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An Open-Label, FIH Study Evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With T1D

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ClinicalTrials.gov Identifier: NCT05565248
Recruitment Status : Recruiting
First Posted : October 4, 2022
Last Update Posted : March 15, 2023
Sponsor:
Collaborator:
ViaCyte
Information provided by (Responsible Party):
CRISPR Therapeutics ( CRISPR Therapeutics AG )

Tracking Information
First Submitted Date  ICMJE September 30, 2022
First Posted Date  ICMJE October 4, 2022
Last Update Posted Date March 15, 2023
Actual Study Start Date  ICMJE January 20, 2023
Estimated Primary Completion Date April 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 30, 2022)
  • Incidence of adverse events with causality related to VCTX211 units, the surgical procedures and/or medical interventions required to implant and explant the VCTX211 units. [ Time Frame: From implantation up to 12 months post implantation ]
  • Assess the clinical efficacy of VCTX211 units via evaluation of C-peptide increase from the baseline. [ Time Frame: From implantation up to 12 months post implantation ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 30, 2022)
  • Incidence of adverse events reported in patients implanted with VCTX211 units. [ Time Frame: From implantation up to 12 months post implantation ]
  • Assess the clinical efficacy of VCTX211 units via evaluation of changes in exogenous insulin use from baseline. [ Time Frame: From implantation up to 12 months post implantation ]
  • Assess the clinical efficacy of VCTX211 units via evaluation of changes in number of hypoglycemic evens from baseline. [ Time Frame: From implantation up to 12 months post implantation ]
  • Assess the clinical efficacy of VCTX211 units via evaluation of changes in hemoglobin A1C levels from baseline. [ Time Frame: From implantation up to 12 months post implantation ]
  • Assess the clinical efficacy of VCTX211 units via evaluation of percentage of time in pre-defined glycemic ranges, as measured by a continuous glucose monitor, from baseline. [ Time Frame: From implantation up to 12 months post implantation ]
  • Qualitative evaluation of immune response to VCTX211 units assessed by histological staining for markers of host adaptive immune cells within the graft. [ Time Frame: From implantation up to 12 months post implantation ]
  • Incidence of new alloreactive antibodies found in the blood of patients post implantation. [ Time Frame: From implantation up to 12 months post implantation ]
  • Incidence of new autoreactive antibodies found in the blood of patients post implantation. [ Time Frame: From implantation up to 12 months post implantation ]
  • The percentage of viable graft cells per unit using immunohistochemical staining. [ Time Frame: From implantation up to 12 months post implantation ]
  • The percentage of graft cells per unit that have differentiated into endocrine/beta cells as determined by immunohistochemical staining. [ Time Frame: From implantation up to 12 months post implantation ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE An Open-Label, FIH Study Evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With T1D
Official Title  ICMJE An Open-Label, First-in-Human Study Evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With Type 1 Diabetes Mellitus (T1D)
Brief Summary This is an open-label, multicenter, Phase 1/2 study evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects with T1D
Detailed Description VCTX211 combination product (unit) compromises 2 components: (1) allogeneic pancreatic endoderm cells (PEC211) genetically modified using Cluster Regularly Interspaced Short Palindromic Repeats/ CRISPR-associated protein 9 (CRISPR/Cas9) to promote immune evasiveness and survival, and (2) a durable, removable, perforated device designed to deliver and retain PEC211 cells.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Diabetes Mellitus
  • Diabetes Mellitus, Type 1
  • Glucose Metabolism Disorders
  • Metabolic Disease
  • Endocrine System Diseases
  • Autoimmune Diseases
  • Immune System Diseases
Intervention  ICMJE Combination Product: VCTX211
CRISPR-Cas9 genetically modified PEC211 cells loaded into a delivery device
Study Arms  ICMJE Experimental: VCTX211 unit
Intervention: Combination Product: VCTX211
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: September 30, 2022)		 40
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 2025
Estimated Primary Completion Date April 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Diagnosis of T1D for a minimum of 5 years
  • Stable diabetes regimen for at least 3 months prior to enrollment.

Exclusion Criteria:

  • Medical history of islet cell, kidney, and/or pancreas transplant
  • Occurrence of 2 or more severe, unexplained hypoglycemic events within 6 months prior to enrollment
  • Known causes of diabetes other than T1D
  • Immunosuppressant therapy in the previous 30 days and/or requirements for chronic immunosuppressive therapy during the study
  • Prior treatment with gene therapy or edited product
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Clinical Trials +1 (877) 214-4634 MedicalAffairs@crisprtx.com
Listed Location Countries  ICMJE Canada
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05565248
Other Study ID Numbers  ICMJE VCTX211-101
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party CRISPR Therapeutics ( CRISPR Therapeutics AG )
Original Responsible Party Same as current
Current Study Sponsor  ICMJE CRISPR Therapeutics AG
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE ViaCyte
Investigators  ICMJE
Study Director: Manasi Jaiman, MD, MPH ViaCyte
Study Director: Sandeep Soni, MD CRISPR Therapeutics
PRS Account CRISPR Therapeutics
Verification Date March 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP