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

Phase 3

Recruiting

Conditions: CALD
cerebral adrenoleukodystrophy
10029299

Registration Number: NL-OMON54368

Lead Sponsor: bluebird bio, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 3

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 in a parent clinical study
3.Able to comply with study requirements

Exclusion Criteria

There are no exclusion criteria for this Study.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>1. Safety Endpoints include:<br /><br>- Proportion of subjects who experience graft versus host disease (GVHD)<br /><br>- Proportion of subjects who undergo subsequent stem cell transplantation (i.e.<br /><br>second HSC infusion)<br /><br>- All drug product-related AEs through 15 years post-drug product infusion<br /><br>- All serious adverse events (SAEs) through 15 years post-drug product infusion<br /><br>(regardless of relatedness to drug product)<br /><br>- Immune-related AEs and new or worsening hematologic or neurologic disorders<br /><br>or malignancies through 15 years post-drug product infusion<br /><br>- Incidence of vector-derived RCL, assessed from archived samples as clinically<br /><br>indicated.<br /><br>- The number of subjects with insertional oncogenesis (myelodysplasia,<br /><br>leukemia, lymphoma, etc.)<br /><br>- The number of subjects with persistent oligoclonality<br /><br><br /><br>2. Efficacy Endpoints include:<br /><br>-MFD-free survival</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Secondary efficacy endpoints include the following:<br /><br>- Overall survival.<br /><br>- Change from Baseline (defined in parent study) in NFS.<br /><br>- Gadolinium enhancement (GdE) status.</p><br>