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Trial record 1 of 1 for:    SIG-001-121
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Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04541628
Recruitment Status : Recruiting
First Posted : September 9, 2020
Last Update Posted : September 9, 2020
Sponsor:
Information provided by (Responsible Party):
Sigilon Therapeutics, Inc.

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.

Condition or disease Intervention/treatment Phase
Hemophilia A Combination Product: SIG-001 Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Estimated Study Start Date : September 2020
Estimated Primary Completion Date : September 2026
Estimated Study Completion Date : September 2026

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arm Intervention/treatment
Experimental: SIG-001
B-Domain Deleted Human Factor VIII (BDD-hFVIII) Producing Spheres
Combination Product: SIG-001
Laparoscopic administration of SIG-001 spheres, an encapsulated allogeneic cell therapy genetically modified with a non-viral vector to produce BDD-hFVIII.




Primary Outcome Measures :
  1. Number and proportion of participants with treatment emergent adverse events (TEAEs) overall and by dose/cohort. [ Time Frame: From SIG-001 administration until study completion (5 years) ]
  2. Number and proportion of participants with serious treatment emergent adverse events (TEAEs) overall and by dose/cohort. [ Time Frame: From SIG-001 administration until study completion (5 years) ]

Secondary Outcome Measures :
  1. Number and proportion of participants with inhibitor titer values assessed by Nijmegen Bethesda Assay overall and by dose/cohort. [ Time Frame: From SIG-001 administration until study completion (5 years) ]
  2. FVIII activity levels assessed by one-stage and chromogenic assays overall and by dose/cohort. [ Time Frame: From SIG-001 administration until study completion (5 years) ]
  3. Annualized Bleeding Rate (ABR) for all bleeds following SIG-001 administration presented by bleed type and location. [ Time Frame: From SIG-001 administration until study completion (5 years) ]
  4. Total number of replacement FVIII therapies administered overall and by dose/cohort. [ Time Frame: From SIG-001 administration until study completion (5 years) ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04541628


Contacts
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Contact: Sigilon Clinical Trials Contact 1-617-586-2822 clinicaltrials@sigilon.com

Locations
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United States, Indiana
Clinical Study Site Not yet recruiting
Indianapolis, Indiana, United States, 46260
United States, Massachusetts
Clinical Study Site Not yet recruiting
Boston, Massachusetts, United States, 02116
United States, Washington
Clinical Study Site Not yet recruiting
Seattle, Washington, United States, 98104
United Kingdom
Clinical Study Site Recruiting
London, United Kingdom
Clinical Study Site Not yet recruiting
Manchester, United Kingdom
Clinical Study Site Not yet recruiting
Southampton, United Kingdom
Sponsors and Collaborators
Sigilon Therapeutics, Inc.
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Responsible Party: Sigilon Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04541628    
Other Study ID Numbers: SIG-001-121
First Posted: September 9, 2020    Key Record Dates
Last Update Posted: September 9, 2020
Last Verified: September 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sigilon Therapeutics, Inc.:
Haemophilia A
Cell therapy
Gene therapy
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn