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A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria (PNH)

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ClinicalTrials.gov Identifier: NCT03406507
Recruitment Status : Recruiting
First Posted : January 23, 2018
Last Update Posted : March 11, 2019
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Description
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Brief Summary:
The purpose of this study is to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and efficacy of ALXN1210 in pediatric patients with paroxysmal nocturnal hemoglobinuria (PNH).

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

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Actual Study Start Date : February 22, 2018
Estimated Primary Completion Date : August 2019
Estimated Study Completion Date : June 2021

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Arms and Interventions
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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.


Outcome Measures
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Primary Outcome Measures :
  1. Cmax (pharmacokinetic parameter) [ Time Frame: Baseline, Week 2, 10, 18, and 26 ]
    Maximum serum concentration

  2. Ctrough (pharmacokinetic parameter) [ Time Frame: Baseline, Week 2, 10, 18, and 26 ]
    Trough serum concentration (measured at end of dosing interval at steady state

  3. accumulation ratio (pharmacokinetic parameter) [ Time Frame: Baseline, Week 2, 10, 18, and 26 ]
  4. free C5 concentrations (pharmacodynamic parameter) [ Time Frame: Baseline, Week 2, 10, 18, and 26 ]
    Change in free C5 concentrations over time

  5. cRBC (pharmacodynamic parameter) [ Time Frame: Baseline, Week 2, 10, 18, and 26 ]
    Change in chicken red blood cell (cRBC) hemolytic activity over time


Secondary Outcome Measures :
  1. Change in lactate dehydrogenase ( LDH) level [ Time Frame: 26 weeks ]
    The percentage change from baseline to Week 26 in serum lactate dehydrogenase (LDH) levels will be compared

  2. Proportion of patients who achieve transfusion avoidance (TA) [ Time Frame: 26 weeks ]
    TA is defined as the proportion of patients who remain transfusion-free and do not require a transfusion according to protocol-specified guidelines.


Eligibility Criteria
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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients from birth up to < 18 years of age and weighing ≥ 5 kg at the time of consent.
  2. PNH diagnosis confirmed by documented by high-sensitivity flow cytometry
  3. Presence of 1 or more of the following PNH-related signs or symptoms within 3 months of Screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia, history of a major adverse vascular event (including thrombosis), dysphagia, or erectile dysfunction; or history of pRBC transfusion due to PNH.
  4. LDH level ≥ 1.5 × ULN for patients not being treated with eculizumab at screening and LDH level ≤ 1.5 × ULN for patients taking eculizumab.
  5. Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and Haemophilus influenzae
  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

Exclusion Criteria:

  1. History of Bone Marrow transplantation
  2. History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease that, in the opinion of the investigator or sponsor, would preclude participation.
  3. 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)
  4. Females who are pregnant, breastfeeding or who have a positive pregnancy test at screening or Day 1
  5. 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.
Contacts and Locations
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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): NCT03406507


Contacts
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Contact: Alexion Pharmaceuticals, Inc 1.855.585.8266 clinicaltrials@alexion.com

Locations
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United States, Georgia
Clinical Trial Site Recruiting
Atlanta, Georgia, United States, 30322
United States, Wisconsin
Clinical Trial Site Recruiting
Milwaukee, Wisconsin, United States, 53226
France
Clinical Trial Site Recruiting
Paris, France
Netherlands
Clinical Trial Site Recruiting
Utrecht, Netherlands
Norway
Clinical Trial Site Recruiting
Oslo, Norway
United Kingdom
Clinical Trial Site Recruiting
Leeds, United Kingdom
Clinical Trial Site Recruiting
London, United Kingdom
Sponsors and Collaborators
Alexion Pharmaceuticals
More Information
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Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03406507     History of Changes
Other Study ID Numbers: ALXN1210-PNH-304
2017‐002820‐26 ( EudraCT Number )
First Posted: January 23, 2018    Key Record Dates
Last Update Posted: March 11, 2019
Last Verified: March 2019

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

Additional relevant MeSH terms:
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Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases
Ravulizumab
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs