CompletedNCT02588833PADDOCKUpdated Jan 11, 2021Results posted

Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.

A Phase 1 interventional study of Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria, sponsored by Apellis Pharmaceuticals, Inc.. Completed at 7 sites in 4 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-01-11.

Sponsored by Apellis Pharmaceuticals, Inc. · Phase 1, Interventional, and Treatment

Phase: Phase 1
Study type: Interventional
Enrollment: 23
Allocation: Non-randomized
Ages: 18 Years and older
Sex: All

Study summary

The objectives of the study are to assess the safety, tolerability, preliminary efficacy and PK of multiple subcutaneous (SC) doses of pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who have not received treatment with eculizumab in the past.

An exploratory objective of the study is to assess the pharmacodynamics (PD) of multiple SC doses of pegcetacoplan when administered to PNH patients.

Read the detailed description

This is a Phase Ib, open-label, multiple ascending dose, pilot study in patients with PNH who have not received eculizumab (Soliris)® in the past. Two cohorts of 3 subjects are planned for evaluation.

Safety will be assessed throughout the study; serial blood and urine samples will be collected for these assessments. Blood samples will be collected for the assessment of pegcetacoplan PK. Additional samples for assessment of PD will also be collected.

The study will consist of four parts;

Part 1: subjects will receive pegcetacoplan for 28 days.
Part 2A: subjects may continue to receive pegcetacoplan for a further 56 days if there is evidence of perceived clinical benefit following review of available safety, PK and PD data by the investigator and sponsor
Part 2B/Part 2C: subjects may continue to receive daily pegcetacoplan treatment for up to 364 days if there is ongoing evidence of clinical benefit following review of the available safety, PK and PD data. After completion of Part 2B, subjects could continue treatment with pegcetacoplan by transitioning to an open-label extension study or Part 2C, if enrollment into the extension study was not yet available. Subjects entering Part 2C continued their pegcetacoplan dose and regimen until enrollment in the open-label extension study was available.
Part 3: Safety follow up Screening will take place within 30 days prior to the start of dosing on Day 1. If needed (see inclusion criteria), Neisseria menigitides types A, C, W, Y and B (administered as two separate vaccinations), Pneumococcal conjugate vaccine or Pneumococcal polysaccharide vaccine 23 (PCV13 or PPSV23, respectively), and Haemophilus influenzae Type B (Hib) vaccinations will be administered at least 14 days prior to dosing on Day 1.

Subjects will be entered into Part 1 of the study on Day 1 at a time designated by the PI. During Part 1, the first 3 daily SC doses of pegcetacoplan (Day 1 to 3) as well as doses on Day 8, 15 and 22 will be administered at the clinical site. From Day 4 to Day 28 daily doses of pegcetacoplan will be administered off-site by a trained study nurse at the subject's home, workplace, or other location convenient to the subject with the exception of those days where dosing is at the clinical site. Following ongoing review of available safety, PK and PD data by the investigator and sponsor, subjects showing evidence of perceived clinical benefit may progress to Part 2A and then to Part 2B of the study and continue to receive daily doses of pegcetacoplan until Day 84 and then until Day 364.

Cohort 2 will not be initiated until all subjects in Cohort 1 have reached the Day 29 visit and the SMC has reviewed emerging safety and efficacy data and determined that, at the initial dose, pegcetacoplan has an acceptable safety and tolerability profile.

The planned length of participation in the study for each subject in Cohort 2 is approximately 444 days (14.5 months) from Day 30 through completion of the Day 414 Exit visit procedures).

The study is planned to take place over approximately 24 months (from screening of Cohort 1 through completion of Cohort 2).

This time period may change in the event that the study is terminated early, additional cohorts are enrolled, additional time is required to review safety between cohorts, or extended safety and PK sampling is added for a cohort.

Conditions studied

Paroxysmal Nocturnal Hemoglobinuria

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Keywords

PNH
Paroxysmal Nocturnal Hemoglobinuria
Complement inhibitor
Anemia
Hemoglobinuria
Hemolysis
Hematologic diseases
Extravascular hemolysis (EVH)
Intravascular hemolysis (IVH)
C3 inhibitor

In context

Hemoglobinuria

107 studies on the registry are indexed under Hemoglobinuria; 10 are open to participants now.

This study's enrollment of 23 is below the median of 27 across 89 interventional studies indexed under Hemoglobinuria.

Browse Hemoglobinuria studies →

Lead sponsor

Apellis Pharmaceuticals, Inc. is the lead sponsor of 28 studies on the registry; 3 are open to participants now.

Of its 18 completed or terminated interventional studies of FDA-regulated products, 14 (78%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

Male or female
At least 18 years old (inclusive)
Weigh >55 kg and have a body mass index (BMI) ≤38.0 kg/m2
Diagnosed with PNH (white blood cell (WBC) clone >10%)
Lactose dehydrogenase (LD) ≥2 times the upper limit of normal
Last transfusion within 12 months prior to screening
Platelet count of >30,000/mm3
Absolute neutrophil count >cells/500 µL
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
Able to provide documentary evidence of Neisseria meningitidis, Pneumococcal conjugate vaccine (multivalent) or Pneumococcal polysaccharide vaccine 23 (PCV13 or PPSV23) and Haemophilus influenzae Type B (Hib) vaccination within 2 years prior to Day 1 dosing, OR willing to receive vaccinations against Neisseria meningitidis at least two weeks prior to dosing on Day 1 with a booster on Day 57, and PCV13 and Hib vaccines at least two weeks prior to dosing on Day 1.
Willing and able to give informed consent

Exclusion criteria

Exclusion Criteria:

Prior eculizumab (Soliris)® treatment
Active bacterial infection
Known infection with hepatitis B, hepatitis C or human immunodeficiency virus (HIV)
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 screening
Creatinine clearance (CrCl) \<50 mL/min (Cockcroft-Gault formula) at screening
Breast-feeding women
History of meningococcal disease

Study design

Phase: Phase 1
Primary purpose: Treatment
Allocation: Non-randomized
Intervention model: Single group
Masking: None (open label)
Enrollment: 23 participants (actual)

Study arms

Experimental
Cohort 1
180 mg pegcetacoplan/day
Drug: Pegcetacoplan
Experimental
Cohort 2
270 mg pegcetacoplan/day
Drug: Pegcetacoplan

Interventions

DrugPegcetacoplan
Complement (C3) Inhibitor
Also known as: APL-2

What researchers measure

Primary outcomes

Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity
TEAEs were defined as adverse events (AEs) that occurred after dosing on Day 1 and up to 30 days after the last dose of study drug. A treatment-related TEAE is defined as a TEAE with a relationship to study drug of probably, possibly, unlikely, or unrelated. A serious adverse event (SAE) is defined as any AE that resulted in death; was life-threatening; required hospitalization or prolongation of existing hospitalization; resulted in persistent or significant incapacity or substantial disruption of ability to conduct normal life functions; was a congenital anomaly or birth defect. TEAEs were graded according to Common Terminology Criteria for Adverse Events v4.03 based on: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening; Grade 5: Death related to AE.
Time frame: From first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days.
Mean Change From Baseline in Lactate Dehydrogenase (LDH) at Day 365
Serum chemistry assessments of LDH were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. LDH results were summarized for Cohort 2 only.
Time frame: Baseline (Day 1) and Day 365.
Mean Percentage Change From Baseline in LDH at Day 365
Serum chemistry assessments of LDH were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. LDH results were summarized for Cohort 2 only.
Time frame: Baseline (Day 1) and Day 365.
Mean Change From Baseline in Haptoglobin at Day 365
Serum chemistry assessments of haptoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Haptoglobin results were summarized for Cohort 2 only.
Time frame: Baseline (Day 1) and Day 365.
Mean Percentage Change From Baseline in Haptoglobin at Day 365
Serum chemistry assessments of haptoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Haptoglobin results were summarized for Cohort 2 only.
Time frame: Baseline (Day 1) and Day 365.
Mean Change From Baseline in Hemoglobin at Day 365
Hematology assessments of hemoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Hemoglobin results were summarized for Cohort 2 only.
Time frame: Baseline (Day 1) and Day 365.
Mean Percentage Change From Baseline in Hemoglobin at Day 365
Hematology assessments of hemoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Hemoglobin results were summarized for Cohort 2 only.
Time frame: Baseline (Day 1) and Day 365.

