BridgeMD
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
EU CTIS ID: 2023-504550-35-00
What this study is testing
To assess the proportion of participants free of TMA manifestation during the 12 months of iptacopan treatment following the switch of treatment from an anti-C5 antibody to iptacopan treatment
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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 ε (DGKE) nephropathy) and non-aHUS kidney disease have been excluded.
- Currently on the recommended (as per label) dosage regimen of anti-C5 antibody treatment (eg., eculizumab or ravulizumab), for at least 3 months prior to entering the screening period .
- In the opinion of the investigator the participant has responded to anti-C5 antibody treatment prior to screening and has clinical evidence of response (in absence of PE/PI) during the Screening period. Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI) confirmed during the Screening period by central laboratory at two visits 12 weeks apart. Clinical evidence of response is defined as: • Hematological normalization in platelet count ≥150 x 109/L and LDH below upper limit of normal [ULN], and Stable kidney function as defined by serum creatinine values within ±15% during the Screening period .
You likely can't join if
- 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.
See the full eligibility criteria
Who can join
- 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 ε (DGKE) nephropathy) and non-aHUS kidney disease have been excluded.
- Currently on the recommended (as per label) dosage regimen of anti-C5 antibody treatment (eg., eculizumab or ravulizumab), for at least 3 months prior to entering the screening period .
- In the opinion of the investigator the participant has responded to anti-C5 antibody treatment prior to screening and has clinical evidence of response (in absence of PE/PI) during the Screening period. Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI) confirmed during the Screening period by central laboratory at two visits 12 weeks apart. Clinical evidence of response is defined as: • Hematological normalization in platelet count ≥150 x 109/L and LDH below upper limit of normal [ULN], and Stable kidney function as defined by serum creatinine values within ±15% during the Screening period .
- 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.
What rules you out
- 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.
- Women of child-bearing potential (WCBP), defined as all women physiologically capable of becoming pregnant from menarche until becoming post-menopausal, unless they are using effective methods of contraception during dosing of iptacopan and for 1 week after stopping of iptacopan. Women are considered post-menopausal if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g., hormonal profile confirming menopause and/or age-appropriate history of vasomotor symptoms).
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Japan
- China
- United States
- United Kingdom
- Turkey
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Japan; China; United States; United Kingdom; Turkey. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.