Neuropeptide Therapy of Recent Onset Type 1 Diabetes
Primary Purpose
Diabetes Mellitus, Type 1
Status
Unknown status
Phase
Phase 1
Locations
Canada
Study Type
Interventional
Intervention
Substance P
Sponsored by
About this trial
This is an interventional treatment trial for Diabetes Mellitus, Type 1 focused on measuring Neuropeptide, Therapy, Onset
Eligibility Criteria
Inclusion Criteria:
- Recent onset T1D (CDA 2013 guidelines: See link in links section
- Age 10-18 years
- Disease Duration 3-30 months
- Fasting C-Peptide (measured at screening) greater than or equal to 33 pmol/L
- Post honeymoon phase based on the following criteria: History of HbA1c values and insulin requirements from diagnosis indicating a honeymoon period followed by increased insulin requirements which at time of recruitment are > 0.50 units/Kg together with an HbA1c value > 7.2 %; Patients with diabetes duration > 3 months who never experienced a honeymoon period as reflected by consistent HbA1c values > 7.2 % from the time of diagnosis and at the time of recruitment are using an insulin dose > 0.50 units/Kg.
- The presence of one or more of the TRPV1 alleles similar to those found in patients currently enrolled in the TRPV1-North American - European Study.
- Stimulated C-Peptide (measured at mixed meal tolerance at Stage A and Stage B of sP trial) ≥ 200 pmol/L and less than or equal to 1500 pmol/L.
Exclusion Criteria:
- Patients with known co-morbidities, including ACE-inhibitor treated hypertension as well as chromosomal abnormalities, involving one or more organ systems.
- Type 2 Diabetes Mellitus
- Patients with a known radiographic contrast allergy
- Pregnancy.
Sites / Locations
- Hospital for Sick ChildrenRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Experimental
Experimental
Experimental
Experimental
Arm Label
Substance P - 1nmol/kg
Substance P - 5nmol/kg
Substance P - 15nmol/kg
Substance P - 45nmol/kg
Arm Description
Substance P 1nmol/kg intra-celiac artery, single treatment
Substance P 5nmol/kg intra-celiac artery, single treatment
Substance P 15nmol/kg intra-celiac artery, single treatment
Substance P 45nmol/kg intra-celiac artery, single treatment
Outcomes
Primary Outcome Measures
Stage A Safety: Side effects reported for entire cohort
To determine if there are unexpected adverse events with intra arterial delivery of sP into the celiac artery in individuals with Type 1 Diabetes, and evidence of residual beta cell function as reflected by a peak C---peptide levels of > 200 pmol/L. Multiple measurements per patient will be aggregated to arrive at one reported value: Number of participants with abnormal laboratory values, adverse events and/or peak C-peptide levels >200pmol/L that are related to treatment.
Secondary Outcome Measures
C-Peptide Levels (small cohort)
To determine in an initial small cohort (n=12, toxicity and dose finding cohort) if one or more sP doses significantly increases the basal or stimulated c---Peptide levels at Day 20---27 post---injection using data from Mixed Meal Tolerance Test (MMTT).
C-Peptide Levels (large cohort)
To assess in a larger cohort (n=40, continued safety and efficacy) whether sP dose, determined from the dose finding cohort, significantly increases basal or stimulated C-- peptide levels at Day 20---27 post sP---injection using MMTT data.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02820558
Brief Title
Neuropeptide Therapy of Recent Onset Type 1 Diabetes
Official Title
A Phase I Study of Safety and Pharmacological Activity of Substance P (sP) in the Reversal of Recent-Onset Type 1 Diabetes (T1D)
Study Type
Interventional
2. Study Status
Record Verification Date
May 2020
Overall Recruitment Status
Unknown status
Study Start Date
May 2016 (undefined)
Primary Completion Date
December 2022 (Anticipated)
Study Completion Date
December 2022 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vanilloid Genetics Inc.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This study evaluates the delivery of substance P via the celiac artery in the treatment of recent onset type 1 diabetes.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Diabetes Mellitus, Type 1
Keywords
Neuropeptide, Therapy, Onset
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
12 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Substance P - 1nmol/kg
Arm Type
Experimental
Arm Description
Substance P 1nmol/kg intra-celiac artery, single treatment
Arm Title
Substance P - 5nmol/kg
Arm Type
Experimental
Arm Description
Substance P 5nmol/kg intra-celiac artery, single treatment
Arm Title
Substance P - 15nmol/kg
Arm Type
Experimental
Arm Description
Substance P 15nmol/kg intra-celiac artery, single treatment
Arm Title
Substance P - 45nmol/kg
Arm Type
Experimental
Arm Description
Substance P 45nmol/kg intra-celiac artery, single treatment
Intervention Type
Drug
Intervention Name(s)
Substance P
Primary Outcome Measure Information:
Title
Stage A Safety: Side effects reported for entire cohort
Description
To determine if there are unexpected adverse events with intra arterial delivery of sP into the celiac artery in individuals with Type 1 Diabetes, and evidence of residual beta cell function as reflected by a peak C---peptide levels of > 200 pmol/L. Multiple measurements per patient will be aggregated to arrive at one reported value: Number of participants with abnormal laboratory values, adverse events and/or peak C-peptide levels >200pmol/L that are related to treatment.
