A Phase 1, Randomized, Placebo-Controlled, Double-Blind Study in Healthy Volunteers and a Phase 2a, Open-Label Study in Patients With Hereditary Angioedema to Evaluate the Safety, Tolerability, PK and PD of ADX-324
Overview
- Phase
- Phase 1
- Intervention
- Placebo
- Conditions
- Hereditary Angioedema
- Sponsor
- ADARx Pharmaceuticals, Inc.
- Enrollment
- 53
- Locations
- 2
- Primary Endpoint
- Safety in Hereditary Angioedema
- Status
- Active, not recruiting
- Last Updated
- 5 days ago
Overview
Brief Summary
The first-in-human Phase 1 study will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-324 in healthy volunteers (HV) and in patients with Hereditary Angioedema (HAE).
Detailed Description
The clinical study described in this protocol is a Phase 1, single-center study evaluating safety, tolerability, PK, and PD of ADX-324. The study consists of 2 parts: * Randomized, double-blind, placebo-controlled, parallel group, single ascending dose (SAD) in HV with up to 6 dose cohorts. For SAD cohorts and planned dosing; and, * Expansion cohort in participants with Hereditary Angioedema (HAE) at selected dose from Part A and will be open label.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Part A - HV
- •Inclusion Criteria:
- •Male and female adults 18 to 55 years old
- •Body mass index (BMI) between 18 and 30 kg/m2
- •Contraception use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
- •Willing and able to provide informed consent and comply with all study visits
Exclusion Criteria
- •Any significant medical history
- •Active malignancy and/or history of malignancy in the past 5 years
- •History of liver disease, Gilbert's syndrome, or abnormal liver function test
- •Estimated creatinine clearance \<60 mL/min or serum creatinine \> 1.5-fold upper limit of normal.
- •Any active infection or acute illness
- •Major surgery or significant traumatic injury occurring within 3 months
- •Have any other conditions that, in the opinion of the Investigator or Sponsor, would make the participant unsuitable for inclusion, or could interfere with the participant participating in or completing the study.
- •Positive serology tests (HepB, Hep C, HIV)
- •Use of any prescription, vaccines, supplements/vitamins, or over-the counter medication
- •Treatment with another investigational product within 30 days prior to the first study drug administration
Arms & Interventions
PART A- Placebo administered to HV
For each cohort in Part A (SAD), 8 participants will be randomized in a 3:1 ratio; 6 participants to active (ADX-324): 2 participants to control (matched placebo). Randomization will be on Day 1. Initially, 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed. The sentinel participants will be evaluated for safety. The investigator's assessment and the independent medical monitor will decide upon the randomization and dosing of the 6 remaining participants (5 active and 1 placebo) according to the randomization schedule.
Intervention: Placebo
PART A - Active ADX-324 administered to HV
For each cohort in Part A (SAD), 8 participants will be randomized in a 3:1 ratio; 6 participants to active (ADX-324): 2 participants to control (matched placebo). Randomization will be on Day 1. Initially, 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed. The sentinel participants will be evaluated for safety. The investigator's assessment and the independent medical monitor will decide upon the randomization and dosing of the 6 remaining participants (5 active and 1 placebo) according to the randomization schedule.
Intervention: ADX-324
PART B - ADX-324 administered to HAE participants
This will be initiated at the dose level determined by the Safety Review Committee from SAD in HVs. The treatment of HAE participants is an open-label study.
Intervention: ADX-324
Outcomes
Primary Outcomes
Safety in Hereditary Angioedema
Time Frame: 365 days
To evaluate the safety and tolerability of ADX-324 in HAE by incidence, relationship, and severity of adverse events and serious adverse events
Safety in Healthy Volunteers
Time Frame: 365 days
To evaluate the safety and tolerability of ADX-324 in HVs by incidence, relationship, and severity of adverse events and serious adverse events
Secondary Outcomes
- Pharmacokinetics in Healthy Volunteers(8 days)
- Pharmacodynamics in Healthy Volunteers(365 days)
- Pharmacokinetics in Hereditary Angioedema(8 days)
- Pharmacodynamics in Hereditary Angioedema(365 days)
- Pharmacokinetics in Hereditary Angioedema(365 days)