A study to investigate the safety and efficacy of study drug OMS721 in adults and adolescents with Atypical Hemolytic Uremic Syndrome.
- Conditions
- Atypical Hemolytic Uremic Syndrome (aHUS)MedDRA version: 20.0Level: LLTClassification code 10019515Term: Hemolytic uremic syndromeSystem Organ Class: 100000004851Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2017-002057-11-PL
- Lead Sponsor
- Omeros Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 80
1. Competent to provide informed consent or, if a minor, have at least one parent or legal guardian to provide informed consent with written assent from the subject.
2. If an adult, voluntarily provide informed consent in accordance with regulations and governing ethics committee requirements prior to any procedures or evaluations performed specifically for the sole purpose of the study. If a minor, at least one parent or legal guardian must provide informed consent and the subject must provide assent in accordance with local regulations and governing ethics committee requirements prior to any procedures or evaluations performed specifically for the sole purpose of the study.
3. Willing and able to comply with study procedures.
4. Are age =12 at screening (Visit 1).
5. Have a primary aHUS, diagnosed clinically, and have ADAMTS13 activity > 5% in plasma. Patients are eligible with or without a documented complement mutation or anti-CFH antibody. Patients are categorized according to their response to plasma therapy (plasma exchange or plasma infusion):
o Plasma therapy-resistant aHUS patients must have all of the following:
? Screening platelet count < 150,000/µL despite at least four plasma therapy treatments in a 7-day period prior to screening
? Evidence of microangiopathic hemolysis (at least one of: (1) presence of schistocytes, (2) serum LDH > 1.5 times upper limit of normal (ULN), and (3) haptoglobin < LLN)
? Serum creatinine > ULN
o Plasma therapy-responsive aHUS patients must have all of the following:
? Have a documented history of requiring plasma therapy to prevent aHUS exacerbation defined as all of the following:
- decrease in platelet count > 25% when plasma therapy frequency has been decreased (including discontinuation of plasma therapy)
- LDH > 1.5 times ULN when plasma therapy frequency has been decreased (including discontinuation of plasma therapy)
? Have received plasma therapy at least once every 2 weeks at an unchanged frequency for at least 8 weeks before first dose of OMS721
6. If sexually active and of childbearing potential, must agree to practice a highly effective method of birth control until the end of the study, defined as one that results in a low failure rate (i.e., less than 1% per year) when used consistently and correctly, such as implants, injectables, combined oral contraceptives, some intrauterine devices, sexual abstinence or vasectomized partner.
7. Do not have access to eculizumab treatment, have not derived therapeutic benefit from eculizumab treatment, or have not been able to tolerate eculizumab treatment.
Are the trial subjects under 18? yes
Number of subjects for this age range: 4
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 72
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 4
1. Have STEC-HUS.
2. Have a positive direct Coombs test.
3. Have a history of hematopoietic stem cell transplant.
4. Have HUS from an identified drug.
5. History of vitamin B12 deficiency-related HUS.
6. History of Systemic Lupus Erythematosus.
7. History of antiphospholipid syndrome.
8. Active cancer or history of cancer (except non-melanoma skin cancers) within 5 years of screening.
9. Have been on hemodialysis or peritoneal dialysis for = 12 weeks.
10. Have an active systemic bacterial or fungal infection requiring systemic antimicrobial therapy (prophylactic antimicrobial therapy administered as standard of care is allowed).
11. Baseline resting heart rate < 45 beats per minute or > 115 beats per minute.
12. Baseline QTcF > 470 milliseconds.
13. Have malignant hypertension (diastolic blood pressure [BP] > 120 mm Hg with bilateral hemorrhages or cotton-wool” exudates on funduscopic examination).
14. Have a poor prognosis with a life expectancy of less than three months in the opinion of the Investigator.
15. Are pregnant or lactating.
16. Have received treatment with an investigational drug or device within four weeks prior to screening.
17. Have abnormal liver function tests defined as ALT or AST > five times ULN.
18. Have HIV infection.
19. History of cirrhosis of the liver.
20. Are an employee of Omeros, an Investigator, a study staff member, or their immediate family member.
21. Have a known hypersensitivity to any constituent of the product.
22. Presence of any condition that the Investigator believes would put the subject at risk or confound the interpretation of the data.
23. Have previously completed treatment in an OMS721study.
24. Have received intravenous immunoglobulin (IVIG) treatment within 8 weeks of screening visit.
25. Have received rituximab within 24 weeks of screening visit.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method