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This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1. The aims are to investigate the safety/tolerability and efficacy of FLT201, and to investigate the relationship of FLT201 dose to augmentation of residual glucocerebrosidase (GCase) expression (activity and concentration), and its potential to improve the clinical phenotype by reduction and prevention of cellular accumulation of GCase substrate.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| FLT201 (4.5 × 10^11 vg/kg) | Experimental | FLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| FLT201 | Genetic | FLT201 is a replication-incompetent single-stranded (ss) recombinant adeno-associated virus (AAV) vector. The vector is composed of a ss DNA genome packaged in an AAV-derived protein capsid. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events Over Time | Treatment-emergent adverse events (including dose-limiting toxicities), with AEs graded as mild/moderate/severe. | Day 1 (dosing) through transfer to the long-term follow-up (LTFU) study, which was up to 15 months. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Lyso-Gb1 in Plasma | Change from baseline to week 38/month 9 of lyso-Gb1 in plasma. | From baseline to week 38/9 months. |
| Change From Baseline in Spleen Volume Measured by MRI |
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Inclusion Criteria:
Treated with either enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) and started this treatment at least 2 years prior to dosing with no change in regimen for the prior 3 months. ERT dose ≥15 U/kg and ≤60 U/kg every other week.
Exclusion Criteria:
Diagnosed or suspected Type 2 or Type 3 Gaucher disease (including any patient with eye movement abnormality on clinical examination).
Positive for neutralising antibodies to AAVS3 at screening.
Evidence of significant and persistent liver dysfunction at Screening defined as >1.5 x upper limit of normal (ULN) in alanine aminotransferase (ALT), aspartate aminotransferase (AST) or total bilirubin.
Evidence of any of the following at screening:
Hepatitis B surface antigen (HBsAg) positive at screening.
Hepatitis C antibody (Hep C Ab) positive and hepatitis C RNA polymerase chain reaction (PCR) (as follow-up test if Hep C Ab-positive)-positive at screening.
Cytomegalovirus (CMV) immunoglobulin G (IgG) and CMV DNA PCR-positive at screening.
Human immunodeficiency virus (HIV)-1 or -2 antibody positive at screening.
Patient has received live attenuated vaccination within 12 weeks prior to screening or intends to receive such vaccination during the study.
History of clinically-advanced liver disease e.g. cirrhosis, portal hypertension.
History of bone marrow transplant.
History of splenectomy (partial or total).
History of splenic infarct within 12 months of screening.
History of receiving any gene transfer medicinal product.
History of receiving any investigational therapy for Gaucher disease within 60 days of screening.
Participation in any other clinical study of an investigational medicinal product (IMP), and/or receiving any other IMP during the study.
History of idiopathic thrombocytopaenic purpura, thrombotic thrombocytopaenic purpura, thrombocytopaenia, anaemia, hepatomegaly, splenomegaly, and/or osteoporosis, unrelated to Gaucher disease.
History of, or active neoplastic disease within 5 years of screening (except for basal or squamous cell carcinoma of the skin or carcinoma in situ which has been definitively treated).
Subjects with uncontrolled cardiac failure, unstable angina, myocardial infarction, pulmonary hypertension or cardiac presentations including cardiac instability deemed significant by the investigator in the past 6 months
History of acute myocarditis or presence of acute myocarditis during screening.
History of substance abuse, including alcohol abuse or alcohol dependence.
Known or suspected intolerance, hypersensitivity or contraindication to the investigational medicinal product (IMP) and non-investigational medicinal products (NIMPs) or their excipients.
History of anaphylaxis or infusion related reactions to ERT.
Contraindication(s) to MRI. (e.g. ferromagnetic metallic implants, some types of pacing and defibrillator devices, nerve stimulators).
Any clinical condition (medical or psychiatric) that, in the opinion of the investigator, could jeopardise safety or compromise ability of the patient to participate in this study.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Kaiser Permanente | Los Angeles | California | 90027 | United States | ||
| Lysosomal Rare Disorders Research and Treatment Center |
Participants were to undergo screening assessments for up to 16 weeks prior to Day 1 (gene therapy infusion). Treatment-eligible participants reported to the infusion trial site on the day prior to receiving the gene therapy infusion (Day -1).
