- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05795140
Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS
A Multi-center, Single Arm, Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Iptacopan in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Who Have Completed a Preceding Iptacopan Phase 3 Study in aHUS
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Estimated)
Phase
- Phase 3
Expanded Access
Contacts and Locations
Study Contact
- Name: Novartis Pharmaceuticals
- Phone Number: +41613241111
- Email: novartis.email@novartis.com
Study Contact Backup
- Name: Novartis Pharmaceuticals
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- 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)
Exclusion Criteria:
- 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 Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Iptacopan 200 mg
Open label , single arm
|
Open label, participant specific kits, hard gelatin capsules to be taken twice a day
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of participants with adverse events and serious adverse events
Time Frame: Throughout the study duration, up to 4 years
|
Number of participants with adverse events and serious adverse events will be provided
|
Throughout the study duration, up to 4 years
|
Number of participants with abnormal safety laboratory parameters, vital signs and ECGs
Time Frame: Throughout the study duration, up to 4 years
|
Number of participants with abnormal safety laboratory parameters , vital signs and ECGs will be provided
|
Throughout the study duration, up to 4 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of participants with absence of aHUS relapse without the use of anti-C5 antibody
Time Frame: 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:
|
Throughout the study duration, up to 4 years
|
Number of participants with complete TMA response status without the use of anti-C5 antibody therapy
Time Frame: 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)
|
Throughout the study duration, up to 4 years
|
Estimated glomerular filtration rate (eGFR)
Time Frame: 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. |
Throughout study duration, up to 4 years
|
Chronic kidney disease (CKD) stage
Time Frame: Throughout study duration, up to 4 years
|
Chronic kidney disease (CKD) stage (1-5) based on eGFR categories will be provided:
|
Throughout study duration, up to 4 years
|
Number of participants by dialysis requirement status
Time Frame: Throughout the study duration, up to 4 years
|
Dialysis requirement status will be provided
|
Throughout the study duration, up to 4 years
|
Number of participants with Thrombotic Microangiopathy (TMA) related adverse events
Time Frame: Throughout study duration, up to 4 years
|
TMA related events during the study defined as any of the following:
|
Throughout study duration, up to 4 years
|
Collaborators and Investigators
Sponsor
Investigators
- Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals
Study record dates
Study Major Dates
Study Start (Estimated)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Pathologic Processes
- Kidney Diseases
- Urologic Diseases
- Disease
- Hematologic Diseases
- Anemia
- Thrombocytopenia
- Blood Platelet Disorders
- Anemia, Hemolytic
- Thrombotic Microangiopathies
- Uremia
- Female Urogenital Diseases
- Female Urogenital Diseases and Pregnancy Complications
- Urogenital Diseases
- Male Urogenital Diseases
- Cytopenia
- Syndrome
- Azotemia
- Hemolysis
- Hemolytic-Uremic Syndrome
- Atypical Hemolytic Uremic Syndrome
Other Study ID Numbers
- CLNP023F12001B
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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