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

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04747613
• Other study ID #: CLNP023C12001B
• Status: Recruiting
• Phase: Phase 3
• Start date: July 27, 2021
• Est. completion date: January 10, 2028

Study information

• Verified date: May 2024
• Source: Novartis
• Contact: Novartis Pharmaceuticals
• Phone: 1-888-669-6682
• Email: novartis.email[@]novartis.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 250
• Est. completion date: January 10, 2028
• Est. primary completion date: January 10, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria

Intervention

Drug:
• Iptacopan
Taken orally b.i.d. Dosage supplied: 200 mg Dosage form: hard gelatin capsule Route of administration: oral

Locations

Country	Name	City	State
Brazil	Novartis Investigative Site	Santo Andre	SP
Brazil	Novartis Investigative Site	Sao Paulo	SP
China	Novartis Investigative Site	Beijing
China	Novartis Investigative Site	Tianjin
China	Novartis Investigative Site	Tianjin
Czechia	Novartis Investigative Site	Brno - Bohunice
Czechia	Novartis Investigative Site	Ostrava	Poruba
France	Novartis Investigative Site	Lille Cedex
France	Novartis Investigative Site	Nice
France	Novartis Investigative Site	Paris 10
France	Novartis Investigative Site	Toulouse
Germany	Novartis Investigative Site	Aachen
Germany	Novartis Investigative Site	Essen
Germany	Novartis Investigative Site	Hamburg
Germany	Novartis Investigative Site	Riesa
Germany	Novartis Investigative Site	Ulm
Italy	Novartis Investigative Site	Ascoli Piceno	AP
Italy	Novartis Investigative Site	Avellino	AV
Italy	Novartis Investigative Site	Bassano Del Grappa	VI
Italy	Novartis Investigative Site	Firenze	FI
Italy	Novartis Investigative Site	Milano	MI
Italy	Novartis Investigative Site	Roma	RM
Italy	Novartis Investigative Site	Torino	TO
Japan	Novartis Investigative Site	Fukushima city	Fukushima
Japan	Novartis Investigative Site	Isehara	Kanagawa
Japan	Novartis Investigative Site	Kanazawa-city	Ishikawa
Japan	Novartis Investigative Site	Kyoto
Japan	Novartis Investigative Site	Nagoya	Aichi
Japan	Novartis Investigative Site	Niigata
Japan	Novartis Investigative Site	Shinjuku-ku	Tokyo
Japan	Novartis Investigative Site	Suita	Osaka
Japan	Novartis Investigative Site	Suwa	Nagano
Korea, Republic of	Novartis Investigative Site	Seoul
Korea, Republic of	Novartis Investigative Site	Seoul
Lithuania	Novartis Investigative Site	Vilnius
Malaysia	Novartis Investigative Site	Kota Kinabalu	Sabah
Malaysia	Novartis Investigative Site	Kuching	Sarawak
Netherlands	Novartis Investigative Site	Nijmegen
Singapore	Novartis Investigative Site	Singapore
Spain	Novartis Investigative Site	Barcelona	Catalunya
Spain	Novartis Investigative Site	San Sebastian	Pais Vasco
Spain	Novartis Investigative Site	Santiago De Compostela	Galicia
Taiwan	Novartis Investigative Site	Hualien
Taiwan	Novartis Investigative Site	Taipei
Turkey	Novartis Investigative Site	Istanbul
United Kingdom	Novartis Investigative Site	Leeds
United Kingdom	Novartis Investigative Site	London
United States	Augusta University	Augusta	Georgia
United States	Montefiore Medical Center .	Bronx	New York
United States	Cleveland Clinic Foundation	Cleveland	Ohio
United States	City Of Hope	Duarte	California
United States	Prisma Health Upstate	Greenville	South Carolina
United States	USC Norris Cancer Center	Los Angeles	California
United States	Lakes Research	Miami Lakes	Florida
United States	Univ of California Irvine (Chao Family Comprehensive Cancer Center)	Orange	California

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Brazil,  China,  Czechia,  France,  Germany,  Italy,  Japan,  Korea, Republic of,  Lithuania,  Malaysia,  Netherlands,  Singapore,  Spain,  Taiwan,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	60 months
Secondary	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	60 months
Secondary	Proportion of participants who remain free from transfusions	Proportion of participants who remain free from transfusions evaluated over yearly intervals	60 months
Secondary	Rate of breakthrough hemolysis (BTH)	Rate of breakthrough hemolysis (BTH)	60 months
Secondary	Proportion of participants with Major Adverse Vascular Events MAVEs	MAVEs (incl. thrombosis) evaluated over yearly intervals	60 months