NCT04174105 (FORTIS) is a Phase 1, PHASE2 clinical trial of zocaglusagene nuzaparvovec in Pompe Disease (Late-onset), sponsored by Astellas Gene Therapies.

Who is sponsoring NCT04174105?

NCT04174105 is sponsored by Astellas Gene Therapies.

What drugs are being tested in NCT04174105?

Interventions in NCT04174105: zocaglusagene nuzaparvovec.

What condition does NCT04174105 study?

NCT04174105 studies Pompe Disease (Late-onset).

Is NCT04174105 still recruiting?

NCT04174105 is currently active, enrolled. Last updated 2 February 2026.

Last reviewed 2026-02-02 · How we verify

NCT04174105: FORTIS

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Gene Transfer Study in Patients With Late Onset Pompe Disease

Active, enrolled Phase 1, PHASE2 Last updated 2 February 2026

What this trial tests

Phase 1, PHASE2 trial testing zocaglusagene nuzaparvovec in Pompe Disease (Late-onset) in 11 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

27 October 2020

Primary endpoint
28 February 2035

28 February 2035

Quick facts

Lead sponsor	Astellas Gene Therapies
Phase	Phase 1, PHASE2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	11
Start date	27 October 2020
Primary completion	28 February 2035
Estimated completion	28 February 2035
Sites	4 locations across United Kingdom, United States

Drugs / interventions tested

zocaglusagene nuzaparvovec — full drug profile →

Conditions studied

Pompe Disease (Late-onset) — all drugs for Pompe Disease (Late-onset) →

Sponsor

Astellas Gene Therapies — full company profile →

Who can join

Adults 18 to 80, any sex, with Pompe Disease (Late-onset). Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Viral vector-based gene therapies in the clinic.
Zhao Z, Anselmo AC, Mitragotri S. · · 2022 · cited 171× · PMID 35079633 · DOI 10.1002/btm2.10258
Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations.
Mendell JR, Connolly AM, Lehman KJ, Griffin DA, et al · · 2022 · cited 87× · PMID 35356756 · DOI 10.1016/j.omtm.2022.02.011
Novel vectors and approaches for gene therapy in liver diseases.
Maestro S, Weber ND, Zabaleta N, Aldabe R, et al · · 2021 · cited 81× · PMID 34159305 · DOI 10.1016/j.jhepr.2021.100300
Moving towards clinical trials for mitochondrial diseases.
Pitceathly RDS, Keshavan N, Rahman J, Rahman S. · · 2021 · cited 52× · PMID 32618366 · DOI 10.1002/jimd.12281
Pompe Disease: a Clinical, Diagnostic, and Therapeutic Overview.
Stevens D, Milani-Nejad S, Mozaffar T. · · 2022 · cited 51× · PMID 36969713 · DOI 10.1007/s11940-022-00736-1
Gene Therapy for Neurodegenerative Diseases: Slowing Down the Ticking Clock.
Martier R, Konstantinova P. · · 2020 · cited 50× · PMID 33071748 · DOI 10.3389/fnins.2020.580179
Therapeutic Approaches in Lysosomal Storage Diseases.
Fernández-Pereira C, San Millán-Tejado B, Gallardo-Gómez M, Pérez-Márquez T, et al · · 2021 · cited 43× · PMID 34944420 · DOI 10.3390/biom11121775
Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.
Massaro G, Geard AF, Liu W, Coombe-Tennant O, et al · · 2021 · cited 42× · PMID 33924076 · DOI 10.3390/biom11040611

Verify or expand the search:

Other recruiting trials for Pompe Disease (Late-onset)

Currently open trials in the same condition.

NCT07478172 — Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease · NA · recruiting
NCT06150820 — A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease · NA · active not recruiting
NCT03694561 — Developing a Management Approach for Patients With "Late-Onset" Pompe Disease · active not recruiting
NCT00567073 — Pompe Pregnancy Sub-Registry · recruiting

Other Astellas Gene Therapies trials

Trials by the same sponsor.

NCT07052929 — Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators · Phase 1, PHASE2 · recruiting
NCT06581146 — A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition · recruiting
NCT06483802 — A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia · Phase 1 · withdrawn
NCT06150820 — A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease · NA · active not recruiting
NCT03199469 — A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM) · Phase 2, PHASE3 · active not recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04174105 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Astellas Gene Therapies
Last refreshed: 2 February 2026

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04174105.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing