Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2015-002805-13-GB

Enrollment

Registered

2015-09-09

Start date

2015-11-27

Completion date

Unknown

Last updated

2020-10-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0 Level: PT Classification code 10051260 Term: Adrenoleukodystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Pharmaceutical Form:

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: 1.Provision of written informed consent for this study by the subject or subject’s parent(s)/ legal guardian(s) and written informed assent by subject, if applicable 2.Have received eli - cel Product in a parent clinical study. 3.Able to comply with study requirements. Are the trial subjects under 18? yes Number of subjects for this age range: 60 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: There are no exclusion criteria for this Study.

Design outcomes

Primary

Measure	Time frame
Main Objective: - Monitor for long-term safety of the Lenti-D Drug Product (also known as elivaldogene autotemcel; hereafter referred to as eli-cel)administered in parent clinical studies. - Monitor for long-term efficacy of eli-cel administered in parent clinical studies.;Secondary Objective: Not Applicable;Primary end point(s): 1. Safety evaluations include: •Proportion of subjects who experience graft versus host disease (GVHD) •Proportion of subjects who undergo subsequent stem cell transplantation (i.e. second HSC infusion) •All drug product-related AEs through 15 years post-drug-product infusion •All serious adverse events (SAEs) through 15 years post-drug product infusion (regardless of relatedness to drug product) • Immune-related AEs through 15 years post-drug product infusion • Incidence of vector-derived RCL, assessed from archived samples as clinically indicated. •The number of subjects with insertional oncogenesis (myelodysplasia, leukemia, lymphoma, etc.) •The number of subjects with clonal predominance 2. Efficacy Endpoints include: •MFD-free survival;Timepoint(s) of evaluation of this end point: Visits are scheduled every 6 months for years 2 to 5 post-drug-product infusion and then annually through 15 years post-drug-product infusion.	—

Measure

Time frame

Main Objective: - Monitor for long-term safety of the Lenti-D Drug Product (also known as elivaldogene autotemcel; hereafter referred to as eli-cel)administered in parent clinical studies. - Monitor for long-term efficacy of eli-cel administered in parent clinical studies.;Secondary Objective: Not Applicable;Primary end point(s): 1. Safety evaluations include: •Proportion of subjects who experience graft versus host disease (GVHD) •Proportion of subjects who undergo subsequent stem cell transplantation (i.e. second HSC infusion) •All drug product-related AEs through 15 years post-drug-product infusion •All serious adverse events (SAEs) through 15 years post-drug product infusion (regardless of relatedness to drug product) • Immune-related AEs through 15 years post-drug product infusion • Incidence of vector-derived RCL, assessed from archived samples as clinically indicated. •The number of subjects with insertional oncogenesis (myelodysplasia, leukemia, lymphoma, etc.) •The number of subjects with clonal predominance 2. Efficacy Endpoints include: •MFD-free survival;Timepoint(s) of evaluation of this end point: Visits are scheduled every 6 months for years 2 to 5 post-drug-product infusion and then annually through 15 years post-drug-product infusion.

—

Secondary

Measure	Time frame
Secondary end point(s): Secondary efficacy endpoints include the following: - Overall survival. - Change from Baseline (defined in parent study) in NFS. - Gadolinium enhancement (GdE) status ;Timepoint(s) of evaluation of this end point: As above	—

Countries

Algeria, Argentina, Australia, Brazil, France, United Kingdom, United States

Contacts

Public ContactClinical Trial Information

bluebird bio, Inc.

clinicaltrials@bluebirdbio.com001 339 4999300

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026