This was a multicenter study with 15 study sites in USA, Brazil, Paraguay, Spain, Germany, UK, and Israel. Ten participants met all the inclusion criteria for the trial and were enrolled, with 6 participants ultimately being dosed with FLT201 at 4 trial sites in Brazil, Spain, UK, and USA.
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| ID | Title | Description |
|---|---|---|
| FG000 | FLT201 | FLT201 (4.5 × 10^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. Investigation has not been conducted with additional dose cohorts. Duration of treatment is 38 week/9 month. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | FLT201 | FLT201 (4.5 × 10^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Treatment-emergent Adverse Events Over Time | Treatment-emergent adverse events (including dose-limiting toxicities), with AEs graded as mild/moderate/severe. | Posted | Count of Participants | Participants | Day 1 (dosing) through transfer to the long-term follow-up (LTFU) study, which was up to 15 months. |
|
The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit.
Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | FLT201 | FLT201 (4.5 × 10^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Neutralising antibodies | Investigations | MedDRA (27.1) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (27.1) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Spur Clinical Trials Contact | Spur Therapeutics (formerly known as Freeline Therapeutics Ltd) | +44 (0)1438 906870 | clinicaltrials@spurtherapeutics.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 16, 2023 | Jan 28, 2026 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 16, 2025 | Jan 28, 2026 | SAP_001.pdf |
| ID | Term |
|---|---|
| D005776 | Gaucher Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
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Change from baseline to week 38/month 9 in spleen volume measured by MRI.
| From baseline to week 38/9 months. |
| Change From Baseline in Liver Volume Measured by MRI | Change from baseline to week 38/month 9 in liver volume measured by MRI. | From baseline to week 38/9 months. |
| Change From Baseline in Hemoglobin | Change from baseline to week 38/month 9 in hemoglobin. | From baseline to week 38/9 months. |
| Change From Baseline in Platelet Count | Change from baseline to week 38/month 9 in platelet count. | From baseline to week 38/9 months. |
| Fairfax |
| Virginia |
| 22030-6066 |
| United States |
| Hospital de Clinicas de Porto Alegre (HCPA) | Porto Alegre | Brazil |
| SphinCS | Höchheim | Germany |
| Shaare Zedek Medical Center | Jerusalem | Israel |
| Rabin Medical Center - PPDS | Petah Tikva | Israel |
| Tel Aviv Sourasky Medical Center | Tel Aviv | Israel |
| Hospital Quironsalud Zaragoza | Zaragoza | Spain |
| Royal Free Hospital | London | United Kingdom |
| Salford Royal Hospital | Salford | United Kingdom |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | Participants |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Secondary | Change From Baseline in Lyso-Gb1 in Plasma | Change from baseline to week 38/month 9 of lyso-Gb1 in plasma. | Posted | Mean | Standard Deviation | ng/ml | From baseline to week 38/9 months. |
|
|
|
| Secondary | Change From Baseline in Spleen Volume Measured by MRI | Change from baseline to week 38/month 9 in spleen volume measured by MRI. | Posted | Mean | Standard Deviation | mL | From baseline to week 38/9 months. |
|
|
|
| Secondary | Change From Baseline in Liver Volume Measured by MRI | Change from baseline to week 38/month 9 in liver volume measured by MRI. | Posted | Mean | Standard Deviation | mL | From baseline to week 38/9 months. |
|
|
|
| Secondary | Change From Baseline in Hemoglobin | Change from baseline to week 38/month 9 in hemoglobin. | Posted | Mean | Standard Deviation | g/dL | From baseline to week 38/9 months. |
|
|
|
| Secondary | Change From Baseline in Platelet Count | Change from baseline to week 38/month 9 in platelet count. | Posted | Mean | Standard Deviation | 10^9 platelets/L | From baseline to week 38/9 months. |
|
|
|
| 0 |
| 6 |
| 2 |
| 6 |
| 6 |
| 6 |
| Neutralising antibodies positive | Investigations | MedDRA (27.1) | Systematic Assessment |
|
| Blood pressure fluctuation | Vascular disorders | MedDRA (27.1) | Systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA (27.1) | Systematic Assessment |
|
| Hypotension | Vascular disorders | MedDRA (27.1) | Systematic Assessment |
|
| Peripheral coldness | Vascular disorders | MedDRA (27.1) | Systematic Assessment |
|
| Adverse drug reaction | General disorders | MedDRA (27.1) | Systematic Assessment |
|
