Trial · NCT04088734
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
NCT04088734, Phase 1, Phase 2, Terminated, Ultragenyx Pharmaceutical Inc; results first posted 2023-07-25
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease. Trials, record NCT04088734. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04088734
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
- NCT number
- NCT04088734
- Status
- Terminated
- Phases
- Phase 1, Phase 2
- Enrollment
- 5 participants, actual
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Conditions
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
- Interventions
- ABO-102
- First posted
- 2019-09-13
- Last updated
- 2023-07-25
- Other registries' IDs
- eudract:2018-000504-42
- Results first posted
- 2023-07-25
Source ClinicalTrials.gov
Registered outcomes22
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame | From the first dose of study drug to <30 days postdose, Day 30, 60, 90, 180 and Month 12 | An adverse event (AE) is any untoward medical occurrence or unintended change from the time informed consent form (ICF) is signed, including inter-current illness that occurs during the course of a clinical trial after treatment has started, whether considered related to treatment or not. TEAEs are those that occurred after the start of study drug. Related adverse events were categorized as possible, probable, or definitely. |
| Primary | Incidence, Type and Severity of Serious Adverse Events (SAEs) by Time Frame | From signing of informed consent through Day 60, 90, 180 and up to Day 454 (> 12 months) | An SAE is defined as any untoward medical occurrence that, at any dose: 1. Results in death 2. Is life threatening 3. Requires inpatient hospitalization or prolongation of existing hospitalization 4. Results in persistent disability/incapacity 5. Is a congenital anomaly/birth defect 6. Other situations such as important medical events that may not be immediately life threatening or result in death or hospitalization but may jeopardize the participant or may require medical or surgical intervention to prevent one of the other outcomes listed in the above definition. Relationship to study drug was defined as unrelated, unlikely, possible, probable, or definitely. |
| Primary | Change From Baseline (BL) in Multiples of Normal of Liver and Spleen Volumes After Treatment | Baseline, Day 30, 180, Month 12 | As Measured by Magnetic Resonance Imaging (MRI). Baseline value of multiple of normal is calculated using the baseline values of the Liver volume/Spleen Volume/Height and Weight. * Body Surface Area (BSA) (m2)=( Height(cm) \* Weight (kg)/3600)1/2. * Normal Liver Volume=(689.9 \* BSA (m)) - 24.7. * Normal Spleen Volume (mL)=(4.6 \* Weight (kg)) + 0.7. * Liver Volume (multiples of normal)=Subject Liver Volume (mL)/Normal Liver Volume (mL). * Spleen Volume (multiples of normal)=Subject Spleen Volume (mL)/Normal Spleen Volume (mL). |
| Primary | Change From BL in Cerebrospinal Fluid (CSF) Heparan Sulfate Levels After Treatment | Baseline, Day 30, Day 180, Month 12 | Change from baseline in CSF heparan sulfate levels after treatment |
| Secondary | Change From Baseline in Plasma Heparan Sulfate After Treatment | Baseline, Day 30, Day 180, Month 12 | |
| Secondary | Change From Baseline in Urine Glycosaminoglycans After Treatment | Baseline, Day 30, Day 180, Month 12 | |
| Secondary | Change From Baseline in Urine Heparan Sulfate After Treatment | Baseline, Day 30, Day 180, Month 12 | |
| Secondary | Change From Baseline in CSF N-Sulfoglucosamine Sulfohydrolase (SGSH) Enzyme Activity Levels After Treatment | Baseline, Day 30, Day 180, and Month 12 | |
| Secondary | Change From Baseline in Heparan N-Sulfatase (Type A) After Treatment | Baseline, Day 30, Day 180, Month 12 | |
| Secondary | Change From Baseline in Plasma SGSH After Treatment | Baseline, Day 30, Day 180, Month 12 |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 registered outcomesOutcomes22
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame | From the first dose of study drug to <30 days postdose, Day 30, 60, 90, 180 and Month 12 | participants | NUMBER | |
| PRIMARY | Incidence, Type and Severity of Serious Adverse Events (SAEs) by Time Frame | From signing of informed consent through Day 60, 90, 180 and up to Day 454 (> 12 months) | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Change From Baseline (BL) in Multiples of Normal of Liver and Spleen Volumes After Treatment | Baseline, Day 30, 180, Month 12 | Participants with an assessment at baseline and given time point | ratio | MEAN |
