Celiac Disease
Conditions
Keywords
Phase 1, Double blind, Multicenter
Brief summary
A safety study of KAN-101 in patients with celiac disease. The study has two parts: 1. Part A - first in human study in which patients receive a single dose of KAN-101 2. Part B - patients will receive three doses of either KAN-101 or placebo
Detailed description
Study KAN-101-01 is a Phase 1, FIH study designed to evaluate the safety and tolerability of KAN-101 in patients with celiac disease on a gluten free diet (GFD). An overview of the two parts and proposed dose groups is given below: 1. Part A (SAD): Patients will receive a single dose of KAN-101. 2. Part B (MAD): Patients will receive three doses of either KAN-101 or placebo.
Interventions
DRUGKAN-101
Intravenous (IV) infusion
DRUGPlacebo
Intravenous (IV) infusion
Sponsors
Kanyos Bio, Inc., a wholly-owned subsidiary of Anokion SA
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Adults aged 18 to 70 years inclusive 2. Diagnosed with celiac disease based on positive serology (eg, tissue transglutaminase IgA antibody and/or deamidated gliadin peptide IgG) and intestinal histology consistent with ≥ Marsh Type II or with evidence of villous atrophy 3. Has HLA-DQ2.5 genotype (HLA-DQA1\*05 and HLA-DQB1\*02) (homozygotes or heterozygotes) 4. Has followed a GFD for \> 12 months immediately prior to study entry 5. Negative or weak positive for tTG-IgA and negative or weak positive for DGP-IgA/IgG during screening 6. Male or female. Females of childbearing potential must use at least 2 acceptable birth control methods 7. Capable of understanding and complying with protocol requirements 8. Patient understands and has signed the informed consent form Key
Exclusion criteria
1. Refractory celiac disease 2. Selective IgA deficiency 3. Positive for HLA-DQ8 (DQA1\*03, DQB1\*0302) 4. Previous treatment with tolerance-inducing therapies for celiac disease 5. Known wheat allergy 6. Part B only: History of hyperacute or prolonged symptoms following gluten exposure 7. Uncontrolled or significant medical conditions (including active infections or chronic hepatitis) which, in the opinion of the Investigator, preclude participation 8. History of dermatitis herpetiformis 9. Pregnant or breastfeeding
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | Up to 28 Days | Incidence and severity of treatment-emergent adverse events (TEAEs) as assessed by the Common Terminology Criteria for Adverse Events (CTCAE) v5.0 or higher |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Cmax | 0, 7, 15, 30, 60, 120, 180, 240, 300, 360 minutes post dose on Days 1 and and 7 | Geometric mean of maximum drug concentration (Cmax) |
| AUC Last | 0, 7, 15, 30, 60, 120, 180, 240, 300, 360 minutes post dose on Days 1 and and 7 | Area under the plasma concentration-time curve from time 0 to the last measurable time point (AUC last) |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| SAD 0.15mg/kg All enrolled patients will receive one dose of 0.15mg/kg KAN-101
KAN-101: Intravenous (IV) infusion | 4 |
| SAD 0.3mg/kg All enrolled patients will receive one dose of 0.3mg/kg KAN-101
KAN-101: Intravenous (IV) infusion | 3 |
| SAD 0.6mg/kg All enrolled patients will receive one dose of 0.6mg/kg KAN-101
KAN-101: Intravenous (IV) infusion | 3 |
| SAD 1.2mg/kg All enrolled patients will receive one dose of 1.2mg/kg KAN-101
KAN-101: Intravenous (IV) infusion | 3 |
| SAD 1.5mg/kg All enrolled patients will receive one dose of 1.5mg/kg KAN-101
KAN-101: Intravenous (IV) infusion | 1 |
| MAD 0.15mg/kg All randomized patients will receive 3 doses of 0.15mg/kg KAN-101
KAN-101: Intravenous (IV) infusion | 6 |
| MAD 0.3mg/kg All randomized patients will receive 3 doses of 0.3mg/kg KAN-101
KAN-101: Intravenous (IV) infusion | 7 |
| MAD 0.6mg/kg All randomized patients will receive 3 doses of 0.6mg/kg KAN-101
KAN-101: Intravenous (IV) infusion | 8 |
| MAD Placebo All randomized patients will receive 3 doses of placebo
Placebo: Intravenous (IV) infusion | 6 |
| Total | 41 |
Baseline characteristics
