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Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria

Primary Purpose

Paroxysmal Nocturnal Hemoglobinuria

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Iptacopan
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria focused on measuring Iptacopan, Paroxysmal Nocturnal Hemoglobinuria (PNH), Hemoglobin, Anemia, LNP023

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion criteria:

  • Male and female participants ≥ 18 years of age with a diagnosis of PNH who have completed the treatment extension period (without tapering down) of Phase II iptacopan studies (CLNP023X2204, CLNP023X2201), Period 4 of LFG316X2201 or Phase III (CLNP023C12302 and CLNP023C12301) clinical studies at the time point of enrollment visit in this roll over extension.
  • Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections
  • Per investigator's clinical judgement benefit from continued treatment with iptacopan and has been clinically stable on iptacopan monotherapy for at least 3 months

Exclusion Criteria:

  • 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 subject at increased risk or potentially confound study data.
  • History of recurrent invasive infections caused by encapsulated organisms, such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae
  • History of hematopoietic stem cell transplantation

Other protocol-defined inclusion/exclusion criteria may apply.

Sites / Locations

  • Novartis Investigative SiteRecruiting
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Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Iptacopan

Arm Description

Participants will be receiving open label oral iptacopan 200 mg b.i.d monotherapy

Outcomes

Primary Outcome Measures

Proportion of participants with adverse events
Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit

Secondary Outcome Measures

Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions evaluated over yearly intervals
Proportion of participants who remain free from transfusions
Proportion of participants who remain free from transfusions evaluated over yearly intervals
Rate of breakthrough hemolysis (BTH)
Rate of breakthrough hemolysis (BTH)
Proportion of participants with Major Adverse Vascular Events MAVEs
MAVEs (incl. thrombosis) evaluated over yearly intervals

Full Information

First Posted
February 5, 2021
Last Updated
October 17, 2023
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT04747613
Brief Title
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
Official Title
An Open Label, Multicenter Roll-over Extension Program (REP) to Characterize the Long-term Safety and Tolerability of Iptacopan (LNP023) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Completed PNH Phase 2 and Phase3 Studies With Iptacopan
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
July 27, 2021 (Actual)
Primary Completion Date
September 16, 2027 (Anticipated)
Study Completion Date
October 14, 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to provide access to iptacopan to patients with PNH who have completed Novartis-sponsored Phase 2 or 3 studies with iptacopan
Detailed Description
The purpose of this phase 3 open-label, single arm, multicenter study is to evaluate the long-term safety, tolerability and efficacy of iptacopan in patients with PNH and to provide access to patients who have completed (without tapering down) Phase 2 and Phase 3 trials and derived benefit from iptacopan treatment.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Nocturnal Hemoglobinuria
Keywords
Iptacopan, Paroxysmal Nocturnal Hemoglobinuria (PNH), Hemoglobin, Anemia, LNP023