| Discomfort | General disorders | MedDRA (27.1) | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA (27.1) | Systematic Assessment |
|
| Malaise | General disorders | MedDRA (27.1) | Systematic Assessment |
|
| Peripheral swelling | General disorders | MedDRA (27.1) | Systematic Assessment |
|
| Temperature intolerance | General disorders | MedDRA (27.1) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
|
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA (27.1) | Systematic Assessment |
|
| Mood altered | Psychiatric disorders | MedDRA (27.1) | Systematic Assessment |
|
| Panic attack | Psychiatric disorders | MedDRA (27.1) | Systematic Assessment |
|
| Activated partial thromboplastin | Investigations | MedDRA (27.1) | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA (27.1) | Systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA (27.1) | Systematic Assessment |
|
| Cardiac murmur | Investigations | MedDRA (27.1) | Systematic Assessment |
|
| Electrocardiogram ST segment elevation | Investigations | MedDRA (27.1) | Systematic Assessment |
|
| Fibrin D dimer increased | Investigations | MedDRA (27.1) | Systematic Assessment |
|
| Glomerular filtration rate decreased | Investigations | MedDRA (27.1) | Systematic Assessment |
|
| Immature granulocyte count increased | Investigations | MedDRA (27.1) | Systematic Assessment |
|
| Mean cell volume increased | Investigations | MedDRA (27.1) | Systematic Assessment |
|
| Neutrophil count increased | Investigations | MedDRA (27.1) | Systematic Assessment |
|
| Prothrombin time prolonged | Investigations | MedDRA (27.1) | Systematic Assessment |
|
| Scan bone marrow abnormal | Investigations | MedDRA (27.1) | Systematic Assessment |
|
| Weight increased | Investigations | MedDRA (27.1) | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
|
| Cardiomegaly | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
|
| Palpitations | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
|
| Action tremor | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
|
| Burning sensation | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
|
| Migraine | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
|
| Tremor | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
|
| Leukocytosis | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
|
| Leukopenia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
|
| Lymphopenia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
|
| Neutropenia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
|
| Neutrophilia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
|
| Splenomegaly | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
|
| Ear pain | Ear and labyrinth disorders | MedDRA (27.1) | Systematic Assessment |
|
| Visual field defect | Eye disorders | MedDRA (27.1) | Systematic Assessment |
|
| Vitreous floaters | Eye disorders | MedDRA (27.1) | Systematic Assessment |
|
| Abdominal distension | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
|
| Dyspepsia | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
|
| Flatulence | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
|
| Frequent bowel movements | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
|
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
|
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
|
| Macule | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
|
| Skin atrophy | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
|
| Micturition urgency | Renal and urinary disorders | MedDRA (27.1) | Systematic Assessment |
|
| Oliguria | Renal and urinary disorders | MedDRA (27.1) | Systematic Assessment |
|
| Pollakiuria | Renal and urinary disorders | MedDRA (27.1) | Systematic Assessment |
|
| Proteinuria | Renal and urinary disorders | MedDRA (27.1) | Systematic Assessment |
|
| Urinary incontinence | Renal and urinary disorders | MedDRA (27.1) | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
|
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
|
| Epstein-Barr virus infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
|
| Folliculitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
|
| Gastrointestinal infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
|
| Hordeolum | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
|
| Pharyngitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
|
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
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| Dehydration | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
|
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
|
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
|
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| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |