglobin, and lactate dehydrogenase
- Change from parent study baseline through Year 15 in the following markers of iron stores: serum ferritin and liver iron content
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
LentiGlobin BB305 Drug Product for Sickle Cell Disease
PRD11215938 · Product
- Active substance
- Lovotibeglogene Autotemcel
- Pharmaceutical form
- DISPERSION FOR INFUSION
- Route of administration
- INTRAVENOUS
- Max daily dose
- 0 Other
- Max total dose
- 0 Other
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Not Authorised
- MA holder
- BLUEBIRD BIO INC.
- Paediatric formulation
- No
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Genetix Biotherapeutics Inc.
- Sponsor organisation
- Genetix Biotherapeutics Inc.
- Address
- 455 Grand Union Boulevard
- City
- Somerville
- Postcode
- 02145-1446
- Country
- United States
Scientific contact point
- Organisation
- Bluebird Bio Inc.
- Contact name
- Clinical Trial Information
Public contact point
- Organisation
- Bluebird Bio Inc.
- Contact name
- Clinical Trial Information
Third parties 9
| Organisation | City, country | Duties |
|---|---|---|
| Unisphere Travel Ltd. Inc. ORG-100043100
|
Stamford, United States | Other |
| CTI Clinical Trial and Consulting Services Europe GmbH ORG-100008276
|
Ulm, Germany | Other |
| Icon Clinical Research Limited ORG-100008322
|
Dublin 18, Ireland | Laboratory analysis |
| ProtaGene CGT GmbH ORG-100041450
|
Heidelberg, Germany | Other |
| Icon Laboratory Services Inc. ORG-100037135
|
Farmingdale, United States | Laboratory analysis |
| Icon Development Solutions LLC ORG-100012400
|
Whitesboro, United States | Laboratory analysis |
| Primevigilance Limited ORG-100027742
|
Guildford, United Kingdom | Code 8 |
| Edetek Inc. ORG-100045957
|
Princeton, United States | Code 10 |
| Voisin Consulting CH SARL ORG-100031396
|
Lausanne, Switzerland | Code 12 |
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ongoing, recruiting | 3 | 1 |
| Rest of world
United States
|
— | 85 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| France | 2020-11-03 | 2020-11-03 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 1 file
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_2024-513901-30-00_For publication | 4.0 |
Application history
3 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-05-14 | France | Acceptable 2024-06-10
|
2024-06-25 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2025-12-18 | France | Acceptable 2024-06-10
|
2025-12-18 |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2026-02-12 | France | Acceptable 2024-06-10
|
2026-02-12 |