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A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With PNH

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ClinicalTrials.gov Identifier: NCT03593200
Recruitment Status : Not yet recruiting
First Posted : July 20, 2018
Last Update Posted : July 20, 2018
Sponsor:
Information provided by (Responsible Party):
Apellis Pharmaceuticals, Inc.

Brief Summary:
This is a Phase IIa, open-label, multiple dose, study in patients with PNH who have not received eculizumab (Soliris ®) in the past. A single cohort of subjects is planned for evaluation.

Condition or disease Intervention/treatment Phase
PNH Drug: APL-2 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase IIa, Open Label, Multiple Dose Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH).
Estimated Study Start Date : August 2018
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019


Arm Intervention/treatment
Experimental: Experimental: Cohort 1
270 mg/day (up to 360 mg/day from Day 29) from Day 1 to Day 364*
Drug: APL-2
Complement (C3) Inhibitor




Primary Outcome Measures :
  1. 1.Number of treatment emergent adverse events (TEAEs) following administration of multiple doses of SC APL-2. Severity of treatment emergent adverse events (TEAEs) following administration of multiple doses of SC APL-2. [ Time Frame: Change from baseline up to week 36 ]
  2. 2. LDH (U/L) [ Time Frame: Change from baseline up to week 32 ]
  3. 3. Haptoglobin (mg/dL) & Hemoglobin (g/dl) [ Time Frame: Change from baseline up to week 32 ]


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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:

  • At least 18 years old (inclusive)
  • Diagnosed with PNH (white blood cell (WBC) clone >10%)
  • Lactose dehydrogenase (LD) ≥2 times the upper limit of normal
  • Screening Ferritin ≥ normal and Total Iron Binding Capacity (TIBC) < LLN based on central lab reference ranges. If a subject is receiving iron supplements at screening, the investigator must ensure that his/her dose has been stable for 8 weeks prior to enrolment and must be maintained throughout the study
  • Last transfusion within 12 months prior to screening
  • Platelet count of >30,000/mm3 at the screening visit
  • Absolute neutrophil count >500/ mm3 at the screening visit
  • Women of child-bearing potential (WOCBP) must have a negative pregnancy test at screening and must agree to use protocol defined methods of contraception for the duration of the study
  • Males must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study
  • 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
  • Willing and able to give informed consent

Exclusion Criteria:

  • Prior eculizumab (Soliris®) treatment
  • Active bacterial infection
  • Hereditary complement deficiency
  • History of bone marrow transplantation
  • Concurrent severe aplastic anemia (SAA), defined as currently receiving immunosuppressive therapy for SAA including but not limited to cyclosporin A, tacrolimus, mycophenolate mofetil or anti-thymocyte globulin
  • Participation in any other investigational drug trial or exposure to another investigational agent, device or procedure within 30 days
  • Evidence of QTcF prolongation defined as >450 ms for males and >470 ms for females at screeningBreast-feeding women
  • History of meningococcal disease

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): NCT03593200


Contacts
Contact: Frank Stout 617-959-0021 clinicaltrials@apellis.com

Locations
Bulgaria
Acibadem City Clinic MHAT Tokuda EAD Sofia Active, not recruiting
Sofia, Bulgaria
Specialized Hospital for Active Treatment of Hematologic Diseases EAD, Sofia Active, not recruiting
Sofia, Bulgaria
Greece
Γενικό Νοσοκομείο Αθηνών "Λαικό"
Athens, Greece
Πανεπιστημιακό Γενικό Νοσοκομείο "ΑΤΤΙΚΟΝ" Active, not recruiting
Athens, Greece
Poland
"Samodzielny Publiczny Szpital Kliniczny nr 1 w Lublinie"
Lublin, Poland
"Instytut Hematologii i Transfuzjologii " Active, not recruiting
Warszawa, Poland
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu Active, not recruiting
Wrocław, Poland
Romania
Spitalul Clinic Colentina Active, not recruiting
Bucuresti, Romania
Secția Clinică Hematologie I Active, not recruiting
București, Romania
Institutul Oncologic "Prof. Dr. Ion Chiricuță" Active, not recruiting
Cluj-Napoca, Romania
Spitalul Clinic Judeţean de Urgenţă Timişoara Active, not recruiting
Timişoara, Romania
Serbia
Klinički centar Srbije Active, not recruiting
Belgrade, Serbia
Sponsors and Collaborators
Apellis Pharmaceuticals, Inc.
Investigators
Study Director: Federico Grossi, MD, PhD Study Director

Responsible Party: Apellis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03593200     History of Changes
Other Study ID Numbers: APL2-202
First Posted: July 20, 2018    Key Record Dates
Last Update Posted: July 20, 2018
Last Verified: July 2018

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

Additional relevant MeSH terms:
Hemoglobinuria, Paroxysmal
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases