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A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Primary Purpose

Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2

Status
Active
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
SPK-3006
Sponsored by
Spark Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pompe Disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Provide written informed consent;
  • Males and Females ≥18 years of age with late-onset Pompe disease;
  • Received ERT for at least the previous 24 months
  • Have clinically moderate, late-onset Pompe disease characteristics;
  • Agree to use reliable contraception.

Exclusion Criteria:

  • Active hepatitis B and/or C;
  • Significant underlying liver disease;
  • Human immunodeficiency virus (HIV) infection;
  • Prior hypersensitivity to rhGAA;
  • Pre-existing anti-AAV neutralizing antibody titers;
  • High titer antibody responses to rhGAA;
  • Requires any invasive ventilation or requires noninvasive ventilation while awake and upright;
  • Received any prior vector or gene transfer agent;
  • Active malignancy (except non-melanoma skin cancer);
  • History of liver cancer;
  • Pregnant or nursing women;
  • Any evidence of active infection at the time of SPK-3006 infusion.

Sites / Locations

  • Barrow Neurological Institute
  • University of California Irvine Health
  • Emory University School of Medicine
  • University of Kansas Medical Center Research Institute
  • University of Minnesota
  • Oregon Health & Science University
  • University of Pennsylvania
  • University of Pittsburgh Medical Center
  • University of Utah
  • Lysosomal and Rare Disorders Research & Treatment Center
  • Vancouver General Hospital
  • The Ottawa Hospital
  • Montreal Neurological Hospital
  • Rigshospitalet
  • Centre Hospitalier Universitaire d'Angers
  • CHU Paris IdF Ouest - Hôpital Raymond Poincaré
  • Centre Hospitalier Régional Universitaire de Lille
  • Assistance Publique Hôpitaux de Marseille
  • Nice University Hospital
  • Friedrich-Baur-Institut Neurologische Klinik Ludwig-Maximilians-Universität München
  • Universita Degli Studi Di Messina - Dipartimento di Medicina Clinica e Sperimentale
  • Universita Degli Studi Di Milano, Laboratorio di Biochimica e Genetica della Malattie Neuromuscolari
  • Malattie Metaboliche Universita Degli Studi Di Napoli Federico II
  • UO Neurologia Azienda Ospedaliera Universitaria Pisana
  • Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino - Neurology, Osp. Molinette
  • Erasmus Medical Center
  • New Queen Elizabeth Hospital Birmingham
  • The Royal Free London NHS Foundation Trust
  • Salford Royal MHS Foundation Trust

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

SPK-3006

Arm Description

All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006.

Outcomes

Primary Outcome Measures

Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values.
Adverse events.
Occurrence of immune response against AAV capsid
Occurrence of immune response against GAA transgene

Secondary Outcome Measures

Full Information

First Posted
September 16, 2019
Last Updated
August 23, 2023
Sponsor
Spark Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT04093349
Brief Title
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
Official Title
Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
October 1, 2020 (Actual)
Primary Completion Date
October 2023 (Anticipated)
Study Completion Date
October 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Spark Therapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
SPK-3006
Arm Type
Experimental
Arm Description
All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006.
Intervention Type
Genetic
Intervention Name(s)
SPK-3006
Intervention Description
adeno-associated viral (AAV) vector
Primary Outcome Measure Information:
Title
Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values.
Description
Adverse events.
Time Frame
52 weeks
Title
Occurrence of immune response against AAV capsid
Time Frame
52 weeks
Title
Occurrence of immune response against GAA transgene
Time Frame
52 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Provide written informed consent; Males and Females ≥18 years of age with late-onset Pompe disease; Received ERT for at least the previous 24 months Have clinically moderate, late-onset Pompe disease characteristics; Agree to use reliable contraception. Exclusion Criteria: Active hepatitis B and/or C; Significant underlying liver disease; Human immunodeficiency virus (HIV) infection; Prior hypersensitivity to rhGAA; Pre-existing anti-AAV neutralizing antibody titers; High titer antibody responses to rhGAA; Requires any invasive ventilation or requires noninvasive ventilation while awake and upright; Received any prior vector or gene transfer agent; Active malignancy (except non-melanoma skin cancer); History of liver cancer; Pregnant or nursing women; Any evidence of active infection at the time of SPK-3006 infusion.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Tahseen Mozaffar, MD
Organizational Affiliation
University of California Irvine Health
Official's Role
Principal Investigator
Facility Information:
Facility Name
Barrow Neurological Institute
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85013
Country
United States
Facility Name
University of California Irvine Health
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Emory University School of Medicine
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Facility Name
University of Kansas Medical Center Research Institute
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
University of Pittsburgh Medical Center
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Facility Name
Lysosomal and Rare Disorders Research & Treatment Center
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22030
Country
United States
Facility Name
Vancouver General Hospital
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V5Z 1M9
Country
Canada
Facility Name
The Ottawa Hospital
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1Y 4E9
Country
Canada
Facility Name
Montreal Neurological Hospital
City
Montréal
State/Province
Quebec
ZIP/Postal Code
H3A 2B4
Country
Canada
Facility Name
Rigshospitalet
City
Copenhagen
ZIP/Postal Code
84 2100
Country
Denmark
Facility Name
Centre Hospitalier Universitaire d'Angers
City
Angers
ZIP/Postal Code
49933
Country
France
Facility Name
CHU Paris IdF Ouest - Hôpital Raymond Poincaré
City
Garches
ZIP/Postal Code
92380
Country
France
Facility Name
Centre Hospitalier Régional Universitaire de Lille
City
Lille Cedex
ZIP/Postal Code
59037
Country
France
Facility Name
Assistance Publique Hôpitaux de Marseille
City
Marseille
ZIP/Postal Code
13 13385
Country
France
Facility Name
Nice University Hospital
City
Nice
ZIP/Postal Code
6000
Country
France
Facility Name
Friedrich-Baur-Institut Neurologische Klinik Ludwig-Maximilians-Universität München
City
Munchen
ZIP/Postal Code
D08033
Country
Germany
Facility Name
Universita Degli Studi Di Messina - Dipartimento di Medicina Clinica e Sperimentale
City
Messina
ZIP/Postal Code
98122
Country
Italy
Facility Name
Universita Degli Studi Di Milano, Laboratorio di Biochimica e Genetica della Malattie Neuromuscolari
City
Milano
ZIP/Postal Code
35 20122
Country
Italy
Facility Name
Malattie Metaboliche Universita Degli Studi Di Napoli Federico II
City
Naples
ZIP/Postal Code
80138
Country
Italy
Facility Name
UO Neurologia Azienda Ospedaliera Universitaria Pisana
City
Pisa
ZIP/Postal Code
56126
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino - Neurology, Osp. Molinette
City
Torino
ZIP/Postal Code
10126
Country
Italy
Facility Name
Erasmus Medical Center
City
Rotterdam
ZIP/Postal Code
3015 CE
Country
Netherlands
Facility Name
New Queen Elizabeth Hospital Birmingham
City
Birmingham
State/Province
GBR
ZIP/Postal Code
B15 2WB
Country
United Kingdom
Facility Name
The Royal Free London NHS Foundation Trust
City
London
State/Province
GBR
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
Salford Royal MHS Foundation Trust
City
Salford
ZIP/Postal Code
M6 8HD
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
Links:
URL
https://resolutestudy.com/
Description
Related Info

Learn more about this trial

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

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