A Phase 1 Study to Evaluate the Safety, Tolerability, and Immunogenicity of UB-313 in Healthy Participants

Study Overview
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Brief Summary
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This first in-human (FIH) study of UB-313, an anti-calcitonin gene-related peptide based immunotherapeutic candidate, is designed to assess the safety, tolerability, and immunogenicity of 4 selected UB-313 dose regimens in healthy adults.

Detailed Description
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This is a single-site, randomized, double-blind, placebo-controlled, multidose regimen, FIH study of UB-313, an anti-CGRP peptide-based immunotherapy candidate. The double-blind period will include dose escalation and cohort staggering for up to 4 planned dose levels of UB-313 or placebo in 4 cohorts of healthy participants. All eligible participants will be enrolled in the study for up to 44 weeks, consisting of IM injections of UB-313 or placebo at Week 0 (Baseline, Day 1), Week 4, and Week 12. The End of Treatment (EoT) is defined as Week 16, followed by a follow-up period up to Week 44.

Official Title
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A Phase 1 Randomized, Double-Blind, Placebo-Controlled, First-in-Human Study to Evaluate the Safety, Tolerability, and Immunogenicity of UBITh® CGRP Immunotherapy (UB-313) in Healthy Participants

Conditions
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Migraine

Intervention / Treatment
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* Biological: UB-313
* Biological: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Is a male or female aged 18 to 55 years old, inclusive, at time of informed consent. Has a body mass index between 18 and 30 kg/m2, inclusive at Screening, and with a minimum weight of 50 kg. Male participants and their partners of childbearing potential must commit to the use of highly effective contraceptives for the study duration and for at least 12 weeks after the last dose. Men must refrain from donating sperm during this same period. Female participants must be of nonchildbearing potential, or, for women of childbearing potential, must be willing to practice highly effective contraception throughout the duration of the study and for at least 24 weeks following the last dose. Other inclusion criteria apply Exclusion Criteria: Has a history of clinically significant medical or psychiatric conditions, which in the opinion of the Investigator may compromise the participant's safety or the scientific value of the study, posing an unacceptable risk to the participant or interfere with the participant's ability to comply with study procedures or abide by study restrictions. Presents any concern by the Investigator regarding safe participation in the study or for any other reason (including contraindication to MRI) that the Investigator considers the participant inappropriate for participation in the study. Has a recent history (within the past year of Screening) of migraine headache. Has unsuitable skin characteristics for the dermal capsaicin challenge as determined by the Investigator. Has not demonstrated at least a 100% increase in dermal blood flow following capsaicin challenge as part of Screening procedures and measured through laser speckle contrast imaging. Other exclusion criteria apply

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 55 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: UB-313 Cohort 1<br>UB-313 100mcg administered by intramuscular (IM) injection at Week 0, Week 4 and Week 12 | Biological: UB-313<br>* A synthetic peptide-based immunotherapy<br>|
| Experimental: UB-313 Cohort 2<br>UB-313 300mcg administered by IM injection at Week 0 and 100mcg at Week 4 and Week 12 | Biological: UB-313<br>* A synthetic peptide-based immunotherapy<br>|
| Experimental: UB-313 Cohort 3<br>UB-313 300mcg administered by IM injection at Week 0, Week 4 and Week 12 | Biological: UB-313<br>* A synthetic peptide-based immunotherapy<br>|
| Experimental: UB-313 Cohort 4<br>UB-313 600mcg administered by IM injection at Week 0 and 100mcg at Week 4 and Week 12 | Biological: UB-313<br>* A synthetic peptide-based immunotherapy<br>|
| Placebo Comparator: Placebo Comparator<br>Placebo (normal saline), administered by IM injection at Week 0, Week 4 and Week 12 | Biological: Placebo<br>* Normal saline<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequency of adverse events | Number of AEs will be assessed | 44 weeks | 
| Immunogenicity will be measured by serum anti-CGRP antibodies in blood | Immunogenicity will be measured by change from baseline of blood anti-CGRP antibody titers. | Weeks 0, 1, 4, 5, 8, 12, 13, 16, 20, 28, 36 and 44 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacodynamics of the immune response | The inhibition of capsaicin-induced increase in dermal blood flow | Baseline to Week 16 and either until return to baseline or until Week 44, whichever comes first. |