Cerebral Adrenoleukodystrophy (CALD)StargazerNCT06224413
A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
An observational study of Cerebral Adrenoleukodystrophy (CALD), run by Genetix Biotherapeutics Inc. at 3 sites in United States.
Trial summary
The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel in the post-marketing setting (tradename Skysona) and to describe major functional disability (MFD)-free survival over time in participants with more advanced… Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Cerebral Adrenoleukodystrophy (CALD)
- Type of treatment
- Injection or infusion
- How many people
- 120 participants
- Where
- 3 sitesUnited States
- Study dates
- 27 Mar 2024to 30 Dec 2047
- How long will I take part?
- 15 Years
- 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 Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants with CALD treated with eli-cel in the post marketing setting at a center in the US that participates in the Registry Study.
Who may be able to join
- Participants who fulfill the following criteria will be eligible for inclusion in this Registry Study.
- Participant must be treated with eli-cel in the post marketing setting at a center in the United States (US) that participates in the Registry Study.
- Participant must have provided an informed consent and/or assent to participate in Center for International Blood and Marrow Transplant Research (CIBMTR) registry.
- Participant must have provided an informed consent and/or assent to participate in the Registry Study.
- Participant must receive follow up care by a US-based physician with the ability to submit REG-502 data.
- Registry Study Subpopulation inclusion:
- Twenty-four of the 120 patients in the Registry Study must meet the following inclusion criteria which will be used to create the more advanced early active CALD subpopulation:
- Participant must meet the above inclusion criteria and have a Loes score of 4.5 through 9.0 with GdE+ (gadolinium enhancement positivity) from an MRI performed before treatment with eli-cel and with NFS of 0 or 1 at baseline.
Who cannot join
- There are no exclusion criteria for this Registry Study.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02115
- Recruiting
UT Southwestern Medical Center
Dallas, Texas, 75390
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official title: A Postmarketing, Prospective, Multicenter, Observational, Long-Term Safety and Effectiveness Registry Study of Patients With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (Stargazer)
The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel in the post-marketing setting (tradename Skysona) and to describe major functional disability (MFD)-free survival over time in participants with more advanced early active CALD. All enrolled participants with CALD treated with eli-cel in the post-marketing setting will be followed in this study for 15 years. No investigational drug product will be administered in this study. This study will enroll 120 participants with CALD treated with eli-cel in the post-marketing setting. A subpopulation of 24 participants with more advanced early active CALD will be specifically enrolled as required by the US FDA as a condition of accelerated approval and will be considered as a separate cohort for effectiveness outcomes.
- Conditions
- Cerebral Adrenoleukodystrophy (CALD)
- Being tested
- No Intervention
- Keywords
- Adrenoleukodystrophy, Adrenoleukodystrophy protein
- Medical subject headings
- Adrenoleukodystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
This is non-interventional study.
| Study group | Role | What they receive |
|---|---|---|
| All Participants | Participants with CALD treated with eli-cel in the post marketing setting will be followed in this registry study for up to 15 years after eli-cel infusion to collect real-world longitudinal data, and evaluate the outcomes. Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)
The Sponsor considers the following events to be AEIs (which should be reported as a medically significant Serious Adverse Events [SAEs]):
- Any newly diagnosed malignancy
- Neutrophil engraftment failure: defined as health care provider (HCP) decision to administer back-up cells or subsequent hematopoietic stem cell transplantation (HSCT) due to neutrophil recovery failure
- Newly acquired HIV-1 or HIV-2 infection
- Any newly diagnosed autoimmune disorders
- Opportunistic infections
- Grade 3 or higher cytopenias occurring more than 60 days after eli-cel infusion
Measured: Through 15 years postinfusion
Number of Participants with Newly Diagnosed Malignancies
Measured: Through 15 years postinfusion
Number of Participants with Insertional Oncogenesis
Measured: Through 15 years postinfusion
Major Functional Disability (MFD)-Free Survival
MFD-free survival is defined as time from drug product infusion to either a rescue cell administration or second transplant, MFD, or death due to any cause, whichever occurs first.
Measured: Through 15 years postinfusion
Secondary outcomes (9)
Overall Survival (OS)
Measured: Through 15 years postinfusion
Number of Participants with Serious Adverse Events (SAEs)
Measured: Through 15 years postinfusion
Number of Participants with Eli-cel Related AEs
Measured: Through 15 years postinfusion
Number of Participants with Presence of Insertional Oncogenesis in Subpopulation with Newly Diagnosed Hematologic Malignancy
Measured: Through 15 years postinfusion
Number of Participants with Complete Remission in Subpopulation with Newly Diagnosed Hematologic Malignancy
Measured: Through 15 years postinfusion
Relapse-free Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy
Measured: Through 15 years postinfusion
Overall Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy
Measured: Through 15 years postinfusion
Number of Participants with Clinically Significant Abnormalities by Karyotyping in Bone Marrow Aspirate in Subpopulation with Newly Diagnosed Hematologic Malignancy
Measured: Through 15 years postinfusion
Number of Participants with Clinically Significant Abnormalities on Fluorescence In-situ Hybridization (FISH) and Next Generation Sequencing (NGS) in Subpopulation with Newly Diagnosed Hematologic Malignancy
Measured: Through 15 years postinfusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2024
- Study start27 Mar 2024
- Primary completion30 Dec 2047 (estimated)
- Study completion30 Dec 2047 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 120 participants (estimated)
- Target duration
- 15 Years
Sponsor and investigators
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Center for International Blood and Marrow Transplant Research
- Responsible party
- Sponsor
- Investigators
- Kiran Bhirangi, MD, Study director, bluebird bio, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06224413
- Sponsor's protocol ID
- REG-502
- Acronym
- Stargazer
- Status last verified
- Jun 2025
- First submitted
- 16 Jan 2024
- Last updated
- 2 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Bluebird bio is committed to transparency and appropriately de-identified patient-level datasets and supporting documents may be shared (if contractually or otherwise legally permitted ) following completion of this study, submission of all applicable regulatory submissions and consistent with criteria established by bluebird bio, our collaborators (CIBMTR) and/or industry best practices to protect confidential information and maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Postmarketing, Prospective, Multicenter, Observational, Long-Term Safety and Effectiveness Registry Study of Patients With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (Stargazer)
- ClinicalTrials.gov ID
- NCT06224413
- Acronym / sponsor ID
- Stargazer / REG-502
- Conditions
- Cerebral Adrenoleukodystrophy (CALD)
- Keywords
- Adrenoleukodystrophy, Adrenoleukodystrophy protein
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- Genetix Biotherapeutics Inc.Industry (pharma, biotech, devices)
- Collaborators
- Center for International Blood and Marrow Transplant Research
- Study start
- 27 Mar 2024 (actual)
- Primary completion
- 30 Dec 2047 (estimated)
- Study completion
- 30 Dec 2047 (estimated)
- First posted
- 25 Jan 2024 (actual)
- 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.
The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel in the post-marketing setting (tradename Skysona) and to describe major functional disability (MFD)-free survival over time in participants with more advanced early active CALD. All enrolled participants with CALD treated with eli-cel in the post-marketing setting will be followed in this study for 15 years. No investigational drug product will be administered in this study. This study will enroll 120 participants with CALD treated with eli-cel in the post-marketing setting. A subpopulation of 24 participants with more advanced early active CALD will be specifically enrolled as required by the US FDA as a condition of accelerated approval and will be considered as a separate cohort for effectiveness outcomes.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 15 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All Participants | – | Other: No InterventionParticipants with CALD treated with eli-cel in the post marketing setting will be followed in this registry study for up to 15 years after eli-cel infusion to collect real-world longitudinal data, and evaluate the outcomes. |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants with CALD treated with eli-cel in the post marketing setting at a center in the US that participates in the Registry Study.
