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

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

4

Participants

Timeline

Start Date

October 1, 2020

Primary Completion Date

April 30, 2032

Study Completion Date

April 30, 2032

Conditions
Pompe DiseasePompe Disease (Late-onset)Glycogen Storage Disease Type 2Glycogen Storage Disease Type IILOPDLysosomal Storage DiseasesAcid Maltase Deficiency
Interventions
GENETIC

SPK-3006

adeno-associated viral (AAV) vector

Trial Locations (29)

6000

Nice University Hospital, Nice

10126

Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino - Neurology, Osp. Molinette, Torino

15213

University of Pittsburgh Medical Center, Pittsburgh

19104

University of Pennsylvania, Philadelphia

22030

Lysosomal and Rare Disorders Research & Treatment Center, Fairfax

30329

Emory University School of Medicine, Atlanta

49933

Centre Hospitalier Universitaire d'Angers, Angers

55455

University of Minnesota, Minneapolis

56126

UO Neurologia Azienda Ospedaliera Universitaria Pisana, Pisa

59037

Centre Hospitalier Régional Universitaire de Lille, Lille

66160

University of Kansas Medical Center Research Institute, Kansas City

80138

Malattie Metaboliche Universita Degli Studi Di Napoli Federico II, Naples

84108

University of Utah, Salt Lake City

85013

Barrow Neurological Institute, Phoenix

92380

CHU Paris IdF Ouest - Hôpital Raymond Poincaré, Garches

92868

University of California Irvine Health, Orange

97239

Oregon Health & Science University, Portland

98122

Universita Degli Studi Di Messina - Dipartimento di Medicina Clinica e Sperimentale, Messina

V5Z 1M9

Vancouver General Hospital, Vancouver

K1Y 4E9

The Ottawa Hospital, Ottawa

H3A 2B4

Montreal Neurological Hospital, Montreal

84 2100

Rigshospitalet, Copenhagen

13 13385

Assistance Publique Hôpitaux de Marseille, Marseille

D08033

Friedrich-Baur-Institut Neurologische Klinik Ludwig-Maximilians-Universität München, München

35 20122

Universita Degli Studi Di Milano, Laboratorio di Biochimica e Genetica della Malattie Neuromuscolari, Milan

3015 CE

Erasmus Medical Center, Rotterdam

B15 2WB

New Queen Elizabeth Hospital Birmingham, Birmingham

NW3 2QG

The Royal Free London NHS Foundation Trust, London

M6 8HD

Salford Royal MHS Foundation Trust, Salford

All Listed Sponsors
lead

Spark Therapeutics, Inc.

INDUSTRY

NCT04093349 - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) | Biotech Hunter | Biotech Hunter