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Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A

Primary Purpose

Hemophilia A

Status

Terminated

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

SIG-001

About this trial

This is an interventional treatment trial for Hemophilia A focused on measuring Haemophilia A, Cell therapy, Gene therapy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

Males aged 18 years or older
Diagnosis of Haemophilia A defined as ≤2% FVIII activity
Greater than 150 exposure days to treatment with FVIII products
Use of reliable barrier contraception if applicable
Normal levels of von Willebrand factor (VWF) antigen
Able and willing to provide informed consent
Willing to withdraw from FVIII prophylaxis during specified periods in the study

Exclusion Criteria:

Body mass index (BMI) ≥35
Current FVIII inhibitors (>0.6 Nijmegen Bethesda Units/mL) or prior Immune Tolerance Induction (ITI)
History of allergic reaction or anaphylaxis to recombinant FVIII products or SIG-001 components
Evidence of any bleeding disorder in addition to haemophilia A
Abnormal laboratory values as defined in the protocol
Active infection with Hepatitis B or Hepatitis C virus or currently managed with antiviral medications for Hepatitis B or C
Uncontrolled HIV infection
Active alcoholism or drug addiction during the 12 months before the screening visit
Active malignancy or history of malignancy in the 5 years prior to study entry
Participation in another investigational medicine or device study
Prior administration of a gene therapy product
Significant underlying disease or comorbidities that are a contraindication for general anaesthesia or laparoscopic procedure

Sites / Locations

Clinical Study Site
Clinical Study Site
Clinical Study Site
Clinical Study Site
Clinical Study Site
Clinical Study Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

SIG-001

Arm Description

B-Domain Deleted Human Factor VIII (BDD-hFVIII) Producing Spheres

Outcomes

Primary Outcome Measures

Number and proportion of participants with treatment emergent adverse events (TEAEs) overall and by dose/cohort.

Number and proportion of participants with serious treatment emergent adverse events (TEAEs) overall and by dose/cohort.

Secondary Outcome Measures

Number and proportion of participants with inhibitor titer values assessed by Nijmegen Bethesda Assay overall and by dose/cohort.

FVIII activity levels assessed by one-stage and chromogenic assays overall and by dose/cohort.

Annualized Bleeding Rate (ABR) for all bleeds following SIG-001 administration presented by bleed type and location.

Total number of replacement FVIII therapies administered overall and by dose/cohort.

Full Information

NCT ID

NCT04541628

First Posted

August 21, 2020

Last Updated

November 7, 2022

Sponsor

Sigilon Therapeutics, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT04541628

Brief Title

Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A

Official Title

A Phase 1/2 Open-Label, Dose-Escalation, Safety, Tolerability, and Efficacy Study of SIG-001 in Adult Patients With Severe or Moderately-Severe Haemophilia A Without Inhibitors (SIG-001-121)

Study Type

Interventional

2. Study Status

Record Verification Date

November 2022

Overall Recruitment Status

Terminated

Why Stopped

SIG-001 programme terminated

Study Start Date

September 28, 2020 (Actual)

Primary Completion Date

October 28, 2022 (Actual)

Study Completion Date

October 28, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Sigilon Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

SIG-001-121 is a first-in-human (FIH), phase 1/2, multi-centre, open-label, dose escalation study to assess the safety, tolerability, and preliminary efficacy of SIG-001 in adults with severe or moderately severe haemophilia A without inhibitors. Up to three dose cohorts (3 patients each) are planned. Cohort expansions (up to 3 additional patients) may be triggered to collect additional information about safety and efficacy.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hemophilia A

Keywords

Haemophilia A, Cell therapy, Gene therapy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

3 (Actual)

8. Arms, Groups, and Interventions

Arm Title

SIG-001

Arm Type

Experimental

Arm Description

B-Domain Deleted Human Factor VIII (BDD-hFVIII) Producing Spheres

Intervention Type

Combination Product

Intervention Name(s)

SIG-001

Intervention Description

Laparoscopic administration of SIG-001 spheres, an encapsulated allogeneic cell therapy genetically modified with a non-viral vector to produce BDD-hFVIII.

Primary Outcome Measure Information:

Title

Number and proportion of participants with treatment emergent adverse events (TEAEs) overall and by dose/cohort.

Time Frame