A multicenter, single-arm, open-label study to evaluate efficacy and safety of switching from anti-C5 antibody treatment to iptacopan treatment in study participants with aHUS
- Conditions
- Atypical Hemolytic Uremic Syndrome (aHUS)MedDRA version: 20.1Level: LLTClassification code: 10079841Term: Atypical hemolytic uremic syndrome Class: 10005329Therapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- CTIS2023-504550-35-00
- Lead Sponsor
- ovartis Pharma AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 33
Signed informed consent must be obtained prior to participation in the study, Male and female participants = 18 years of age at the time of consent., Willing and able to comply with the study visit schedule, Participants with diagnosis of aHUS for whom etiologies of other types of TMA (eg., Thrombotic Thrombocytopenic Purpura (TTP), Shiga Toxin producing Escherichia Coli (STEC)-TMA, Pneumococcal hemolytic uremic syndrome, TMA secondary to cobalamin C defect, TMA related to Diacylglycerol kinase e (DGKE) nephropathy) and non-aHUS kidney disease have been excluded., Currently on the recommended weight-based dosage regimen of anti-C5 antibody treatment (eg., eculizumab or ravulizumab), for at least 3 months prior to the screening visit., Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI) for at least 3 months prior to entering the screening period is defined as: •Hematological normalization in platelet count =150 x 109/L and LDH below upper limit of normal [ULN], and •Stable or improving kidney function as defined by =15% increase in serum creatinine., Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is required prior to the start of treatment with iptacopan. If the participant has not been previously vaccinated or if a booster is required, the vaccine(s) should be given, according to local regulations, at least 2 weeks prior to first iptacopan dosing. If iptacopan treatment has to start earlier than 2 weeks post-vaccination, prophylactic antibiotic treatment must be initiated at the start of iptacopan study treatment and for at least 2 weeks after vaccination, If not received previously or if a booster is required, vaccination against Haemophilus influenzae infection, should be given, if available and according to local regulations, at least 2 weeks prior to first iptacopan dosing.
History of aHUS disease relapse while on anti-C5 antibody treatment., Uncontrolled arterial hypertension (systolic blood pressure >160 mmHg)., Active infection or history of recurrent invasive infections caused by encapsulated bacteria, i.e. meningococcus, pneumococcus (eg., N. meningitidis, S. pneumoniae) or H. influenzae., Participants with sepsis or active systemic bacterial, viral (including COVID-19) or fungal infection within 14 days prior to study treatment administration., Human immunodeficiency virus (HIV) infection (known history of HIV or test positive for HIV at screening)., Kidney, bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT), heart, lung, small bowel, pancreas, liver transplantation or any other cell or solid organ transplantation., History of drug or alcohol abuse within the 12 months prior to dosing., Major concurrent comorbidities including but not limited to advanced cardiac disease (e.g. NYHA class IV), severe pulmonary disease (e.g., severe pulmonary hypertension (WHO class IV)), or hepatic disease (e.g. active hepatitis) that in the opinion of the Investigator precludes participant's participation in the study., Participants committed to an institution by virtue of an order issued either by the judicial or the administrative authorities, Participants dependent on the sponsor, the study site or the Investigator, Liver disease, such as active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection defined as hepatitis B virus surface antibody (HBsAg) positive or HCV RNA positive, or liver injury as indicated by abnormal liver function tests at Screening as defined below: •Any single parameter of alanine amino transferase (ALT), gamma-glutamyl transferase (GGT), alkaline phosphatase must not exceed 3×upper limit of normal (ULN)., Any medical condition deemed likely to interfere with the participant’s participation in the study., Use of other investigational drugs within 5 half-lives of enrollment, or within 30 days, whichever is longer; or longer if required by local regulations., History of hypersensitivity to iptacopan or any of its excipients or to drugs of similar chemical classes., History of malignancy of any organ system (other than localized basal cell carcinoma of the skin or in situ cervical cancer) treated or untreated within the past 5 years, regardless of whether there is evidence of local recurrence or metastases, Female participants who are pregnant or breastfeeding, or intending to conceive during the course of the study., Women of child-bearing potential (WCBP), defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of iptacopan and for 1 week after stopping of iptacopan., eGFR < 30 ml/min/1.73m2.
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