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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05795140
Other study ID # CLNP023F12001B
Secondary ID
Status Recruiting
Phase Phase 3
First received
Last updated
Start date May 8, 2024
Est. completion date January 3, 2033

Study information

Verified date May 2024
Source Novartis
Contact Novartis Pharmaceuticals
Phone +41613241111
Email novartis.email@novartis.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


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 information / eligibility

Status Recruiting
Enrollment 150
Est. completion date January 3, 2033
Est. primary completion date December 23, 2032
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria: 1. Signed informed consent must be obtained prior to participation in the open label extension study 2. Willing and able to comply with the study Schedule of Activities 3. 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 4. 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: 1. Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the prohibited drugs 2. 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 3. Active infection or history of recurrent invasive infections caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae 4. History of hypersensitivity to iptacopan or its excipients or to drugs of similar chemical classes 5. Pregnant or nursing (lactating) women 6. 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


Related Conditions & MeSH terms


Intervention

Drug:
Iptcaopan 200 mg
Open label, participant specific kits, hard gelatin capsules to be taken twice a day

Locations

Country Name City State
Brazil Novartis Investigative Site Sao Paulo SP

Sponsors (1)

Lead Sponsor Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

Brazil, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of participants with adverse events and serious adverse events Number of participants with adverse events and serious adverse events will be provided Throughout the study duration, up to 4 years
Primary Number of participants with abnormal safety laboratory parameters, vital signs and ECGs Number of participants with abnormal safety laboratory parameters , vital signs and ECGs will be provided Throughout the study duration, up to 4 years
Secondary Number of participants with absence of aHUS relapse without the use of anti-C5 antibody 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)
Throughout the study duration, up to 4 years
Secondary Number of participants with complete TMA response status without the use of anti-C5 antibody therapy 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
Secondary Estimated glomerular filtration rate (eGFR) 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
Secondary Chronic kidney disease (CKD) stage 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
Throughout study duration, up to 4 years
Secondary Number of participants by dialysis requirement status Dialysis requirement status will be provided Throughout the study duration, up to 4 years
Secondary Number of participants with Thrombotic Microangiopathy (TMA) related adverse events 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.
Throughout study duration, up to 4 years
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