Cerebral AdrenoleukodystrophyPhase 1NCT06030648
MT2022-01: MSCs for ALD
A Phase 1 study testing Mesenchymal stem cells (IV-MSC) in people with Cerebral Adrenoleukodystrophy, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
This is a single-institution study to evaluate the use of intravenously administered allogeneic, 3rd party mesenchymal stem cells (IV-MSC) in patients with active, cerebral adrenoleukodystrophy (CALD), as a bridge to hematopoietic stem cell transplant or gene therapy, or in patients who are too advanced for gene… Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Cerebral Adrenoleukodystrophy
- What is being tested?
- Mesenchymal stem cells (IV-MSC)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Aug 2023to 1 May 2029
- Phase
- Phase 1
- 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 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- age ≥ 3 years
- diagnosis of ALD, as established by elevation of very long chain fatty acid levels or gene mutation
- evidence of active cerebral disease as determined by the presence of gadolinium enhancement
- ALD MRI (Loes) score ≥ 1
- Patients who have not received prior gene therapy or transplant
- Life expectancy of > 6 months as determined by the enrolling researcher
- Have adequate organ function confirmed by laboratory values obtained within 28 days prior to enrollment
Who cannot join
- Inability to undergo sedation or MRI studies for any reason
- Other concurrent life-threatening disease (life expectancy <6 months) or eligible for hospice care
- Known or suspected hypersensitivity to human serum albumin, diphenhydramine, acetaminophen, methylprednisolone, or any of the components of IMS001.
Trial locations and contacts
1 site in 1 country.
United States1 site
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, 55455
About this study
Official title: Intravenous Administration of Mesenchymal Stem Cells (IV-MSC) for the Treatment of Cerebral Adrenoleukodystrophy (cALD)
This is a single-institution study to evaluate the use of intravenously administered allogeneic, 3rd party mesenchymal stem cells (IV-MSC) in patients with active, cerebral adrenoleukodystrophy (CALD), as a bridge to hematopoietic stem cell transplant or gene therapy, or in patients who are too advanced for gene therapy or HSCT. The intervention will occur in the time between diagnosis of active CALD and transplant which is currently 8-12 weeks.
- Conditions
- Cerebral Adrenoleukodystrophy
- Being tested
- Mesenchymal stem cells (IV-MSC)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mesenchymal stem cells (IV-MSC)Biological
Patients will receive 1 infusion (1 x 106 cells/kg on day 0) then be followed for 8 weeks from the infusion.
| Study group | Role | What they receive |
|---|---|---|
| IV-MSC for cALD (Early Disease/Bridge Therapy) | Experimental | Patients with active, cerebral adrenoleukodystrophy (cALD) as a bridge to hematopoietic stem cell transplant or gene therapy. Receives: Biological: Mesenchymal stem cells (IV-MSC) |
| IV-MSC for cALD (Advanced Disease) | Experimental | Patients with active, cerebral adrenoleukodystrophy (cALD) who are too advanced for gene therapy or HSCT. Receives: Biological: Mesenchymal stem cells (IV-MSC) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).
Safety and tolerability of cerebral adrenoleukodystrophy (cALD) patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC) assessed by incidence of adverse events.
Measured: 6 months
Secondary outcomes (1)
Incidence of radiographic response in patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).
Radiographic response is defined as the resolution of intravenous gadolinium contrast enhancement on brain MRI.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Sep 2023
- Study start28 Aug 2023
- Primary completion1 May 2027 (estimated)
- Study completion1 May 2029 (estimated)
Why it stopped: Awaiting supplies
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cerebral AdrenoleukodystrophyStudies 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06030648
- Sponsor's protocol ID
- 2022LS001
- Status last verified
- Jun 2026
- First submitted
- 31 Aug 2023
- Last updated
- 18 Jun 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intravenous Administration of Mesenchymal Stem Cells (IV-MSC) for the Treatment of Cerebral Adrenoleukodystrophy (cALD)
- ClinicalTrials.gov ID
- NCT06030648
- Acronym / sponsor ID
- 2022LS001
- Conditions
- Cerebral Adrenoleukodystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: Awaiting supplies
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 28 Aug 2023 (actual)
- Primary completion
- 1 May 2027 (estimated)
- Study completion
- 1 May 2029 (estimated)
- First posted
- 11 Sep 2023 (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.
This is a single-institution study to evaluate the use of intravenously administered allogeneic, 3rd party mesenchymal stem cells (IV-MSC) in patients with active, cerebral adrenoleukodystrophy (CALD), as a bridge to hematopoietic stem cell transplant or gene therapy, or in patients who are too advanced for gene therapy or HSCT. The intervention will occur in the time between diagnosis of active CALD and transplant which is currently 8-12 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| IV-MSC for cALD (Early Disease/Bridge Therapy) | Experimental | Biological: Mesenchymal stem cells (IV-MSC)Patients with active, cerebral adrenoleukodystrophy (cALD) as a bridge to hematopoietic stem cell transplant or gene therapy. |
| IV-MSC for cALD (Advanced Disease) | Experimental | Biological: Mesenchymal stem cells (IV-MSC)Patients with active, cerebral adrenoleukodystrophy (cALD) who are too advanced for gene therapy or HSCT. |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- age ≥ 3 years
- diagnosis of ALD, as established by elevation of very long chain fatty acid levels or gene mutation
- evidence of active cerebral disease as determined by the presence of gadolinium enhancement
- ALD MRI (Loes) score ≥ 1
- Patients who have not received prior gene therapy or transplant
Key exclusion criteria (3 listed)
- Inability to undergo sedation or MRI studies for any reason
- Other concurrent life-threatening disease (life expectancy <6 months) or eligible for hospice care
- Known or suspected hypersensitivity to human serum albumin, diphenhydramine, acetaminophen, methylprednisolone, or any of the components of IMS001.
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 |
|---|---|---|
| Mesenchymal stem cells (IV-MSC)Patients will receive 1 infusion (1 x 106 cells/kg on day 0) then be followed for 8 weeks from the infusion. | Biological | IV-MSC for cALD (Advanced Disease), IV-MSC for cALD (Early Disease/Bridge Therapy) |
Primary outcome
Safety and tolerability of intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).
Time frame: 6 months
Safety and tolerability of cerebral adrenoleukodystrophy (cALD) patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC) assessed by incidence of adverse events.
1 secondary outcome
Incidence of radiographic response in patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2026.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Mesenchymal stem cells (IV-MSC) in people with Cerebral Adrenoleukodystrophy, run by Masonic Cancer Center, University of Minnesota at 1 site 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 7 inclusion and 3 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: paused, as of the sponsor's last update on 18 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
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?
Safety and tolerability of intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC). (time frame: 6 months). 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 18 Jun 2026.