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Comparison · NCT02534844-0cc100f6

Adrabetadex vs Sham Control: Parts A/B: Number of Participants Treated for at Least 6 Months Who Qualified for the Rescue Option

Calculated by ConductScience from the counts the trial posted to ClinicalTrials.gov.

2 sourcestrial-comparisons-2026-03-23-0.1.0

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ConductScience Library. Adrabetadex vs Sham Control: Parts A/B: Number of Participants Treated for at Least 6 Months Who Qualified for the Rescue Option. Trial comparisons, record NCT02534844-0cc100f6. Release trial-comparisons-2026-03-23-0.1.0, 23 Mar 2026. https://conductscience.org/library/comparisons/NCT02534844-0cc100f6

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Measures6

Calculated by ConductScience from the counts the trial posted to ClinicalTrials.gov.

MeasureValue95% interval, lower95% interval, upperMethodNote
Risk in group 10.05260.01460.173wilson
Risk in group 20.1110.0310.328wilson
Risk difference, group 1 minus group 2-0.0585-0.2790.086newcombe hybrid score
Risk ratio, group 1 over group 20.4740.07243.1log
Odds ratio, group 1 over group 20.4440.05743.44log
Number needed to treat, for one fewer participant with the outcome on Adrabetadex than on Sham Control17.1from the risk difference intervalthe interval includes no difference

Groups2

Group 2 names the comparator: sham.

GroupTitleDescriptionWith the outcomeAnalysedClinicalTrials.gov code
Group 1AdrabetadexParticipants received lumbar IT infusions of adrabetadex once every 2 weeks for a total of 52 weeks.238OG001
Group 2Sham ControlParticipants received sham control procedures once every 2 weeks for a total of 52 weeks.218OG000

Outcome

Type
SECONDARY
Title
Parts A/B: Number of Participants Treated for at Least 6 Months Who Qualified for the Rescue Option
Description
Participants who manifested significant disease progression according to predefined clinical criteria after treatment of 26 weeks or more had the option to rescue. Number of participants who qualified for the rescue option following a minimum of 26 weeks of treatment were analyzed.
Time frame
Baseline up to Week 26
Population
mITT population included all randomized participants who received at least one treatment (sham or adrabetadex). As per planned analysis, efficacy data were collected and presented by overall randomized treatment assignment (either sham or adrabetadex). These two treatment arms (either sham or adrabetadex) combine data from Parts A and B of the study.

Source ClinicalTrials.gov

Trial

Title
VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease
NCT number
NCT02534844
Phases
PHASE2, PHASE3
Conditions
Niemann-Pick Disease, Type C
Interventions
Parts A/B: Adrabetadex, Parts A/B: Sham Control
Lead sponsor
Mandos LLC
Results first posted
2023-02-22

Source ClinicalTrials.gov

Source

Release
trial-comparisons-2026-03-23-0.1.0
Release manifest SHA-256
f66225897fbf258b57ea10bdacfd0347d3cdfda93f7f5685234b7e06ea36fa57
AACT copy
2026-03-23
Tables read
outcome_counts.parquet, outcome_measurements.parquet, outcomes.parquet, reported_event_totals.parquet, reported_events.parquet, result_groups.parquet, studies.parquet, ctgov_trial.parquet

Source ClinicalTrials.gov

Sources 2

  • ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
  • ClinicalTrials.govNo license stated
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

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions. The measures are ConductScience's calculation from them.

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