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Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS

Studies

Study First Submitted Date 2023-03-21
Study First Posted Date 2023-04-03
Last Update Posted Date 2023-07-11
Start Month Year February 12, 2024
Primary Completion Month Year August 16, 2029
Verification Month Year July 2023
Verification Date 2023-07-31
Last Update Posted Date 2023-07-11

Detailed Descriptions

Sequence: 20647650
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.

Conditions

Sequence: 51979733
Name Atypical Hemolytic Uremic Syndrome
Downcase Name atypical hemolytic uremic syndrome

Id Information

Sequence: 40009458
Id Source org_study_id
Id Value CLNP023F12001B

Design Groups

Sequence: 55381407
Group Type Experimental
Title Iptacopan 200 mg
Description Open label , single arm

Interventions

Sequence: 52290819
Intervention Type Drug
Name Iptcaopan 200 mg
Description Open label, participant specific kits, hard gelatin capsules to be taken twice a day

Keywords

Sequence: 79566934 Sequence: 79566935 Sequence: 79566936 Sequence: 79566937 Sequence: 79566938 Sequence: 79566939 Sequence: 79566940 Sequence: 79566941 Sequence: 79566942 Sequence: 79566943 Sequence: 79566944 Sequence: 79566945
Name aHUS Name iptacopan Name atypical hemolytic uremic syndrome Name thrombotic microangiopathy Name TMA Name dialysis Name CKD Name eGFR Name PE/PI Name Complement 3 Glomerulopathy (C3G) Name IgAN Name PNH
Downcase Name ahus Downcase Name iptacopan Downcase Name atypical hemolytic uremic syndrome Downcase Name thrombotic microangiopathy Downcase Name tma Downcase Name dialysis Downcase Name ckd Downcase Name egfr Downcase Name pe/pi Downcase Name complement 3 glomerulopathy (c3g) Downcase Name igan Downcase Name pnh

Design Outcomes

Sequence: 176699617 Sequence: 176699618 Sequence: 176699619 Sequence: 176699620 Sequence: 176699621 Sequence: 176699622 Sequence: 176699623 Sequence: 176699624
Outcome Type primary Outcome Type primary Outcome Type secondary Outcome Type secondary Outcome Type secondary Outcome Type secondary Outcome Type secondary Outcome Type secondary
Measure Number of participants with adverse events and serious adverse events Measure Number of participants with abnormal safety laboratory parameters, vital signs and ECGs Measure Number of participants with absence of aHUS relapse without the use of anti-C5 antibody Measure Number of participants with complete TMA response status without the use of anti-C5 antibody therapy Measure Estimated glomerular filtration rate (eGFR) Measure Chronic kidney disease (CKD) stage Measure Number of participants by dialysis requirement status Measure Number of participants with Thrombotic Microangiopathy (TMA) related adverse events
Time Frame Throughout the study duration, up to 4 years Time Frame Throughout the study duration, up to 4 years Time Frame Throughout the study duration, up to 4 years Time Frame Throughout the study duration, up to 4 years Time Frame Throughout study duration, up to 4 years Time Frame Throughout study duration, up to 4 years Time Frame Throughout the study duration, up to 4 years Time Frame Throughout study duration, up to 4 years
Description Number of participants with adverse events and serious adverse events will be provided Description Number of participants with abnormal safety laboratory parameters , vital signs and ECGs will be provided Description 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) Description 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) Description 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. Description 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 dialysis Description Dialysis requirement status will be provided Description 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.

Browse Conditions

Sequence: 192729106 Sequence: 192729107 Sequence: 192729108 Sequence: 192729109 Sequence: 192729110 Sequence: 192729111 Sequence: 192729112 Sequence: 192729113 Sequence: 192729114 Sequence: 192729115 Sequence: 192729116 Sequence: 192729117 Sequence: 192729118 Sequence: 192729119 Sequence: 192729120 Sequence: 192729121 Sequence: 192729122 Sequence: 192729123 Sequence: 192729124 Sequence: 192729125
Mesh Term Azotemia Mesh Term Hemolytic-Uremic Syndrome Mesh Term Atypical Hemolytic Uremic Syndrome Mesh Term Syndrome Mesh Term Hemolysis Mesh Term Disease Mesh Term Pathologic Processes Mesh Term Uremia Mesh Term Kidney Diseases Mesh Term Urologic Diseases Mesh Term Female Urogenital Diseases Mesh Term Female Urogenital Diseases and Pregnancy Complications Mesh Term Urogenital Diseases Mesh Term Male Urogenital Diseases Mesh Term Anemia, Hemolytic Mesh Term Anemia Mesh Term Hematologic Diseases Mesh Term Thrombotic Microangiopathies Mesh Term Thrombocytopenia Mesh Term Blood Platelet Disorders
Downcase Mesh Term azotemia Downcase Mesh Term hemolytic-uremic syndrome Downcase Mesh Term atypical hemolytic uremic syndrome Downcase Mesh Term syndrome Downcase Mesh Term hemolysis Downcase Mesh Term disease Downcase Mesh Term pathologic processes Downcase Mesh Term uremia Downcase Mesh Term kidney diseases Downcase Mesh Term urologic diseases Downcase Mesh Term female urogenital diseases Downcase Mesh Term female urogenital diseases and pregnancy complications Downcase Mesh Term urogenital diseases Downcase Mesh Term male urogenital diseases Downcase Mesh Term anemia, hemolytic Downcase Mesh Term anemia Downcase Mesh Term hematologic diseases Downcase Mesh Term thrombotic microangiopathies Downcase Mesh Term thrombocytopenia Downcase Mesh Term blood platelet disorders
Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor

Sponsors

Sequence: 48139622
Agency Class INDUSTRY
Lead Or Collaborator lead
Name Novartis Pharmaceuticals

Overall Officials

Sequence: 29175379
Role Study Director
Name Novartis Pharmaceuticals
Affiliation Novartis Pharmaceuticals

Central Contacts

Sequence: 11965769 Sequence: 11965770
Contact Type primary Contact Type backup
Name Novartis Pharmaceuticals Name Novartis Pharmaceuticals
Phone +1-888-669-6682 Phone +41613241111
Email novartis.email@novartis.com
Role Contact Role Contact

Design Group Interventions

Sequence: 67890691
Design Group Id 55381407
Intervention Id 52290819

Eligibilities

Sequence: 30652690
Gender All
Minimum Age 18 Years
Maximum Age N/A
Healthy Volunteers No
Criteria 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.
Adult True
Child False
Older Adult True

Calculated Values

Sequence: 254256860
Registered In Calendar Year 2023
Were Results Reported False
Has Single Facility False
Minimum Age Num 18
Minimum Age Unit Years
Number Of Primary Outcomes To Measure 2
Number Of Secondary Outcomes To Measure 6

Designs

Sequence: 30399547
Allocation N/A
Intervention Model Single Group Assignment
Observational Model
Primary Purpose Treatment
Time Perspective
Masking None (Open Label)

Intervention Other Names

Sequence: 26570764
Intervention Id 52290819
Name LNP023

Responsible Parties

Sequence: 28766049
Responsible Party Type Sponsor