This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. (NCT02350816), a randomized phase 2 study. The primary outcome is Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Based on Type, Severity and Relationship to Treatment Drug.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02350816 |
| Phase | Phase 2 |
| Condition | Sanfilippo Syndrome, Mucopolysaccharidosis (MPS) |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 17 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Shire |
| Results posted | Yes |
2. Clinical Question
The central clinical question is whether continued or newly initiated HGT-1410 treatment in pediatric patients with MPS Type IIIA is accompanied by treatment-emergent adverse events, and how those events are characterized by seriousness, severity, and relationship to treatment. The registry also identifies developmental and imaging outcomes as secondary measures, but numerical results for those outcomes are not reported in the registry record.
Population
Pediatric patients with mucopolysaccharidosis type IIIA who participated in Study HGT-SAN-093. The safety population was defined as participants who had an intrathecal drug delivery device implanted or received at least one dose of investigational product in the extension study.
Intervention
HGT-1410, administered intrathecally through an indwelling intrathecal drug delivery device. The described regimens used a dose of 45 mg, given every 2 weeks or every 4 weeks, depending on group.
Comparator
No untreated or placebo comparator is described for the extension. Participants who had previously received HGT-1410 continued treatment, while participants previously assigned to no treatment began HGT-1410 in the extension.
Primary question
How many participants experienced treatment-emergent adverse events, characterized by event type, severity, and relationship to the investigational product, device, or procedure?
This is therefore chiefly a safety question in an extension setting. It differs from a controlled comparison of treatment against no treatment: the described extension groups received active treatment, and the registry does not report a concurrent untreated group. The record lists changes in Vineland Adaptive Behavior Scales Second Edition scores, developmental quotient assessed by neurocognitive tests, and total cortical grey matter volume as secondary outcomes. Those measures indicate areas of interest, but without posted outcome estimates they do not answer whether, or by how much, those measures changed.
3. Trial Design
This was a Phase 2, randomized, parallel-group, open-label extension study. The registry lists an enrollment of 17 and identifies pediatric patients with Sanfilippo Syndrome or mucopolysaccharidosis as the condition population. The study began on 2015-04-08, had a primary completion date of 2019-04-12, and is listed as terminated. The reason for termination is not reported in the registry record.
The extension followed Study HGT-SAN-093. Participants previously assigned to HGT-1410 every 2 weeks or every 4 weeks continued their respective regimens. Group 1 continued 45 mg every 2 weeks starting at Week 50, and Group 2 continued 45 mg every 4 weeks starting at Week 52. Both continuation groups were described as having a cumulative treatment period of up to 42 months (168 weeks). Administration was intrathecal through an indwelling device.
Participants previously assigned to no treatment in the earlier study began HGT-1410 in the extension. Group 3A began 45 mg every 2 weeks, and Group 3B began 45 mg every 4 weeks, with treatment starting at Week 0 of the extension and described as lasting up to 30 months. The descriptions state that these participants would receive a device and be randomized in a 1:1 allocation ratio to the two schedules. More detail about the randomization procedure is not reported in the registry record.
Masking was none, so participants and study personnel were not described as blinded. The design supports descriptive reporting of safety during treatment, but the extension does not provide a concurrent untreated comparison in the posted group descriptions. No protocol-level rationale for the design, sample-size justification, or planned statistical analysis is reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: A total of 17 participants were enrolled and completed treatment period in the study.
Overall Study
| Stage | Group 1 | Group 2 | Group 3A | Group 3B |
|---|---|---|---|---|
| STARTED | 7 | 6 | 2 | 2 |
| COMPLETED | 7 | 6 | 2 | 2 |
| NOT COMPLETED | 0 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Safety population consisted of all participants who had the IDDD implant or received at least 1 dose of investigational product in the extension study (SHP610-201 \[NCT02350816\]).
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
Population: Safety population consisted of all participants who had the IDDD implant or received at least 1 dose of investigational product in the extension study (SHP610-201 \[NCT02350816\]).
