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Trial record 4 of 8 for:    ALXN1210

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

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ClinicalTrials.gov Identifier: NCT03056040
Recruitment Status : Active, not recruiting
First Posted : February 16, 2017
Last Update Posted : March 26, 2018
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Brief Summary:
The purpose of this study is to assess ALXN1210 compared to eculizumab in adult patients with PNH who are clinically stable on eculizumab for at least 6 months

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH) Biological: ALXN1210 Biological: Eculizumab Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 197 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : June 5, 2017
Actual Primary Completion Date : March 8, 2018
Estimated Study Completion Date : March 2020


Arm Intervention/treatment
Experimental: ALXN1210 Biological: ALXN1210

Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight.

  • ≥ 40 to <60 kg: 2400 mg loading, then 3000 mg every 8 weeks
  • ≥ 60 to <100 kg: 2700 mg loading, then 3300 mg every 8 weeks
  • ≥100 kg: 3000 mg loading, then 3600 mg every 8 weeks

Active Comparator: Eculizumab Biological: Eculizumab

Regular maintenance dosing beginning on Day 1, then every 2 weeks to Day 183.

Single loading dose of ALXN1210 on Day 183, followed by regular maintenance dosing beginning on Day 197, based on weight.

  • ≥ 40 to <60 kg: 2400 mg loading, then 3000 mg every 8 weeks
  • ≥ 60 to <100 kg: 2700 mg loading, then 3300 mg every 8 weeks
  • ≥100 kg: 3000 mg loading, then 3600 mg every 8 weeks




Primary Outcome Measures :
  1. Hemolysis as directly measured by percentage change in LDH levels [ Time Frame: 26 weeks ]
    The difference between treatment arms in LDH-PCHG from Baseline to Day 183


Secondary Outcome Measures :
  1. Change from baseline in quality of life as assessed by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue [ Time Frame: 26 weeks ]
    Change in quality of life as assessed by the FACIT-Fatigue scale will be compared between the treatment groups from Baseline to Day 183

  2. Percentage of patients who achieve Transfusion Avoidance (TA) [ Time Frame: 26 weeks ]
    The proportion of patients who remain transfusion-free will be compared between treatment groups from Baseline to Day 183

  3. Percentage of patients with stabilized hemoglobin [ Time Frame: 26 weeks ]
    The percentage of patients with stabilized hemoglobin will be compared between the treatment groups from Baseline to Day 183.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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. LDH level ≤ 1.5 × 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

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03056040


  Show 47 Study Locations
Sponsors and Collaborators
Alexion Pharmaceuticals

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03056040     History of Changes
Other Study ID Numbers: ALXN1210-PNH-302
2016-002026-36 ( EudraCT Number )
First Posted: February 16, 2017    Key Record Dates
Last Update Posted: March 26, 2018
Last Verified: March 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases