Adrenoleukodistrophy
Conditions
Interventions
The study consists of two parts after infusion of SBT101:
• Part 1: A 24 month core study period to evaluate the safety and potential
impact of SBT101 on disease progression. Part 1 will consist of
Sponsors
SwanBio Therapeutics, Ltd.
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: 1. Male adults aged >=18 and =1 in the Functional System Score of the EDSS. Signs of pyramidal tract dysfunction don't include hyperreflexia. 4. Must agree to use reliable double-barrier contraception methods (e.g., XML File Identifier: kPpg2r7PH4BP7A3Dw3ckGhWrK7g= Page 10/28 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. 5. 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. 6. The patient provided written informed consent prior to any study procedures being performed
Exclusion criteria
Exclusion criteria: 1. 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 =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 = 2 × upper limit of normal (ULN). 9. 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). 10. Presence of a clinically significant active bacterial, viral, fungal, parasitic, or prion-associated infection. 11. History of diabetes or abnormal fasting plasma glucose (>=126 mg/dL) or hemoglobin A1C >=6.5%. 12. Patients who have received a gene therapy. 13. Current use of medications that could potentially lead to changes in intracranial pressure (e.g., levothyroxine, vitamin A supplementation, oral contraceptives, tetracycline, acetazolamide [Diamox]). 14. 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. 15. 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. 16. Patients with unstable, clinically significant neurologic (other t
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percentage of patients with at least 1 Grade III or Grade IV AE that is at least possibly related to SBT101 at Month 24, as reported by the patients or observed by the Investigator, after SBT101 treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Changes in the 6-minute Walk Test (6MWT) at Month 12 and Month 24 compared to controls, and compared to the reference changes determined by the prospective SBTNHX-CT901 study. • Changes in the 2-minute Walk Test (2MWT) at Month 12 and Month 24 compared to controls, and compared to the reference changes determined by the prospective SBTNHX-CT901 study. • Changes in the 5 times Sit-to-Stand Test (5XSST) at Month 12 and Month 24 compared to controls, and compared to the reference changes determined by the prospective SBTNHX-CT901 study. • Changes in postural body sway amplitudes at Month 12 and Month 24 compared to controls, and compared to the reference changes determined by the prospective SBTNHX-CT901 study. • Proportions of patients with Expanded Disability Status Score (EDSS) of >=5.5 at Month 12 and Month 24 compared to controls, and compared to the reference changes determined by the prospective SBTNHX-CT901 study. • Proportions of patients with increased anti-spasticity medication dose at Month 12 and Month 24 compared to controls, and compared to the reference changes determined by the prospective SBTNHX-CT901 study. • Changes in Timed Up and Go (TUG) at Month 12 and Month 24 compared to controls, and compared to the changes observed in the prospective SBTNHX-CT901 study. • Changes in Dual-task TUG (TUG-DT) at Month 12 and Month 24 compared to controls, and compared to the changes observed in the prospective SBTNHX-CT901 study. • Changes in the Balance Evaluation System Test (miniBESTest) at Month 12 and Month 24 compared to controls, and compared to the changes observed in the prospective SBTNHX-CT901 study. • Changes in the Clinical Global Impression (CGI; performed by a qualified professional) score at Month 12 and Month 24 compared to controls. Changes in the Patient Global Impression of Severity (PGI-S), Patient Global Impression of Severity - Balance (PGI-S Balance), and Patient Global Impression of Change - Bal | — |
Countries
Netherlands
Outcome results
None listed