| CTRI Number |
CTRI/2024/07/070874 [Registered on: 19/07/2024] Trial Registered Prospectively |
| Last Modified On: |
22/04/2026 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Single Arm Study |
|
Public Title of Study
|
To study if iptacopan is safe, tolerable and effective in patients with aHUS |
|
Scientific Title of Study
|
A multicenter, 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 |
| Trial Acronym |
NIL |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| 2022-502965-34-00 |
EudraCT |
| CLNP023F12001B version 1.0 dated 28 Jul 2023 |
Protocol Number |
| NCT05795140 |
ClinicalTrials.gov |
|
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
|
| Name |
Murugananthan K |
| Designation |
Country Monitoring Head |
| Affiliation |
Novartis Healthcare Private Limited |
| Address |
Novartis Healthcare Private Limited 7 floor Inspire BKC G
Block BKC Main Road Bandra Kurla Complex Bandra (East)
Mumbai
MAHARASHTRA
400051
India
Mumbai
MAHARASHTRA
400051
India |
| Phone |
912250243544 |
| Fax |
|
| Email |
murugananthan.k@novartis.com |
|
Details of Contact Person
Scientific Query
|
| Name |
Murugananthan K |
| Designation |
Country Monitoring Head |
| Affiliation |
Novartis Healthcare Private Limited |
| Address |
Novartis Healthcare Private Limited 7 floor Inspire BKC G
Block BKC Main Road Bandra Kurla Complex Bandra (East)
Mumbai
MAHARASHTRA
400051
India
Mumbai
MAHARASHTRA
400051
India |
| Phone |
912250243544 |
| Fax |
|
| Email |
murugananthan.k@novartis.com |
|
Details of Contact Person
Public Query
|
| Name |
Murugananthan K |
| Designation |
Country Monitoring Head |
| Affiliation |
Novartis Healthcare Private Limited |
| Address |
Novartis Healthcare Private Limited 7 floor Inspire BKC G
Block BKC Main Road Bandra Kurla Complex Bandra (East)
Mumbai
MAHARASHTRA
400051
India
Mumbai
MAHARASHTRA
400051
India |
| Phone |
912250243544 |
| Fax |
|
| Email |
murugananthan.k@novartis.com |
|
|
Source of Monetary or Material Support
|
| Novartis Pharma AG, Novartis Campus 4056 – Basel, Switzerland |
|
|
Primary Sponsor
|
| Name |
Novartis Healthcare Pvt Ltd |
| Address |
7 floor, Inspire BKC, G Block, BKC Main Road, Bandra Kurla
Complex, Bandra (East), Mumbai – 400051,India |
| Type of Sponsor |
Pharmaceutical industry-Global |
|
|
Details of Secondary Sponsor
|
|
|
Countries of Recruitment
|
Austria
Brazil
China
Czech Republic
France
Germany
Greece
India
Italy
Japan
Republic of Korea
Slovakia
Slovenia
South Africa
Spain
Taiwan
Turkey
United Kingdom
United States of America |
|
Sites of Study
|
| No of Sites = 5 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr Jeethu Joseph Eapen |
Christian Medical College |
Department of Nephrology, A Block, 6th Floor,
Christian Medical College Vellore Ranipet Campus, Kilminnal Village, Ranipet District 632517
Vellore
TAMIL NADU |
9042849392
jjeapen@gmail.com |
| Dr Vipul Chakurkar |
KEM Hospital |
Renal Unit 2nd floor, KEM Hospital, Diamond jubilee Building, Sardar Moodliar Road, Rasta Peth, Pune, 411011
Pune
MAHARASHTRA |
9403207328
chakurkarvipul@gmail.com |
| Dr Raja Ramchandran |
Post Graduate Institute of Medical Education and Research (PGIMER) |
Department of Nephrology, Ground floor, C Block, Nehru Hospital, Post Graduate Institute of Medical Education and Research (PGIMER), Sector 12, Chandigarh 160012
Chandigarh
CHANDIGARH |
9216958874
drraja1980@gmail.com |
| Dr Dharmendra Singh Bhadauria |
Sanjay Gandhi Postgraduate Institute of Medical Sciences |
Department of Nephrology, Sanjay Gandhi Postgraduate Institute of Medical Sciences, Raebareli Road Lucknow
Lucknow
UTTAR PRADESH |
8004904367
drdharm1@rediffmail.com |
| Dr Amit Pasari |
Saraswati Kidney Care Center |
Saraswati Kidney Care Center ( A Unit of SS Multispecialty) 13, New Sneh Nagar, Nagpur- 440015
Nagpur
MAHARASHTRA |
9422164630
dramit28@yahoo.co.in |
|
Details of Ethics Committee
Modification(s)
|
| No of Ethics Committees= 5 |
| Name of Committee |
Approval Status |
| Institutional Ethics Committee, PGIMER |
Submittted/Under Review |
| Institutional ETHICS COMMITTEE, SGPGI |
Submittted/Under Review |
| Institutional Review Board (IRB) & The Ethics Committee, Christian Medical College |
Submittted/Under Review |
| KEM Hospital Research Centre Ethics Committee |
Approved |
| SKCC Institutional Ethics Committee |
Approved |
|
|
Regulatory Clearance Status from DCGI
|
|
|
Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: D593||Hemolytic-uremic syndrome, |
