Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruitment ended
Participants planned
45
Countries
1
Sites
1
Fabry Disease (X-linked lysosomal storage disease).
To evaluate long-term safety of ST-920
Key facts
- Sponsor
- Sangamo Therapeutics Inc.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male
- Therapeutic area
- Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Trial duration
- 3 Apr 2025 → ongoing
- Decision date (initial)
- 2025-01-31
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
- Funding sources
- Sangamo Therapeutics
External identifiers
- EU CT number
- 2024-512700-18-00
- WHO UTN
- U1111-1305-1998
- ClinicalTrials.gov
- NCT05039866
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy
To evaluate long-term safety of ST-920
Conditions and MedDRA coding
Fabry Disease (X-linked lysosomal storage disease).
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 24.1 | PT | 10016016 | Fabry´s disease | 100000004850 |
Regulatory references
- Plan to share IPD
- No
- IPD plan description
- Undecided
| EU CT number | Title | Sponsor |
|---|---|---|
| 2024-512695-34-00 | A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease. | Sangamo Therapeutics Inc. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- Subjects who received ST-920 therapy in a separate parent trial
- Subjects who have consented to participate in this LTFU study.
Exclusion criteria 1
- This study has no exclusion criteria
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- To evaluate long-term safety of ST-920 by assessment of incidence and severity of adverse events (AEs)
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD7634978 · Product
- Active substance
- Isaralgagene Civaparvovec
- Substance synonyms
- ST-920, Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA
- Pharmaceutical form
- SOLUTION FOR INJECTION/INFUSION
- Route of administration
- OTHER USE
- Max daily dose
- 0 Other
- Max total dose
- 0 Other
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Not Authorised
- MA holder
- SANGAMO THERAPEUTICS, INC.
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/19/2241
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Sangamo Therapeutics Inc.
- Sponsor organisation
- Sangamo Therapeutics Inc.
- Address
- 501 Canal Boulevard Suite A100
- City
- Richmond
- Postcode
- 94804-3522
- Country
- United States
Scientific contact point
- Organisation
- Sangamo Therapeutics Inc.
- Contact name
- Clinical Trials Information Desk
Public contact point
- Organisation
- Sangamo Therapeutics Inc.
- Contact name
- Clinical Trials Information Desk
Third parties 3
| Organisation | City, country | Duties |
|---|---|---|
| Medpace Finland Oy ORG-100009147
|
Helsinki, Finland | On site monitoring, Code 10, Code 12, Code 2, Code 5, Data management, E-data capture, Code 8, Code 9 |
| PPD Development LP ORG-100011560
|
Richmond, United States | Laboratory analysis |
| Frontage Laboratories Inc. ORG-100011515
|
Exton, United States | Laboratory analysis |
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Germany | Ongoing, recruitment ended | 1 | 1 |
| Rest of world
United Kingdom, Australia, Canada, United States
|
— | 44 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Germany | 2025-04-03 | 2025-04-15 | 2025-04-15 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 8 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_EN_2024-512700_18_Sangamo_redacted | 4.1 |
| Protocol (for publication) | D4_Patient facing documents_DE_Questionnaire 1_Sangamo_blank | 1 |
| Protocol (for publication) | D4_Patient facing documents_DE_Questionnaire 2_Sangamo_blank | 1 |
| Protocol (for publication) | D4_Patient facing documents_DE_Questionnaire 3_Sangamo_blank | 1 |
| Protocol (for publication) | D4_Patient facing documents_DE_SF-36_Sangamo_blank | 2 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_DE_Sangamo | N/A |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_SIS and ICF adults_Sangamo_redacted | 4.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_SIS and ICF Pregnancy Follow-up_Sangamo_blank | 1 |
Application history
5 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-10-25 | Germany | Acceptable with conditions 2025-01-28
|
2025-01-31 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2025-02-25 | Germany | Acceptable with conditions 2025-01-28
|
2025-02-25 |
| 3 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-02-26 | Germany | Acceptable with conditions | 2025-03-28 |
| 4 | SUBSTANTIAL MODIFICATION | SM-2 | 2025-05-14 | Germany | Acceptable 2025-06-13
|
2025-06-18 |
| 5 | SUBSTANTIAL MODIFICATION | SM-3 | 2026-04-01 | Germany | Acceptable 2026-05-19
|
2026-05-19 |