A Phase 1/2 interventional study of SBT101 Treatment in AMN, AMN Gene Mutation and X-ALD, sponsored by SwanBio Therapeutics, Inc.. Terminated at 2 sites in 2 countries. Open to male participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-08-26.
Sponsored by SwanBio Therapeutics, Inc. · Phase 1/2, Interventional, and Treatment
This is a Phase 1/2 randomized, blinded, dose-escalation study to evaluate the safety and efficacy of intrathecal (IT) administration of SBT101, a recombinant adeno-associated virus serotype 9 (AAV9) containing a functional copy of the human adenosine triphosphate (ATP)-binding cassette transporter subfamily D member 1 (ABCD1; hABCD1) gene, in adult patients with adrenomyeloneuropathy (AMN) aged 18-65 years.
Patients will receive a single dose of SBT101 via IT route (or an imitation procedure) and will be followed for safety and efficacy for 2 years. Patients receiving SBT101 will be followed for an additional 3 years (5 total) for Safety. Patients receiving an imitation procedure will be offered the opportunity to receive SBT101 after 2 years, as data indicate.
The study consists of two parts after infusion of SBT101:
Part 1: A blinded 24-month core study period to evaluate the safety and potential impact of SBT101 on disease progression. Part 1 will consist of 2 phases:
Phase 1: Dose-Escalation Phase: Two (2) doses of SBT101 (Dose level 1 cohort and Dose Level 2 cohort) will be evaluated to establish the maximum tolerated dose (MTD).
Phase 2: Dose-Expansion Phase: Additional patients will be enrolled to receive SBT101 at the MTD
Part 2: An unblinded 3-year long-term safety follow-up period with annual follow-up visits to evaluate the safety of SBT101 and disease progression.
68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.
This study's enrollment of 9 is below the median of 31 across 40 interventional studies indexed under Adrenoleukodystrophy.
Browse Adrenoleukodystrophy studies →SwanBio Therapeutics, Inc. is the lead sponsor of 2 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients treated with SBT101 (1.0E14 vector genomes)
Genetic: SBT101 Treatment
Patients treated with SBT101 (3.0E14 vector genomes).
Genetic: SBT101 Treatment
SBT101 Treatment
Adverse Events to SBT101. Any Serious TEAE. Any Serious TEAE Related to the Study Procedure or Study Drug.
Any Treatment Emergent Adverse Event (TEAE). TEAE was defined as any adverse event which started during or after the administration of IMP or the immunosuppressant pre-medication.
Time frame: 2 years
| Milestone | Dose Level 1 | Dose Level 2 |
|---|---|---|
| Started | 4 | 5 |
| Enrolled at study closure | 3 | 5 |
| Completed | 3 | 5 |
| Not completed | 1 | 0 |
| Withdrew: Death | 1 | 0 |
Any Treatment Emergent Adverse Event (TEAE). TEAE was defined as any adverse event which started during or after the administration of IMP or the immunosuppressant pre-medication.
| Participants | Cohort 1 - Dose Level 1 | Cohort 2 - Dose Level 2 |
|---|---|---|
| Any TEAE | 4 | 4 |
| Any Grade 3 or 4 TEAE related to study procedure or study drug | 2 | 0 |
Collected over From enrollment until 30 days after the patient's last visit (up to 24 months).. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1 - Dose Level 1 | 1/4 (25%) | 3/4 (75%) | 4/4 (100%) |
| Cohort 2 - Dose Level 2 | 0/4 (0%) | 2/4 (50%) | 4/4 (100%) |
| Event | Cohort 1 - Dose Level 1 | Cohort 2 - Dose Level 2 |
|---|---|---|
| GastroenteritisInfections and infestations | 1/4 | 0/4 |
| PyelonephritisInfections and infestations | 1/4 | 0/4 |
| Adrenocortical insufficiency acuteEndocrine disorders | 0/4 | 1/4 |
| Muscular weaknessMusculoskeletal and connective tissue disorders | 1/4 | 0/4 |
| Myelitis transverseNervous system disorders | 1/4 | 0/4 |
| AspirationRespiratory, thoracic and mediastinal disorders | 1/4 | 0/4 |
| Deep vein thrombosisVascular disorders | 0/4 | 1/4 |
| Event | Cohort 1 - Dose Level 1 | Cohort 2 - Dose Level 2 |
|---|---|---|
| Nervous system disordersMusculoskeletal and connective tissue disorders | 4/4 | 4/4 |
| HeadacheNervous system disorders | 3/4 | 4/4 |
| ParaesthesiaNervous system disorders | 0/4 | 4/4 |
| Muscular weaknessMusculoskeletal and connective tissue disorders | 2/4 | 3/4 |
| Micturition urgencyRenal and urinary disorders | 3/4 | 2/4 |
| FatigueGeneral disorders | 1/4 | 3/4 |
| Back painMusculoskeletal and connective tissue disorders | 2/4 | 2/4 |
| Musculoskeletal stiffnessMusculoskeletal and connective tissue disorders | 2/4 | 2/4 |
| Muscle spasmsMusculoskeletal and connective tissue disorders | 0/4 | 2/4 |
| HypoaesthesiaNervous system disorders | 1/4 | 2/4 |
Safety Analysis Set (all participants who received any amount of SBT101 as part of the study).
| Age, Continuous(Years) | Cohort 1 - Dose Level 1 | Cohort 2 - Dose Level 2 | Total |
|---|---|---|---|
| Mean | 28.3 ± 7.41 | 35 ± 1.41 | 31.6 ± 6.12 |
| Sex: Female, Male(Participants) | Cohort 1 - Dose Level 1 | Cohort 2 - Dose Level 2 | Total |
|---|---|---|---|
| Female | 0 | 0 | 0 |
| Male | 4 | 4 | 8 |
| Race (NIH/OMB)(Participants) | Cohort 1 - Dose Level 1 | Cohort 2 - Dose Level 2 | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 1 | 0 | 1 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 3 | 4 | 7 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1 - Dose Level 1 | Cohort 2 - Dose Level 2 | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 |
| Not Hispanic or Latino | 4 | 4 | 8 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Confirmed Mutation in the ABCD1 Gene(Participants) | Cohort 1 - Dose Level 1 | Cohort 2 - Dose Level 2 | Total |
|---|---|---|---|
| Count of participants | 4 | 4 | 8 |
| Adrenal Insufficiency(Participants) | Cohort 1 - Dose Level 1 | Cohort 2 - Dose Level 2 | Total |
|---|---|---|---|
| Yes | 3 | 2 | 5 |
| No | 1 | 2 | 3 |
| Time Since AMN Diagnosis(Years) | Cohort 1 - Dose Level 1 | Cohort 2 - Dose Level 2 | Total |
|---|---|---|---|
| Mean | 2.41 ± 1.720 | 1.74 ± 0.811 | 2.14 ± 1.333 |
| Baseline EDSS(Score on a scale) | Cohort 1 - Dose Level 1 | Cohort 2 - Dose Level 2 | Total |
|---|---|---|---|
| Mean | 3.25 ± 0.645 | 2.88 ± 0.854 | 3.06 ± 0.729 |
PDFs hosted by the registry, ClinicalTrials.gov.
This study is terminated, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.
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SwanBio Therapeutics, Inc.