| PRIMARY | Change From BL in Cerebrospinal Fluid (CSF) Heparan Sulfate Levels After Treatment | Baseline, Day 30, Day 180, Month 12 | Participants with an assessment at given time point | nmol/mL | MEAN |
| SECONDARY | Change From Baseline in Plasma Heparan Sulfate After Treatment | Baseline, Day 30, Day 180, Month 12 | Participants with an assessment at given time point | pmol/mL | MEAN |
| SECONDARY | Change From Baseline in Urine Glycosaminoglycans After Treatment | Baseline, Day 30, Day 180, Month 12 | Participants with an assessment at given time point | mg/mmol creatinine | MEAN |
| SECONDARY | Change From Baseline in Urine Heparan Sulfate After Treatment | Baseline, Day 30, Day 180, Month 12 | Participants with an assessment at given time point | μmol/mmol creatinine | MEAN |
| SECONDARY | Change From Baseline in CSF N-Sulfoglucosamine Sulfohydrolase (SGSH) Enzyme Activity Levels After Treatment | Baseline, Day 30, Day 180, and Month 12 | Participants with an assessment at given time point | nmol/17 h/mL | MEAN |
| SECONDARY | Change From Baseline in Heparan N-Sulfatase (Type A) After Treatment | Baseline, Day 30, Day 180, Month 12 | Participants with an assessment at given time point | nmol/17 h/mL | MEAN |
| SECONDARY | Change From Baseline in Plasma SGSH After Treatment | Baseline, Day 30, Day 180, Month 12 | Participants with an assessment at given time point | nmol/17 h/mL | MEAN |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 outcomesResults by group194
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame | scAAV9.U1a.hSGSH, 3x1013 vg/kg | < 30 days : Possible; Mild | 1 | participants | |
| Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame | scAAV9.U1a.hSGSH, 3x1013 vg/kg | < 30 days : Possible; Moderate | 0 | participants | |
| Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame | scAAV9.U1a.hSGSH, 3x1013 vg/kg | < 30 days : Possible; Severe | 0 | participants | |
| Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame | scAAV9.U1a.hSGSH, 3x1013 vg/kg | < 30 days : Probable; Mild | 2 | participants | |
| Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame | scAAV9.U1a.hSGSH, 3x1013 vg/kg | < 30 days : Probable; Moderate | 0 | participants | |
| Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame | scAAV9.U1a.hSGSH, 3x1013 vg/kg | < 30 days : Probable; Severe | 1 | participants | |
| Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame | scAAV9.U1a.hSGSH, 3x1013 vg/kg | < 30 days : Definitely; Mild | 1 | participants | |
| Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame | scAAV9.U1a.hSGSH, 3x1013 vg/kg | < 30 days : Definitely; Moderate | 0 | participants | |
| Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame | scAAV9.U1a.hSGSH, 3x1013 vg/kg | < 30 days : Definitely; Severe | 0 | participants | |
| Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 30 - < 60 days : Possible; Mild | 0 | participants |
1 to 10 of 194Source ClinicalTrials.gov
See all 194 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 5 | |
| Overall Study | COMPLETED | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 0 | |
| Overall Study | NOT COMPLETED | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 5 |
Source ClinicalTrials.gov
Adverse events54
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Neutropenia | Blood and lymphatic system disorders | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 1 | 5 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 1 | 5 | |
| other | Mixed deafness | Ear and labyrinth disorders | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 1 | 5 | |
| other | Cushingoid | Endocrine disorders | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 1 | 5 | |
| other | Constipation | Gastrointestinal disorders | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 1 | 5 | |
| other | Diarrhoea | Gastrointestinal disorders | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 1 | 5 | |
| other | Dysphagia | Gastrointestinal disorders | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 1 | 5 | |
| other | Nausea | Gastrointestinal disorders | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 2 | 5 | |
| other | Vomiting | Gastrointestinal disorders | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 4 | 5 | |
| other | Gait disturbance | General disorders | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 1 | 5 |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 0 | 5 |
| Total, other adverse events | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 5 | 5 |
| Total, serious adverse events | scAAV9.U1a.hSGSH, 3x1013 vg/kg | 3 | 5 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04088734
GET https://conductscience.org/api/trials/v1/NCT04088734/registered?page=2