ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03593200
Recruitment Status : Recruiting
First Posted : July 20, 2018
Last Update Posted : October 16, 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).
Actual Study Start Date : August 16, 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 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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 Recruiting
Sofia, Bulgaria
Contact: Ismail Amine         
Contact       ismail.amin@acibademcityclinic.bg   
Specialized Hospital for Active Treatment of Hematologic Diseases EAD, Sofia Recruiting
Sofia, Bulgaria
Contact: Kalina Ignatova         
Greece
Γενικό Νοσοκομείο Αθηνών "Λαικό" Not yet recruiting
Athens, Greece
Contact: Maria Aggelopoulou         
Contact       mkangelop@gmail.com   
Πανεπιστημιακό Γενικό Νοσοκομείο "ΑΤΤΙΚΟΝ" Not yet recruiting
Athens, Greece
Contact: Vasiliki Vasiliki Pappa         
Contact       vas_pappa@yahoo.com   
Poland
"Samodzielny Publiczny Szpital Kliniczny nr 1 w Lublinie" Not yet recruiting
Lublin, Poland
Contact: Marek Hus         
Contact       marhus@go2.pl   
"Instytut Hematologii i Transfuzjologii " Not yet recruiting
Warszawa, Poland
Contact: Ewa Lech-Marańda         
Contact       emaranda@ihit.waw.pl   
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu Not yet recruiting
Wrocław, Poland
Contact: Tomasz Wróbel         
Contact       tomasz.wrobel@umed.wroc.pl   
Romania
Spitalul Clinic Colentina Not yet recruiting
Bucuresti, Romania
Contact: Viola Popov         
Contact       violamariap@gmail.com   
Secția Clinică Hematologie I Not yet recruiting
București, Romania
Contact: Iulia Ursuleac         
Contact       iuliaursuleac@yahoo.com   
Institutul Oncologic "Prof. Dr. Ion Chiricuță" Not yet recruiting
Cluj-Napoca, Romania
Contact: Mihnea Zdrenghea, MD, PhD         
Contact       mzdrenghea@umfcluj.ro   
Spitalul Clinic Judeţean de Urgenţă Timişoara Not yet recruiting
Timişoara, Romania
Contact: Mihaela Adriana, MD, PhD         
Contact       dsimu@mail.dnttm.ro   
Serbia
Klinički centar Srbije Recruiting
Belgrade, Serbia
Contact: Andrija Bogdanović         
Contact       ebogdano@eunet.rs   
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: October 16, 2018
Last Verified: October 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