| Characteristic | Unit | Group 1 | Group 2 | Group 3A | Group 3B | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 7 | 6 | 2 | 2 | 17 | |
| Age, Continuous | Months | 29.64 (SD 9.989) | 31.62 (SD 8.598) | 40.40 (SD 5.798) | 36.95 (SD 7.142) | 32.47 (SD 8.941) |
| Sex: Female, Male: Female | Participants | 5 | 3 | 2 | 0 | 10 |
| Sex: Female, Male: Male | Participants | 2 | 3 | 0 | 2 | 7 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 1 | 0 | 0 | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 7 | 5 | 2 | 2 | 16 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 7 | 6 | 2 | 2 | 17 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Based on Type, Severity and Relationship to Treatment Drug, measured over From start of study drug administration up to follow-up (Week 276).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants with treatment-emergent adverse events (TEAEs), with events characterized by type, severity, and relationship to treatment. The registry defines an adverse event as an untoward medical occurrence during a clinical investigation that does not necessarily have a causal relationship with the treatment. TEAEs were categorized by seriousness, including serious or life-threatening events; by severity, including mild, moderate, or severe events; and by relationship to the investigational product, the intrathecal drug delivery device, the device surgical procedure, or intrathecal administration of HGT-1410.
Counting affected participants is a direct way to describe how many people in a safety population had at least one event in a specified category. It can provide a practical overview of safety experience, while event details help distinguish the kinds of occurrences being reported. A participant count is not the same as an event count: one participant may experience more than one event, and the registry’s endpoint is framed as participants with events.
The stated timeframe is from the start of study-drug administration up to follow-up at Week 276. The endpoint definition also describes collection from initial device implantation or first dose if earlier in either the earlier study or extension, through a stated data cutoff, with an additional follow-up rule for early termination. These definitions describe the intended safety accounting; they do not establish that every participant was followed for the same duration. Exposure duration by participant is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted safety results show variation in the number of participants reported with serious adverse events across the four groups. Group 1 had 6 of 7 participants affected, Group 2 had 6 of 6, Group 3A had 0 of 2, and Group 3B had 1 of 2. For other adverse events, the reported counts were 7 of 7 in Group 1, 6 of 6 in Group 2, 2 of 2 in Group 3A, and 2 of 2 in Group 3B. These are descriptive counts, not estimates adjusted for differences in treatment duration, prior treatment, or other characteristics.
There were 0 deaths reported in each group. The record also lists serious-event terms with percentages: cerebrospinal fluid leakage, 66.7%; pneumonia aspiration, 50.0%; implant site extravasation, 28.6%; leukopenia, 16.7%; vomiting, 16.7%; and device breakage, 16.7%. These percentages should be read as the posted event summaries, not as evidence of a comparison between treatment schedules. The record does not provide the group assignment for each listed term in this summary, nor does it provide the event-level data needed to assess overlap among terms or repeated events in an individual participant.
The groups are small, and some reported group counts are based on only 2 participants. With small denominators, a change in the event experience of a single participant can substantially alter a group’s descriptive proportion. This makes apparent differences between groups imprecise and sensitive to individual observations. The registry does not report confidence intervals, hypothesis tests, or a statistical comparison between groups, so the counts do not establish that one schedule has a higher or lower event risk than another.
The groups also differ in their treatment history and when treatment began in the extension. Groups 1 and 2 continued treatment received in the earlier study, whereas Groups 3A and 3B began treatment in the extension after previously receiving no treatment. These pathways are not interchangeable. The posted counts alone cannot separate effects associated with the schedule, prior treatment, duration of exposure, device implantation, or the underlying clinical course.
The extension was open-label and has no described untreated comparison group. Thus, its safety summaries can describe events occurring during the study’s defined observation period, but cannot by themselves establish that an event was caused by HGT-1410 or the device. The endpoint definition explicitly notes that an adverse event need not have a causal relationship with treatment. No numerical results for the listed developmental or imaging outcomes are reported in the registry record, so the posted safety findings do not provide an estimate of efficacy.
9. Statistical Methods Explained
The registry reports participant counts rather than a comparative effect estimate for the primary safety endpoint. For each group, the count of participants affected is presented alongside the number at risk. This makes the denominator visible and helps readers understand how much information supports each count. The groups are small, so the descriptive summaries should not be treated as precise estimates of event frequency in a wider population.
What does a participant count tell us?
A count of participants with an event answers how many people experienced at least one event in the reporting category. It does not necessarily tell how many events occurred, because one participant may have more than one event. Nor does it indicate how long each participant was observed or exposed to treatment. Event rates accounting for person-time would require information about duration of exposure and follow-up, which is not reported in the registry record.
Seriousness and severity are different classifications. Seriousness identifies events meeting a regulatory or clinical seriousness category, while severity describes the intensity of an event. The endpoint description lists serious and life-threatening event types as seriousness categories, and mild, moderate, and severe as severity categories. A serious event is not automatically severe, and a severe event is not automatically classified as serious. The registry summaries do not provide the full cross-classification of these categories by participant.
Why is a confidence interval useful?
In general, a confidence interval expresses uncertainty around an estimate and indicates which values are reasonably compatible with the observed data under the method used. It is especially informative when the sample is small, because a point estimate alone can give a misleading impression of precision. No confidence intervals for the group safety results are reported in the registry record. Accordingly, the counts should be treated as descriptive observations rather than precise estimates of underlying risks.
In general, a hypothesis test assesses how compatible observed data are with a specified statistical model or null hypothesis. A p-value is not a measure of clinical importance, and it does not establish causation. The record reports no statistical comparison or p-value for the primary endpoint. The presence of different counts among groups should therefore not be interpreted as a statistically established difference.
Why does treatment exposure matter?
Participants may have different periods of exposure and follow-up. Groups 1 and 2 continued treatment that had begun in the earlier study, while Groups 3A and 3B initiated treatment in the extension. The endpoint’s timeframe refers to a long follow-up period, but individual exposure durations are not reported in the registry record. A raw count does not adjust for unequal time at risk. In general, a comparison intended to account for different observation times would need an appropriate time-to-event or rate-based method and sufficient information about follow-up. No such analysis is reported in the registry record.
In general, time-to-event methods can account for differing observation periods and censoring, but they rely on documented event times and follow-up status. No time-to-event analysis is reported for these adverse-event summaries. Likewise, a regression model could adjust for measured participant or treatment characteristics, but the registry does not report such a model or its inputs. It is not possible to infer that an adjustment was performed.
What can the design establish?
Randomization is listed for the study, and the descriptions specify random assignment between the two treatment schedules for participants beginning therapy in Groups 3A and 3B. However, the extension includes participants continuing regimens assigned in the earlier study as well as participants starting treatment after no treatment. The posted safety summaries are not presented as a randomized comparison of all four groups. In addition, the study was open-label, and no untreated comparison group is described in the extension. These features limit interpretation of between-group differences as treatment effects.
The registry’s planning assumptions describe sample-size calculations as illustrations based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The intended effect is described as a fraction of the observed effect. Such planning calculations are not observed trial outcomes, do not constitute observed power, and do not establish that the study was designed or powered for a particular comparison. No numerical planning estimates are reported in the registry record. The record does not report the study’s own sample-size justification or statistical plan.
Overall, the statistical evidence available for the primary endpoint is descriptive: group-level participant counts, at-risk counts, adverse-event terms, and deaths. It does not include uncertainty intervals or an inferential analysis that quantifies a comparative treatment effect.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists several secondary outcomes: change from baseline in Vineland Adaptive Behavior Scales Second Edition scores, change from baseline in developmental quotient assessed by neurocognitive tests, and change from baseline in total cortical grey matter volume. It also identifies 9 outcome measures in the record. When a study examines multiple outcomes, there are multiple opportunities to observe apparently notable results by chance. In general, a prespecified approach to multiplicity can help define how such results should be interpreted. The multiplicity strategy is not reported in the registry record.
No numerical efficacy results or statistical analyses for the secondary outcomes are reported in the registry record. The record also reports no inferential analysis for the primary safety endpoint. It is therefore not possible to determine which analyses, if any, were planned, whether analyses were adjusted for multiple outcomes, or how missing outcome measurements were handled. A missing-data method, imputation approach, or sensitivity analysis is not reported in the registry record.
The safety population is defined as participants who had an intrathecal drug delivery device implanted or received at least one dose of investigational product in the extension. The posted adverse-event groups include at-risk counts, but the registry does not report additional analysis populations for efficacy outcomes or explain how participants with incomplete measurements were treated. The number randomized, treated, and included in each outcome analysis is not reported in the registry record beyond the group-level safety counts.
In general, analysis populations matter because different inclusion rules can change the interpretation of an estimate. Missing outcomes can also make results less representative if the reasons for missingness are related to health status or treatment experience. Without an account of missing observations, participant flow, and analysis rules, no assumption should be made about how complete or comparable the outcome data were.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Group 1 | 6 | 7 | 85.7% |
| Group 2 | 6 | 6 | 100.0% |
| Group 3A | 0 | 2 | 0.0% |
| Group 3B | 1 | 2 | 50.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Group 1 | 0 of 7 |
| Group 2 | 0 of 6 |
| Group 3A | 0 of 2 |
| Group 3B | 0 of 2 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Group 1 | 7 of 7 |
| Group 2 | 6 of 6 |
| Group 3A | 2 of 2 |
| Group 3B | 2 of 2 |
Most frequent serious adverse event terms
| Event term | Organ system | Group 1 | Group 2 | Group 3A | Group 3B |
|---|---|---|---|---|---|
| Cerebrospinal fluid leakage | Nervous system disorders | 2 of 7 | 4 of 6 | 0 of 2 | 0 of 2 |
| Device failure | General disorders | 1 of 7 | 1 of 6 | 0 of 2 | 0 of 2 |
| Implant site extravasation | General disorders | 2 of 7 | 0 of 6 | 0 of 2 | 0 of 2 |
| Leukopenia | Blood and lymphatic system disorders | 0 of 7 | 1 of 6 | 0 of 2 | 0 of 2 |
| Vomiting | Gastrointestinal disorders | 0 of 7 | 1 of 6 | 0 of 2 | 0 of 2 |
| Adverse drug reaction | General disorders | 1 of 7 | 0 of 6 | 0 of 2 | 0 of 2 |
| Device breakage | General disorders | 0 of 7 | 1 of 6 | 0 of 2 | 0 of 2 |
| Implant site pain | General disorders | 1 of 7 | 0 of 6 | 0 of 2 | 0 of 2 |
| Pyrexia | General disorders | 0 of 7 | 1 of 6 | 0 of 2 | 0 of 2 |
| Central nervous system infection | Infections and infestations | 0 of 7 | 1 of 6 | 0 of 2 | 0 of 2 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Group 1 | Group 2 | Group 3A | Group 3B |
|---|---|---|---|---|---|
| Pyrexia | General disorders | 6 of 7 | 3 of 6 | 2 of 2 | 2 of 2 |
| Upper respiratory tract infection | Infections and infestations | 6 of 7 | 2 of 6 | 1 of 2 | 1 of 2 |
| Vomiting | Gastrointestinal disorders | 5 of 7 | 3 of 6 | 0 of 2 | 1 of 2 |
| Rhinitis | Infections and infestations | 5 of 7 | 1 of 6 | 1 of 2 | 0 of 2 |
| Fall | Injury, poisoning and procedural complications | 4 of 7 | 1 of 6 | 1 of 2 | 1 of 2 |
| Ear infection | Infections and infestations | 0 of 7 | 4 of 6 | 1 of 2 | 1 of 2 |
| Procedural pain | Injury, poisoning and procedural complications | 5 of 7 | 0 of 6 | 0 of 2 | 1 of 2 |
| Diarrhoea | Gastrointestinal disorders | 3 of 7 | 1 of 6 | 1 of 2 | 0 of 2 |
| Gastroenteritis | Infections and infestations | 3 of 7 | 1 of 6 | 0 of 2 | 1 of 2 |
| Otitis media | Infections and infestations | 4 of 7 | 1 of 6 | 0 of 2 | 0 of 2 |
Serious events with the highest arm-level rate: cerebrospinal fluid leakage (66.7%), pneumonia aspiration (50.0%), implant site extravasation (28.6%), leukopenia (16.7%), vomiting (16.7%).
For serious adverse events, the registry reports Group 1 as 6 participants affected among 7 at risk, Group 2 as 6 among 6, Group 3A as 0 among 2, and Group 3B as 1 among 2. The record reports 0 deaths in each group. For other adverse events, it reports 7 among 7 in Group 1, 6 among 6 in Group 2, 2 among 2 in Group 3A, and 2 among 2 in Group 3B.
These figures count participants, not necessarily the number of events. They do not show how many events any participant experienced, how long each participant was followed, or whether participants experienced events in more than one category. The listed serious-event terms and percentages are cerebrospinal fluid leakage, 66.7%; pneumonia aspiration, 50.0%; implant site extravasation, 28.6%; leukopenia, 16.7%; vomiting, 16.7%; and device breakage, 16.7%. The registry summary does not specify the group for each term or show how the terms overlap.
The small group denominators make these summaries particularly sensitive to individual cases. They describe what was recorded in this study, not a stable estimate of risk for other patients. The registry does not report an adjusted comparison or causal attribution for the events.
12. What the Result Does, and Does Not, Mean
The posted results provide a descriptive account of adverse events recorded in an open-label extension of HGT-SAN-093. They show participant counts for serious and other adverse events in each of the four groups, report no deaths in any group, and list several serious-event terms with percentages. These observations are relevant to understanding what was recorded among participants in the extension’s safety population.
