Clinical trial NCT04747613: 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
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Keywords: Iptacopan
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Hemoglobin
Anemia
LNP023
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2021-02-10
Enrollment: 208
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Proportion of participants with adverse events
timeFrame: 60 months
Secondary outcomes:
measure: Proportion of participants achieving sustained hemoglobin levels ≥ 12 g/dL in the absence of red blood cell transfusions
timeFrame: 60 months
measure: Proportion of participants who remain free from transfusions
timeFrame: 60 months
measure: Rate of breakthrough hemolysis (BTH)
timeFrame: 60 months
measure: Proportion of participants with Major Adverse Vascular Events MAVEs
timeFrame: 60 months
Other outcomes:
Locations:
city: Duarte
country: United States
facility: City Of Hope
state: California
city: Los Angeles
country: United States
facility: USC Norris Cancer Center
state: California
city: Orange
country: United States
facility: Univ of California Irvine (Chao Family Comprehensive Cancer Center)
state: California
city: Miami Lakes
country: United States
facility: Lakes Research
state: Florida
city: Augusta
country: United States
facility: Augusta University
state: Georgia
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation
state: Ohio
city: Greenville
country: United States
facility: Prisma Health Upstate
state: South Carolina
city: Santo Andre
country: Brazil
facility: Novartis Investigative Site
state: SP
city: Sao Paulo
country: Brazil
facility: Novartis Investigative Site
state: SP
city: Beijing
country: China
facility: Novartis Investigative Site
city: Tianjin
country: China
facility: Novartis Investigative Site
city: Tianjin
country: China
facility: Novartis Investigative Site
city: Ostrava
country: Czechia
facility: Novartis Investigative Site
state: Poruba
city: Brno Bohunice
country: Czechia
facility: Novartis Investigative Site
city: Lille
country: France
facility: Novartis Investigative Site
city: Nantes Cedex 1
country: France
facility: Novartis Investigative Site
city: Nice
country: France
facility: Novartis Investigative Site
city: Paris 10
country: France
facility: Novartis Investigative Site
city: Toulouse
country: France
facility: Novartis Investigative Site
city: Aachen
country: Germany
facility: Novartis Investigative Site
city: Dresden
country: Germany
facility: Novartis Investigative Site
city: Essen
country: Germany
facility: Novartis Investigative Site
city: Hamburg
country: Germany
facility: Novartis Investigative Site
city: Riesa
country: Germany
facility: Novartis Investigative Site
city: Ulm
country: Germany
facility: Novartis Investigative Site
city: Ascoli Piceno
country: Italy
facility: Novartis Investigative Site
state: AP
city: Avellino
country: Italy
facility: Novartis Investigative Site
state: AV
city: Firenze
country: Italy
facility: Novartis Investigative Site
state: FI
city: Milano
country: Italy
facility: Novartis Investigative Site
state: MI
city: Roma
country: Italy
facility: Novartis Investigative Site
state: RM
city: Torino
country: Italy
facility: Novartis Investigative Site
state: TO
city: Bassano Del Grappa
country: Italy
facility: Novartis Investigative Site
state: Vicenza
city: Nagoya
country: Japan
facility: Novartis Investigative Site
state: Aichi
city: Fukushima city
country: Japan
facility: Novartis Investigative Site
state: Fukushima
city: Kanazawa
country: Japan
facility: Novartis Investigative Site
state: Ishikawa
city: Isehara
country: Japan
facility: Novartis Investigative Site
state: Kanagawa
city: Suwa
country: Japan
facility: Novartis Investigative Site
state: Nagano
city: Suita
country: Japan
facility: Novartis Investigative Site
state: Osaka
city: Shinjuku Ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Kyoto
country: Japan
facility: Novartis Investigative Site
city: Niigata
country: Japan
facility: Novartis Investigative Site
city: Seoul
country: Korea, Republic of
facility: Novartis Investigative Site
city: Seoul
country: Korea, Republic of
facility: Novartis Investigative Site
city: Vilnius
country: Lithuania
facility: Novartis Investigative Site
city: Kota Kinabalu
country: Malaysia
facility: Novartis Investigative Site
state: Sabah
city: Kuching
country: Malaysia
facility: Novartis Investigative Site
state: Sarawak
city: Nijmegen
country: Netherlands
facility: Novartis Investigative Site
city: Singapore
country: Singapore
facility: Novartis Investigative Site
city: Barcelona
country: Spain
facility: Novartis Investigative Site
state: Catalunya
city: Santiago De Compostela
country: Spain
facility: Novartis Investigative Site
state: Galicia
city: San Sebastian
country: Spain
facility: Novartis Investigative Site
state: Pais Vasco
city: Hualien
country: Taiwan
facility: Novartis Investigative Site
city: Taipei
country: Taiwan
facility: Novartis Investigative Site
city: Istanbul
country: Turkey
facility: Novartis Investigative Site
city: Leeds
country: United Kingdom
facility: Novartis Investigative Site
state: West Yorkshire
city: London
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: