X-Linked Adrenoleukodystrophy (X-ALD)NCT02204904
Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)
An observational study of X-Linked Adrenoleukodystrophy (X-ALD) and Cerebral Adrenoleukodystrophy (CALD), run by Genetix Biotherapeutics Inc. at 13 sites in 7 countries.
Trial summary
Study ALD-103 will be a multi-site, global, prospective and retrospective data collection study that is designed to evaluate outcomes of allo-HSCT in male subjects with CALD ≤17 years of age. Read more about this study
- Who is it for?
- Ages Up to 17 years, men only
- Conditions the study is for
- X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
- What is being tested?
- Allo-HSCT
- Type of treatment
- Injection or infusion
- How many people
- 59 participants
- Where
- 13 sitesin 7 countries
- Study dates
- Apr 2015to 6 Dec 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
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 17 years
- Men only
- No healthy volunteers
- Child
Study population: Boys aged 17 or younger receiving allogeneic hematopoietic stem cell transplantation for the treatment of cerebral adrenoleukodystrophy prospectively or partially prospective/retrospective
Who may be able to join
- Provide informed consent from a competent custodial parent or guardian with legal capacity to execute a local Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved consent. In addition, informed assent will be sought from capable subjects, in accordance with the directive of the institution's IRB/IEC and all other local requirements.
- Be male and ≤17 years of age at the time of treatment, for retrospective and partial prospective/retrospective subjects, or at the time of parental/guardian consent and, where appropriate, subject assent, for prospective subjects.
- Have a confirmed diagnosis of CALD as defined by abnormal VLCFA profile and cerebral lesion on brain MRI.
- Depending on the cohort, the subject must:
- Be scheduled for allo-HSCT evaluation at a study site (prospective cohort only),
- Have received an allo-HSC infusion and be consented in time to complete the Month 24 Visit on study (partial prospective/retrospective cohort only), or
- Have received their most recent allo-HSC infusion on or after January 1, 2013 (retrospective cohort only).
Who cannot join
- Previous treatment with a gene therapy product.
- Receipt of an experimental transplantation procedure.
Trial locations and contacts
13 sites in 7 countries.
13 sites in 7 countries
United States6 sites
Boston Children's Hospital/Massachusetts General Hospital
Boston, Massachusetts, 02141
Duke University Medical Center
Durham, North Carolina, 27705
Children's Hospital Los Angeles
Los Angeles, California, 90027
University of Minnesota
Minneapolis, Minnesota, 55455
Stanford University
Palo Alto, California, 94304
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United Kingdom2 sites
Great Ormond Street Hospital
London
Central Manchester University Hospitals NHS Foundation Trust
Manchester, M13 9WL
Netherlands1 site
Princess Maxima Center for Pediatric Oncology (PMC)
Utrecht
About this study
Official title: A Prospective and Retrospective Data Collection Study to Evaluate Outcomes in Males ≤17 Years of Age Undergoing Allogeneic Hematopoietic Stem Cell Transplantation for the Treatment of Cerebral Adrenoleukodystrophy
Study ALD-103 will be a multi-site, global, prospective and retrospective data collection study that is designed to evaluate outcomes of allo-HSCT in male subjects with CALD ≤17 years of age.
- Conditions
- X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
- Being tested
- Allo-HSCT
- Keywords
- 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.
Allo-HSCTGenetic
Allogeneic Hematopoietic Stem Cell Transplantation
| Study group | Role | What they receive |
|---|---|---|
| Allo-HSCT prospective | Subjects who will be consented before they received an allo-HSC infusion. They will be consented and enrolled on the study during the Screening Period. Receives: Genetic: Allo-HSCT | |
| Allo-HSCT partial prospective/retrospective | Subjects who will be consented after they received an allo-HSC infusion but before they reach 24 months post-infusion on study. Subjects in this cohort will participate prospectively in at least the Month 24 Visit in order to obtain prospective on-study data for this and all visits after Month 24 Receives: Genetic: Allo-HSCT | |
| Allo-HSCT retrospective | Subjects who received an allo-HSC infusion on or after January 1, 2013 and died before study data collection. Receives: Genetic: Allo-HSCT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of transplant-related mortality (TRM).
TRM is defined as death due to any transplantation-related cause other than disease progression.
Measured: Through 100 and 365 days post allo-HSC infusion
Incidence and timing of neutrophil engraftment.
Measured: 1-48 (± 1) months post allo-HSC infusion
Incidence and timing of platelet engraftment
Measured: 1-48 (± 1) months post allo-HSC infusion
Incidence of engraftment failure or allograft rejection.
Measured: 1-48 (± 1) months post allo-HSC infusion
Incidence of primary donor-derived chimerism of ≥50%.