They do not show that HGT-1410 caused every event. The registry definition explicitly allows an adverse event to occur without a causal relationship to treatment, and the endpoint separately considered relationship to the drug, device, procedure, or administration. The posted group counts do not establish those causal relationships. Nor do the counts establish that one dosing schedule is safer than another: there is no reported comparative analysis, confidence interval, or statistical test, and some groups have very small denominators.
The results also do not establish efficacy. The registry names developmental and imaging measures as secondary outcomes, but numerical results for those measures are not reported in the registry record. Without those results, no conclusion about change in developmental quotient, adaptive behavior, or cortical grey matter volume can be drawn from the posted information.
The extension’s design and treatment histories further limit causal interpretation. Some participants continued treatment from the earlier study, while others began treatment in the extension after previously receiving no treatment. There is no untreated extension group described, and masking was none. The group summaries cannot separate treatment schedule from prior treatment, duration of exposure, device-related factors, or other differences among participants.
In general, results from a small extension study can help characterize events observed during treatment and follow-up, but they cannot alone answer every question about comparative safety, long-term risk, or benefit. Those broader questions require appropriate comparisons, complete outcome reporting, and analyses that account for uncertainty and follow-up. Such analyses are not reported in the registry record.
13. Why This Trial Matters Statistically
This study is statistically informative in part because it illustrates the distinction between a safety description and a comparative treatment effect. The primary endpoint is framed as the number of participants with treatment-emergent adverse events, a useful way to organize safety observations. Yet a set of participant counts, even when displayed by group, is not automatically a randomized comparison. Interpretation depends on how groups were formed, how long participants were observed, and what analyses were conducted.
The extension combines two treatment histories: continuation of schedules from the earlier study and initiation of treatment among participants previously assigned to no treatment. That structure matters because the groups may differ in prior exposure and timing. The open-label design and absence of a described untreated extension group also mean that the observed event counts cannot isolate the effect of HGT-1410 from other explanations. These are design considerations, not reasons to dismiss the descriptive information.
The small group counts show why uncertainty matters. A summary can be factually accurate while still giving an imprecise picture of event frequency beyond the participants observed. Confidence intervals and suitable comparative methods would help communicate uncertainty, but they are not reported in the registry record. The posted summaries should therefore be read as counts from this study rather than as definitive estimates of population risk.
The record also lists clinically relevant secondary outcome domains, while providing no numerical efficacy results. This distinction highlights the importance of complete results reporting: naming an endpoint does not reveal its observed change or the uncertainty around it. Overall, the study is useful for understanding the registry’s safety reporting and the limits of descriptive evidence in a small extension study. It does not, from the posted results alone, resolve comparative safety or efficacy questions.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
Several trial-specific limitations affect interpretation. First, the registry lists the study as terminated, but the reason for termination is not reported in the registry record. It is also not reported whether termination changed planned follow-up or outcome collection. The listed primary completion date does not by itself describe individual participant follow-up.
Second, participant exposure differs by group history. Groups 1 and 2 continued schedules from the earlier study, whereas Groups 3A and 3B began treatment in the extension. The summaries do not provide participant-level exposure duration, time at risk, or the timing of individual adverse events. Group counts therefore cannot be adjusted for unequal observation or exposure.
Third, the primary endpoint definition spans the earlier study and the extension and includes specific data-cutoff and early-termination follow-up rules, while the stated timeframe refers to follow-up up to Week 276. The registry does not provide enough detail to reconcile each participant’s actual observation window with these descriptions. The reason for including events across the earlier study in the endpoint definition is not reported in the registry record.
Fourth, the record does not report confidence intervals, inferential comparisons, detailed event-level data, missing-data methods, or an analysis plan. It also does not provide numerical results for the secondary developmental and imaging outcomes. These omissions prevent assessment of precision, adjusted group differences, and the completeness of efficacy reporting. The available safety counts remain useful as descriptive observations, but should not be interpreted beyond their scope.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. — NCT02350816.
- Registry record last updated 2021-06-11; status last verified 2021-05.
- Results first posted 2020-04-20.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
NCT02350816 is a Phase 2, randomized, parallel-group, open-label extension study of HGT-1410 in pediatric patients with MPS Type IIIA who participated in Study HGT-SAN-093. The registry lists enrollment of 17, a start date of 2015-04-08, a primary completion date of 2019-04-12, and terminated status.
The described regimens used intrathecal administration through an indwelling device, with 45 mg given every 2 weeks or every 4 weeks. The primary endpoint counted participants with treatment-emergent adverse events. Group-level safety counts and serious-event terms are reported, with 0 deaths in each group. Numerical results for the listed secondary outcomes and inferential statistical analyses are not reported in the registry record.