Haemophilia A MedDRA version: 20.0 Level: LLT Classification code 10018937 Term: Haemophilia A System Organ Class: 100000004850
Conditions
Interventions
Product Name: BDD-hFVIII Producing Spheres
Product Code: SIG-001
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: S14001
Current Sponsor code: S14001
Other descriptive name: BDD-hFVI
Sponsors
Sigilon Therapeutics, Inc.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Males aged 18 years or older 2. Diagnosis of Haemophilia A as follows: a) Severe (=65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: 1. Morbid obesity defined as body mass index (BMI) =35 2. Current FVIII inhibitors (>0.6 NBU/mL) or prior Immune Tolerance Induction (ITI) Note: Patients with a history of low and/or transient inhibitors require Sponsor review and approval. 3. History of allergic reaction or anaphylaxis to recombinant FVIII products, alginate (including seaweed and algae), or barium and barium products (including barium contrast agents) 4. Evidence of any bleeding disorder in addition to haemophilia A 5. Abnormal laboratory values, as follows: a) Platelet count 200 copies/mL 8. Active alcoholism or drug addiction during the 12 months before the screening visit 9. Active malignancy or history of malignancy in the 5 years prior to study entry, exclusive of surgically removed non-melanoma skin cancer 10. Participation in another investigational medicine or device study within 90 days of screening or 5 investigational product half-lives, whichever is longer 11. Prior administration of a gene therapy product 12. Treatment with emicizumab less than 6 months prior to screening. Note: Patients with emicizumab exposure may be eligible before 6 months of prior exposure if FVIII activity assessed by one stage assay is back to pre-emicizumab treatment levels. 13. Unable to tolerate general anaesthesia required for the laparoscopic procedure 14. History of: a) Abdominal adhesions due to prior large, median laparotomy(ies) Note: Uncomplicated prior laparoscopic procedures are not exclusionary (e.g. cholecystectomy, laparoscopic appendectomy or diagnostic laparoscopy). b) Septic peritonitis c) Intraperitoneal mesh d) Inflammatory Bowel Disease (IBD), including ulcerative colitis or Crohn’s disease 15. Other comorbidities that in the opinion of the Investigator increase the risk of abdominal adhesions 16. Any disease or medical condition that in the Investigator’s opinion is a contraindication for anaesthesia or the laparoscopic procedure (e.g. prior significant cardiovascular events) 17. In the Investigator’s judgment, the patient is unlikely to complete all protocol-required study visits, procedures, or comply with the study requirements for participation
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of up to 3 dose levels of SIG-001 administered laparoscopically into the intraperitoneal space.;Secondary Objective: To evaluate development of FVIII inhibitors after administration of SIG-001 To characterize FVIII activity levels after administration of SIG-001 To assess frequency of bleeding episodes following SIG-001 administration To assess effects of SIG-001 on usage of FVIII products;Primary end point(s): Clinically significant changes from baseline in vital signs, clinical laboratory tests, and treatment emergent adverse events (TEAE);Timepoint(s) of evaluation of this end point: Vitals – Screening, SIG-001 Administration, Follow-Up up to 5 years. TEAEs – Screening, SIG-001 Administration, Follow-Up up to 5 years. See Table 2 Schedule of Assessment in the Clinical Study Protocol for further details. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Factor VIII inhibitor titers (Nijmegen Bethesda Units [NBU]) FVIII activity levels (one-stage and chromogenic assays) Bleeding rate (annualized; ABR) Total use of factor VIII therapies (annualized);Timepoint(s) of evaluation of this end point: FVIII inhibitor titers - Screening 1, Follow Up up to 5 years. FVIII activity levels - Screening 1, SIG-001 Administration, Follow-Up up to 5 years. ABR and annualized FVIII therapy use - assessed prospectively throughout the study. See Table 2 Schedule of Assessment in the Clinical Study Protocol for further details. | — |
Countries
Germany, United Kingdom, United States
Contacts
Public ContactClinical Trials
Sigilon Therapeutics, Inc.
Outcome results
None listed