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

Sponsor
CRISPR Therapeutics AG (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05565248
Collaborator
ViaCyte (Industry)
40
1
33

Study Details

Study Description

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

Condition or Disease Intervention/Treatment Phase
  • Combination Product: VCTX211
Phase 1/Phase 2

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 Design

Study Type:
Interventional
Anticipated Enrollment :
40 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
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)
Anticipated Study Start Date :
Nov 1, 2022
Anticipated Primary Completion Date :
Apr 1, 2025
Anticipated Study Completion Date :
Aug 1, 2025

Arms and Interventions

Arm Intervention/Treatment
Experimental: VCTX211 unit

Combination Product: VCTX211
CRISPR-Cas9 genetically modified PEC211 cells loaded into a delivery device

Outcome Measures

Primary Outcome Measures

  1. 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. [From implantation up to 12 months post implantation]

  2. Assess the clinical efficacy of VCTX211 units via evaluation of C-peptide increase from the baseline. [From implantation up to 12 months post implantation]

Secondary Outcome Measures

  1. Incidence of adverse events reported in patients implanted with VCTX211 units. [From implantation up to 12 months post implantation]

  2. Assess the clinical efficacy of VCTX211 units via evaluation of changes in exogenous insulin use from baseline. [From implantation up to 12 months post implantation]

  3. Assess the clinical efficacy of VCTX211 units via evaluation of changes in number of hypoglycemic evens from baseline. [From implantation up to 12 months post implantation]

  4. Assess the clinical efficacy of VCTX211 units via evaluation of changes in hemoglobin A1C levels from baseline. [From implantation up to 12 months post implantation]

  5. 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. [From implantation up to 12 months post implantation]

  6. Qualitative evaluation of immune response to VCTX211 units assessed by histological staining for markers of host adaptive immune cells within the graft. [From implantation up to 12 months post implantation]

  7. Incidence of new alloreactive antibodies found in the blood of patients post implantation. [From implantation up to 12 months post implantation]

  8. Incidence of new autoreactive antibodies found in the blood of patients post implantation. [From implantation up to 12 months post implantation]

  9. The percentage of viable graft cells per unit using immunohistochemical staining. [From implantation up to 12 months post implantation]

  10. The percentage of graft cells per unit that have differentiated into endocrine/beta cells as determined by immunohistochemical staining. [From implantation up to 12 months post implantation]

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years to 65 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

  • CRISPR Therapeutics AG
  • ViaCyte

Investigators

  • Study Director: Manasi Jaiman, MD, MPH, ViaCyte
  • Study Director: Sandeep Soni, MD, CRISPR Therapeutics

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
CRISPR Therapeutics AG
ClinicalTrials.gov Identifier:
NCT05565248
Other Study ID Numbers:
  • VCTX211-101
First Posted:
Oct 4, 2022
Last Update Posted:
Oct 4, 2022
Last Verified:
Sep 1, 2022
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
Keywords provided by CRISPR Therapeutics AG
Additional relevant MeSH terms:

Study Results

No Results Posted as of Oct 4, 2022