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A Phase III Study to Evaluate the Efficacy and Safety of APL-2 in Patients With PNH

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ClinicalTrials.gov Identifier: NCT04085601
Recruitment Status : Recruiting
First Posted : September 11, 2019
Last Update Posted : September 11, 2019
Sponsor:
Information provided by (Responsible Party):
Apellis Pharmaceuticals, Inc.

Brief Summary:
Evaluation of the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria .

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria Drug: APL-2 Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 54 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Actual Study Start Date : August 14, 2019
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : September 30, 2020


Arm Intervention/treatment
No Intervention: Standard of Care (SOC) excluding complement inhibitors
Experimental: 1,080mg APL-2 administered subcutaneously twice weekly Drug: APL-2
Complement (C3) Inhibitor




Primary Outcome Measures :
  1. Hemoglobin stabilization defined as avoidance of a > 1 g/dl decrease in hemoglobin levels in the absence of transfusion. [ Time Frame: Baseline through Week 26 ]
  2. Reduction in lactate dehydrogenase (LDH) level [ Time Frame: Baseline to Week 26 ]


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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. Be at least 18 years old (inclusive).
  2. Have LDH ≥1.5 x ULN at the screening visit.
  3. Have PNH diagnosis, confirmed by high sensitivity flow cytometry (granulocyte or monocyte clone >10%).
  4. Have Hb less than the lower limit of normal (LLN) at the screening visit.
  5. Have ferritin greater than/equal to the LLN, or total iron binding capacity (TIBC) less than/equal to ULN at the screening visit, based on central laboratory reference ranges. If a subject is receiving iron supplements at screening, the Investigator must ensure that the subject's dose has been stable for 4 weeks prior to screening, and it must be maintained throughout the study. Subjects not receiving iron at screening must not start iron supplementation during the course of the study.
  6. Body mass index (BMI) ≤ 35 kg/m2 at the screening visit.
  7. Have a platelet count of >50,000/mm3 at the screening visit.
  8. Have an absolute neutrophil count >500/mm3 at the screening visit.

Exclusion Criteria:

  1. Treatment with any complement inhibitor (eg, eculizumab) within 3 months prior to screening.
  2. Hereditary complement deficiency.
  3. History of bone marrow transplantation.
  4. Concomitant use of any of the following medications is prohibited if not on a stable regimen for the time period indicated below prior to screening:

    • Erythropoietin or immunosuppressants for at least 8 weeks
    • Systemic corticosteroids for at least 4 weeks
    • Vitamin K antagonists (eg, warfarin) with a stable international normalized ratio (INR) for at least 4 weeks
    • Iron supplements, vitamin B12, or folic acid for at least 4 weeks
    • Low-molecular-weight heparin for at least 4 weeks

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): NCT04085601


Contacts
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Contact: Apellis Clinical Trial Information Line 617-977-5700 clinicaltrials@apellis.com

Locations
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Malaysia
Hospital Ampang Recruiting
Ampang, Selangor, Malaysia, 68000
University Malaya Medical Centre Recruiting
Kuala Lumpur, Malaysia, 59100
Poland
EMC Medical Institute Recruiting
Wrocław, Poland, 50-220
Sponsors and Collaborators
Apellis Pharmaceuticals, Inc.

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Responsible Party: Apellis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04085601     History of Changes
Other Study ID Numbers: APL2-308
First Posted: September 11, 2019    Key Record Dates
Last Update Posted: September 11, 2019
Last Verified: September 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