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Stem Cell Therapy

MSC Therapy for Adrenoleukodystrophy

Phase 1

Recruiting

Research Sponsored by Masonic Cancer Center, University of Minnesota

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of ALD, as established by elevation of very long chain fatty acid levels or gene mutation

Age ≥ 3 years

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 months

Awards & highlights

Study Summary

This trial evaluates 3rd party stem cells as a bridge to stem cell transplant or gene therapy for cerebral adrenoleukodystrophy (CALD), between diagnosis and transplant (currently 8-12 weeks).

Who is the study for?

This trial is for patients with active cerebral adrenoleukodystrophy (CALD) who are over 3 years old, have not had gene therapy or a transplant yet, and are expected to live more than 6 months. They must have certain MRI findings and blood tests showing proper organ function.Check my eligibility

What is being tested?

The study is testing the use of mesenchymal stem cells given through an IV as a temporary treatment for CALD while waiting for a stem cell transplant or gene therapy. This intervention fills the gap between CALD diagnosis and these longer-term treatments.See study design

What are the potential side effects?

Potential side effects may include reactions related to immune response, such as inflammation, or issues due to sedation required for administering the treatment. Specific side effects will depend on individual patient responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with ALD based on specific tests.

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I am at least 3 years old.

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I have never had gene therapy or a transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Safety and tolerability of intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).

Secondary outcome measures

Incidence of radiographic response in patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).

Trial Design

2Treatment groups

Experimental Treatment

Group I: IV-MSC for cALD (Early Disease/Bridge Therapy)Experimental Treatment1 Intervention

Patients with active, cerebral adrenoleukodystrophy (cALD) as a bridge to hematopoietic stem cell transplant or gene therapy.

Group II: IV-MSC for cALD (Advanced Disease)Experimental Treatment1 Intervention

Patients with active, cerebral adrenoleukodystrophy (cALD) who are too advanced for gene therapy or HSCT.

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Masonic Cancer Center, University of MinnesotaLead Sponsor

272 Previous Clinical Trials

14,643 Total Patients Enrolled

8 Trials studying Adrenoleukodystrophy

1,324 Patients Enrolled for Adrenoleukodystrophy

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are enrollment spots still available for this investigation?

"Affirmative. According to the information on clinicaltrials.gov, this medical experiment is currently accepting applicants; it was first posted on August 28th 2023 and most recently updated on September 7th 2023. Ten volunteers are needed from one particular research facility."

Answered by AI

What risks do patients face when undergoing intravenous mesenchymal stem cell (IV-MSC) therapy for cALD?

"As this is an early-stage clinical trial, our team at Power has placed IV-MSC for cALD (Early Disease/Bridge Therapy) on the lower end of safety scale, with a score of 1. Thus far, there is restricted data that supports its efficacy and security."

Answered by AI

What is the current enrollment of participants in this research?

"Affirmative. The clinicaltrials.gov page states that this trial is now recruiting participants, with the first posting on August 28th 2023 and the most recent update occuring September 7th of the same year. 10 enrollees are being sought from a single healthcare facility."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

MT2022-01: MSCs for ALD

2023. "MT2022-01: MSCs for ALD". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06030648.

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