Secondary outcomes

Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Day 365
The FACIT-Fatigue Scale is a 13 item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). There are 13 statements with the total score ranging from 0 to 52 and higher scores indicating better quality of life. The FACIT-Fatigue Scale results were summarized for Cohort 2 only.
Time frame: Baseline (Day 1) and Day 365.
Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at Day 365
Hematology assessments of ARC were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. ARC results were summarized for Cohort 2 only.
Time frame: Baseline (Day 1) and Day 365.
Mean Percentage Change From Baseline in ARC at Day 365
Hematology assessments of ARC were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. ARC results were summarized for Cohort 2 only.
Time frame: Baseline (Day 1) and Day 365.
Mean Change From Baseline in Total Bilirubin at Day 365
Serum chemistry assessments of total bilirubin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the 3 parts of the treatment period. Total bilirubin results were summarized for Cohort 2 only.
Time frame: Baseline (Day 1) and Day 365.
Mean Percentage Change From Baseline in Total Bilirubin at Day 365
Serum chemistry assessments of total bilirubin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the 3 parts of the treatment period. Total bilirubin results were summarized for Cohort 2 only.
Time frame: Baseline (Day 1) and Day 365.
Number of Subjects Receiving Red Blood Cell (RBC) Transfusions
The number of on-study RBC transfusions were monitored throughout the treatment period.
Time frame: From Day 1 up to Day 533.

Results

Posted Jan 11, 2021

Participant flow

This Phase 1b, pilot study was conducted at 9 sites in 5 countries (Hong Kong, Malaysia, New Zealand, Thailand and United States) in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who had not received treatment with eculizumab (Soliris®) in the past, between 01 December 2015 and 26 August 2019. A total of 22 subjects were enrolled.

Part 1

Participant flow — Part 1
Milestone	Cohort 1	Cohort 2
Started	3	20
Completed	2	20
Not completed	1	0
Withdrew: Withdrawal by subject	1	0

Part 2A

Participant flow — Part 2A
Milestone	Cohort 1	Cohort 2
Started	0	20
Completed	0	18
Not completed	0	2
Withdrew: Physician decision	0	1
Withdrew: Withdrawal by subject	0	1

Part 2B

Participant flow — Part 2B
Milestone	Cohort 1	Cohort 2
Started	0	18
Completed	0	17
Not completed	0	1
Withdrew: Adverse event	0	1

Part 2C

Participant flow — Part 2C
Milestone	Cohort 1	Cohort 2
Started	0	14
Completed	0	12
Not completed	0	2
Withdrew: Adverse event	0	1
Withdrew: Withdrawal by subject	0	1

Part 3 (Safety Follow-up)

Participant flow — Part 3 (Safety Follow-up)
Milestone	Cohort 1	Cohort 2
Started	3	4
Completed	3	2
Not completed	0	2
Withdrew: Other	0	2

Outcome measures

PrimaryNumber of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity

TEAEs were defined as adverse events (AEs) that occurred after dosing on Day 1 and up to 30 days after the last dose of study drug. A treatment-related TEAE is defined as a TEAE with a relationship to study drug of probably, possibly, unlikely, or unrelated. A serious adverse event (SAE) is defined as any AE that resulted in death; was life-threatening; required hospitalization or prolongation of existing hospitalization; resulted in persistent or significant incapacity or substantial disruption of ability to conduct normal life functions; was a congenital anomaly or birth defect. TEAEs were graded according to Common Terminology Criteria for Adverse Events v4.03 based on: Grade 1: Mild; Grade 2: Moderate; Grade 3: Severe; Grade 4: Life-threatening; Grade 5: Death related to AE.

Time frame:: From first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days.
Reported as:: Count of participants · Participants

Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity
Participants	Cohort 1	Cohort 2
All TEAEs	2	18
Treatment related TEAEs	2	9
Serious AEs	1	6
TEAEs leading to study drug discontinuation	1	2
TEAEs leading to death	0	1
TEAEs with mild intensity	0	3
TEAEs with moderate intensity	2	8
TEAEs with severe intensity	0	5
TEAEs with life threatening intensity	0	1

PrimaryMean Change From Baseline in Lactate Dehydrogenase (LDH) at Day 365

Serum chemistry assessments of LDH were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. LDH results were summarized for Cohort 2 only.

Time frame:: Baseline (Day 1) and Day 365.
Reported as:: Mean · units per liter (U/L)

Mean Change From Baseline in Lactate Dehydrogenase (LDH) at Day 365
units per liter (U/L)	Cohort 2
Mean Change From Baseline in Lactate Dehydrogenase (LDH) at Day 365	-2105.2 ± 1078.79

PrimaryMean Percentage Change From Baseline in LDH at Day 365

Time frame:: Baseline (Day 1) and Day 365.
Reported as:: Mean · percent change

Mean Percentage Change From Baseline in LDH at Day 365
percent change	Cohort 2
Mean Percentage Change From Baseline in LDH at Day 365	-84.8 ± 14.04

PrimaryMean Change From Baseline in Haptoglobin at Day 365

Serum chemistry assessments of haptoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Haptoglobin results were summarized for Cohort 2 only.

Time frame:: Baseline (Day 1) and Day 365.
Reported as:: Mean · gram per liter (g/L)

Mean Change From Baseline in Haptoglobin at Day 365
gram per liter (g/L)	Cohort 2
Mean Change From Baseline in Haptoglobin at Day 365	0.066 ± 0.1245

PrimaryMean Percentage Change From Baseline in Haptoglobin at Day 365

Time frame:: Baseline (Day 1) and Day 365.
Reported as:: Mean · percent change

Mean Percentage Change From Baseline in Haptoglobin at Day 365
percent change	Cohort 2
Mean Percentage Change From Baseline in Haptoglobin at Day 365	166.176 ± 311.3656

PrimaryMean Change From Baseline in Hemoglobin at Day 365

Hematology assessments of hemoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. Hemoglobin results were summarized for Cohort 2 only.

Time frame:: Baseline (Day 1) and Day 365.
Reported as:: Mean · g/L

Mean Change From Baseline in Hemoglobin at Day 365
g/L	Cohort 2
Mean Change From Baseline in Hemoglobin at Day 365	3.68 ± 2.690

PrimaryMean Percentage Change From Baseline in Hemoglobin at Day 365

Time frame:: Baseline (Day 1) and Day 365.
Reported as:: Mean · percent change

Mean Percentage Change From Baseline in Hemoglobin at Day 365
percent change	Cohort 2
Mean Percentage Change From Baseline in Hemoglobin at Day 365	49.86 ± 43.254

SecondaryMean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Day 365

The FACIT-Fatigue Scale is a 13 item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). There are 13 statements with the total score ranging from 0 to 52 and higher scores indicating better quality of life. The FACIT-Fatigue Scale results were summarized for Cohort 2 only.

Time frame:: Baseline (Day 1) and Day 365.
Reported as:: Mean · score on a scale

Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Day 365
score on a scale	Cohort 2
Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Day 365	7.1 ± 11.09

SecondaryMean Change From Baseline in Absolute Reticulocyte Count (ARC) at Day 365

Hematology assessments of ARC were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the different parts of the treatment period. ARC results were summarized for Cohort 2 only.

Time frame:: Baseline (Day 1) and Day 365.
Reported as:: Mean · 10^9 cells/L

Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at Day 365
10^9 cells/L	Cohort 2
Mean Change From Baseline in Absolute Reticulocyte Count (ARC) at Day 365	-105.9 ± 70.28

SecondaryMean Percentage Change From Baseline in ARC at Day 365

Time frame:: Baseline (Day 1) and Day 365.
Reported as:: Mean · percent change

Mean Percentage Change From Baseline in ARC at Day 365
percent change	Cohort 2
Mean Percentage Change From Baseline in ARC at Day 365	-47.5 ± 26.86

SecondaryMean Change From Baseline in Total Bilirubin at Day 365

Serum chemistry assessments of total bilirubin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the 3 parts of the treatment period. Total bilirubin results were summarized for Cohort 2 only.

Time frame:: Baseline (Day 1) and Day 365.
Reported as:: Mean · micromole per liter (umol/L)

Mean Change From Baseline in Total Bilirubin at Day 365
micromole per liter (umol/L)	Cohort 2
Mean Change From Baseline in Total Bilirubin at Day 365	-29.9 ± 24.34

SecondaryMean Percentage Change From Baseline in Total Bilirubin at Day 365

Time frame:: Baseline (Day 1) and Day 365.
Reported as:: Mean · percent change

Mean Percentage Change From Baseline in Total Bilirubin at Day 365
percent change	Cohort 2
Mean Percentage Change From Baseline in Total Bilirubin at Day 365	-60.9 ± 19.00

SecondaryNumber of Subjects Receiving Red Blood Cell (RBC) Transfusions

The number of on-study RBC transfusions were monitored throughout the treatment period.

