Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS
- Conditions
- Atypical Hemolytic Uremic Syndrome
- Interventions
- Drug: Iptcaopan 200 mg
- Registration Number
- NCT05795140
- Lead Sponsor
- Novartis Pharmaceuticals
- Brief Summary
This is a multicenter, single arm, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of iptacopan in participants with aHUS.
- Detailed Description
The extension study Baseline/Day 1 visit is equivalent to the End of Treatment visit of the parent study. The study will begin on Day 1 followed by on-site visits every 4 months during the study treatment period. A Safety Follow Up tele-visit must be conducted 7 days after last study treatment to collect information on Adverse Events.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 125
- Signed informed consent must be obtained prior to participation in the open label extension study
- Willing and able to comply with the study Schedule of Activities
- Participants who have completed the full study treatment period of any prior "Novartis sponsored" iptacopan Phase 3 clinical trial in aHUS, are still on iptacopan study treatment and derive benefit from it as per Investigator's judgement
- Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections should be up to date (i.e., any boosters required should be administered according to local guidelines)
- Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the prohibited drugs
- Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the Investigator could put the participant at risk
- Active infection or history of recurrent invasive infections caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
- History of hypersensitivity to iptacopan or its excipients or to drugs of similar chemical classes
- Pregnant or nursing (lactating) women
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of investigational drug and for 1 week after stopping of investigational drug.
Other protocol-defined inclusion/exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Iptacopan 200 mg Iptcaopan 200 mg Open label , single arm
- Primary Outcome Measures
Name Time Method Number of participants with adverse events and serious adverse events Throughout the study duration, up to 4 years Number of participants with adverse events and serious adverse events will be provided
Number of participants with abnormal safety laboratory parameters, vital signs and ECGs Throughout the study duration, up to 4 years Number of participants with abnormal safety laboratory parameters , vital signs and ECGs will be provided
- Secondary Outcome Measures
Name Time Method Number of participants with absence of aHUS relapse without the use of anti-C5 antibody Throughout the study duration, up to 4 years Atypical hemolytic uremic syndrome (aHUS) relapse is defined by the coexistence of at least two of the following at the same visit:
* thrombocytopenia (platelet count \< 150 x 109 /L),
* microangiopathic hemolytic anemia (hemoglobin \< 10 g/dl, LDH \> upper limit of normal, undetectable haptoglobin, presence of schistocyte on blood smear),
* worsening kidney function (serum creatinine or urine protein to creatinine ratio (UPCR) \> upper limit of normal and an increase of ≥ 15% compared to baseline levels)Number of participants with complete TMA response status without the use of anti-C5 antibody therapy Throughout the study duration, up to 4 years Complete thrombotic microangiopathy (TMA) Response is defined as (1) hematological normalization in platelet count (platelet count ≥150 x 109/L) and LDH (below ULN), and (2) improvement in kidney function (≥ 25% serum creatinine reduction from baseline or ≥ 25% serum creatinine reduction compared to serum creatinine values prior to initiation of anti-C5 antibody therapy)
Estimated glomerular filtration rate (eGFR) Throughout study duration, up to 4 years Estimated glomerular filtration rate (eGFR) based on eGFR categories will be collected.
Serum creatinine as measured in mg/dL as part of the clinical chemistry panel through the central laboratory will be used to calculate the eGFR applying the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula.Chronic kidney disease (CKD) stage Throughout study duration, up to 4 years Chronic kidney disease (CKD) stage (1-5) based on eGFR categories will be provided:
* Stage 1 (G1): Kidney damage with normal kidney function
* Stage 2 (G2): Mild loss of kidney function
* Stage 3 (G3): 3a: Mild to moderate loss of kidney function; 3b: Moderate to severe loss of kidney function
* Stage 4 (G4): Severe loss of kidney function
* Stage 5 End stage renal disease (kidney failure): Kidney failure and need for transplant or dialysisNumber of participants by dialysis requirement status Throughout the study duration, up to 4 years Dialysis requirement status will be provided
Number of participants with Thrombotic Microangiopathy (TMA) related adverse events Throughout study duration, up to 4 years TMA related events during the study defined as any of the following:
* Irreversible (\>3 months) reduction in eGFR rate by ≥20%, not attributable to another cause
* An episode of acute kidney injury (AKI) attributed to a TMA that requires renal replacement therapy
* A non-renal manifestation of a TMA that requires hospitalization, or causes irreversible organ damage or death.
Trial Locations
- Locations (1)
Novartis Investigative Site
🇮🇳Pune, Maharashtra, India