|
|
Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Intervention |
LNP023 (iptacopan) |
dose of 200 mg b.i.d for 48 months of treatment period. |
| Comparator Agent |
NA |
NA |
|
|
Inclusion Criteria
|
| Age From |
18.00 Year(s) |
| Age To |
99.00 Year(s) |
| Gender |
Both |
| Details |
1. Approximately 100 male and female participants, ≥18 years of age, with a diagnosis of aHUS prior to screening.
2. Participants must have completed the full study treatment period of a Novartis sponsored iptacopan Phase 3 clinical trial in aHUS, are still on iptacopan study treatment and derive benefit from it as per Investigators judgement.
3. Already vaccinated against bacteria such as Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae. If the patient has not been previously vaccinated, or if a booster is required, vaccine will be given if available and according to local regulations, at least 2 weeks prior to first dosing of iptacopan.
|
|
| ExclusionCriteria |
| Details |
1. Concomitant treatment with any complement inhibitor as well as concomitant treatment with any of the drugs listed in Section 6.8.2
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.
|
|
|
Method of Generating Random Sequence
|
Not Applicable |
|
Method of Concealment
|
Not Applicable |
|
Blinding/Masking
|
Open Label |
|
Primary Outcome
|
| Outcome |
TimePoints |
| Is iptacopan safe, tolerable and effective in participants with aHUS |
Vitals, adverse events and laboratory parameters are followed in all visits since day 1 to end of tretment visit.
ECG is carried out on Day 1, Month 12,24,36,48 till End of traetment To be performed once in a year (i.e. at Month 12, Month 24, Month 36, Month 48 etc. until the EoT visit) |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
| To evaluate the proportion of participants who were free of aHUS signs and symptoms at time of enrollment and do not have these return during their participation in the study |
Absences of aHUS signs and symptoms without the use of anti C5 antibody treatment are carried out in all visits since day 1 to end of study treatment.
|
|
|
Target Sample Size
|
Total Sample Size="100"
Sample Size from India="10"
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" |
|
Phase of Trial
|
Phase 3 |
|
Date of First Enrollment (India)
|
31/07/2024 |
| Date of Study Completion (India) |
Applicable only for Completed/Terminated trials |
| Date of First Enrollment (Global) |
12/02/2024 |
| Date of Study Completion (Global) |
Applicable only for Completed/Terminated trials |
|
Estimated Duration of Trial
|
Years="4"
Months="0"
Days="0" |
Recruitment Status of Trial (Global)
Modification(s)
|
Open to Recruitment |
| Recruitment Status of Trial (India) |
Open to Recruitment |
|
Publication Details
|
N/A |
|
Individual Participant Data (IPD) Sharing Statement
|
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
|
|
Brief Summary
|
The purpose of this Phase 3b study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in participants with atypical hemolytic uremic syndrome (aHUS). All participants should have successfully completed the full study treatment period in a Novartis-sponsored Phase 3 study of iptacopan in aHUS. aHUS is a very rare disease that happens when there is an uncontrolled activation of an important system in your body called the alternative complement pathway (AP). The AP usually provides protection against infections. However, in aHUS, the over-activation of AP can lead to destruction of red blood cells, low platelets and damage small blood vessels in your kidneys causing inflammation. This is known as thrombotic microangiopathy (TMA). The constant inflammation causes continued damage to your kidneys.
Iptacopan is a new treatment tested in aHUS. It is believed that it may improve aHUS by reducing inflammation and helping your kidneys. Iptacopan acts on a protein called factor B in your immune system. Inhibition of factor B has the potential to prevent the destruction of red blood cells by your own immune system resulting in maintenance of normal hemoglobin levels. |