EUCTR2017-001082-24-LTActive, not recruitingPhase 1
A Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients with Atypical Hemolytic Uremic Syndrome
Conditions
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Enrollment
- 24
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •1. Willing to provide written informed consent and to comply with the study requirements
- •2. Age 18 years or older
- •3. Clinical presentation consistent with a diagnosis of primary aHUS
- •4. Clinical thrombotic microangiopathy (TMA) activity as defined by:
- •a. Evidence of low platelet count and
- •b. Evidence of hemolysis and
- •c. Evidence of impaired renal function
- •5. ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13) >10% (at pre-Screening, Screening, or historically documented in their chart) in a plasma sample that was obtained prior to start of plasma therapy.
- •a. Patients who have already started plasma therapy and who do not have a documented pre-plasma ADAMTS13 result will not be eligible for the study unless they are proven to have a documented aHUS-associated mutation.
- •6. Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use a highly effective method of contraception
- •7. Vaccinated with meningococcal group ACWY conjugate vaccine and meningococcal group B vaccine or willingness to receive these vaccinations as well as prophylactic antibiotic treatment for at least 2 weeks after completing the recommended vaccination series or longer, if required by local standard of care.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 22
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
Exclusion Criteria
- •1. Abnormal liver function tests
- •2. Positive Shiga toxin producing Escherichia coli test
- •3. Suspected secondary aHUS, in the opinion of the Investigator, unless the patient has a documented aHUS-associated genetic mutation.
- •4. Positive direct Coombs test
- •5. Prior eculizumab exposure within 3 months
- •6. Patients considered non-eculizumab responders (clinical or laboratory non-response),
- •except for those with known C5 polymorphisms
- •7. Patients who have received hemodialysis for >3 months
- •8. Patients who require hemodialysis or plasma exchange more frequently than every
- •9. Clinical laboratory test results considered clinically relevant and unacceptable in the
- •opinion of the Investigator
- •10. Malignancy (except for non-melanoma skin cancers, cervical in-situ carcinoma, breast
- •ductal carcinoma in situ, or stage 1 prostate carcinoma) within the last 5 years
- •11. Patients with active systemic bacterial or fungal infections, as demonstrated by a positive
- •culture result, that require systemic treatment with antibiotics or antifungals
- •a. Patients receiving empiric or prophylactic antibiotics are not excluded
- •12. Patients with sepsis or a life-threatening infection
- •13. Received an investigational agent within the last 90 days or are in follow-up of another
- •clinical study before the first ALN-CC5 administration
- •14. Active psychiatric disorder, including, but not limited to, schizophrenia, bipolar disorder,
- •or severe depression requiring current pharmacological intervention
- •15. Bone marrow transplant recipients
- •16. Organ transplant recipients will be excluded, except for kidney transplant recipients with
- •primary aHUS
- •a. For kidney transplant recipients, primary aHUS will be defined by known genetic
- •mutation and no confounding diagnosis (eg, antibody-mediated rejection or viral
- •infection) by routine kidney biopsy, as judged by the Investigator
Investigators
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