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

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ClinicalTrials.gov Identifier: NCT03500549
Recruitment Status : Recruiting
First Posted : April 17, 2018
Last Update Posted : October 9, 2018
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 Drug: Soliris Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Actual Study Start Date : June 14, 2018
Estimated Primary Completion Date : July 2019
Estimated Study Completion Date : December 2019


Arm Intervention/treatment
Experimental: Group 1 - Monotherapy APL-2 (n=35)
Complement (C3) Inhibitor
Drug: APL-2
Complement (C3) Inhibitor

Active Comparator: Group 2 - Monotherapy Eculizumab (n=35)
Complement (C5) Inhibitor
Drug: APL-2
Complement (C3) Inhibitor

Drug: Soliris
Complement (C5) Inhibitor




Primary Outcome Measures :
  1. Hemoglobin [ Time Frame: 16 weeks ]
    Week 16 change from baseline in hemoglobin level



Information from the National Library of Medicine

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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. At least 18 years of age
  2. Primary diagnosis of PNH confirmed by high-sensitivity flow cytometry
  3. On treatment with eculizumab. Dose of eculizumab must have been stable for at least 3 months prior to the Screening Visit
  4. Hb <10.5 g/dL at the Screening Visit
  5. Absolute reticulocyte count > 1.0x ULN at the Screening Visit
  6. Platelet count of >50,000/mm3 at the Screening Visit
  7. Absolute neutrophil count >500/mm3 at the Screening Visit
  8. Vaccination against Neisseria meningitides types A, C, W, Y and B, Streptococcus pneumoniae and Haemophilus influenzae Type B (Hib) either within 2 years prior to Day 1 dosing, or within 14 days after starting treatment with APL-2. Unless documented evidence exists that subjects are non-responders to vaccination as evidenced by titers or display titer levels within acceptable local limits
  9. Women of child-bearing potential (WOCBP) must have a negative pregnancy test at the Screening and Day -28 Visit (Run-in Period) and must agree to use protocol defined methods of contraception for the duration of the study and 90 days after their last dose of study drug
  10. Males must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study and 90 days after their last dose of study drug
  11. Willing and able to give informed consent
  12. Willing and able to self-administer APL-2 (administration by caregiver will be allowed)
  13. Have a body mass index (BMI) ≤40.0 kg/m2

Exclusion Criteria:

  1. Active bacterial infection that has not resolved within 1 week of Day -28 (first dose of APL-2)
  2. Receiving iron, folic acid, vitamin B12 and EPO, unless the dose is stable, in the 4 weeks prior to Screening
  3. Hereditary complement deficiency
  4. History of bone marrow transplantation
  5. History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration
  6. Participation in any other investigational drug trial or exposure to other investigational agent within 30 days or 5 half-lives (whichever is longer)
  7. Currently breast-feeding women
  8. Inability to cooperate or any condition that, in the opinion of the investigator, could increase the subject's risk of participating in the study or confound the outcome of the study

    This study includes cardiac safety evaluations. The following cardiac eligibility criteria are necessary to avoid confounding the cardiac safety outcomes:

  9. History or family history of Long QT Syndrome or torsade de pointes, unexplained syncope, syncope from an uncorrected cardiac etiology, or family history of sudden death
  10. Myocardial infarction, CABG, coronary or cerebral artery stenting and /or angioplasty, stroke, cardiac surgery, or hospitalization for congestive heart failure within 3 months or greater than Class 2 Angina Pectoris or NYHA Heart Failure Class >2
  11. QTcF > 470 ms, PR > 280 ms
  12. Mobitz II 2nd degree AV Block, 2:1 AV Block, High Grade AV Block, or Complete Heart Block unless the patient has an implanted pacemaker or implantable cardiac defibrillator (ICD) with backup pacing capabilities
  13. Receiving Class 1 or Class 3 antiarrhythmic agents, or arsenic, methadone, ondansetron or pentamidine at screening
  14. Receiving any other QTc-prolonging drugs (see Appendix 4 in Section 18.4), at a stable dose for less than 3 weeks prior to dosing
  15. Receiving prophylactic ciprofloxacin, erythromycin or azithromycin for less than one week prior to the first dose of study medication (must have a repeat screening ECG after one week of prophylactic antibiotics with QTcF < 470 ms)

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): NCT03500549


Contacts
Contact: Allen Papazian 617-977-5700 clinicaltrials@apellis.com

Locations
United States, Florida
Apellis Investigational Site Recruiting
Orange City, Florida, United States, 32763
Sponsors and Collaborators
Apellis Pharmaceuticals, Inc.

Responsible Party: Apellis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03500549     History of Changes
Other Study ID Numbers: APL2-302
First Posted: April 17, 2018    Key Record Dates
Last Update Posted: October 9, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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
Complement System Proteins
Immunologic Factors
Physiological Effects of Drugs