Measured: by 100 days post allo-HSC infusion
Frequency and severity of Criteria for Adverse Events (CTCAE) ≥Grade 3 AEs, CTCAE ≥Grade 3 infections, and all SAEs.
Measured: 1-48 (± 1) months post allo-HSC infusion
Proportion of subjects who experience either ≥Grade II acute (Graft versus Host Disease) GVHD or chronic GVHD.
Measured: 1-48 (± 1) months post allo-HSC infusion
Incidence of ≥Grade II acute GVHD.
Measured: 1-48 (± 1) months post allo-HSC infusion
Incidence of chronic GVHD.
Measured: 1-48 (± 1) months post allo-HSC infusion
Number of emergency room visits.
Measured: 1-48 (± 1) months post allo-HSC infusion
Number and duration of intensive care unit stay.
Measured: 1-48 (± 1) months post allo-HSC infusion
Number and duration of in-patient hospitalization.
Measured: 1-48 (± 1) months post allo-HSC infusion
Secondary outcomes (6)
Incidence of Major Functional Disabilities (MFDs).
MFDs is defined as any of the following: loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement.
Measured: 1-48 (± 2) months post allo-HSC infusion
Change from Baseline in Loes score
Measured: 1-48 (± 2) months post allo-HSC infusion
Change from Baseline in Neurological Function Score (NFS)
Measured: 1-48 (± 2) months post allo-HSC infusion
Frequency and timing of resolution of gadolinium enhancement on MRI, if applicable
Measured: 1-48 (± 2) months post allo-HSC infusion
MFD-free survival
Measured: 48 (± 2) months post allo-HSC infusion
Overall survival
Measured: 48 (± 2) months post allo-HSC infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2014 (estimated)
- Study startApr 2015
- Primary completion6 Dec 2019
- Study completion6 Dec 2019
Why it stopped: Sufficient enrollment for Month 24 safety and efficacy endpoints
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for X-Linked Adrenoleukodystrophy (X-ALD)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
- Other
- Enrolment
- 59 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Elizabeth McNeil, MD MSc, Study director, bluebird bio, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02204904
- Sponsor's protocol ID
- ALD-103
- Status last verified
- Dec 2019
- First submitted
- 28 Jul 2014
- Last updated
- 21 May 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes
Publications
Chiesa R, Boelens JJ, Duncan CN, Kuhl JS, Sevin C, Kapoor N, Prasad VK, Lindemans CA, Jones SA, Amartino HM, Algeri M, Bunin N, Diaz-de-Heredia C, Loes DJ, Shamir E, Timm A, McNeil E, Dietz AC, Orchard PJ. Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy. Blood Adv. 2022 Mar 8;6(5):1512-1524. doi: 10.1182/bloodadvances.2021005294.
PubMed 34781360
Study at a glance
From the registry record, last updated by the sponsor on 21 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective and Retrospective Data Collection Study to Evaluate Outcomes in Males ≤17 Years of Age Undergoing Allogeneic Hematopoietic Stem Cell Transplantation for the Treatment of Cerebral Adrenoleukodystrophy
- ClinicalTrials.gov ID
- NCT02204904
- Acronym / sponsor ID
- ALD-103
- Conditions
- X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
- Keywords
- X-linked Adrenoleukodystrophy, Hematopoietic Stem Cells
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Sufficient enrollment for Month 24 safety and efficacy endpoints
- Enrolment
- 59 participants (actual)
- Lead sponsor
- Genetix Biotherapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- Apr 2015 (actual)
- Primary completion
- 6 Dec 2019 (actual)
- Study completion
- 6 Dec 2019 (actual)
- First posted
- 31 Jul 2014 (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.
Study ALD-103 will be a multi-site, global, prospective and retrospective data collection study that is designed to evaluate outcomes of allo-HSCT in male subjects with CALD ≤17 years of age.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Allo-HSCT prospective | – | Genetic: Allo-HSCTSubjects who will be consented before they received an allo-HSC infusion. They will be consented and enrolled on the study during the Screening Period. |
| Allo-HSCT partial prospective/retrospective | – | Genetic: Allo-HSCTSubjects who will be consented after they received an allo-HSC infusion but before they reach 24 months post-infusion on study. Subjects in this cohort will participate prospectively in at least the Month 24 Visit in order to obtain prospective on-study data for this and all visits after Month 24 |
| Allo-HSCT retrospective | – | Genetic: Allo-HSCTSubjects who received an allo-HSC infusion on or after January 1, 2013 and died before study data collection. |
Who may qualify
- Ages
- Up to 17 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Boys aged 17 or younger receiving allogeneic hematopoietic stem cell transplantation for the treatment of cerebral adrenoleukodystrophy prospectively or partially prospective/retrospective
Key inclusion criteria (7 listed)
- Provide informed consent from a competent custodial parent or guardian with legal capacity to execute a local Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved consent. In addition, informed assent will be sought from capable subjects, in accordance with the directive of the institution's IRB/IEC and all other local requirements.
