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A Phase 1/2 Randomized, Blinded, Dose-escalation Study to Evaluate the Safety and Efficacy of Intrathecal Administration of AAV9-ABCD1 Gene Therapy (SBT101) in Adult Patients with Adrenomyeloneuropathy
EU CTIS ID: 2024-518451-39-00
What this study is testing
To characterize the safety and tolerability of one-time IT administered SBT101 in adults diagnosed with AMN
- Human Pharmacology (Phase I)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male adults aged ≥18 and ≤65 years
- Diagnosed with X-linked adrenoleukodystrophy (ALD)), including proven mutation in the ABCD1 gene through confirmatory genetic testing, and supported by historically elevated VLCFA.
- Clinical evidence of spinal cord involvement with EDSS score between 1 and 4.5 and a pyramidal function score of ≥1 in the Functional System Score of the EDSS. Signs of pyramidal tract dysfunction don't include hyperreflexia.
- Must agree to use double-barrier contraception methods (e.g., condom and 1 other form of contraception for female sexual partners that are of childbearing potential, such as diaphragm, intrauterine device, spermicidal jelly, and/or hormonal contraceptive) and to not donate sperm for at least 6 months following the IMP procedure.
- For patients who are receiving any other treatment for ALD, including off-label medications and/or supplements (e.g., antioxidants, Lorenzo's oil, statins, etc.) or physical rehabilitation support, such treatments must have been at a stable dose and/or frequency for ≥4 weeks prior to Screening and patients must agree to continue at the same dose and/or frequency through Part 1 of the study.
- The patient provided written informed consent prior to any study procedures being performed
You likely can't join if
- Presence of inflammatory cerebral disease, established by radiographic review of brain MRI demonstrating a Loes score ≥0.5 on the 34-point scale, except for the abnormalities that can be observed in patients with AMN without inflammatory cerebral demyelination with Loes score ≤4.
- Presence of clinically significant active bacterial, viral, fungal, parasitic, or prior - associated infection.
- History of diabetes or abnormal fasting serum glucose (≥126 mg/dL) or hemoglobin A1C ≥6.5%.
- Patients who have received a gene therapy.
- Current use of medications that could potentially lead to changes in intracranial pressure (e.g., levothyroxine, vitamin A supplementation, oral contraceptives, tetracycline, acetazolamide [Diamox]).
- Patients who are currently using strong CYP3A4 inducers or strong CYP3A4 inhibitors, and are unwilling or unable to stop prior to and during the immunosuppression regimen.
See the full eligibility criteria
Who can join
- Male adults aged ≥18 and ≤65 years
- Diagnosed with X-linked adrenoleukodystrophy (ALD)), including proven mutation in the ABCD1 gene through confirmatory genetic testing, and supported by historically elevated VLCFA.
- Clinical evidence of spinal cord involvement with EDSS score between 1 and 4.5 and a pyramidal function score of ≥1 in the Functional System Score of the EDSS. Signs of pyramidal tract dysfunction don't include hyperreflexia.
- Must agree to use double-barrier contraception methods (e.g., condom and 1 other form of contraception for female sexual partners that are of childbearing potential, such as diaphragm, intrauterine device, spermicidal jelly, and/or hormonal contraceptive) and to not donate sperm for at least 6 months following the IMP procedure.
- For patients who are receiving any other treatment for ALD, including off-label medications and/or supplements (e.g., antioxidants, Lorenzo's oil, statins, etc.) or physical rehabilitation support, such treatments must have been at a stable dose and/or frequency for ≥4 weeks prior to Screening and patients must agree to continue at the same dose and/or frequency through Part 1 of the study.
- The patient provided written informed consent prior to any study procedures being performed
What rules you out
- Presence of inflammatory cerebral disease, established by radiographic review of brain MRI demonstrating a Loes score ≥0.5 on the 34-point scale, except for the abnormalities that can be observed in patients with AMN without inflammatory cerebral demyelination with Loes score ≤4.
- Presence of clinically significant active bacterial, viral, fungal, parasitic, or prior - associated infection.
- History of diabetes or abnormal fasting serum glucose (≥126 mg/dL) or hemoglobin A1C ≥6.5%.
- Patients who have received a gene therapy.
- Current use of medications that could potentially lead to changes in intracranial pressure (e.g., levothyroxine, vitamin A supplementation, oral contraceptives, tetracycline, acetazolamide [Diamox]).
- Patients who are currently using strong CYP3A4 inducers or strong CYP3A4 inhibitors, and are unwilling or unable to stop prior to and during the immunosuppression regimen.
- Patients who are currently receiving or have received an investigational drug or procedure within 3 months prior to Screening. The use of investigational drugs is prohibited throughout Part 1 of the study.
- Patients with unstable, clinically significant neurologic (other than AMN), psychiatric, cardiovascular, ophthalmologic, pulmonary, hepatic, renal, metabolic, gastrointestinal, urologic, immunologic, hematopoietic, and/or endocrine disease (other than adrenal insufficiency) and/or other abnormality that may impact the ability to participate in the study or that may potentially confound the study results. It is the responsibility of the Investigator to assess clinical significance; however, consultation with the Sponsor's Medical Monitor may be warranted.
- Patients who, in the opinion of the Investigator, have any other medical or psychological condition or social circumstance that would impair their ability to participate reliably in the assessments, or who may increase the risk to themselves or others by participating.
- Patients who are an immediate family member, study site employee, or are in a dependent relationship with a study team member involved in the conduct of this study (e.g., spouse, parent, child, sibling).
- Pathological changes identified on brain MRI including all lesions from previous diagnosis of inflammatory cerebral disease identified on brain MRI as contrast-enhancing (gadolinium-enhancing) lesion with Loes score ≥ 0.5 on the 34-point scale, except for the abnormalities that can be observed in patients with AMN without inflammatory cerebral demyelination with Loes score ≤4
- 15 years or more have elapsed since the initial onset of myeloneuropathy manifestations, such as walking or running difficulties, bladder dysfunction, increased muscular tone, spasticity, weakness, balance problems, etc.
- Contraindications for MRI procedure and/or contrast materials.
- History of a brain or spinal cord disease that would interfere with lumbar puncture procedures, CSF circulation, and/or safety assessments.
- Contraindication to steroids, sirolimus, tacrolimus, and/or anesthetic medications.
- Contraindication to SBT101 and/or any of its ingredients.
- Unstable adrenal function (e.g., untreated or inappropriately treated adrenal insufficiency). a. Adrenal function will be evaluated through laboratory assessments of cortisol, plasma adrenocorticotropic hormone (ACTH), plasma renin, aldosterone, sodium, and potassium levels at Screening. Monitoring of replacement therapy will be mainly clinical. Determination of inappropriately treated adrenal insufficiency will be made by the Investigator in consultation with the Medical Monitor. b. Patients who meet the following criteria will be considered to have adrenal insufficiency: (i) basal cortisol concentration <275 nmol (10 μg/dL) in the morning (i.e., 6 AM to 10 AM) and (ii) plasma ACTH >2 × upper limit of normal (ULN).
- Positive for human immunodeficiency virus (HIV) type 1 or 2 (HIV-1, HIV-2), hepatitis B virus (HBV), or hepatitis C virus (HCV). a. Patients who have been vaccinated against HBV (e.g., HBV surface antibody-positive) who are negative for other markers of prior HBV infection (e.g., HBV core antibody-negative) are eligible. b. Patients who are positive for anti-HCV antibodies are eligible, as long as they have a negative HCV load as measured by quantitative polymerase chain reaction (qPCR).
The study team makes the final eligibility decision.
Where it's taking place
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.