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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 7 December 2020
Main ID:  EUCTR2019-004665-40-NL
Date of registration: 09/11/2020
Prospective Registration: Yes
Primary sponsor: Novartis Pharma AG
Public title: Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatment
Scientific title: A randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody
Date of first enrolment: 20/11/2020
Target sample size: 91
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-004665-40
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: yes
Randomised: yes
Open: yes
Single blind: no
Double blind: no
Parallel group: yes
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: no
Other: yes
Other specify the comparator: Ravulizumab and Eculizumab
Number of treatment arms in the trial: 2
 
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Brazil Canada Czechia France Germany Hungary Italy Japan
Korea, Republic of Netherlands Spain Taiwan United Kingdom United States
Contacts
Name: Clinical Trial Information Desk   
Address:  Forum 1, Novartis Campus 4056 Basel Switzerland
Telephone: +41613241 111
Email: clinicaltrial.enquiries@novartis.com
Affiliation:  Novartis Pharma AG
Name: Clinical Trial Information Desk   
Address:  Forum 1, Novartis Campus 4056 Basel Switzerland
Telephone: +41613241 111
Email: clinicaltrial.enquiries@novartis.com
Affiliation:  Novartis Pharma AG
Key inclusion & exclusion criteria
Inclusion criteria:
- Male and female participants = 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with clone size = 10%
- Stable regimen of anti-C5 antibody treatment (either eculizumab or ravulizumab) for at least 6 months prior to randomization
- Mean hemoglobin level <10 g/dL
- Vaccination against Neisseria meningitidis infection is required prior to the start of treatment.
- If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given

Other protocol-defined inclusion criteria may apply
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 82
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 9

Exclusion criteria:
- Participants on a stable eculizumab dose but with a dosing interval of 11 days or less
- Known or suspected hereditary complement deficiency at screening
- History of hematopoietic stem cell transplantation
- Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L; platelets <30x109/L; neutrophils <500x106/L).
- Active systemic bacterial, viral or fungal infection within 14 days prior to study drug administration
- A history of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
- Major concurrent comorbidities including but not limited to severe kidney disease (e.g., dialysis), advanced cardiac disease (e.g., NYHA class IV), severe pulmonary disease (e.g., severe pulmonary) hypertension (WHO class IV)), or hepatic disease (e.g., active hepatitis) that in the opinion of the investigator precludes participant's participation in the study.

Other protocol-defined exclusion criteria may apply


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1 Level: PT Classification code 10034042 Term: Paroxysmal nocturnal haemoglobinuria System Organ Class: 10038359 - Renal and urinary disorders
Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Intervention(s)

Product Name: LNP023
Product Code: LNP023
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: not yet established
Current Sponsor code: LNP023
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-

Product Name: LNP023
Product Code: LNP023
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: not yet established
Current Sponsor code: LNP023
Other descriptive name:  LNP023 HYDROCHLORIDE SALT
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 200-

Trade Name: Soliris
Product Name: Eculizumab
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: ECULIZUMAB
CAS Number: 219685-50-4
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 300-

Trade Name: Ultomiris
Product Name: Ravulizumab
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: RAVULIZUMAB
Other descriptive name: ravulizumab
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 300-

Primary Outcome(s)
Main Objective: - Proportion of participants achieving a sustained increase in hemoglobin levels of = 2 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.
Primary end point(s): - Proportion of participants achieving a sustained increase in hemoglobin levels from baseline of = 2 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.
Secondary Objective: - Proportion of participants who remain free from transfusions
- Average change in hemoglobin
- Change in fatigue score, using the FACIT-Fatigue questionnaire
- Average change in reticulocyte counts
- Average percent change in LDH
- Rate of breakthrough hemolysis (BTH)
- Rates of Major Adverse Vascular Events (MAVEs incl. thrombosis
Timepoint(s) of evaluation of this end point: Day 126 and Day 168
Secondary Outcome(s)
Secondary end point(s): 1. Proportion of participants who remain free from transfusions
2. Change from baseline in hemoglobin (g/dL) as mean of visits between Day 126 and D168
3. Change from baseline in FACIT-Fatigue scores as mean of visits between Day 126 and Day 168
4. Change from baseline in reticulocyte counts as mean of visits between Day 126 and Day 168
5. Percent change from baseline in LDH levels (U/L) as mean of visits between Day 126 and Day 168
6. Rate of breakthrough hemolysis (BTH)
7. Rates of Major Adverse Vascular Events (MAVEs incl. thrombosis
Timepoint(s) of evaluation of this end point: 1. Day 14 and Day 168
2. Baseline and as mean of visit Day 126, 140, 154 and 168.
3. Baseline and as mean of visit Day 126, 140, 154 and 168.
4. Baseline and as mean of visit Day 126, 140, 154 and 168.
5. Baseline and as mean of visit Day 126, 140, 154 and 168.
6. Day 1 and Day 168.
7. Day 1 and Day 168.
Secondary ID(s)
2019-004665-40-HU
CLNP023C12302
Source(s) of Monetary Support
Novartis Pharma AG
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 20/11/2020
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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