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Iptcaopan

Phase 3

Atypical Hemolytic Uremic Syndrome | Small molecule | Rare Disease |Novartis AG|Last Updated: Jun 4, 2026

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment65

FDA Designations

No designations recorded

Clinical trial landscape

Iptcaopan · 1 trial · 1 indication

Phase 3 1
NCT05795140Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUSAtypical Hemolytic Uremic Syndrome
RECRUITING65 Analytics
PHASE3RECRUITING
Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS
Atypical Hemolytic Uremic Syndrome
NCT0579514065
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Study Endpoints

Primary Endpoints

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, including abnormal safety laboratory parameters, vital signs and ECGs

Secondary Endpoints

Number of participants with absence of TMA manifestation without the use of anti-C5 antibody
Throughout the study duration, up to 4 years
Number of participants with complete TMA response status without the use of anti-C5 antibody therapy
Throughout the study duration, up to 4 years
Estimated glomerular filtration rate (eGFR)
Throughout study duration, up to 4 years
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Iptacopan 200 mgEXPERIMENTALOpen label , single arm

Interventions

NameTypeDescription
Iptcaopan 200 mgDRUGOpen label, participant specific kits, hard gelatin capsules to be taken twice a day
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Eligibility Criteria

Age Range18 Years to 100 Years
SexALL
Healthy VolunteersNo
Study Sites9

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...

Countries:BrazilChinaCzechiaIndiaJapanTurkey (Türkiye)
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Frequently asked questions about Iptcaopan

What is Iptacopan used for?

Iptacopan is an investigational small molecule being developed for atypical hemolytic uremic syndrome (aHUS), a rare disease. It is currently in Phase 3 clinical development and is not yet approved by regulatory authorities.

Who is developing Iptacopan?

Iptacopan is being developed by Novartis AG, a multinational pharmaceutical company listed on the stock exchange under the ticker NVS. The company is conducting a Phase 3 clinical trial to evaluate the drug's long-term safety, tolerability, and efficacy in patients with atypical hemolytic uremic syndrome.

What phase is Iptacopan in?

Iptacopan is in Phase 3 clinical development. A Phase 3 trial is currently recruiting participants to evaluate the long-term safety, tolerability, and efficacy of the drug in patients with atypical hemolytic uremic syndrome (aHUS).

What clinical trials is Iptacopan in?

Iptacopan is being studied in a Phase 3 clinical trial with the identifier NCT05795140. This trial is recruiting 65 adult participants with atypical hemolytic uremic syndrome across multiple countries, including Brazil, China, Czechia, India, Japan, and Turkey. The study is open-label and uncontrolled.

Is Iptacopan the same as Iptacopan 200 mg?

Yes, Iptacopan is also known as Iptacopan 200 mg, which refers to the specific dosage strength being studied. The drug is being evaluated in a Phase 3 clinical trial for atypical hemolytic uremic syndrome.