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

NCT02082860 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2014-12-18

No results posted yet for this study

Summary

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.

Conditions

  • Acute Intermittent Porphyria

Interventions

GENETIC

rAAV2/5-PBGD vector dosage 1

Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1.

GENETIC

rAAV2/5-PBGD vector dosage 2

Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1.

GENETIC

rAAV2/5-PBGD vector dosage 3

Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1.

GENETIC

rAAV2/5-PBGD vector dosage 4

Intravenous injection of rAAV2/5-PBGD vector, single administration on Day 1.

Sponsors & Collaborators

  • Porphyria Centre Sweden

    collaborator UNKNOWN

  • University of Navarra

    collaborator OTHER

  • UniQure N.V.

    collaborator INDUSTRY

  • Nationales Centrum für Tumorerkrankungen

    collaborator UNKNOWN

  • Digna Biotech S.L.

    lead INDUSTRY

Principal Investigators

  • Juan Ruiz, MD · Digna Biotech S.L.

  • Jesus Prieto, MD · Clinica Universidad de Navarra

  • Rafael Enriquez de Salamanca, MD · Hospital 12 de Octubre

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
64 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-11-30
Primary Completion
2014-07-31
Completion
2014-11-30

Countries

  • Spain

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02082860 on ClinicalTrials.gov