Phase 1 Placebo Controlled Study of the Safety, Activity and Pharmacokinetics of HQK-1001 in Healthy Subjects

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the safety, activity and pharmacokinetics of a new investigational drug (HQK-1001) in healthy human volunteers. This study will also evaluate if there are differences in these parameters when HQK-1001 is administered in a fed versus a fasted state.

Official Title
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A Phase 1, Double-Blind, Placebo Controlled, Repeat-Dose Study of the Safety, Activity and Pharmacokinetics of HQK-1001 in Healthy Volunteers

Conditions
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Healthy

Intervention / Treatment
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* Drug: HQK-1001
* Drug: placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Healthy male or female Between the ages of 18 and 45 years old Able and willing to give informed consent Able to comply with all study procedures If female, must not be of childbearing potential or must agree to use one or more of the following forms of contraception during screening and throughout the study: hormonal (i.e., oral, transdermal, implant or injection); double barrier (i.e., condom, diaphragm with spermicide); intrauterine device (IUD); vasectomized partner (6 months minimum); or abstinence Male subjects must also agree to use one or more of the above forms of birth control for either themselves or their partner, as appropriate, throughout the course of the study Not receiving medications within the 2 weeks before the first dose of study medication, except for multivitamins and contraception Complete blood count (CBC) with white blood cell (WBC) count hemoglobin, hematocrit, reticulocyte count and platelet count within normal range for the testing facility or not clinically significant Serum ferritin level > 50 ng/ml in Cohort 1 Serum ferritin level > 30 mg/ml in Cohorts 2, 3, 4 and 5 Serum chemistry values, coagulation tests and urinalysis values within the normal range for the testing facility or not clinically significant Negative urine test for substances of abuse including marijuana, cocaine, opiates, and methadone Exclusion Criteria: Prior participation in HQP 2007-001 Clinically significant abnormal vital signs Blood donation within 2 months of study medication administration Blood transfusion within 3 months of study medication administration An acute febrile illness or upper respiratory tract illness within 72 hours prior to administration of study medication Received another investigational agent within 4 weeks before administration of study medication Receiving any other investigational agent during this study Any acute or chronic disease (e.g., history of hepatitis B or C or HIV-1) Heart disease including an abnormal electrocardiogram, clinical significant, (ECG) or cardiac arrhythmia History of neurological disease, such as a seizure disorder Currently pregnant or breast feeding a child A smoker in the past 12 months Body Mass Index (BMI) >33 kg/m2

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 45 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: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br>HQK-1001 | Drug: HQK-1001<br>* HQK-1001 (5, 10 or 15 mg/kg) capsules administered once a day, orally, for 14 days.<br>|
| Placebo Comparator: 2<br> | Drug: placebo<br>* Matching placebo capsules administered once a day, orally, for 14 days.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam |  | 41 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacokinetics assessed by plasma drug concentration levels |  | 15 days | 
| Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin |  | 41 days | 
| Comparison of safety, PK and PD under fed versus fasting conditions |  | 41 days | 
| Comparison of safety and PK when oral iron is administered with HQK-1001. |  | 41 days |