A Safety, Tolerability, and Efficacy Study of VC-01™ Combination Product in Subjects With Type I Diabetes Mellitus

• Sponsor: ViaCyte (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT02239354
• Collaborator: California Institute for Regenerative Medicine (CIRM) (Other)
• Enrollment: 19
• Locations: 2
• Arms: 2
• Actual Duration (Months): 39
• Patients Per Site: 9.5
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

The purpose of this trial is to test if VC-01™ combination product can be implanted subcutaneously in subjects with Type 1 Diabetes and maintained safely for two years. It will also test if VC-01 is an effective treatment for subjects with Type 1 Diabetes.

Condition or Disease	Intervention/Treatment	Phase
Type 1 Diabetes Mellitus	Combination Product: VC-01™ Combination Product	Phase 1/Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 19 participants
• Allocation: Non-Randomized
• Intervention Model: Sequential Assignment
• Masking: None (Open Label)
• Primary Purpose: Other
• Official Title: A Prospective, Multicenter, Open-Label, First-in-Human Phase 1/2 Study With Two Cohorts to Evaluate the Safety, Tolerability, and Efficacy of Various Doses of VC-01™ Combination Product in Subjects With Type 1 Diabetes Mellitus
• Actual Study Start Date: Sep 1, 2014
• Actual Primary Completion Date: Dec 1, 2017
• Actual Study Completion Date: Dec 1, 2017

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cohort 1 2 VC-01™ Combination Product implants	Combination Product: VC-01™ Combination Product; Biologic and Device
Experimental: Cohort 2 4 or 6 VC-01™ Combination Product implants	Combination Product: VC-01™ Combination Product; Biologic and Device

Outcome Measures

Primary Outcome Measures

1. Number of Adverse Events Reported During the Study. [Thru the Month 24 Visit]

2. Change in C-peptide [Baseline to the Month 6 Visit]

   The Full Analysis Set (FAS) is the intent-to-treat (ITT) set of subjects. This set is defined as all T1DM subjects who were enrolled into the study and received implantation of at least one VC-01-250 or sentinel unit on Study Day 1. The FAS (Cohort 2 T1DM subjects who meet the FAS criteria) will be used to analyze the primary efficacy endpoint.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 55 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Men and women (non-pregnant and non-childbearing potential)

• Diagnosis of type 1 diabetes mellitus for at least 3 years

• Stable diabetic treatment

• Willingness to use a continuous glucose meter

• Acceptable candidate for implantation

Exclusion Criteria:

• Advanced complications associated with diabetes

• Immunosuppressive therapy

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of California at San Diego	San Diego	California	United States
2	University of Alberta Hospital	Edmonton	Alberta	Canada	T6G 2B7

Sponsors and Collaborators

• ViaCyte
• California Institute for Regenerative Medicine (CIRM)

Investigators

None specified.

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Mar 1, 2016

More Information

Publications

None provided.

• Responsible Party: ViaCyte
• ClinicalTrials.gov Identifier: NCT02239354
• Other Study ID Numbers: VC01-101
• First Posted: Sep 12, 2014
• Last Update Posted: Mar 24, 2022
• Last Verified: Feb 1, 2022

• Additional relevant MeSH terms: Diabetes Mellitus; Diabetes Mellitus, Type 1

Study Results

Participant Flow

Arm/Group Title	Cohort 1	Cohort 2
Arm/Group Description	Up to 2 VC-01-250™ Combination Product implants Up to 6 VC-01-20 sentinel units VC-01™ Combination Product: Biologic and Device	Up to 4 or 6 VC-01™ Combination Product implants Up to 3 VC-01-20 sentinel units VC-01™ Combination Product: Biologic and Device
Period Title: Overall Study
STARTED	19	0
COMPLETED	9	0
NOT COMPLETED	10	0

Baseline Characteristics

Arm/Group Title	Cohort 1	Cohort 2	Total
Arm/Group Description	Up to 2 VC-01-250™ Combination Product implants Up to 6 VC-01-20 sentinel units VC-01™ Combination Product: Biologic and Device	Up to 4 or 6 VC-01™ Combination Product implants Up to 3 VC-01-20 sentinel units VC-01™ Combination Product: Biologic and Device	Total of all reporting groups
Overall Participants	19	0	19
Age (years) [Median (Standard Deviation) ]
Median (Standard Deviation) [years]	29.0 (11.92)	29.0 (11.92)
Sex: Female, Male (Count of Participants)
Female	1 5.3%	1 Infinity
Male	18 94.7%	18 Infinity
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	1 5.3%	1 Infinity
Not Hispanic or Latino	18 94.7%	18 Infinity
Unknown or Not Reported	0 0%	0 NaN
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	0 NaN
Asian	0 0%	0 NaN
Native Hawaiian or Other Pacific Islander	0 0%	0 NaN
Black or African American	0 0%	0 NaN
White	19 100%	19 Infinity
More than one race	0 0%	0 NaN
Unknown or Not Reported	0 0%	0 NaN

Outcome Measures

1. Primary Outcome

Title	Number of Adverse Events Reported During the Study.
Time Frame	Thru the Month 24 Visit

Outcome Measure Data

Analysis Population Description

The Safety Analysis Set (SAS) will include all T1DM subjects who were enrolled into the study and in whom an implant surgery was attempted, regardless if any VC-01-250 units or sentinel-sized units were actually implanted. This includes all subjects in both Cohort 1 and Cohort 2.

Arm/Group Title	Cohort 1
Arm/Group Description	Up to 2 VC-01-250™ Combination Product implants Up to 6 VC-01-20 sentinel units VC-01™ Combination Product: Biologic and Device
Measure Participants	19
Number [events]	114

2. Primary Outcome

Title	Change in C-peptide
Description	The Full Analysis Set (FAS) is the intent-to-treat (ITT) set of subjects. This set is defined as all T1DM subjects who were enrolled into the study and received implantation of at least one VC-01-250 or sentinel unit on Study Day 1. The FAS (Cohort 2 T1DM subjects who meet the FAS criteria) will be used to analyze the primary efficacy endpoint.
Time Frame	Baseline to the Month 6 Visit

Outcome Measure Data

Analysis Population Description

No subjects were enrolled in Cohort 2. Therefore, no data was collected for this endpoint.

Arm/Group Title	Cohort 2
Arm/Group Description	Up to 4 or 6 VC-01™ Combination Product implants Up to 3 VC-01-20 sentinel units VC-01™ Combination Product: Biologic and Device
Measure Participants	0

Adverse Events

Time Frame	AEs are recorded on the CRF from Visit 2 (Week-3) through the last subject visit (out to two years). At the conclusion of the trial, any ongoing AEs were followed up for 28 days after the explantation procedure.
Adverse Event Reporting Description	All observed or volunteered AEs regardless of suspected causal relationship to the investigational product were reported. For all AEs, the Investigator obtained information adequate to determine the outcome of the AE, whether it meets the criteria for classification as a serious adverse event(SAE), the causality, and the severity,
Arm/Group Title	Cohort 1		Cohort 2
Arm/Group Description	Up to 2 VC-01-250™ Combination Product implants Up to 6 VC-01-20 sentinel units VC-01™ Combination Product: Biologic and Device		Up to 4 or 6 VC-01™ Combination Product implants Up to 3 VC-01-20 sentinel units VC-01™ Combination Product: Biologic and Device
All Cause Mortality
	Cohort 1		Cohort 2
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/19 (0%)		0/0 (NaN)
Serious Adverse Events
	Cohort 1		Cohort 2
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	2/19 (10.5%)		0/0 (NaN)
Infections and infestations
Cellulitis	1/19 (5.3%)	1	1/0 (Infinity)	1
Right middle lobe pneumonia	1/19 (5.3%)	1	1/0 (Infinity)	1
Other (Not Including Serious) Adverse Events
	Cohort 1		Cohort 2
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	18/19 (94.7%)		0/0 (NaN)
Blood and lymphatic system disorders
Anaemia	1/19 (5.3%)	1	1/0 (Infinity)	1
Lymphadenitis	1/19 (5.3%)	1	1/0 (Infinity)	1
Thrombocytopenia	1/19 (5.3%)	1	1/0 (Infinity)	1
White blood cell disorder	1/19 (5.3%)	1	1/0 (Infinity)	1
Gastrointestinal disorders
Nausea	3/19 (15.8%)	3	3/0 (Infinity)	3
Constipation	2/19 (10.5%)	2	2/0 (Infinity)	2
Vomiting	2/19 (10.5%)	2	2/0 (Infinity)	2
Colitis ulcerative	1/19 (5.3%)	1	1/0 (Infinity)	1
Diarrhoea	1/19 (5.3%)	1	1/0 (Infinity)	1
General disorders
Device dislocation	7/19 (36.8%)	10	7/0 (Infinity)	10
Device breakage	5/19 (26.3%)	5	5/0 (Infinity)	5
Implant site extravasation	5/19 (26.3%)	7	5/0 (Infinity)	7
Implant site haemorrhage	2/19 (10.5%)	3	2/0 (Infinity)	3
Implant site hypoaesthesia	2/19 (10.5%)	2	2/0 (Infinity)	2
Implant site swelling	2/19 (10.5%)	2	2/0 (Infinity)	2
Calcinosis	1/19 (5.3%)	1	1/0 (Infinity)	1
Complication of device removal	1/19 (5.3%)	1	1/0 (Infinity)	1
Device difficult to use	1/19 (5.3%)	1	1/0 (Infinity)	1
Device extrusion	1/19 (5.3%)	1	1/0 (Infinity)	1
Implant site oedema	1/19 (5.3%)	1	1/0 (Infinity)	1
Implant site pain	1/19 (5.3%)		1/0 (Infinity)
Implant site rash	1/19 (5.3%)	1	1/0 (Infinity)	1
Localised oedema	1/19 (5.3%)	1	1/0 (Infinity)	1
Oedema peripheral	1/19 (5.3%)	1	1/0 (Infinity)	1
Immune system disorders
Sensitisation	1/19 (5.3%)	1	1/0 (Infinity)	1
Infections and infestations
Cellulitis	2/19 (10.5%)	2	2/0 (Infinity)	2
Pneumonia	1/19 (5.3%)	1	1/0 (Infinity)	1
Wound infection	1/19 (5.3%)	1	1/0 (Infinity)	1
Injury, poisoning and procedural complications
Procedural pain	14/19 (73.7%)	30	14/0 (Infinity)	30
Contusion	2/19 (10.5%)	2	2/0 (Infinity)	2
Incision site haematoma	2/19 (10.5%)	2	2/0 (Infinity)	2
Post procedural haematoma	2/19 (10.5%)	2	2/0 (Infinity)	2
Joint dislocation	1/19 (5.3%)	1	1/0 (Infinity)	1
Post procedural inflammation	1/19 (5.3%)	1	1/0 (Infinity)	1
Post procedural swelling	1/19 (5.3%)	1	1/0 (Infinity)	1
Procedural complication	1/19 (5.3%)	1	1/0 (Infinity)	1
Tooth fracture	1/19 (5.3%)	1	1/0 (Infinity)	1
Venous injury	1/19 (5.3%)	1	1/0 (Infinity)	1
Wound	1/19 (5.3%)	1	1/0 (Infinity)	1
Wound dehiscence	1/19 (5.3%)	1	1/0 (Infinity)	1
Musculoskeletal and connective tissue disorders
Musculoskeletal pain	1/19 (5.3%)	1	1/0 (Infinity)	1
Nervous system disorders
Hypoaesthesia	1/19 (5.3%)	1	1/0 (Infinity)	1
Paraesthesia	1/19 (5.3%)	1	1/0 (Infinity)	1
Respiratory, thoracic and mediastinal disorders
Apnoea	1/19 (5.3%)	1	1/0 (Infinity)	1
Oropharyngeal pain	1/19 (5.3%)	1	1/0 (Infinity)	1
Skin and subcutaneous tissue disorders
Ecchymosis	2/19 (10.5%)	2	2/0 (Infinity)	2
Erythema	2/19 (10.5%)	2	2/0 (Infinity)	2
Acne	1/19 (5.3%)	1	1/0 (Infinity)	1
Angioedema	1/19 (5.3%)	1	1/0 (Infinity)	1
Eczema	1/19 (5.3%)	1	1/0 (Infinity)	1
Rash erythematous	1/19 (5.3%)	1	1/0 (Infinity)	1
Skin irritation	1/19 (5.3%)	1	1/0 (Infinity)	1
Vascular disorders
Thrombophlebitis superficial	1/19 (5.3%)	1	1/0 (Infinity)	1

Limitations/Caveats

Study did not proceed to Cohort 2 to evaluate efficacy.

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

If a multi-center publication is not published within 14 to 18 months (depends on the individual PI's contract) after completion of the study, the PI will have the right to publish and present results. The PI will submit any proposed publication or presentation to the Sponsor for review at least 30 to 60 days (depending on the individual PI's contract) prior to submitting any such proposed publication to a publisher, proceeding with such proposed presentation or making any other disclosure.