Time Frame
Reported during the first 20-27 days following sP administration
Secondary Outcome Measure Information:
Title
C-Peptide Levels (small cohort)
Description
To determine in an initial small cohort (n=12, toxicity and dose finding cohort) if one or more sP doses significantly increases the basal or stimulated c---Peptide levels at Day 20---27 post---injection using data from Mixed Meal Tolerance Test (MMTT).
Time Frame
Day 20-27 post sP injection
Title
C-Peptide Levels (large cohort)
Description
To assess in a larger cohort (n=40, continued safety and efficacy) whether sP dose, determined from the dose finding cohort, significantly increases basal or stimulated C-- peptide levels at Day 20---27 post sP---injection using MMTT data.
Time Frame
Day 20-27 post sP injection
Other Pre-specified Outcome Measures:
Title
sP Longevity
Description
To determine an estimate of sP longevity in the entire cohort of patients, by monitoring HbA1c values, exogenous insulin requirement, daily recorded blood sugar levels and adverse event recording at 3 and 6 months post injection of sP. Multiple measurements per patient will be aggregated to arrive at one reported value for the aforementioned outcome measures.
Time Frame
3 and 6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
10 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Recent onset T1D (CDA 2013 guidelines: See link in links section
Age 10-18 years
Disease Duration 3-30 months
Fasting C-Peptide (measured at screening) greater than or equal to 33 pmol/L
Post honeymoon phase based on the following criteria: History of HbA1c values and insulin requirements from diagnosis indicating a honeymoon period followed by increased insulin requirements which at time of recruitment are > 0.50 units/Kg together with an HbA1c value > 7.2 %; Patients with diabetes duration > 3 months who never experienced a honeymoon period as reflected by consistent HbA1c values > 7.2 % from the time of diagnosis and at the time of recruitment are using an insulin dose > 0.50 units/Kg.
The presence of one or more of the TRPV1 alleles similar to those found in patients currently enrolled in the TRPV1-North American - European Study.
Stimulated C-Peptide (measured at mixed meal tolerance at Stage A and Stage B of sP trial) ≥ 200 pmol/L and less than or equal to 1500 pmol/L.
Exclusion Criteria:
Patients with known co-morbidities, including ACE-inhibitor treated hypertension as well as chromosomal abnormalities, involving one or more organ systems.
Type 2 Diabetes Mellitus
Patients with a known radiographic contrast allergy
Pregnancy.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Holly Tschirhart
Phone
416-813-7654
Ext
204517
Email
holly.tschirhart@sickkids.ca
First Name & Middle Initial & Last Name or Official Title & Degree
Catherine Pastor
Phone
416-813-7654
Ext
204396
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Etienne Sochett, MD
Organizational Affiliation
Hospital for Sick Children, Toronto Ontario
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hospital for Sick Children
City
Toronto
State/Province
Ontario
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Holly Tschirhart
Phone
416-813-7654
Ext
204517
Email
holly.tschirhart@sickkids.ca
First Name & Middle Initial & Last Name & Degree
Catherine Pastor
Phone
416-813-7654
Ext
204396
Email
catherine.pastor@sickkids.ca
12. IPD Sharing Statement
Plan to Share IPD
Yes
Links:
URL
http://guidelines.diabetes.ca/Browse/Chapter3
Description
CDA 2013 guidelines
Learn more about this trial
Neuropeptide Therapy of Recent Onset Type 1 Diabetes
We'll reach out to this number within 24 hrs