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Complement Inhibitor
Pegcetacoplan for TA-TMA After Stem Cell Transplant
Phase 2
Recruiting
Research Sponsored by Swedish Orphan Biovitrum
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Diagnosis of TA-TMA established, as per laboratory markers indicating TMA
Have a diagnosis of TA-TMA that persists despite initial management of any triggering condition
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from treatment start to end of study, an average of 6 months
Awards & highlights
Study Summary
This trialwill study how a new drug affects people with a rare blood disorder after a stem cell transplant.
Who is the study for?
Adults over 18 with TA-TMA after a stem cell transplant can join. They must have specific lab markers for TMA, agree to use contraception, and not be pregnant or fathering children. Excluded are those with certain blood disorders, active infections, known genetic conditions affecting the blood, uncontrolled bleeding or weight outside 30-100 kg range.Check my eligibility
What is being tested?
The trial is testing Pegcetacoplan's effects on patients with TA-TMA post-stem cell transplant. It looks at how the body processes it (PK), its impact on disease (PD), effectiveness in treating symptoms of TMA and overall safety.See study design
What are the potential side effects?
Possible side effects of Pegcetacoplan include allergic reactions to ingredients, potential increased risk of infections due to immune system changes, and other unspecified impacts that will be monitored throughout the study.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My tests show I have thrombotic microangiopathy.
Select...
My TA-TMA condition persists despite initial treatments.
Select...
I am not pregnant and agree to use birth control as required by the study.
Select...
I have received a stem cell transplant from a donor.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ from treatment start to end of study, an average of 6 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from treatment start to end of study, an average of 6 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Pegcetacoplan PK parameter AUC0-tau
Pegcetacoplan PK parameter Cmax
Pegcetacoplan PK parameter Ctrough
+1 moreSecondary outcome measures
Absolute levels, change from baseline, and percent change in sC5b-9
Duration of TMA response.
Duration of clinical response.
+8 moreOther outcome measures
Number of participants with antibodies to polyethylene glycol (PEG) and pegcetacoplan throughout treatment and follow-up periods.
Number of participants with clinically significant changes in abnormal electrocardiogram findings.
Number of participants with clinically significant changes in vital signs.
+1 moreSide effects data
From 2020 Phase 3 trial • 80 Patients • NCT0350054917%
Haemolysis
16%
Nasopharyngitis
14%
Diarrhoea
12%
Injection site erythema
12%
Cough
10%
Headache
10%
Fatigue
9%
Urinary tract infection
9%
Upper respiratory tract infection
8%
Oral herpes
8%
Pyrexia
8%
Oropharyngeal pain
8%
Arthralgia
6%
Injection site induration
6%
Injection site pruritus
6%
Pain in extremity
5%
Myalgia
5%
Asthenia
5%
Injection site pain
5%
Anxiety
5%
Abdominal distension
5%
Acute kidney injury
5%
Contusion
4%
Dizziness
4%
Vomiting
4%
Hyperbilirubinaemia
4%
Injection site bruising
4%
Hypertension
4%
Thrombocytopenia
4%
Abdominal pain
4%
Erythema
4%
Sinusitis
3%
Chromaturia
3%
Injection site reaction
3%
Nausea
3%
Gastroenteritis
3%
Vaccination complication
3%
Anaemia
3%
Dyspnoea
3%
Constipation
3%
Back pain
3%
Decreased appetite
1%
Injection site swelling
1%
Intestinal ischaemia
1%
Allergy to immunoglobulin therapy
1%
Haemolytic anaemia
1%
Cholelithiasis
1%
Haematoma muscle
1%
COVID-19
1%
Insomnia
1%
Haemoglobinuria
1%
Cytopenia
1%
Small intestinal obstruction
1%
Epistaxis
1%
Ovarian cyst
1%
Hypersensitivity pneumonitis
1%
Oedematous pancreatitis
1%
Biliary sepsis
1%
Diverticulitis
1%
Post procedural sepsis
1%
Sepsis
1%
Palpitations
1%
Diffuse large B-cell lymphoma
1%
Deep vein thrombosis
1%
Acute myeloid leukaemia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Open-label Period: Pegcetacoplan
Run-in Period: Pegcetacoplan + Eculizumab
RCP: Eculizumab
RCP: Pegcetacoplan
Trial Design
1Treatment groups
Experimental Treatment
Group I: PegcetacoplanExperimental Treatment1 Intervention
sterile solution in stoppered glass vial given as 1080 mg infusion 3 times weekly for 12 weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Pegcetacoplan
2015
Completed Phase 3
~380
Find a Location
Who is running the clinical trial?
Swedish Orphan BiovitrumLead Sponsor
92 Previous Clinical Trials
12,502 Total Patients Enrolled
Apellis Pharmaceuticals, Inc.Industry Sponsor
24 Previous Clinical Trials
4,013 Total Patients Enrolled
Luis López LazaroStudy DirectorSwedish Orphan Biovitrum AB
1 Previous Clinical Trials
57 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My tests show I have thrombotic microangiopathy.My TA-TMA condition persists despite initial treatments.I agree to use contraception and not father a child during the study.I have or might have hereditary fructose intolerance.I am not currently experiencing any active bleeding from my stomach or intestines.I have a known ADAMTS13 deficiency.I am not pregnant and agree to use birth control as required by the study.I can sign and follow the study's consent form.I have been treated with a drug for my immune system.I have been diagnosed with veno-occlusive disease.My body weight is either below 30 kg or above 100 kg.I have been diagnosed with a condition related to Shiga toxin affecting my kidneys.I have been diagnosed with bone marrow failure or a failed bone marrow transplant.I have been diagnosed with a blood clotting disorder.I am 18 years or older.I have received a stem cell transplant from a donor.I have chronic inactive hepatitis B with a high viral load.I do not have an uncontrolled infection or sepsis.
Research Study Groups:
This trial has the following groups:- Group 1: Pegcetacoplan
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any noteworthy precedents involving Pegcetacoplan's use in scientific research?
"Currently, 10 clinical trials are researching Pegcetacoplan with 5 of those in the final phase. Although most studies for this treatment occur within London and California, there is a total of 576 sites conducting research on it."
Answered by AI
How many participants have signed up to take part in this clinical investigation?
"Affirmative. Data provided by clinicaltrials.gov implies that this medical investigation is presently recruiting patients; first posted on February 1st 2022 and most recently revised on October 19th, it seeks to enrol 12 volunteers at 6 distinct sites."
Answered by AI
Is there currently an open enrollment for this research endeavor?
"Affirmative. According to the information found on clinicaltrials.gov, this medical experiment is actively recruiting patients; it was first posted in February 2022 and updated most recently in October 2022. The study requires 12 participants from 6 separate sites."
Answered by AI
How many sites are currently managing this research project?
"This medical study is welcoming patients at Ryotaro Nakamura, MD in Duarte, California, Clinical Trial Site in Rochester, Minnesota and Hassan AlKhateeb, MD in Augusta Georgia. As well as four additional clinical trial centres."
Answered by AI
Has the Federal Drug Administration sanctioned Pegcetacoplan for medical use?
"Due to a lack of efficacy data, the safety rating for Pegcetacoplan is 2. This score stems from this being Phase 2 trial and hence having only some evidence that it can be safely used in patients."
Answered by AI
Is this an unprecedented clinical trial?
"As of now, 10 trials for Pegcetacoplan are taking place in 149 cities and 26 countries. This medication was first approved for testing back in 2018 by Apellis Pharmaceuticals, Inc.. The Phase 3 study included 600 participants with 8 subsequent studies concluding since then."
Answered by AI
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