Cerebral Adrenoleukodystrophy (CALD)NCT02698579
Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
An observational study of Cerebral Adrenoleukodystrophy (CALD) and Adrenoleukodystrophy (ALD), run by Genetix Biotherapeutics Inc. at 13 sites in 9 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages Up to 19 years, men only
- Conditions the study is for
- Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
- Type of treatment
- Injection or infusion
- How many people
- 64 participants
- Where
- 13 sitesin 9 countries
- Study dates
- 22 Jan 2016to Aug 2038
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 19 years
- Men only
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
- 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
Who cannot join
- There are no exclusion criteria for this study
Trial locations and contacts
13 sites in 9 countries.
13 sites in 9 countries
United States4 sites
Boston Children's Hospital/Massachusetts General Hospital
Boston, Massachusetts, 02115
Mattel Children's Hospital-UCLA
Los Angeles, California, 90095
University of Minnesota
Minneapolis, Minnesota, 55455
Lucile Packard Children's Hospital - Stanford
Palo Alto, California, 94304
United Kingdom2 sites
Royal Free London Hospital
London, England, NW3 2QG
Great Ormond Street Hospital
London, England
Brazil1 site
Hospital das Clínicas da Universidade de São Paulo
São Paulo, 05403-000
About this study
Official title: Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
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.
- Conditions
- Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
- Being tested
- No interventional drug product utilized in this follow-up study
- Keywords
- Adrenoleukodystrophy, X-linked Adrenoleukodystrophy, Hematopoietic Stem Cells
- Medical subject headings
- Adrenoleukodystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventional drug product utilized in this follow-up studyGenetic
Participants received a single IV infusion of Lenti-D Drug Product (also known as elivaldogene autotemcel or eli-cel) in either parent Study ALD-102 or ALD-104.
The objectives of this long-term follow-up study are to assess long-term safety and efficacy following completion of participation in parent studies. Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant are conducted in this study.
| Study group | Role | What they receive |
|---|---|---|
| Long-term followup | Participants who have received Lenti-D Drug Product in a parent clinical study (bluebird bio-sponsored clinical studies ALD-102 and ALD-104) and who meet the eligibility criteria for the Study LTF-304 will be followed in this long-term followup study for 13 years (for a total of 15 years of follow-up after drug product infusion in the parent studies). Receives: Genetic: No interventional drug product utilized in this follow-up study |
What is being measured
The primary outcome is the main question the study is designed to answer.
Major functional disability (MFD)-free survival
The MFDs are loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, complete loss of voluntary movement.
Measured: 15 years post-drug-product infusion
Number of participants with malignancies
Measured: 15 years post-drug-product infusion
Number of participants who experience graft versus host disease (GVHD)
Measured: 15 years post-drug-product infusion
Number of participants with immune-related adverse events (AEs)
Measured: 15 years post-drug-product infusion
Number of participants with new or worsening hematologic disorders
Measured: 15 years post-drug-product infusion
Number of participants with new or worsening neurologic disorders
Measured: 15 years post-drug-product infusion
Secondary outcomes (3)
Number of participants who undergo subsequent stem cell transplantation
Measured: 15 years post-drug-product infusion
Change from baseline in neurological function score (NFS)
The NFS is a 25-point score used to evaluate the severity of gross neurologic dysfunction in CALD by scoring 15 symptoms (functional domains) across 6 categories. Listed here are the 15 symptoms followed by their maximal score out of 25 points: a) Hearing / auditory processing problems-1, b) Aphasia / apraxia-1, c) Loss of communication-3, d) Vision impairment /field cut-1, e) Cortical blindness-2, f) Swallowing / other central nervous system (CNS) dysfunctions-2, g) Tube feeding-2, h) Running difficulties / hyperreflexia-1, i) Walking difficulties / spasticity / spastic gait (no assistance)-1, j) Spastic gait (needs assistance)-2, k) Wheelchair dependence-2, l) Complete loss of voluntary movement-3, m) Episodes of incontinence -1, n) Total incontinence-2, o) Nonfebrile seizures-1. A score of "0" denotes absence of clinical signs of cerebral disease. Maximal signs within a domain score the total of all grades within that domain.
Measured: 15 years post-drug-product infusion
Number of participants without gadolinium enhancement (GdE) status on magnetic resonance imaging (MRI)
Contrast enhancement (gadolinium enhancement; GdE+) on brain MRI represents a clinically important radiographic biomarker of active neuroinflammatory disease and poor prognosis (in untreated patients). As such, assessment of the number of participants who remained negative for gadolinium enhancement (GdE-) was conducted for this outcome measure.
Measured: 15 years post-drug-product infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2016 (estimated)
- Study start22 Jan 2016
- Primary completionAug 2038 (estimated)
- Study completionAug 2038 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Cerebral Adrenoleukodystrophy (CALD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Vinod K Prasad, MD, FRCP, Study director, Genetix Biotherapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02698579
- Sponsor's protocol ID
- LTF-304
- Other IDs
- 2024-513904-33-00, 2015-002805-13, 2015
- Status last verified
- Sep 2026
- First submitted
- 17 Feb 2016
- Last updated
- 14 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: bluebird bio is committed to transparency. Appropriately de-identified patient-level datasets and supporting documents may be shared following attainment of applicable marketing approvals associated with this study and consistent with criteria established by bluebird bio and/or industry best practices to maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.
