Overview

This trial is active, not recruiting.

Conditions paroxysmal nocturnal hemoglobinuria, pnh
Treatments study drug- alxn1210
Phase phase 2
Sponsor Alexion Pharmaceuticals
Start date November 2015
End date February 2017
Trial size 26 participants
Trial identifier NCT02605993, 2015-002674-20, ALXN1210-PNH-201

Summary

The primary purpose of this study is to evaluate the safety, tolerability and efficacy of multiple IV doses of ALXN1210 administered to complement inhibitor treatment-naïve patients with PNH.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation non-randomized
Intervention model single group assignment
Primary purpose treatment
Masking no masking
Arm
(Experimental)
Study Drug- ALXN1210
study drug- alxn1210
Cohort 1: ascending dose escalation to therapeutic dose level At Day 477, the dose and dosing interval for all patients will change to an every 8 week, weight-based regimen, as follows: ≥ 40 to < 60 kg: 3000 mg every 8 weeks ≥ 60 to < 100 kg: 3300 mg every 8 weeks ≥ 100 kg: 3600 mg every 8 weeks
(Experimental)
Study Drug- ALXN1210
study drug- alxn1210
Cohort 2: ascending dose escalation to therapeutic dose level At Day 463, the dose and dosing interval for all patients will change to an every 8 week, weight-based regimen, as follows: ≥ 40 to < 60 kg: 3000 mg every 8 weeks ≥ 60 to < 100 kg: 3300 mg every 8 weeks ≥ 100 kg: 3600 mg every 8 weeks
(Experimental)
Study Drug- ALXN1210
study drug- alxn1210
Cohort 3: ascending dose escalation to therapeutic dose level At Day 477, the dose and dosing interval for all patients will change to an every 8 week, weight-based regimen, as follows: ≥ 40 to < 60 kg: 3000 mg every 8 weeks ≥ 60 to < 100 kg: 3300 mg every 8 weeks ≥ 100 kg: 3600 mg every 8 weeks
(Experimental)
Study Drug- ALXN1210
study drug- alxn1210
Cohort 4: ascending dose escalation to therapeutic dose level

Primary Outcomes

Measure
To evaluate the safety as measured by change in the number of treatment-emergent and related adverse events as assessed by CTCAE (v4.03).
time frame: Treatment Period (36 weeks) and Follow-up Period (week 36 to week 144)
Efficacy as measured by the percentage change in lactate dehydrogenase or LDH levels from baseline to Day 253 during treatment with ALXN1210.
time frame: 36 weeks

Secondary Outcomes

Measure
Time to maximal concentration of ALXN1210 in blood (tmax)
time frame: 36 weeks
Area Under the plasma concentration versus time Curve (AUC) of ALXN1210
time frame: 36 weeks
Peak Plasma Concentration (Cmax) of ALXN1210
time frame: 36 weeks
ALXN1210 elimination half-life (t1/2) in blood
time frame: 36 weeks
Change from baseline in concentration of complement factor 5 (C5)
time frame: Treatment Period (36 weeks) and Follow-up Period (week 36 to week 144)
Change from baseline in serum levels of terminal complement activity
time frame: Treatment Period (36 weeks) and Follow-up Period (week 36 to week 144)
Development of antibodies against ALXN1210
time frame: Up to 144 weeks

Eligibility Criteria

All participants at least 18 years old.

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 investigational product, whichever is greater 4. History of allergy to excipients of ALXN1210 or known allergy to Chinese hamster ovary (CHO) 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 patient unsuitable for enrollment

Additional Information

Official title A Phase 2, Open-label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal Hemoglobinuria
Trial information was received from ClinicalTrials.gov and was last updated in March 2017.
Information provided to ClinicalTrials.gov by Alexion Pharmaceuticals.