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

Sponsor

Spark Therapeutics (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT04093349

Collaborator

(none)

Enrollment

Locations

Arm

Anticipated Duration (Months)

Patients Per Site

Patients Per Site Per Month

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Pompe Disease Pompe Disease (Late-onset) Glycogen Storage Disease Type 2 Glycogen Storage Disease Type II LOPD Lysosomal Storage Diseases Acid Maltase Deficiency	Genetic: SPK-3006	Phase 1/Phase 2

Study Design

Study Type:

Interventional

Anticipated Enrollment :

30 participants

Allocation:

N/A

Intervention Model:

Sequential Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Oct 1, 2020

Anticipated Primary Completion Date :

Oct 1, 2023

Anticipated Study Completion Date :

Oct 1, 2027

Arms and Interventions

Arm	Intervention/Treatment
Experimental: SPK-3006 All participants who meet the eligibility criteria will receive a single intravenous (i.v.) administration of SPK-3006.	Genetic: SPK-3006 adeno-associated viral (AAV) vector

Arm

Intervention/Treatment

Experimental: SPK-3006

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

Genetic: SPK-3006

adeno-associated viral (AAV) vector

Outcome Measures

Primary Outcome Measures

Number of adverse and serious adverse events (AEs/SAEs), including clinically significant abnormal laboratory values. [52 weeks]
Adverse events.
Occurrence of immune response against AAV capsid [52 weeks]
Occurrence of immune response against GAA transgene [52 weeks]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Barrow Neurological Institute	Phoenix	Arizona	United States	85013
2	University of California Irvine Health	Orange	California	United States	92868
3	Emory University School of Medicine	Atlanta	Georgia	United States	30329
4	University of Kansas Medical Center Research Institute	Kansas City	Kansas	United States	66160
5	University of Minnesota	Minneapolis	Minnesota	United States	55455
6	Oregon Health & Science University	Portland	Oregon	United States	97239
7	University of Pennsylvania	Philadelphia	Pennsylvania	United States	19104
8	University of Pittsburgh Medical Center	Pittsburgh	Pennsylvania	United States	15213
9	University of Utah	Salt Lake City	Utah	United States	84108
10	Lysosomal and Rare Disorders Research & Treatment Center	Fairfax	Virginia	United States	22030
11	Vancouver General Hospital	Vancouver	British Columbia	Canada	V5Z 1M9
12	The Ottawa Hospital	Ottawa	Ontario	Canada	K1Y 4E9
13	Montreal Neurological Hospital	Montréal	Quebec	Canada	H3A 2B4
14	Rigshospitalet	Copenhagen		Denmark	84 2100
15	Centre Hospitalier Universitaire d'Angers	Angers		France	49933
16	CHU Paris IdF Ouest - Hôpital Raymond Poincaré	Garches		France	92380
17	Centre Hospitalier Régional Universitaire de Lille	Lille Cedex		France	59037
18	Assistance Publique Hôpitaux de Marseille	Marseille		France	13 13385
19	Nice University Hospital	Nice		France	6000
20	Friedrich-Baur-Institut Neurologische Klinik Ludwig-Maximilians-Universität München	Munchen		Germany	D08033
21	Universita Degli Studi Di Messina - Dipartimento di Medicina Clinica e Sperimentale	Messina		Italy	98122
22	Universita Degli Studi Di Milano, Laboratorio di Biochimica e Genetica della Malattie Neuromuscolari	Milano		Italy	35 20122
23	Malattie Metaboliche Universita Degli Studi Di Napoli Federico II	Naples		Italy	80138
24	UO Neurologia Azienda Ospedaliera Universitaria Pisana	Pisa		Italy	56126
25	Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino - Neurology, Osp. Molinette	Torino		Italy	10126
26	Erasmus Medical Center	Rotterdam		Netherlands	3015 CE
27	New Queen Elizabeth Hospital Birmingham	Birmingham	GBR	United Kingdom	B15 2WB
28	The Royal Free London NHS Foundation Trust	London	GBR	United Kingdom	NW3 2QG
29	Salford Royal MHS Foundation Trust	Salford		United Kingdom	M6 8HD