Publications
Eichler F, Duncan CN, Musolino PL, Lund TC, Gupta AO, De Oliveira S, Thrasher AJ, Aubourg P, Kuhl JS, Loes DJ, Amartino H, Smith N, Folloni Fernandes J, Sevin C, Sankar R, Hussain SA, Gissen P, Dalle JH, Platzbecker U, Downey GF, McNeil E, Demopoulos L, Dietz AC, Thakar HL, Orchard PJ, Williams DA. Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2024 Oct 10;391(14):1302-1312. doi: 10.1056/NEJMoa2400442.
PubMed 39383459Duncan CN, Bledsoe JR, Grzywacz B, Beckman A, Bonner M, Eichler FS, Kuhl JS, Harris MH, Slauson S, Colvin RA, Prasad VK, Downey GF, Pierciey FJ, Kinney MA, Foos M, Lodaya A, Floro N, Parsons G, Dietz AC, Gupta AO, Orchard PJ, Thakar HL, Williams DA. Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2024 Oct 10;391(14):1287-1301. doi: 10.1056/NEJMoa2405541.
PubMed 39383458
Study at a glance
From the registry record, last updated by the sponsor on 14 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
- ClinicalTrials.gov ID
- NCT02698579
- Acronym / sponsor ID
- LTF-304
- Conditions
- Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
- Keywords
- Adrenoleukodystrophy, X-linked Adrenoleukodystrophy, Hematopoietic Stem Cells
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 64 participants (actual)
- Lead sponsor
- Genetix Biotherapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 22 Jan 2016 (actual)
- Primary completion
- Aug 2038 (estimated)
- Study completion
- Aug 2038 (estimated)
- First posted
- 3 Mar 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Long-term followup | – | Genetic: No interventional drug product utilized in this follow-up studyParticipants who have received Lenti-D Drug Product in a parent clinical study (bluebird bio-sponsored clinical studies ALD-102 and ALD-104) and who meet the eligibility criteria for the Study LTF-304 will be followed in this long-term followup study for 13 years (for a total of 15 years of follow-up after drug product infusion in the parent studies). |
Who may qualify
- Ages
- Up to 19 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (1 listed)
- There are no exclusion criteria for this study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No interventional drug product utilized in this follow-up studyParticipants received a single IV infusion of Lenti-D Drug Product (also known as elivaldogene autotemcel or eli-cel) in either parent Study ALD-102 or ALD-104. The objectives of this long-term follow-up study are to assess long-term safety and efficacy following completion of participation in parent studies. Vector copy number (VCN) measurement, safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant are conducted in this study. | Genetic | Long-term followup |
Primary outcome
Major functional disability (MFD)-free survival
Time frame: 15 years post-drug-product infusion
The MFDs are loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, complete loss of voluntary movement.
Number of participants with malignancies
Time frame: 15 years post-drug-product infusion
Number of participants who experience graft versus host disease (GVHD)
Time frame: 15 years post-drug-product infusion
Number of participants with immune-related adverse events (AEs)
Time frame: 15 years post-drug-product infusion
Number of participants with new or worsening hematologic disorders
Time frame: 15 years post-drug-product infusion
Number of participants with new or worsening neurologic disorders
Time frame: 15 years post-drug-product infusion
3 secondary outcomes
Number of participants who undergo subsequent stem cell transplantation
Time frame: 15 years post-drug-product infusion
Change from baseline in neurological function score (NFS)
Time frame: 15 years post-drug-product infusion
Number of participants without gadolinium enhancement (GdE) status on magnetic resonance imaging (MRI)
Time frame: 15 years post-drug-product infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Sep 2026.
- Derived from this study: PubMed 39383459Eichler F, Duncan CN, Musolino PL, Lund TC, Gupta AO, De Oliveira S, Thrasher AJ, Aubourg P, Kuhl JS, Loes DJ, Amartino H, Smith N, Folloni Fernandes J, Sevin C, Sankar R, Hussain SA, Gissen P, Dalle JH, Platzbecker U, Downey GF, McNeil E, Demopoulos L, Dietz AC, Thakar HL, Orchard PJ, Williams DA. Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2024 Oct 10;391(14):1302-1312. doi: 10.1056/NEJMoa2400442.
- Derived from this study: PubMed 39383458Duncan CN, Bledsoe JR, Grzywacz B, Beckman A, Bonner M, Eichler FS, Kuhl JS, Harris MH, Slauson S, Colvin RA, Prasad VK, Downey GF, Pierciey FJ, Kinney MA, Foos M, Lodaya A, Floro N, Parsons G, Dietz AC, Gupta AO, Orchard PJ, Thakar HL, Williams DA. Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2024 Oct 10;391(14):1287-1301. doi: 10.1056/NEJMoa2405541.
- Parent Study ALD-102 protocol registrations and summary result disclosureLink provided by the sponsor.
- Parent Study ALD-104 protocol registrations and summary result disclosureLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. bluebird bio is committed to transparency. Appropriately de-identified patient-level datasets and supporting documents may be shared following attainment of applicable marketing approvals associated with this study and consistent with criteria established by bluebird bio and/or industry best practices to maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Vinod K Prasad, MD, FRCP study director
Genetix Biotherapeutics Inc.
13 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cerebral Adrenoleukodystrophy (CALD) and Adrenoleukodystrophy (ALD), run by Genetix Biotherapeutics Inc. at 13 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 14 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States, United Kingdom, Argentina, Australia, Brazil and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Major functional disability (MFD)-free survival (time frame: 15 years post-drug-product infusion). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02698579, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Sep 2026.