| Characteristic | SAD 0.15mg/kg | Total | MAD Placebo | MAD 0.6mg/kg | MAD 0.3mg/kg | MAD 0.15mg/kg | SAD 1.5mg/kg | SAD 1.2mg/kg | SAD 0.6mg/kg | SAD 0.3mg/kg |
|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | 47.8 years STANDARD_DEVIATION 19.05 | 38 years STANDARD_DEVIATION 13.9 | 30.3 years STANDARD_DEVIATION 10.88 | 36.3 years STANDARD_DEVIATION 16.19 | 40.7 years STANDARD_DEVIATION 10.69 | 47.2 years STANDARD_DEVIATION 12.32 | 46 years STANDARD_DEVIATION 0 | 29.3 years STANDARD_DEVIATION 11.37 | 35.3 years STANDARD_DEVIATION 9.29 | 30.3 years STANDARD_DEVIATION 15.72 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 4 Participants | 41 Participants | 6 Participants | 8 Participants | 7 Participants | 6 Participants | 1 Participants | 3 Participants | 3 Participants | 3 Participants |
| Region of Enrollment United States | 4 participants | 41 participants | 6 participants | 8 participants | 7 participants | 6 participants | 1 participants | 3 participants | 3 participants | 3 participants |
| Sex: Female, Male Female | 4 Participants | 31 Participants | 3 Participants | 7 Participants | 5 Participants | 5 Participants | 1 Participants | 1 Participants | 2 Participants | 3 Participants |
| Sex: Female, Male Male | 0 Participants | 10 Participants | 3 Participants | 1 Participants | 2 Participants | 1 Participants | 0 Participants | 2 Participants | 1 Participants | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk | EG006 affected / at risk | EG007 affected / at risk | EG008 affected / at risk |
|---|---|---|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 4 | 0 / 3 | 0 / 3 | 0 / 3 | 0 / 1 | 0 / 6 | 0 / 7 | 0 / 8 | 0 / 6 |
| other Total, other adverse events | 4 / 4 | 3 / 3 | 2 / 3 | 3 / 3 | 1 / 1 | 6 / 6 | 5 / 7 | 7 / 8 | 4 / 6 |
| serious Total, serious adverse events | 0 / 4 | 0 / 3 | 0 / 3 | 0 / 3 | 0 / 1 | 0 / 6 | 0 / 7 | 0 / 8 | 0 / 6 |
Outcome results
Primary
Incidence and Severity of Treatment-emergent Adverse Events (TEAEs)
Incidence and severity of treatment-emergent adverse events (TEAEs) as assessed by the Common Terminology Criteria for Adverse Events (CTCAE) v5.0 or higher
Time frame: Up to 28 Days
Population: All patients who received any amount of study drug with treatment group based on the dose level received.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| SAD 0.15mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | TEAE | 4 Participants |
| SAD 0.15mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | Grade 3 or higher TEAE | 0 Participants |
| SAD 0.15mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | SAE | 0 Participants |
| SAD 0.15mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | no TEAE | 0 Participants |
| SAD 0.3mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | no TEAE | 0 Participants |
| SAD 0.3mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | TEAE | 3 Participants |
| SAD 0.3mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | Grade 3 or higher TEAE | 0 Participants |
| SAD 0.3mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | SAE | 0 Participants |
| SAD 0.6mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | Grade 3 or higher TEAE | 0 Participants |
| SAD 0.6mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | no TEAE | 1 Participants |
| SAD 0.6mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | SAE | 0 Participants |
| SAD 0.6mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | TEAE | 2 Participants |
| SAD 1.2mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | TEAE | 3 Participants |
| SAD 1.2mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | Grade 3 or higher TEAE | 0 Participants |
| SAD 1.2mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | SAE | 0 Participants |
| SAD 1.2mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | no TEAE | 0 Participants |
| SAD 1.5mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | SAE | 0 Participants |
| SAD 1.5mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | Grade 3 or higher TEAE | 0 Participants |
| SAD 1.5mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | TEAE | 1 Participants |
| SAD 1.5mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | no TEAE | 0 Participants |
| MAD 0.15mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | no TEAE | 0 Participants |
| MAD 0.15mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | TEAE | 6 Participants |
| MAD 0.15mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | SAE | 0 Participants |
| MAD 0.15mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | Grade 3 or higher TEAE | 0 Participants |
| MAD 0.3mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | SAE | 0 Participants |
| MAD 0.3mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | Grade 3 or higher TEAE | 0 Participants |
| MAD 0.3mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | no TEAE | 2 Participants |
| MAD 0.3mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | TEAE | 5 Participants |
| MAD 0.6mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | Grade 3 or higher TEAE | 0 Participants |
| MAD 0.6mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | no TEAE | 1 Participants |
| MAD 0.6mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | SAE | 0 Participants |
| MAD 0.6mg/kg | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | TEAE | 7 Participants |
| MAD Placebo | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | TEAE | 4 Participants |
| MAD Placebo | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | no TEAE | 2 Participants |
| MAD Placebo | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | SAE | 0 Participants |
| MAD Placebo | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) | Grade 3 or higher TEAE | 0 Participants |
Secondary
AUC Last
Area under the plasma concentration-time curve from time 0 to the last measurable time point (AUC last)
Time frame: 0, 7, 15, 30, 60, 120, 180, 240, 300, 360 minutes post dose on Days 1 and and 7
Population: All patients who received at least 1 dose of KAN-101 and have at least 1 drug concentration value. A patient may have been excluded from summary statistics due to insufficient data or failure to meet acceptability criteria
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| SAD 0.15mg/kg | AUC Last | Day 1 | 366.8 h*ng/mL | Standard Deviation 147.7 |
| SAD 0.3mg/kg | AUC Last | Day 1 | 646.3 h*ng/mL | Standard Deviation 526.7 |
| SAD 0.6mg/kg | AUC Last | Day 1 | 5365 h*ng/mL | Standard Deviation 62.85 |
| SAD 1.2mg/kg | AUC Last | Day 1 | 13920 h*ng/mL | Standard Deviation 3033 |
| SAD 1.5mg/kg | AUC Last | Day 1 | 16340 h*ng/mL | — |
| MAD 0.15mg/kg | AUC Last | Day 7 | 205 h*ng/mL | Standard Deviation 152 |
| MAD 0.15mg/kg | AUC Last | Day 1 | 322.5 h*ng/mL | Standard Deviation 290.7 |
| MAD 0.3mg/kg | AUC Last | Day 7 | 1258 h*ng/mL | Standard Deviation 751 |
| MAD 0.3mg/kg | AUC Last | Day 1 | 1180 h*ng/mL | Standard Deviation 653.7 |
| MAD 0.6mg/kg | AUC Last | Day 7 | 3914 h*ng/mL | Standard Deviation 760 |
| MAD 0.6mg/kg | AUC Last | Day 1 | 3462 h*ng/mL | Standard Deviation 986.4 |
Secondary
Cmax
Geometric mean of maximum drug concentration (Cmax)
Time frame: 0, 7, 15, 30, 60, 120, 180, 240, 300, 360 minutes post dose on Days 1 and and 7
Population: All patients who received at least 1 dose of KAN-101 and have at least 1 drug concentration value. A patient may have been excluded from summary statistics due to insufficient data or failure to meet acceptability criteria
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| SAD 0.15mg/kg | Cmax | Day 1 | 839 ng/mL | Standard Deviation 498 |
| SAD 0.3mg/kg | Cmax | Day 1 | 1796 ng/mL | Standard Deviation 1443 |
| SAD 0.6mg/kg | Cmax | Day 1 | 10900 ng/mL | Standard Deviation 1140 |
| SAD 1.2mg/kg | Cmax | Day 1 | 19470 ng/mL | Standard Deviation 3336 |
| SAD 1.5mg/kg | Cmax | Day 1 | 22350 ng/mL | — |
| MAD 0.15mg/kg | Cmax | Day 1 | 967 ng/mL | Standard Deviation 917 |
| MAD 0.15mg/kg | Cmax | Day 7 | 588 ng/mL | Standard Deviation 375 |
| MAD 0.3mg/kg | Cmax | Day 1 | 2527 ng/mL | Standard Deviation 1299 |
| MAD 0.3mg/kg | Cmax | Day 7 | 2937 ng/mL | Standard Deviation 1539 |
| MAD 0.6mg/kg | Cmax | Day 7 | 6609 ng/mL | Standard Deviation 754.7 |
| MAD 0.6mg/kg | Cmax | Day 1 | 5753 ng/mL | Standard Deviation 1623 |