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

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

3 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-09-28

Study Completion Date

2023-01-09

Brief Summary

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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.

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Detailed Description

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Conditions

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Hemophilia A

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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SIG-001

Participants received a single dose of 50 milliliter (mL), 78.5 mL and 133 mL of SIG-001 spheres \[an encapsulated allogeneic cell therapy genetically modified with a non-viral vector to produce B-Domain Deleted Human Factor VIII (BDD-hFVIII) producing Spheres\] administered laparoscopically into the peritoneal cavity.

Group Type EXPERIMENTAL

SIG-001

Intervention Type COMBINATION_PRODUCT

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

Interventions

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SIG-001

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

Intervention Type COMBINATION_PRODUCT

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No