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Safety and Early Benefit of Allogeneic Bone Marrow‑Derived Mesenchymal Stromal Cells in Children and Adolescents with X‑Linked Adrenoleukodystrophy

InvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

The trial focuses on children and adolescents who have Adrenoleukodystrophy, a rare inherited condition that damages the brain and can affect the adrenal glands, leading to loss of movement, speech, and other abilities. The study uses a type of stem cell called allogeneic bone marrow-derived mesenchymal stromal cells, which are taken from a donor’s bone marrow and prepared for injection. These cells are delivered by an intrathecal method, meaning they are placed directly into the fluid that surrounds the spinal cord.

The purpose of the study is to assess the safety and possible benefit of this stem‑cell infusion. Participants will receive a single infusion of the cells and will then be observed for several weeks to check for any serious side effects and to see if there are early signs of improvement. Follow‑up visits include physical examinations and basic tests, and doctors will specifically look for serious reactions known as dose‑limiting toxicities, which are severe side effects that could require medical attention.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Baseline Assessments

    You will undergo a series of examinations to determine your current health status. This includes a physical examination, blood tests, and imaging studies such as magnetic resonance imaging (MRI) to evaluate brain involvement.

    The results will be used to confirm eligibility for the trial and to establish a reference point for later comparisons.

  2. Step 2

    Cell Preparation

    The study medication consists of allogeneic bone marrow‑derived mesenchymal stromal cells (MSC). These cells are stored frozen and will be thawed, washed, and placed in a sterile saline solution containing a small amount of human serum albumin before they are given to you.

  3. Step 3

    Intrathecal Infusion

    You will receive a single infusion of 5 × 10⁶ MSCs through an intrathecal injection, which means the cells are delivered directly into the fluid surrounding the spinal cord.

    The dose is administered once and is not repeated during the study.

  4. Step 4

    Immediate Post‑Infusion Observation

    After the infusion, you will remain in the clinical unit for at least 24 hours. During this time, vital signs and any immediate side effects will be closely monitored.

    Medical staff will assess you for signs of discomfort, fever, headache, or any other reactions.

  5. Step 5

    Follow‑Up Visits for Safety Assessment

    You will attend scheduled follow‑up visits on day 7, day 14, and day 28 after the infusion. Each visit includes a physical exam, laboratory tests, and a review of any symptoms you may have experienced.

    The primary focus of these visits is to detect dose‑limiting toxicities, which are serious side effects defined as grade 3 or higher according to standard safety criteria. This monitoring continues for the first 28 days to evaluate the safety of the treatment.

Who can join the trial?

7 criteria

  • Male patients who are younger than 16 years old.
  • Must have a diagnosis of X‑linked adrenoleukodystrophy that has been confirmed by a genetic test (a lab test that looks at the DNA) and by measuring long‑chain fatty acids (a type of fat in the blood that is abnormal in this disease).
  • The child must not be able to receive other standard treatments such as a bone‑marrow transplant (called haematopoietic progenitor transplantation) because they do not have a matching donor, or because their disease scores (Loes and NFS) are higher than the limits set by the centre, and they also cannot receive the approved gene‑therapy drug Skysona®.
  • The child must have a low enough disease severity, shown by an NFS score (a neurological function rating) of 6 out of 25 or less.
  • The brain scan called CMR (magnetic resonance imaging of the brain) must show a contrast uptake (called GIS) of at least 1, meaning the disease is active enough for the study.
  • It must be possible to perform a CMR scan and to do neuropsychological assessments (tests that check thinking, memory, and behavior) on the child.
  • The parents or legal guardian must sign the informed consent form, showing they understand the study and agree to take part.

Who cannot join the trial?

6 criteria

  • If the boy is a candidate for haematopoietic stem cell transplantation (a bone‑marrow transplant) from a matching donor and his disease scores (Loes and NFS values, which measure how far the disease has progressed on scans and neurological function) are suitable, he cannot join the study.
  • If he is a candidate for gene therapy (a treatment that changes the patient’s genes) such as Skysona®, which is only approved in the United States, he cannot join the study.
  • If his disease is in a “stopped” form (the brain scans have been stable for more than 12 months) or if he has no symptoms yet but was diagnosed because a family member has the disease, he cannot join the study.
  • If he has other serious illnesses at the same time, such as an active CNS infection (infection in the brain or spinal cord), an active systemic infection (infection elsewhere in the body), or an active tumour (cancer), he cannot join the study.
  • If his blood or bone‑marrow tests show problems that make a lumbar puncture (a spinal tap) unsafe, he cannot join the study.
  • If the patient or his family refuses to attend the required follow‑up visits, he cannot join the study.
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Investigated drugs

Allogeneic bone marrow‑derived mesenchymal stromal cells
These are living cells taken from the bone marrow of a healthy donor. The cells are grown in the lab, frozen, and then thawed before they are given to the patient. In this trial the cells are placed in a small amount of saline with a tiny amount of human albumin and are injected directly into the spinal fluid (intrathecal injection). The purpose of using these cells is to see if they are safe and whether they might help slow or improve the brain disease that occurs in children with X‑linked adrenoleukodystrophy. The trial will watch closely for any side effects and look for early signs that the cells may be having a beneficial effect.

What is already known about the treatment

Allogeneic bone marrow-derived mesenchymal stromal cells - This product is given as a sterile suspension that is injected directly into the spinal fluid (intrathecal injection). It is currently an investigational cell‑based therapy being studied in early‑phase clinical trials and is not yet approved for routine medical use. The main purpose is to treat children with the cerebral form of X‑linked adrenoleukodystrophy, a rare genetic brain disease. The cells work by releasing anti‑inflammatory proteins and growth factors that calm the immune system and help repair damaged brain tissue, and they are classified as a mesenchymal stromal cell biologic therapy.

Investigated diseases

X-linked adrenoleukodystrophy (X-ALD) - It is a rare inherited disorder that mainly affects boys. The disease causes accumulation of very‑long‑chain fatty acids in the brain and adrenal glands. This leads to loss of the protective myelin sheath around nerve fibers, resulting in gradually worsening neurological function. Children may develop problems with coordination, vision, hearing, and cognition as the disease advances. The adrenal glands may also become unable to produce essential hormones, adding to the clinical picture.
Trial detailsLast updated 15 Oct 2026
Age0-17PhasePhase ITrial ID2026-526701-14-00Protocol codeALDXyCelEstimated enrolment5 patientsSponsorFundacion Para La Investigacion Biomedica Hospital Infantil Universitario Nino Jesus

sourced from the EU Clinical Trials Register and site verification

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