Pegcetacoplan for Thrombotic Microangiopathies

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Thrombotic Microangiopathies+1 More
Pegcetacoplan - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

The purpose of the study is to assess PK, Pharmacodynamics (PD), Efficacy and safety of pegcetacoplan in patients with TA-TMA after HSCT.

Eligible Conditions
  • Thrombotic Microangiopathies
  • Transplant-Associated Thrombotic Microangiopathy

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Thrombotic Microangiopathies

Study Objectives

4 Primary · 11 Secondary · Reporting Duration: From treatment start to end of study, an average of 6 months

From the first observed TMA response until the response criteria is no longer fulfilled or until end of study
Duration of TMA response.
From the first observed clinical response until the response criteria is no longer fulfilled or until end of study
Duration of clinical response.
Month 6
Number of participants with treatment-related adverse events.
From treatment start to first documentation of attainment of a TMA response
Time to TMA response.
From treatment start to first documentation of attainment of a clinical response
Time to clinical response.
Week 12
Number of participants reaching TMA response at week 12
Number of participants reaching clinical response at week 12
Week 24
Absolute levels, change from baseline, and percent change in sC5b-9
Number of participants reaching TMA response at week 24
Number of participants reaching clinical response at week 24
Number of participants with clinically significant changes in abnormal electrocardiogram findings.
Number of participants with clinically significant changes in vital signs.
Pegcetacoplan PK parameter AUC0-tau
Pegcetacoplan PK parameter Cmax
Pegcetacoplan PK parameter Ctrough
Pegcetacoplan PK parameter Tmax
TA-TMA relapse at Week 24.
Week 24
Overall survival
Month 6
Number of participants with antibodies to polyethylene glycol (PEG) and pegcetacoplan throughout treatment and follow-up periods.

Trial Safety

Safety Progress

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

Other trials for Thrombotic Microangiopathies

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%Oral herpes
8%Arthralgia
8%Pyrexia
8%Oropharyngeal pain
6%Injection site pruritus
6%Injection site induration
6%Pain in extremity
5%Contusion
5%Myalgia
5%Abdominal distension
5%Anxiety
5%Injection site pain
5%Acute kidney injury
5%Asthenia
4%Abdominal pain
4%Injection site bruising
4%Hypertension
4%Vomiting
4%Hyperbilirubinaemia
4%Sinusitis
4%Dizziness
4%Thrombocytopenia
4%Erythema
3%Constipation
3%Anaemia
3%Vaccination complication
3%Dyspnoea
3%Decreased appetite
3%Nausea
3%Injection site reaction
3%Gastroenteritis
3%Back pain
3%Chromaturia
1%Haemoglobinuria
1%Post procedural sepsis
1%Diverticulitis
1%Sepsis
1%Cholelithiasis
1%Hypersensitivity pneumonitis
1%Haemolytic anaemia
1%Epistaxis
1%Biliary sepsis
1%Oedematous pancreatitis
1%Injection site swelling
1%Deep vein thrombosis
1%Diffuse large B-cell lymphoma
1%Intestinal ischaemia
1%Acute myeloid leukaemia
1%Allergy to immunoglobulin therapy
1%Ovarian cyst
1%Cytopenia
1%COVID-19
1%Small intestinal obstruction
1%Haematoma muscle
1%Insomnia
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

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: from treatment start to end of study, an average of 6 months

Who is running the clinical trial?

Apellis Pharmaceuticals, Inc.Industry Sponsor
21 Previous Clinical Trials
3,504 Total Patients Enrolled
Swedish Orphan BiovitrumLead Sponsor
79 Previous Clinical Trials
11,458 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 10 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
Note: Postmenopausal is defined as having had 12 consecutive months with no menses without an alternative medical cause.
You have received allogeneic HSCT.
You have a diagnosis of TA-TMA, as per laboratory markers indicating TMA.
You have a diagnosis of TA-TMA that persists despite initial management of any triggering condition.
You have a positive rUPCR test.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 25th, 2021

Last Reviewed: October 13th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.