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Multiple Ascending Dose Study in Healthy Subjects

Phase 1
Terminated
Conditions
Amyloidosis
Interventions
Registration Number
NCT03879278
Lead Sponsor
Proclara Biosciences, Inc.
Brief Summary

This will be a Phase 1, randomized, double-blind, single center, placebo-controlled, multiple ascending dose (MAD) study in a maximum of 3 cohorts of 8 healthy male and female subjects each. Subjects in Cohorts 1, 2 and 3 will receive ascending multiple IV doses of NPT189 or matching placebo.

Detailed Description

This study is a Phase 1, randomized, double-blind, placebo-controlled, multiple dose, dose escalation safety, tolerability and PK study of NPT189 in healthy subjects. Three dose cohorts are planned each with a maximum of 8 subjects (6 active: 2 placebo) enrolled per cohort. Eligible subjects will be administered NPT189 by IV infusions at the dose specified for their cohort (i.e., 2 mg/kg, 5 mg/kg, and 12.5 mg/kg) or a matching placebo. Subjects will receive a total of 5 doses of NPT189, with doses administered at weekly intervals. Safety and tolerability will be assessed by analysis of adverse events (AEs), vital signs, electrocardiograms (ECG), laboratory and physical examinations. PK will be assessed by analysis of serum NPT189 concentrations.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
8
Inclusion Criteria
  1. Age 18-65 years, inclusive, at screening
  2. Weight 45-120 kg, inclusive
  3. Body Mass Index of 18.0-32.0 kg/m2, inclusive
  4. Female subjects of childbearing potential (defined as not surgically sterile or at least 2 years postmenopausal confirmed by a Screening Follicular Stimulating Hormone level of > 40) must agree to use two of the following forms of contraception from 60 days prior through 90 days following the dose of study drug: hormonal (oral, transdermal, implant, or injection), barrier (condom, diaphragm with spermicide), IUD, complete abstinence, or vasectomized partner (6 months minimum) or male subjects who are sexually active with women of childbearing potential must agree to complete abstinence or to use a condom for 90 days following the dose of study drug.
  5. No clinically significant abnormal findings on physical examination, vital signs, electrocardiogram (ECG), or clinical laboratory evaluation during screening; and systolic blood pressure between 140 mm and 90 mm Hg and diastolic blood pressure between 90 mm and 45 mm Hg.
  6. Can understand and sign the informed consent document, can communicate with the Investigator, and can understand and comply with the requirements of the protocol.
Exclusion Criteria
  1. Have a past or present serious medical illness or other medical or social reason that in the opinion of the Principal Investigator, would jeopardize the safety of the subject or impact the validity of the study results.
  2. Participation in a clinical trial (other than a screening visit) within 60 days of study drug administration.
  3. Use of any over-the-counter or prescription medication , vitamin preparations and other food supplements, or herbal medications (e.g., St. John's Wort) within 14 days prior to the dose of study drug or during the study, with the exception of hormonal contraceptives for women of childbearing potential, unless approved by the Principal Investigator or use of beta-blockers within 30 days prior to dose of study drug or during the study.
  4. Donation of blood or plasma within 30 days of the dose of study drug and throughout the duration of the study and for male subjects, donation of sperm through the duration of the study.
  5. Women who are nursing, pregnant, suspected of being pregnant, or trying to become pregnant, or are lactating, have a positive serum pregnancy test at screening or urine pregnancy test prior to administration of study drug regardless of childbearing potential.
  6. Positive blood screen for HIV, hepatitis B surface antigen (HbSAg), or hepatitis C as Screening, or a positive urine screen for alcohol (the day before each dose administration only) or, drugs of abuse, or cotinine at Screening and on the day before each dose administration.
  7. Have clinically significant abnormalities in laboratory values as judged by the clinical investigator.
  8. Have a history of alcoholism and/or drug abuse.
  9. Unsuitable veins for infusion or blood sampling.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Treatment 2 mg/kgNPT189Five IV doses of 2 mg/kg NPT189 (n = 6) or matching placebo (n = 2) administered once per week
Treatment 5 mg/kgNPT189Five IV doses of 5 mg/kg NPT189 (n = 6) or matching placebo (n = 2) administered once per week
Treatment 12.5 mg/kgNPT189Five IV doses of 12.5 mg/kg NPT189 (n = 6) or matching placebo (n = 2) administered once per week
Primary Outcome Measures
NameTimeMethod
Adverse event assessment in subjects receiving multiple intravenous infusions of NPT189Day 1 through Day 112

Summary of the frequency and percentage of adverse events

Secondary Outcome Measures
NameTimeMethod
Profile of pharmacokinetics (PK) of multiple intravenous (IV) infusions of NPT189 (Cmax)Day 1 through Day 112

Observed maximum concentration (Cmax)

Profile of pharmacokinetics (PK) of multiple intravenous (IV) infusions of NPT189 (Tmax)Day 1 through Day 112

Time of the maximum measured concentration (Tmax)

Profile of pharmacokinetics (PK) of multiple (IV) infusions of NPT189 (AUC 0-tau)Day 1 through Day 112

Area under the serum concentration-time curve over a dosing interval, tau

Profile of pharmacokinetics (PK) of multiple intravenous (IV) infusions of NPT189. Terminal elimination half-life (t 1/2)Day 1 through DAy 112

Terminal elimination half-life (t 1/2)

Profile of pharmacokinetics (PK) of multiple (IV) infusions of NPT189. Clearance (CL)Day 1 through Day 112

Clearance (CL)

Profile of pharmacokinetics (PK) of multiple (IV) infusions of NPT189. Volume of distribution (Vd)Day 1 through Day 112

Apparent volume of distribution (Vd)

Trial Locations

Locations (1)

PRA Health Sciences

🇳🇱

Groningen, Netherlands

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