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

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: NCT05565248
Recruitment Status : Not yet recruiting
First Posted : October 4, 2022
Last Update Posted : October 4, 2022
Sponsor:
Collaborator:
ViaCyte
Information provided by (Responsible Party):
CRISPR Therapeutics ( CRISPR Therapeutics AG )

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
Diabetes Mellitus Diabetes Mellitus, Type 1 Glucose Metabolism Disorders Metabolic Disease Endocrine System Diseases Autoimmune Diseases Immune System Diseases 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.

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Study Type : Interventional  (Clinical Trial)
Estimated 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)
Estimated Study Start Date : November 2022
Estimated Primary Completion Date : April 2025
Estimated Study Completion Date : August 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Diabetes Type 1

Arm Intervention/treatment
Experimental: VCTX211 unit Combination Product: VCTX211
CRISPR-Cas9 genetically modified PEC211 cells loaded into a delivery device




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. [ Time Frame: 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. [ Time Frame: From implantation up to 12 months post implantation ]

Secondary Outcome Measures :
  1. Incidence of adverse events reported in patients implanted with VCTX211 units. [ Time Frame: 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. [ Time Frame: 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. [ Time Frame: 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. [ Time Frame: 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. [ Time Frame: 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. [ Time Frame: From implantation up to 12 months post implantation ]
  7. Incidence of new alloreactive antibodies found in the blood of patients post implantation. [ Time Frame: From implantation up to 12 months post implantation ]
  8. Incidence of new autoreactive antibodies found in the blood of patients post implantation. [ Time Frame: From implantation up to 12 months post implantation ]
  9. The percentage of viable graft cells per unit using immunohistochemical staining. [ Time Frame: 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. [ Time Frame: From implantation up to 12 months post implantation ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

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): NCT05565248


Contacts
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Contact: Clinical Trials +1 (877) 214-4634 MedicalAffairs@crisprtx.com

Sponsors and Collaborators
CRISPR Therapeutics AG
ViaCyte
Investigators
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Study Director: Manasi Jaiman, MD, MPH ViaCyte
Study Director: Sandeep Soni, MD CRISPR Therapeutics
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Responsible Party: CRISPR Therapeutics AG
ClinicalTrials.gov Identifier: NCT05565248    
Other Study ID Numbers: VCTX211-101
First Posted: October 4, 2022    Key Record Dates
Last Update Posted: October 4, 2022
Last Verified: September 2022

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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 ( CRISPR Therapeutics AG ):
Type 1 Diabetes
T1D
Allogeneic
Combination Device
CRISPR-Cas9
Cell Therapy
T1DM
Diabetes
VCTX
Additional relevant MeSH terms:
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Diabetes Mellitus
Diabetes Mellitus, Type 1
Metabolic Diseases
Glucose Metabolism Disorders
Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases