A First-in-Human (FIH), Randomized, Dose-Escalation, Double-Blind, Placebo-Controlled Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SPC5001 Administered to Healthy Subjects and Subjects with Familial Hypercholesterolemia (FH)

A First-in-Human (FIH), Randomized, Dose-Escalation, Double-Blind, Placebo-Controlled Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SPC5001 Administered to Healthy Subjects and Subjects with Familial Hypercholesterolemia (FH) - SPC5001 in healthy subjects and subjects with FH

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON36203

Enrollment

Registered

2011-04-06

Start date

2011-04-11

Completion date

Unknown

Last updated

2024-04-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

familial hypercholesterolemia Increased blood cholesterol level

Interventions

Multiple doses of SPC5001 (0.5 mg/kg, 1.5mg/kg, 5mg/kg or 10mg/kg (dose to be determined for FH subjects, highest dose that was well-tolerated in healthy subjects)) will be evaluated in healthy subj

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

Inclusion criteria: Age: 18-65 years (healthy subjects) Age: 18-45 years (FH subjects) BMI: 18-33 kg/m2 LDL >= 2.59 mmol/L (>=100 mg/dL) Triglycerides (fasted)

Exclusion criteria

Exclusion criteria: Any uncontrolled or active major systemic disease. History or presence of malignancy in the past year. Active acute or chronic infection. Clinically significant illness within 30 days prior to the planned first drug administration. See protocol for other criteria.

Design outcomes

Primary

Measure	Time frame
Safety will be evaluated from reported adverse events, scheduled physical examinations, hepatic ultrasounds, vital signs, 12-lead ECGs, and clinical laboratory test results.	—

Secondary

Measure	Time frame
Pharmacokinetics Plasma blood samples and urine samples will be collected to determine the pharmacokinetics (PK) of SPC5001. Plasma blood samples (4mL) will be collected on day 1, 2, 8, 15, 16, 18, 22, 29, 36, 50, 64 and 78 (or at early termination) at different timepoints. Urine samples will be collected on day 1,2, 15 and 16 at different timepoints. Pharmacodynamics Pharmacodynamic (PD) blood samples (4ml) will be collected for the purpose of exploratory analysis related to the effects, mechanism, dynamics and/or adverse events of SPC5001. Total cholesterol, HDL-C, LDL-C, VLDL-C, apoB, ApoA1, triglycerides, lipoprotein particle size* and lipoprotein sub-type* (*samples will be stored for future analyses) and PCSK9. Pharmacodynamic samples will be collected during screening and on study days -1, 1, 2, 4, 8, 9, 11, 14, 15, 16, 18, 22, 29, 36, 50, 64 and 78 (or at early termination)	—

Measure

Time frame

Pharmacokinetics Plasma blood samples and urine samples will be collected to determine the pharmacokinetics (PK) of SPC5001. Plasma blood samples (4mL) will be collected on day 1, 2, 8, 15, 16, 18, 22, 29, 36, 50, 64 and 78 (or at early termination) at different timepoints. Urine samples will be collected on day 1,2, 15 and 16 at different timepoints. Pharmacodynamics Pharmacodynamic (PD) blood samples (4ml) will be collected for the purpose of exploratory analysis related to the effects, mechanism, dynamics and/or adverse events of SPC5001. Total cholesterol, HDL-C, LDL-C, VLDL-C, apoB, ApoA1, triglycerides, lipoprotein particle size* and lipoprotein sub-type* (*samples will be stored for future analyses) and PCSK9. Pharmacodynamic samples will be collected during screening and on study days -1, 1, 2, 4, 8, 9, 11, 14, 15, 16, 18, 22, 29, 36, 50, 64 and 78 (or at early termination)

—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)