Key inclusion criteria (8 listed)
- Participants who fulfill the following criteria will be eligible for inclusion in this Registry Study.
- Participant must be treated with eli-cel in the post marketing setting at a center in the United States (US) that participates in the Registry Study.
- Participant must have provided an informed consent and/or assent to participate in Center for International Blood and Marrow Transplant Research (CIBMTR) registry.
- Participant must have provided an informed consent and/or assent to participate in the Registry Study.
- Participant must receive follow up care by a US-based physician with the ability to submit REG-502 data.
Key exclusion criteria (1 listed)
- There are no exclusion criteria for this Registry 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 InterventionThis is non-interventional study. | Other | All Participants |
Primary outcome
Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs)
Time frame: Through 15 years postinfusion
The Sponsor considers the following events to be AEIs (which should be reported as a medically significant Serious Adverse Events [SAEs]): * Any newly diagnosed malignancy * Neutrophil engraftment failure: defined as health care provider (HCP) decision to administer back-up cells or subsequent hematopoietic stem cell transplantation (HSCT) due to neutrophil recovery failure * Newly acquired HIV-1 or HIV-2 infection * Any newly diagnosed autoimmune disorders * Opportunistic infections * Grade 3 or higher cytopenias occurring more than 60 days after eli-cel infusion
Number of Participants with Newly Diagnosed Malignancies
Time frame: Through 15 years postinfusion
Number of Participants with Insertional Oncogenesis
Time frame: Through 15 years postinfusion
Major Functional Disability (MFD)-Free Survival
Time frame: Through 15 years postinfusion
MFD-free survival is defined as time from drug product infusion to either a rescue cell administration or second transplant, MFD, or death due to any cause, whichever occurs first.
9 secondary outcomes
Overall Survival (OS)
Time frame: Through 15 years postinfusion
Number of Participants with Serious Adverse Events (SAEs)
Time frame: Through 15 years postinfusion
Number of Participants with Eli-cel Related AEs
Time frame: Through 15 years postinfusion
Number of Participants with Presence of Insertional Oncogenesis in Subpopulation with Newly Diagnosed Hematologic Malignancy
Time frame: Through 15 years postinfusion
Number of Participants with Complete Remission in Subpopulation with Newly Diagnosed Hematologic Malignancy
Time frame: Through 15 years postinfusion
Relapse-free Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy
Time frame: Through 15 years postinfusion
Overall Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy
Time frame: Through 15 years postinfusion
Number of Participants with Clinically Significant Abnormalities by Karyotyping in Bone Marrow Aspirate in Subpopulation with Newly Diagnosed Hematologic Malignancy
Time frame: Through 15 years postinfusion
Number of Participants with Clinically Significant Abnormalities on Fluorescence In-situ Hybridization (FISH) and Next Generation Sequencing (NGS) in Subpopulation with Newly Diagnosed Hematologic Malignancy
Time frame: Through 15 years postinfusion
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 2 Jul 2025.
- Individual participant dataSharing planned: Yes. Bluebird bio is committed to transparency and appropriately de-identified patient-level datasets and supporting documents may be shared (if contractually or otherwise legally permitted ) following completion of this study, submission of all applicable regulatory submissions and consistent with criteria established by bluebird bio, our collaborators (CIBMTR) and/or industry best practices to protect confidential information and 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.
Kiran Bhirangi, MD study director
bluebird bio, Inc.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cerebral Adrenoleukodystrophy (CALD), run by Genetix Biotherapeutics Inc. at 3 sites in United States.
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 8 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: looking for participants now, as of the sponsor's last update on 2 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. 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?
Contact bluebird bio (+1-833-999-6378, clinicaltrials@bluebirdbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs) (time frame: Through 15 years postinfusion). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 2 Jul 2025.