- Be male and ≤17 years of age at the time of treatment, for retrospective and partial prospective/retrospective subjects, or at the time of parental/guardian consent and, where appropriate, subject assent, for prospective subjects.
- Have a confirmed diagnosis of CALD as defined by abnormal VLCFA profile and cerebral lesion on brain MRI.
- Depending on the cohort, the subject must:
Key exclusion criteria (2 listed)
- Previous treatment with a gene therapy product.
- Receipt of an experimental transplantation procedure.
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 |
|---|---|---|
| Allo-HSCTAllogeneic Hematopoietic Stem Cell Transplantation | Genetic | Allo-HSCT partial prospective/retrospective, Allo-HSCT prospective, Allo-HSCT retrospective |
Primary outcome
Incidence of transplant-related mortality (TRM).
Time frame: Through 100 and 365 days post allo-HSC infusion
TRM is defined as death due to any transplantation-related cause other than disease progression.
Incidence and timing of neutrophil engraftment.
Time frame: 1-48 (± 1) months post allo-HSC infusion
Incidence and timing of platelet engraftment
Time frame: 1-48 (± 1) months post allo-HSC infusion
Incidence of engraftment failure or allograft rejection.
Time frame: 1-48 (± 1) months post allo-HSC infusion
Incidence of primary donor-derived chimerism of ≥50%.
Time frame: by 100 days post allo-HSC infusion
Frequency and severity of Criteria for Adverse Events (CTCAE) ≥Grade 3 AEs, CTCAE ≥Grade 3 infections, and all SAEs.
Time frame: 1-48 (± 1) months post allo-HSC infusion
Proportion of subjects who experience either ≥Grade II acute (Graft versus Host Disease) GVHD or chronic GVHD.
Time frame: 1-48 (± 1) months post allo-HSC infusion
Incidence of ≥Grade II acute GVHD.
Time frame: 1-48 (± 1) months post allo-HSC infusion
Incidence of chronic GVHD.
Time frame: 1-48 (± 1) months post allo-HSC infusion
Number of emergency room visits.
Time frame: 1-48 (± 1) months post allo-HSC infusion
Number and duration of intensive care unit stay.
Time frame: 1-48 (± 1) months post allo-HSC infusion
Number and duration of in-patient hospitalization.
Time frame: 1-48 (± 1) months post allo-HSC infusion
6 secondary outcomes
Incidence of Major Functional Disabilities (MFDs).
Time frame: 1-48 (± 2) months post allo-HSC infusion
Change from Baseline in Loes score
Time frame: 1-48 (± 2) months post allo-HSC infusion
Change from Baseline in Neurological Function Score (NFS)
Time frame: 1-48 (± 2) months post allo-HSC infusion
Frequency and timing of resolution of gadolinium enhancement on MRI, if applicable
Time frame: 1-48 (± 2) months post allo-HSC infusion
MFD-free survival
Time frame: 48 (± 2) months post allo-HSC infusion
Overall survival
Time frame: 48 (± 2) months post allo-HSC infusion
Results and safety
The study was stopped early (reason given: Sufficient enrollment for Month 24 safety and efficacy endpoints). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 May 2020.
- Derived from this study: PubMed 34781360Chiesa R, Boelens JJ, Duncan CN, Kuhl JS, Sevin C, Kapoor N, Prasad VK, Lindemans CA, Jones SA, Amartino HM, Algeri M, Bunin N, Diaz-de-Heredia C, Loes DJ, Shamir E, Timm A, McNeil E, Dietz AC, Orchard PJ. Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy. Blood Adv. 2022 Mar 8;6(5):1512-1524. doi: 10.1182/bloodadvances.2021005294.
- Individual participant dataSharing planned: Yes
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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.
Elizabeth McNeil, MD MSc study director
bluebird bio, Inc.
13 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of X-Linked Adrenoleukodystrophy (X-ALD) and Cerebral Adrenoleukodystrophy (CALD), run by Genetix Biotherapeutics Inc. at 13 sites in 7 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 7 inclusion and 2 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: stopped early, as of the sponsor's last update on 21 May 2020. 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, Canada, Germany and 2 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?
The study was stopped early (reason given: Sufficient enrollment for Month 24 safety and efficacy endpoints). Results are not available in the source record.
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?
Incidence of transplant-related mortality (TRM). (time frame: Through 100 and 365 days post allo-HSC infusion). The study lists 12 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 NCT02204904, which also lists 1 linked publication.
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 21 May 2020.