Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02598583
• Date of registration: 02/11/2015
• Prospective Registration: No
• Primary sponsor: Alexion Pharmaceuticals
• Public title: Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Scientific title: An Open-Label, Intrapatient, Dose-Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal Hemoglobinuria
• Date of first enrolment: November 2015
• Target sample size: 13
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT02598583
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Australia, Korea, Republic of

Contacts

• Name: Alexion Pharmaceuticals, Inc.
• Affiliation: Alexion Pharmaceuticals

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Male or female =18 years of age

2. PNH diagnosis confirmed by documented high-sensitivity flow cytometry

3. Documented meningococcal vaccination not more than 3 years prior to dosing

4. 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

5. Willing and able to give written informed consent and comply with the study visit
schedule

Exclusion Criteria:

1. Treatment with a complement inhibitor at any time

2. Females who are pregnant, breastfeeding or who have a positive pregnancy test at
screening or Day 1

3. Participation in an interventional clinical study within 30 days before initiation of
dosing on Day 1, or use of any experimental therapy within 30 days prior to dosing on
Day 1, or within 5 half-lives of the product, whichever is greater

4. History of allergy to excipients of ALXN1210 or known allergy to Chinese hamster ovary
cell proteins

5. Inability to comply with study requirements

6. History of any clinically significant cardiac, hepatic, immunologic, pulmonary, or
rheumatoid disease that, in the Investigator's judgment, would preclude participation

7. Other unspecified reasons that, in the opinion of the Investigator or Sponsor, make
the participant unsuitable for enrollment



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

PNH

Intervention(s)

Biological: ALXN1210

Primary Outcome(s)

Percent Change In Lactate Dehydrogenase (LDH) Levels From Baseline To Day 169 [Time Frame: Baseline, Day 169]

Secondary Outcome(s)

Change In Clinical Manifestations Of PNH From Baseline To Day 169 [Time Frame: Baseline, Day 169]

Percent Change In Haptoglobin Levels From Baseline To Day 169 [Time Frame: Baseline, Day 169]

Percent Change In D-dimer Levels From Baseline To Day 169 [Time Frame: Baseline, Day 169]

Percent Change In Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clones From Baseline To Day 169 [Time Frame: Baseline, Day 169]

Percent Change In Free Hemoglobin Levels From Baseline To Day 169 [Time Frame: Baseline, Day 169]

Percent Change In Reticulocyte/Erythrocyte Count From Baseline To Day 169 [Time Frame: Baseline, Day 169]

Secondary ID(s)

ALXN1210-PNH-103

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 30/01/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02598583