Neuropeptide Therapy of Recent Onset Type 1 Diabetes

Sponsor

Vanilloid Genetics Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT02820558

Collaborator

(none)

Enrollment

Location

Arms

Duration (Months)

0.2

Patients Per Site Per Month

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Diabetes Mellitus, Type 1	Drug: Substance P	Phase 1

Study Design

Study Type:

Interventional

Anticipated Enrollment :

12 participants

Allocation:

Non-Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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 Start Date :

May 1, 2016

Anticipated Primary Completion Date :

Dec 1, 2022

Anticipated Study Completion Date :

Dec 1, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Substance P - 1nmol/kg Substance P 1nmol/kg intra-celiac artery, single treatment	Drug: Substance P
Experimental: Substance P - 5nmol/kg Substance P 5nmol/kg intra-celiac artery, single treatment	Drug: Substance P
Experimental: Substance P - 15nmol/kg Substance P 15nmol/kg intra-celiac artery, single treatment	Drug: Substance P
Experimental: Substance P - 45nmol/kg Substance P 45nmol/kg intra-celiac artery, single treatment	Drug: Substance P

Outcome Measures

Primary Outcome Measures

Stage A Safety: Side effects reported for entire cohort [Reported during the first 20-27 days following sP administration]
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) [Day 20-27 post sP injection]
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) [Day 20-27 post sP injection]
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.

Other Outcome Measures

sP Longevity [3 and 6 months]
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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

10 Years to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.