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NCT00882921

An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

Completed Results posted Last updated 8 June 2021
What this trial tests

trial testing Idursulfase in Hunter Syndrome in 26 participants. Completed in 8 February 2013.

Timeline
14 October 2008
Primary endpoint
8 February 2013
8 February 2013

Quick facts

Lead sponsorShire
StatusCompleted
Study typeOBSERVATIONAL
Enrollment26
Start date14 October 2008
Primary completion8 February 2013
Estimated completion8 February 2013
Sites6 locations across United Kingdom, United States, Brazil

Drugs / interventions tested

Conditions studied

Sponsor

Shire — full company profile →

Who can join

5 and older, male only, with Hunter Syndrome. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Infusion-Related Adverse Event (IRAE) Rates Between IgG Anti-idursulfase Antibody Positive (Ab+) and Anti-idursulfase IgG Antibody Negative (Ab-) Patients Primary · Baseline to 109 Weeks

The primary analysis of how presence of antibodies affected IRAE rates was performed based on a negative binomial regression model. This was done to account for potentially differential follow-up time between antibody groups.

Ab+ (n =13)
GroupValue95% CI
Idursulfase (Elaprase) 0.5 mg/kg Weekly0.0121
Ab- (n = 13)
GroupValue95% CI
Idursulfase (Elaprase) 0.5 mg/kg Weekly0.0042
Ab+ (age adjusted)(n = 13)
GroupValue95% CI
Idursulfase (Elaprase) 0.5 mg/kg Weekly0.0055
Ab- (age adjusted)(n = 13)
GroupValue95% CI
Idursulfase (Elaprase) 0.5 mg/kg Weekly0.0026
Change From Baseline in uGAG Levels to 109 Weeks Secondary · Baseline to 109 Weeks

Urine GAG

GroupValue95% CI
Elaprase-74.07± 246.663

Adverse events — posted to ClinicalTrials.gov

Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Elaprase® (0.5 mg/kg)
Serious: 16/26 (62%)
Deaths:

Serious adverse events (35 terms)

ReactionSystemElaprase® (0.5 mg/kg)
ConvulsionNervous system disorders
Lower respiratory tract infectionInfections and infestations
Central line infectionInfections and infestations
Upper respiratory tract infectionInfections and infestations
Carpal tunnel syndromeNervous system disorders
AgitationPsychiatric disorders
Arnold-Chiari malformationCongenital, familial and genetic disorders
Abdominal distensionGastrointestinal disorders
Abdominal painGastrointestinal disorders
Dental cariesGastrointestinal disorders
Inguinal hernia, obstructiveGastrointestinal disorders
Hernia obstructiveGeneral disorders
Multi-organ failureGeneral disorders
PainGeneral disorders
PneumoniaInfections and infestations
SeromaInjury, poisoning and procedural complications
Brain stem auditory evoked responseInvestigations
Cervical spinal stenosisMusculoskeletal and connective tissue disorders
Grand mal convulsionNervous system disorders
HydrocephalusNervous system disorders
Neurological decompensationNervous system disorders
SyringomyeliaNervous system disorders
Acute respiratory failureRespiratory, thoracic and mediastinal disorders
Bronchial hyperreactivityRespiratory, thoracic and mediastinal disorders
DysphoniaRespiratory, thoracic and mediastinal disorders
Other adverse events (71 terms — click to expand)

ReactionSystemElaprase® (0.5 mg/kg)
PyrexiaGeneral disorders
Upper respiratory tract infectionInfections and infestations
CoughRespiratory, thoracic and mediastinal disorders
Ear infectionInfections and infestations
Lower respiratory tract infectionInfections and infestations
FallInjury, poisoning and procedural complications
ArthralgiaMusculoskeletal and connective tissue disorders
ConstipationGastrointestinal disorders
DiarrhoeaGastrointestinal disorders
VomitingGastrointestinal disorders
ConvulsionNervous system disorders
HeadacheNervous system disorders
Oropharyngeal painRespiratory, thoracic and mediastinal disorders
RashSkin and subcutaneous tissue disorders
Back painMusculoskeletal and connective tissue disorders
Petit mal epilepsyNervous system disorders
RhinorrhoeaRespiratory, thoracic and mediastinal disorders
Abdominal painGastrointestinal disorders
GastroenteritisInfections and infestations
InfluenzaInfections and infestations
Otitis externaInfections and infestations
Pain in extremityMusculoskeletal and connective tissue disorders
Ear painEar and labyrinth disorders
Catheter related complicationGeneral disorders
FatigueGeneral disorders
NasopharyngitisInfections and infestations
Oral candidiasisInfections and infestations
Otitis mediaInfections and infestations
ContusionInjury, poisoning and procedural complications
Musculoskeletal painMusculoskeletal and connective tissue disorders
DizzinessNervous system disorders
InsomniaPsychiatric disorders
Productive coughRespiratory, thoracic and mediastinal disorders
Sleep apnoea syndromeRespiratory, thoracic and mediastinal disorders
DeafnessEar and labyrinth disorders
Eye pruritusEye disorders
Eye swellingEye disorders
Gastrooesophageal reflux diseaseGastrointestinal disorders
NauseaGastrointestinal disorders
Adverse drug reactionGeneral disorders

Most-reported serious reactions: Convulsion, Lower respiratory tract infection, Central line infection, Upper respiratory tract infection, Carpal tunnel syndrome, Agitation, Arnold-Chiari malformation, Abdominal distension.

Data from ClinicalTrials.gov NCT00882921 adverse events section.

Sponsor's own description

The objective of this study is to evaluate the effect of anti-idursulfase antibodies on idursulfase safety (measured by infusion related adverse events) between patients who develop anti-idursulfase antibodies and patients who do not after long-term idursulfase enzyme replacement therapy (ERT).

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II patients.
    Giugliani R, Harmatz P, Jones SA, Mendelsohn NJ, et al · · 2017 · cited 15× · PMID 28243577 · DOI 10.1016/j.ymgmr.2017.01.014
  2. Protection is not always a good thing: The immune system's impact on gene therapy.
    Freitas MV, Frâncio L, Haleva L, Matte UDS. · · 2022 · cited 10× · PMID 35852088 · DOI 10.1590/1678-4685-gmb-2022-0046

Verify or expand the search:

Other trials of Idursulfase

Trials testing the same drug.

Other recruiting trials for Hunter Syndrome

Currently open trials in the same condition.

Other Shire trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00882921.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing