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rAAV2/5-hNAGLU

Phase 1

Sanfilippo Syndrome B | Small molecule | Rare Disease |uniQure N.V.|Last Updated: Dec 2, 2019

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment4

FDA Designations

No designations recorded

Clinical trial landscape

rAAV2/5-hNAGLU · 1 trial · 1 indication

Phase 1 1
NCT03300453Intracerebral Gene Therapy in Children With Sanfilippo Type B SyndromeSanfilippo Syndrome B
COMPLETED4 Analytics
PHASE1COMPLETED
Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome
Sanfilippo Syndrome B
NCT033004534
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Study Endpoints

Primary Endpoints

Number of Participants With Treatment-Related (Serious) Adverse Events as assessed by continuous evaluation of change from baseline
Baseline until end of study (Month 66)

Multiple measurements will be aggregated to derive the number of participants with Abnormal Laboratory Values and/or Adverse Events that are related to Treatment.

Secondary Endpoints

Number of Participants with presence of brain atrophy, white matter lesions and other lesions as assessed by cerebral MRI
Baseline until end of study (Month 66)
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
rAAV2/5-hNAGLUEXPERIMENTALEach patient will receive 960 µL of vector suspension. The vector suspension will be deposited simultaneously at 16 sites, each deposit containing 2.4x 1011 vg (4x1012 vg in total).

Interventions

NameTypeDescription
rAAV2/5-hNAGLUDRUGone-time brain intraparenchymal gene therapy dose
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Eligibility Criteria

Age Range18 Months to 60 Months
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion criteria: * Age: 18 months up to 60 months (5th birthday); * Onset of clinical manifestations related to mucopolysaccharidosis type IIIB (MPSIIIB); * NAGLU activity in peripheral blood cell and/or cultured fibroblast extracts of less than 10% of controls; * Patient affiliated to, or cover...

Countries:France
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Frequently asked questions about rAAV2/5-hNAGLU

What is rAAV2/5-PBGD vector dosage 1 used for?

rAAV2/5-PBGD vector dosage 1 is an investigational gene therapy being studied for two rare diseases: Acute Intermittent Porphyria and Sanfilippo Syndrome B. It is currently in Phase 1 clinical development and is not approved by the FDA.

Who makes rAAV2/5-PBGD vector dosage 1?

rAAV2/5-PBGD vector dosage 1 is developed by uniQure N.V., a biopharmaceutical company traded on NASDAQ under the ticker QURE. The company is conducting clinical trials for this gene therapy in rare disease indications.

What phase is rAAV2/5-PBGD vector dosage 1 in?

rAAV2/5-PBGD vector dosage 1 is in Phase 1 clinical development. Two Phase 1 trials have been completed, one for Acute Intermittent Porphyria and one for Sanfilippo Syndrome B. The drug remains investigational and is not FDA approved.

What clinical trials is rAAV2/5-PBGD vector dosage 1 in?

rAAV2/5-PBGD vector dosage 1 has been studied in two completed Phase 1 trials. NCT02082860 evaluated the vector for Acute Intermittent Porphyria in Spain with 8 participants. NCT03300453 evaluated intracerebral gene therapy for Sanfilippo Syndrome B in France with 4 participants.

Is rAAV2/5-PBGD vector dosage 1 the same as other gene therapy vectors?

rAAV2/5-PBGD vector dosage 1 is a specific gene therapy vector developed by uniQure. It uses an adeno-associated virus serotype 2/5 capsid to deliver the PBGD gene. No alternative names for this specific vector dosage have been disclosed.