Clinical trial NCT02698579: Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
Official title: Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Cerebral Adrenoleukodystrophy (CALD)
Adrenoleukodystrophy (ALD)
X-Linked Adrenoleukodystrophy (X-ALD)
Keywords: Adrenoleukodystrophy
X-linked Adrenoleukodystrophy
Hematopoietic Stem Cells
Lead sponsor: bluebird bio
Sponsor class: INDUSTRY
First posted: 2016-03-03
Enrollment: 64
Sex: MALE
Maximum age (years): 19
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in a parent clinical study will be expected to participate in this long-term follow-up study.

Brief summary: This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical study (Study ALD-102 or Study ALD-104).

After completing a parent clinical study (approximately 2 years), eligible participants will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in this study.


Eligibility criteria: Inclusion Criteria:

* Provision of written informed consent for this study by the participant or participant's parent(s)/ legal guardian(s) and written informed assent by participant, if applicable
* Have received eli-cel in a parent clinical study

Exclusion Criteria:

* There are no exclusion criteria for this study

Primary outcomes:
measure: Major functional disability (MFD)-free survival
timeFrame: 15 years post-drug-product infusion
measure: Number of participants with malignancies
timeFrame: 15 years post-drug-product infusion
measure: Number of participants who experience graft versus host disease (GVHD)
timeFrame: 15 years post-drug-product infusion
measure: Number of participants with immune-related adverse events (AEs)
timeFrame: 15 years post-drug-product infusion
measure: Number of participants with new or worsening hematologic disorders
timeFrame: 15 years post-drug-product infusion
measure: Number of participants with new or worsening neurologic disorders
timeFrame: 15 years post-drug-product infusion
Secondary outcomes:
measure: Number of participants who undergo subsequent stem cell transplantation
timeFrame: 15 years post-drug-product infusion
measure: Change from baseline in neurological function score (NFS)
timeFrame: 15 years post-drug-product infusion
measure: Number of participants without gadolinium enhancement (GdE) status on magnetic resonance imaging (MRI)
timeFrame: 15 years post-drug-product infusion
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Mattel Children's Hospital-UCLA
state: California
city: Palo Alto
country: United States
facility: Lucile Packard Children's Hospital - Stanford
state: California
city: Boston
country: United States
facility: Boston Children's Hospital/Massachusetts General Hospital
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Caba
country: Argentina
facility: Instituto Neurogenia
city: North Adelaide
country: Australia
facility: Women's and Children's Hospital
city: São Paulo
country: Brazil
facility: Hospital das Clínicas da Universidade de São Paulo
city: Le Kremlin-Bicêtre
country: France
facility: Hôpital Bicêtre
state: Cedex
city: Leipzig
country: Germany
facility: Universitätsklinikum Leipzig AöR
city: Rome
country: Italy
facility: Ospedale Pediatrico Bambino Gesù
city: Utrecht
country: Netherlands
facility: Prinses Maxima Center
city: London
country: United Kingdom
facility: Royal Free London Hospital
state: England
city: London
country: United Kingdom
facility: Great Ormond Street Hospital
Study officials:
affiliation: bluebird bio, Inc.
name: Vinod K Prasad, MD, FRCP
role: STUDY_DIRECTOR
References: