EUCTR2011-005400-15-GR
Active, not recruiting
Phase 1
A Multicenter, Single Arm, Open-label Study to Assess the Long-term Safety and Efficacy of AMG 145 on LDL-C in Subjects With Homozygous Familial Hypercholesterolemia or PCSK9 Mutations
ConditionsHomozygous Familial HypercholesterolemiaMedDRA version: 14.1Level: LLTClassification code 10057100Term: Homozygous familial hypercholesterolaemiaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Homozygous Familial Hypercholesterolemia
- Sponsor
- Amgen Inc
- Enrollment
- 75
- Status
- Active, not recruiting
- Last Updated
- 7 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Subjects have completed study 20110233 or another qualifying Amgen protocol (all scheduled visits) and are still taking investigational product at the end of the study
- •If the subjects did not complete study 20110233 or another qualifying Amgen protocol they will be eligible if they:
- •\- Have severe familial hypercholesterolemia due to genetic causes (beyond LDL receptor mutations) which were not studied in 20110233, such as PCSK9 gain of function mutations, as evidenced by genetic or functional evidence.
- •\- Have a diagnosis of homozygous familial hypercholesterolemia and are interested in enrolling after 20110233 has closed. As stated in protocol 20110233, the diagnosis of homozygous familial hypercholesterolemia will be based on genetic confirmation or a clinical diagnosis based on a history of an untreated LDL cholesterol concentration greater than 500
- •mg/dl (13 mmol/L) together with either xanthoma before 10 years of age or evidence of heterozygous familial hypercholesterolemia in both parents.
- •\- Are male or female \= 12 to \= 65 years of age
- •\- Are on a stable on a low\-fat diet and taking pre\-existing lipid\-lowering therapies (such as statins, cholesterol\-absorption inhibitors, bile\-acid sequestrants or nicotinic acid, or combinations thereof) for at least 4 weeks, with fasting central lab LDL cholesterol concentration \>130 (3\.4 mmol/L)
- •\- Fasting triglycerides \= 400 mg/dL (4\.5 mmol/L) by central laboratory at screening
- •\- Bodyweight of 40 kg or greater at screening
- •Are the trial subjects under 18? yes
Exclusion Criteria
- •All subjects will be ineligible for the study if they fulfill any of the following criteria:
- •\- Female subject is not willing to use at least one highly effective method of birth control during treatment and for an additional 15 weeks after the end of treatment unless subject is sterilized or postmenopausal;
- •Menopause is defined as 12 months of spontaneous and continuous amenorrhea in a female \= 55 years old or 12 months of spontaneous and continuous amenorrhea with a follicle\-stimulating hormone level \>40 IU/L (or according to the definition of postmenopausal range for the laboratory involved) in a female \< 55 years old unless the subject has undergone bilateral oophorectomy”
- •Highly effective methods of birth control include abstinence, birth control pills, shots, implants, or patches, intrauterine devices (IUDs), sexual activity with a male partner who has had a vasectomy, condom or occlusive cap (diaphragm or cervical/vault caps) used with spermicide.
- •\- Subject is pregnant or breast feeding, or might become pregnant during treatment and/or within 15 weeks after the end of treatment
- •\- Unreliability as a study participant based on the investigator's (or designee’s) knowledge of the subject (eg, inability or unwillingness to adhere to the protocol)
- •\- Experienced a treatment related serious adverse event in the parent study
- •\- Disorder that would interfere with understanding and giving informed consent or compliance with protocol requirements
- •Have an unstable medical condition, in the judgment of the investigator.
- •\- Currently enrolled in another investigational device or drug study, or less than 30 days since ending another investigational device or drug study(s), or receiving other investigational agent(s)
Outcomes
Primary Outcomes
Not specified
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