Homozygous Familial Hypercholesterolemia HoFH Clinical Trial
— RAMANOfficial title:
A Multicenter, Open-label, Single-arm, Study to Evaluate Safety and Tolerability of Repatha in Patients With Homozygous Familial Hypercholesterolemia (HoFH) in India
| Verified date | May 2024 |
| Source | Amgen |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
To describe the safety and tolerability of evolocumab in participants with homozygous familial hypercholesterolemia (HoFH) in India. All participants will receive evolocumab over an 8-week period.
| Status | Completed |
| Enrollment | 30 |
| Est. completion date | November 27, 2019 |
| Est. primary completion date | November 27, 2019 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 12 Years to 80 Years |
| Eligibility | Inclusion Criteria: - Male or female = 12 to = 80 years of age at the time of signing the informed consent - Diagnosis of HoFH based on low-density lipoprotein cholesterol (LDL-C), familial history and xanthoma - On a low-fat diet and receiving background lipid-lowering therapy stable for 4 weeks prior to screening and during the time frame of the trial - Fasting LDL-C at screening > 130 mg/dL (3.4 mmol/L) - Fasting triglycerides at screening = 400 mg/dL (4.5 mmol/L) Exclusion Criteria: - Use of mipomersen or lomitapide within 6 months of screening. - Known active infection or major hematologic, renal, metabolic, gastrointestinal, hepatic, or endocrine dysfunction - Currently receiving treatment in another investigational device or drug study, or less than 30 days since ending treatment on another investigational device or drug study(ies) - Female subject is pregnant or breastfeeding or planning to become pregnant or breastfeed - Female subjects of childbearing potential unwilling to use an acceptable method of effective contraception - Subject has known sensitivity to any of the products to be administered during dosing - History or evidence of any other clinically significant disorder, condition or disease - Subject has previously received evolocumab or any other proprotein convertase subtilisin/kexin type 9 (PKSK-9)-inhibiting therapy |
| Country | Name | City | State |
|---|---|---|---|
| India | Research Site | Ahmedabad | Gujarat |
| India | Research Site | Bangalore | Karnataka |
| India | Research Site | Belagavi | Karnataka |
| India | Research Site | Kochi | Kerala |
| India | Research Site | Lucknow | Uttar Pradesh |
| India | Research Site | Mumbai | Maharashtra |
| India | Research Site | New Delhi | Delhi |
| India | Research Site | New Delhi | Delhi |
| India | Research Site | New Delhi | Delhi |
| India | Research Site | New Delhi | Delhi |
| India | Research Site | Pune | Maharashtra |
| India | Research Site | Pune | Maharashtra |
| Lead Sponsor | Collaborator |
|---|---|
| Amgen |
India,
Raal FJ, Hegele RA, Ruzza A, Lopez JAG, Bhatia AK, Wu J, Wang H, Gaudet D, Wiegman A, Wang J, Santos RD. Evolocumab Treatment in Pediatric Patients With Homozygous Familial Hypercholesterolemia: Pooled Data From Three Open-Label Studies. Arterioscler Thromb Vasc Biol. 2024 May;44(5):1156-1164. doi: 10.1161/ATVBAHA.123.320268. Epub 2024 Mar 28. — View Citation
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Includes both serious and non-serious adverse events (AEs). AE: any untoward medical occurrence in a participant. SAE: an AE that meets 1 on the following serious criteria: fatal; life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity; congenital anomaly/birth defect; other medically important serious event. TEAE: any AE starting on or after the first dose of study drug and up to and including 30 days after the end of study drug or the end of study date, whichever is earlier. | 12 weeks | |
| Secondary | Percent Change From Baseline to Week 12 in Low-Density Lipoprotein Cholesterol (LDL-C) | baseline, week 12 | ||
| Secondary | Percent Change From Baseline to Week 12 in Apolipoprotein B (ApoB) | baseline, week 12 | ||
| Secondary | Percent Change From Baseline to Week 12 in Lipoprotein(a) (Lp[a]) | baseline, week 12 |