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
250 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Iptacopan
Arm Type
Experimental
Arm Description
Participants will be receiving open label oral iptacopan 200 mg b.i.d monotherapy
Intervention Type
Drug
Intervention Name(s)
Iptacopan
Other Intervention Name(s)
LNP023
Intervention Description
Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral
Primary Outcome Measure Information:
Title
Proportion of participants with adverse events
Description
Safety evaluations including but not limited to adverse events/serious adverse events, safety laboratory parameters, vital signs, etc. through End of Study visit
Time Frame
60 months
Secondary Outcome Measure Information:
Title
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions
Description
Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions evaluated over yearly intervals
Time Frame
60 months
Title
Proportion of participants who remain free from transfusions
Description
Proportion of participants who remain free from transfusions evaluated over yearly intervals
Time Frame
60 months
Title
Rate of breakthrough hemolysis (BTH)
Description
Rate of breakthrough hemolysis (BTH)
Time Frame
60 months
Title
Proportion of participants with Major Adverse Vascular Events MAVEs
Description
MAVEs (incl. thrombosis) evaluated over yearly intervals
Time Frame
60 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion criteria: Male and female participants ≥ 18 years of age with a diagnosis of PNH who have completed the treatment extension period (without tapering down) of Phase II iptacopan studies (CLNP023X2204, CLNP023X2201), Period 4 of LFG316X2201 or Phase III (CLNP023C12302 and CLNP023C12301) clinical studies at the time point of enrollment visit in this roll over extension. Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections Per investigator's clinical judgement benefit from continued treatment with iptacopan and has been clinically stable on iptacopan monotherapy for at least 3 months Exclusion Criteria: 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 subject at increased risk or potentially confound study data. History of recurrent invasive infections caused by encapsulated organisms, such as Neisseria meningitidis, Streptococcus pneumoniae or Haemophilus influenzae History of hematopoietic stem cell transplantation Other protocol-defined inclusion/exclusion criteria may apply.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Novartis Pharmaceuticals
Phone
1-888-669-6682
Email
novartis.email@novartis.com
First Name & Middle Initial & Last Name or Official Title & Degree
Novartis Pharmaceuticals
Phone
+41613241111
Facility Information:
Facility Name
Novartis Investigative Site
City
Duarte
State/Province
California
ZIP/Postal Code
91010
Country
United States
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Santo Andre
State/Province
SP
ZIP/Postal Code
09090-790
Country
Brazil
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Sao Paulo
State/Province
SP
ZIP/Postal Code
01323-900
Country
Brazil
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Tianjin
State/Province
Tianjin
ZIP/Postal Code
300020
Country
China
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Beijing
ZIP/Postal Code
100730
Country
China
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Tianjin
ZIP/Postal Code
300052
Country
China
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Ostrava
State/Province
Poruba
ZIP/Postal Code
708 52
Country
Czechia
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Brno - Bohunice
ZIP/Postal Code
625 00
Country
Czechia
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Lille Cedex
ZIP/Postal Code
59 037
Country
France
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Paris 10
ZIP/Postal Code
75475
Country
France
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Toulouse
ZIP/Postal Code
31059
Country
France
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Aachen
ZIP/Postal Code
52074
Country
Germany
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Essen
ZIP/Postal Code
45147
Country
Germany
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Hamburg
ZIP/Postal Code
20246
Country
Germany
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Riesa
ZIP/Postal Code
01589
Country
Germany
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Ulm
ZIP/Postal Code
89081
Country
Germany
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Ascoli Piceno
State/Province
AP
ZIP/Postal Code
63100
Country
Italy
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Avellino
State/Province
AV
ZIP/Postal Code
83100
Country
Italy
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Firenze
State/Province
FI
ZIP/Postal Code
50134
Country
Italy
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Milano
State/Province
MI
ZIP/Postal Code
20122
Country
Italy
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Roma
State/Province
RM
ZIP/Postal Code
00161
Country
Italy
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Torino
State/Province
TO
ZIP/Postal Code
10126
Country
Italy
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Bassano Del Grappa
State/Province
VI
ZIP/Postal Code
36061
Country
Italy
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Nagoya
State/Province
Aichi
ZIP/Postal Code
453-8511
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Fukushima city
State/Province
Fukushima
ZIP/Postal Code
960 1295
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Kanazawa-city
State/Province
Ishikawa
ZIP/Postal Code
920-8641
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Isehara
State/Province
Kanagawa
ZIP/Postal Code
259-1193
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Suwa
State/Province
Nagano
ZIP/Postal Code
392-8510
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Suita
State/Province
Osaka
ZIP/Postal Code
565 0871
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Shinjuku-ku
State/Province
Tokyo
ZIP/Postal Code
160-0023
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Kyoto
ZIP/Postal Code
606 8507
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Niigata
ZIP/Postal Code
951 8520
Country
Japan
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Seoul
ZIP/Postal Code
03080
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Seoul
ZIP/Postal Code
06351
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Vilnius
ZIP/Postal Code
LT-08661
Country
Lithuania
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Kota Kinabalu
State/Province
Sabah
ZIP/Postal Code
88586
Country
Malaysia
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Kuching
State/Province
Sarawak
ZIP/Postal Code
93586
Country
Malaysia
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Nijmegen
ZIP/Postal Code
6500 MB
Country
Netherlands
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Singapore
ZIP/Postal Code
119228
Country
Singapore
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Barcelona
State/Province
Catalunya
ZIP/Postal Code
08036
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Santiago de Compostela
State/Province
Galicia
ZIP/Postal Code
15706
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
San Sebastian
State/Province
Pais Vasco
ZIP/Postal Code
20080
Country
Spain
Individual Site Status
Active, not recruiting
Facility Name
Novartis Investigative Site
City
Hualien
ZIP/Postal Code
970
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Taipei
ZIP/Postal Code
10002
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
Leeds
ZIP/Postal Code
LS9 7TF
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Novartis Investigative Site
City
London
ZIP/Postal Code
SE5 9RS
Country
United Kingdom
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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

Learn more about this trial

Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria

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