Phase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria

NCTID	NCT02082860 (View at clinicaltrials.gov)
Description	This is a Phase I trial aimed to determine the safety of the investigational gene therapy product (rAAV2/5-PBGD) for the treatment of Acute Intermittent Porphyria (AIP). Up to eight patients fulfilling the eligibility criteria will participate in this multicentre, open label, single dose, dose-ranging Phase I clinical trial. The enrolled patients will be followed up to assess the safety profile of the investigational gene therapy product and to establish the maximum therapeutic safe dose to be administered in future confirmatory/pivotal clinical trial(s). In addition, the biological and clinical response to the treatment with rAAV2/5-PBGD in AIP patients will be assessed. A complete evaluation of the clinical (symptoms and quality of life assessment) and laboratory (blood and urine) data will be performed. (Show More)
Development Status	Inactive
Indication	Acute Intermittent Porphyria
Disease Ontology Term	DOID:3890
Compound Name	RAAV2/5-PBGD
Sponsor	Digna Biotech S.L.
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	8
Results Posted	Not Available

Target Gene/Variant	PBGD
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2/5
Editor Type	none
Dose 1	5.0E11 gc/kg
Dose 2	2E12 gc/kg
Dose 3	6E12 gc/kg
Dose 4	1.8E13 gc/kg
Dose 5

No.of Trial Sites	2
Locations	Spain

Has US IND
FDA Designations
Recent Updates	Therapy was safe, but failed to show efficacy, this product did not advance to Phase II, original developers working on alternatives