Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

JRCT ID: jRCT2031230686

Registered date:11/03/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Atypical Hemolytic Uremic Syndrome
• Date of first enrollment: 23/03/2024
• Target sample size: 1
• Countries of recruitment: China,Japan,France,Japan,Germany,Japan,Italy,Japan,Spain,Japan,Turkey,Japan,United Kingdom,Japan,United Status,Japan
• Study type: Interventional

Intervention(s)

Experimental: iptacopan 200 mg b.i.d.
-open label arm of iptacopan 200 mg b.i.d.
- Interventions:
-Drug: Iptacopan

Outcome(s)

Primary Outcome

Percentage of participants free of TMA manifestation [Time Frame: 12 months]
Absence of thrombotic microangiopathy (TMA) manifestation, without use of anti-C5 antibody, during the 12 months of iptacopan treatment following the switch of treatment from an anti-C5 antibody to iptacopan treatment.

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

- Male and female adult participants >= 18 years of age with diagnosis of aHUS for whom etiologies of other types of TMA and non-aHUS kidney disease have been excluded.
- Currently on the recommended weight-based dosage regimen of anti-C5 antibody treatment 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 as defined by:
1. Hematological normalization in platelet count >=150 x 109/L and LDH below upper limit of normal [ULN], and
2. 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 not received previously or if a booster is required, vaccination against Haemophilus influenzae infection, should be given, if available and according to local regulations.

Exclude criteria

- History of aHUS disease relapse while on anti-C5 antibody treatment.
- eGFR < 30 ml/min/1.73m^2
- 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.
- 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
- Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study
- Any medical condition deemed likely to interfere with the patient's participation in the study

Related Information

• Primary Sponsor: Iwasaki Ryohei
• Secondary ID(s): NCT05935215