Time frame:: From Day 1 up to Day 533.
Reported as:: Count of participants · Participants

Number of Subjects Receiving Red Blood Cell (RBC) Transfusions
Participants	Cohort 1	Cohort 2
Number of Subjects Receiving Red Blood Cell (RBC) Transfusions	1	7

Adverse events

Collected over TEAE data is reported from first dose of study drug (Day 1) up to 30 days after the last dose of study drug, up to approximately 563 days.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Cohort 1	0/3 (0%)	1/3 (33.3%)	2/3 (66.7%)
Cohort 2	1/20 (5%)	6/20 (30%)	17/20 (85%)

Most frequent serious events

Showing 10 of 11

Most frequent serious events
Event	Cohort 1	Cohort 2
HypersensitivityImmune system disorders	1/3	0/20
HaemolysisBlood and lymphatic system disorders	0/3	2/20
Abdominal neoplasmNeoplasms benign, malignant and unspecified (incl cysts and polyps)	0/3	1/20
Aplastic anaemiaBlood and lymphatic system disorders	0/3	1/20
Intravascular haemolysisBlood and lymphatic system disorders	0/3	1/20
Acute kidney injuryRenal and urinary disorders	0/3	1/20
Joint dislocationInjury, poisoning and procedural complications	0/3	1/20
PneumonitisRespiratory, thoracic and mediastinal disorders	0/3	1/20
Paroxysmal nocturnal haemoglobinuriaBlood and lymphatic system disorders	0/3	1/20
Cholecystitis acuteHepatobiliary disorders	0/3	1/20

Most frequent other events

Showing 10 of 74

Most frequent other events
Event	Cohort 1	Cohort 2
Urinary tract infectionInfections and infestations	1/3	0/20
Injection site erythemaGeneral disorders	1/3	4/20
Upper respiratory tract infectionInfections and infestations	0/3	5/20
HypokalaemiaMetabolism and nutrition disorders	0/3	4/20
EpistaxisRespiratory, thoracic and mediastinal disorders	0/3	3/20
Electrocardiogram QT prolongedInvestigations	0/3	3/20
NeutropeniaBlood and lymphatic system disorders	0/3	3/20
PharyngitisInfections and infestations	0/3	2/20
PyrexiaGeneral disorders	0/3	2/20
Rash maculo-papularSkin and subcutaneous tissue disorders	0/3	2/20

Baseline characteristics

The Intent-To-Treat (ITT) population set included all subjects eligible to receive study drug and who received ≥1 dose of study drug. In this study, subjects could participate in more than 1 cohort. Overall, 2 cohorts of 22 unique subjects were evaluated as one subject participated in cohorts 1 and 2.

Age, Categorical
Age, Categorical(Participants)	Cohort 1	Cohort 2	Total
<=18 years	0	0	0
Between 18 and 65 years	3	19	22
>=65 years	0	1	1

Sex: Female, Male
Sex: Female, Male(Participants)	Cohort 1	Cohort 2	Total
Female	1	9	10
Male	2	11	13

Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)	Cohort 1	Cohort 2	Total
White	3	1	4
Asian	0	15	15
Native Hawaiian or Other Pacific Islander	0	1	1
Maori	0	1	1
Other	0	2	2

Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)	Cohort 1	Cohort 2	Total
Not Hispanic or Latino	3	19	22
Unknown	0	1	1

Study locations

7 sites

Prince of Wales Hospital
Hong Kong, Hong Kong
Queen Mary Hospital
Hong Kong, Hong Kong
Hospital Ampang
Ampang, Selangor 68000, Malaysia
Waikato Hospital
Hamilton, Waikato 3240, New Zealand
Ramathibodi Hospital
Bangkok, 10400, Thailand
Siriraj hospital
Bangkok, 10700, Thailand
Phramongkutklao Hospital
Bangkok, Thailand

References and documents

Publications

Wong RSM, Pullon HWH, Amine I, Bogdanovic A, Deschatelets P, Francois CG, Ignatova K, Issaragrisil S, Niparuck P, Numbenjapon T, Roman E, Sathar J, Xu R, Al-Adhami M, Tan L, Tse E, Grossi FV. Inhibition of C3 with pegcetacoplan results in normalization of hemolysis markers in paroxysmal nocturnal hemoglobinuria. Ann Hematol. 2022 Sep;101(9):1971-1986. doi: 10.1007/s00277-022-04903-x. Epub 2022 Jul 22. PubMed 35869170 ↗

Study documents

Study protocol · Oct 25, 2018
Statistical analysis plan · Dec 19, 2019

Documents are hosted by the registry — open the source record to download them.

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Jan 11, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT02588833
Lead sponsor: Apellis Pharmaceuticals, Inc.
Responsible party: Sponsor
First posted: Oct 28, 2015
Start date: Dec 1, 2015
Primary completion: Aug 26, 2019
Completion: Aug 26, 2019
Results posted: Jan 11, 2021
Last update: Jan 11, 2021

Study contacts

Federico Grossi, MD, PhD

study director · Apellis Pharmaceuticals, Inc.

Oversight

Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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