Overview
Sponsor-declared trial summary
Phase
Phase I and Phase II (Integrated) - Other
Status
Ongoing, recruitment ended
Participants planned
5
Countries
1
Sites
1
Gaucher Disease Type 1
To assess the long-term safety of FLT201 in participants with Gaucher disease type 1.
Key facts
- Sponsor
- Spur Therapeutics Limited
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Trial duration
- 10 May 2024 → ongoing
- Decision date (initial)
- 2024-04-29
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Freeline Therapeutics Ltd
External identifiers
- EU CT number
- 2024-511172-33-00
- WHO UTN
- U1111-1303-0375
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Others, Safety
To assess the long-term safety of FLT201 in participants with Gaucher disease type 1.
Secondary objectives 6
- 1. To further assess the safety of FLT201.
- 2. To investigate the durability of endogenous production of glucosylceramidase-β1 (pseudonym βglucocerebrosidase) (GCase) enzyme.
- 3. To investigate the clearance of glucosylsphingosine (lyso-Gb1).
- 4. To assess the impact of FLT201 on hemoglobin, platelet count, spleen size, and liver size.
- 5. To describe the immune responses to the FLT201 transgene product (GCase).
- 6. To assess viral shedding in various body fluids.
Conditions and MedDRA coding
Gaucher Disease Type 1
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 24.1 | PT | 10075697 | Gaucher´s disease type I | 100000004850 |
Regulatory references
- Scientific advice from competent authorities
- Paul-Ehrlich-Institut
- Plan to share IPD
- No
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- 1. Participants who have previously received FLT201 (including those who may have required recommencement or initiation of ERT/substrate reduction therapy [SRT]).
- 2. Participants able to give full informed consent and able to comply with all requirements of the trial.
Exclusion criteria 1
- Not applicable.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Incidence of treatment-emergent adverse events (TEAEs) (including DLTs)
Secondary endpoints 6
- 1. Incidence of AEs, SAEs, and changes from baseline in vital signs, 12-lead ECG, physical examination, and laboratory assessments.
- 2. Change from baseline in GCase activity (Dried Blood Spot [DBS]), plasma GCase activity and relative concentration, and leukocyte GCase activity over time.
- 3. Change from baseline in lyso-Gb1 concentration.
- 4. Change from baseline in hemoglobin, platelet count, spleen volume as measured by MRI, and liver volume as measured by MRI.
- 5. Change from baseline in GCase antibody levels.
- 6. Time to clearance of vector genomes in plasma and semen until three consecutive negative samples, where applicable (this may be completed in the preceding treatment trial and, if so, will not be followed-up within this trial.)
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD9503100 · Product
- Active substance
- Adeno-Associated Viral Vector Serotype S3 Containing Codon-Optimised Expression Cassette Encoding Human Beta-Glucocerebrosidase Variant
- Other product name
- Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human β-glucocerebrosidase variant
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- SOLUTION FOR INFUSION
- Authorisation status
- Not Authorised
- MA holder
- FREELINE THERAPEUTICS LIMITED
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/21/2491
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Spur Therapeutics Limited
- Sponsor organisation
- Spur Therapeutics Limited
- Address
- Sycamore House, Leyden Road Leyden Road
- City
- Stevenage
- Postcode
- SG1 2BP
- Country
- United Kingdom
Scientific contact point
- Organisation
- Spur Therapeutics Limited
- Contact name
- Clinical Trials Information
Public contact point
- Organisation
- Spur Therapeutics Limited
- Contact name
- Clinical Trials Information
Third parties 6
| Organisation | City, country | Duties |
|---|---|---|
| Centogene GmbH ORG-100043695
|
Rostock, Germany | Laboratory analysis |
| Medpace Finland Oy ORG-100009147
|
Helsinki, Finland | On site monitoring, Code 12, Code 13, Other, Code 2, Laboratory analysis, Code 5, Data management, Code 8 |
| Scout Clinical ORG-100042228
|
Dallas, United States | Other |
| Everest Clinical Research Corporation ORG-100041734
|
Markham, Canada | Code 11 |
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | E-data capture |
| PPD International Holdings LLC ORG-100007655
|
Zaventem, Belgium | Laboratory analysis |
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Spain | Ongoing, recruitment ended | 1 | 1 |
| Rest of world
Brazil, United Kingdom, United States
|
— | 4 | — |
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 |
|---|---|---|---|---|---|
| Spain | 2024-05-10 | 2024-05-13 | 2024-05-13 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 14 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol Admin Letter_Spur Therapeutics_redacted | N/A |
| Protocol (for publication) | D1_Protocol_2024-511172-33_Freeline Therapeutics Ltd_redacted | 4.0 |
| Protocol (for publication) | D4_Patient facing documents_BPI-SF-ENG_Freeline Therapeutics Ltd_redacted | N/A |
| Protocol (for publication) | D4_Patient facing documents_BPI-SF-SPA_Freeline Therapeutics Ltd_redacted | N/A |
| Protocol (for publication) | D4_Patient facing documents_FACIT_ENG_Freeline Therapeutics Ltd_redacted | 4.0 |
| Protocol (for publication) | D4_Patient facing documents_FACIT_SPA_Freeline Therapeutics Ltd_redacted | 4.0 |
| Protocol (for publication) | D4_Patient facing documents_SF 36V2 ENG_Freeline Therapeutics Ltd_redacted | 2.0 |
| Protocol (for publication) | D4_Patient facing documents_SF 36V2 SPA_Freeline Therapeutics Ltd_redacted | 2.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_ES_Freeline Therapeutics | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main ICF_Spur Therapeutics | 5.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main ICF_Spur Therapeutics_ENG | 5.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Pregnant Partner ICF_Spur Therapeutics | 2.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_EN_2024-511172-33_Freeline Therapeutics Ltd | 4.0 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_ES_2024-511172-33_Freeline Therapeutics Ltd | 4.0 |
Application history
8 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-02-15 | Spain | Acceptable 2024-04-08
|
2024-04-29 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-06-14 | Spain | Acceptable 2024-07-29
|
2024-07-29 |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2024-12-02 | Spain | Acceptable 2024-07-29
|
2024-12-02 |
| 4 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2025-03-14 | Spain | Acceptable 2024-07-29
|
2025-03-14 |
| 5 | SUBSTANTIAL MODIFICATION | SM-2 | 2025-05-09 | Spain | Acceptable | 2025-06-13 |
| 6 | NON SUBSTANTIAL MODIFICATION | NSM-3 | 2025-06-24 | Spain | Acceptable | 2025-06-24 |
| 7 | NON SUBSTANTIAL MODIFICATION | NSM-4 | 2025-10-15 | Spain | Acceptable | 2025-10-15 |
| 8 | NON SUBSTANTIAL MODIFICATION | NSM-5 | 2026-05-05 | Spain | Acceptable | 2026-05-05 |