Pegcetacoplan for Paroxysmal Hemoglobinuria

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Radboud University Hospital Nijmegen, Nijmegen, NetherlandsParoxysmal Hemoglobinuria+1 MorePegcetacoplan - Drug
Eligibility
12 - 17
All Sexes
What conditions do you have?
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Study Summary

This trial will test the safety and effectiveness of pegcetacoplan in adolescents with PNH.

Eligible Conditions
  • Paroxysmal Nocturnal Hemoglobinuria
  • Paroxysmal Hemoglobinuria

Treatment Effectiveness

Effectiveness Progress

1 of 3

Similar Trials

1
HU-active - NDec plus NDec
1
Extension Period: AG-946 5 mg
1
Pegcetacoplan

Study Objectives

5 Primary · 17 Secondary · Reporting Duration: Week 16-52

16 weeks
Change from baseline to Wk 16 in hemoglobin (Hb)
Change from baseline to wk 16 absolute reticulocyte count (ARC)
Change from baseline to wk 16 lactate dehydrogenase (LDH)
Bacterial Infections
Pegcetacoplan serum concentrations over the course of the 16-week treatment period
48 weeks
Incidence of thromboembolic events (major adverse vascular events [MAVE]) over the course of the 16-week treatment period and over 48 weeks of treatment with pegcetacoplan
52 weeks
Incidence of thromboembolic events (major adverse vascular events [MAVE]) over the course of the 16-week treatment period and over 52 weeks of treatment with pegcetacoplan
Week 16-48
Change from baseline from week 16 to week 48 of C3 deposition on RBC cells
Change from baseline to Week 16 and to Week 48 in Health-Related Quality of Life (HRQOL) assessments
Change from baseline to Week 48, and from Week 16 to Week 48, ARC
Change from baseline to Week 48, and from Week 16 to Week 48, in Hb
Change from baseline to Week 48, and from Week 16 to Week 48, in LDH
Occurrence of breakthrough hemolysis over 16 and 48 weeks of treatment with pegcetacoplan
Total units (mL/kg) transfused over 16 and 48 weeks of treatment with pegcetacoplan
Week 16-52
Erythrocytes
Change from baseline to Week 16 and to Week 52 in Health-Related Quality of Life (HRQOL) assessments
Change from baseline to Week 52, and from Week 16 to Week 52 , in Hb
Change from baseline to Week 52, and from Week 16 to Week 52 ARC
Change from baseline to Week 52, and from Week 16 to Week 52, in LDH
Number of packed red blood cell (PRBC) units
Occurrence of breakthrough hemolysis over 16 and 52 weeks of treatment with pegcetacoplan
Total units (mL/kg) transfused over 16 and 52 weeks of treatment with pegcetacoplan

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Similar Trials

2
HU-active - NDec plus NDec
2
Extension Period: AG-946 5 mg
2
Pegcetacoplan

Side Effects for

Open-label Period: Pegcetacoplan
17%Haemolysis
16%Nasopharyngitis
14%Diarrhoea
12%Injection site erythema
12%Cough
10%Fatigue
10%Headache
9%Urinary tract infection
9%Upper respiratory tract infection
8%Pyrexia
8%Arthralgia
8%Oral herpes
8%Oropharyngeal pain
6%Injection site pruritus
6%Injection site induration
6%Pain in extremity
5%Asthenia
5%Acute kidney injury
5%Anxiety
5%Abdominal distension
5%Injection site pain
5%Contusion
5%Myalgia
4%Thrombocytopenia
4%Hypertension
4%Injection site bruising
4%Dizziness
4%Abdominal pain
4%Vomiting
4%Hyperbilirubinaemia
4%Erythema
4%Sinusitis
3%Anaemia
3%Gastroenteritis
3%Injection site reaction
3%Dyspnoea
3%Nausea
3%Constipation
3%Chromaturia
3%Vaccination complication
3%Back pain
3%Decreased appetite
1%Cytopenia
1%Insomnia
1%Haematoma muscle
1%Oedematous pancreatitis
1%Intestinal ischaemia
1%Haemolytic anaemia
1%Deep vein thrombosis
1%Acute myeloid leukaemia
1%Diffuse large B-cell lymphoma
1%Allergy to immunoglobulin therapy
1%Ovarian cyst
1%Epistaxis
1%Small intestinal obstruction
1%COVID-19
1%Biliary sepsis
1%Diverticulitis
1%Injection site swelling
1%Haemoglobinuria
1%Hypersensitivity pneumonitis
1%Cholelithiasis
1%Post procedural sepsis
1%Sepsis
1%Palpitations
This histogram enumerates side effects from a completed 2020 Phase 3 trial (NCT03500549) in the Open-label Period: Pegcetacoplan ARM group. Side effects include: Haemolysis with 17%, Nasopharyngitis with 16%, Diarrhoea with 14%, Injection site erythema with 12%, Cough with 12%.

Trial Design

1 Treatment Group

Pegcetacoplan
1 of 1

Experimental Treatment

12 Total Participants · 1 Treatment Group

Primary Treatment: Pegcetacoplan · No Placebo Group · Phase 2

Pegcetacoplan
Drug
Experimental Group · 1 Intervention: Pegcetacoplan · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Pegcetacoplan
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: week 16-52

Who is running the clinical trial?

Apellis Pharmaceuticals, Inc.Lead Sponsor
21 Previous Clinical Trials
3,504 Total Patients Enrolled

Eligibility Criteria

Age 12 - 17 · All Participants · 5 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
References

Frequently Asked Questions

Are there other ongoing or completed studies using Pegcetacoplan?

"11 different clinical trials are underway to research the efficacy of Pegcetacoplan. Of these, 5 are in Phase 3. The locations for these trials span from California to London, with a total of 593 sites running studies." - Anonymous Online Contributor

Unverified Answer

Does this research stand alone or has it been done before?

"As of right now, there are 11 ongoing Pegcetacoplan studies in 164 different cities and 26 countries. The first clinical trial for this medication was back in 2018 and it completed Phase 2 drug approval that year. Since then, 8 more trials have been conducted." - Anonymous Online Contributor

Unverified Answer

Has the FDA cleared Pegcetacoplan for use?

"Pegcetacoplan has received a 2 for safety. This is due to the lack of efficacy data, as this is only a Phase 2 trial." - Anonymous Online Contributor

Unverified Answer

How many volunteers are enrolling in this clinical trial?

"The answer is affirmative. According to the information available on clinicaltrials.gov, this particular clinical trial is still recruiting participants. The trial was originally posted on February 4th, 2021 and was updated for the last time on July 18th, 2022. They are looking for a total of 12 participants from 1 site." - Anonymous Online Contributor

Unverified Answer

Are people still being considered for participation in this trial?

"Yes, you're right. The website says that the trial is still looking for patients. They began recruiting on February 4th 2021 and the listing was updated last on July 18th, 2022. So far, 1 site has enrolled 12 people but they need more participants." - Anonymous Online Contributor

Unverified Answer

Does this trial have an age limit?

"This study is enrolling adolescent patients who are between 12 and 17 years old." - Anonymous Online Contributor

Unverified Answer

Could I potentially qualify to participate in this research?

"The inclusion criteria for this clinical trial requires that patients have hemoglobinuria, paroxysmal and are between the ages of 12-17. The study is looking to enroll a total of 12 participants." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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