ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) currently Treated With Eculizumab

• Register: EUCTR
• Last refreshed on: 10 December 2019
• Main ID: EUCTR2016-002026-36-GB
• Date of registration: 14/02/2017
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals Incorporated
• Public title: ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) currently Treated With Eculizumab
• Scientific title: A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
• Date of first enrolment: 30/05/2017
• Target sample size: 192
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-002026-36
• 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: yes; Placebo: no; Other: yes; Other specify the comparator: 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

Germany, Netherlands, Spain, United Kingdom, United States

Contacts

• Name: European Clinical Trial Information
• Affiliation: Alexion Europe SAS

• Name: European Clinical Trial Information
• Affiliation: Alexion Europe SAS

Key inclusion & exclusion criteria

Inclusion criteria:
1. Male or female = 18 years of age
2. Treated with eculizumab for PNH for at least 6 months prior to Day 1
3. Lactate dehydrogenase (LDH) = 1.5 x upper limit of normal (ULN) at Screening
4. PNH diagnosis confirmed by documented by high-sensitivity flow cytometry
5. Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment.
6. Female patients of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ALXN1210
7. Willing and able to give written informed consent and comply with study visit schedule
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 172
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion criteria:
1. History of bone marrow transplantation
2. Body weight < 40 kilograms
3. History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease that, in the opinion of the investigator or sponsor, would preclude participation.
4. Unstable medical conditions (eg, myocardial ischemia, active gastrointestinal bleed, severe congestive heart failure, anticipated need for major surgery within 6 months of randomization, coexisting chronic anemia unrelated to PNH)
5. Females who are pregnant, breastfeeding or who have a positive pregnancy test at screening or Day 1
6. Participation in another interventional clinical study or use of any experimental therapy within 30 days before initiation of study drug on Day 1 in this study or within 5 half-lives of that investigational product, whichever is greater.



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: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Intervention(s)

Product Code: ALXN1210
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Current Sponsor code: ALXN1210
Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-

Trade Name: Soliris
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: ECULIZUMAB
CAS Number: 219685-50-4
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-

Primary Outcome(s)

Secondary Objective: Safety and tolerability of ALXN1210
Additional efficacy measures

Timepoint(s) of evaluation of this end point: Week 26

Main Objective: Efficacy of ALXN1210

Primary end point(s): Hemolysis as directly measured by lactate dehydrogenase percent change (LDH-PCHG)

Secondary Outcome(s)

Secondary end point(s): - Change from baseline in quality of life (QoL) as assessed by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue
- Percentage of patients who achieve transfusion avoidance (TA)
- Proportion of patients with stabilized hemoglobin

Timepoint(s) of evaluation of this end point: Week 26

Secondary ID(s)

ALXN1210-PNH-302

Source(s) of Monetary Support

Alexion Pharmaceuticals Incorporated

Ethics review

Status: Approved
Approval date:
Contact: