Transplant-Associated Thrombotic MicroangiopathyPhase 2NCT05148299

A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation

A Phase 2 study testing Pegcetacoplan in people with Transplant-Associated Thrombotic Microangiopathy, run by Swedish Orphan Biovitrum at 14 sites in 5 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The purpose of the study was to assess pharmacokinetics (PK), pharmacodynamics (PD), efficacy, and safety of pegcetacoplan in patients with TA-TMA after HSCT. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Transplant-Associated Thrombotic Microangiopathy
What is being tested?: Pegcetacoplan
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 12 participants
Where: 14 sitesin 5 countries
Study dates: 1 Feb 2022to 8 Dec 2024
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Male and female patients aged ≥ 18 years at the time of informed consent form (ICF) signature.
Received allogeneic HSCT.
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.
Have random urine protein/creatinine ratio (rUPCR) ≥ 1 mg/mg.
Women of childbearing potential, defined as any women who have experienced menarche and who are NOT permanently sterile or postmenopausal, must have a negative serum pregnancy test at screening and agree to use protocol-defined methods of contraception for the duration of the study and 8 weeks after their last investigational medicinal product (IMP) dose.

Show all 12Show fewer

Note: Postmenopausal is defined as having had 12 consecutive months with no menses without an alternative medical cause.
Men must agree to the following for the duration of the study and 8 weeks after their last dose of IMP:
Avoid fathering a child.
Use protocol-defined methods of contraception.
Refrain from donating sperm.
Patient and/or legally authorized representative must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF.

Who cannot join

Positive direct Coombs test.
Known familial or acquired ADAMTS13 deficiency.
Known Shiga toxin-related hemolytic uremic syndrome.
Known bone marrow or graft failure.
Diagnosis of disseminated intravascular coagulation.
Diagnosis of veno-occlusive disease (VOD).

Show all 17Show fewer

Active GI bleeding (hematemesis or hematochezia) at baseline.
Body weight < 30 kg and > 100 kg.
Uncontrolled systemic bacterial or fungal infection, presence or suspicion of sepsis.
Previously or currently treated with a complement inhibitor (approved or investigational).
Pregnancy or breastfeeding.
Positive human immunodeficiency virus antibody at screening or documented in pre-HSCT medical record.
Hepatitis C virus detectable by polymerase chain reaction at screening or documented in pre-HSCT medical record.
Chronic inactive hepatitis B virus with viral loads > 1000 IU/mL (> 5000 copies/mL) at screening or documented in pre-HSCT medical record. Eligible patients who are chronic active carriers (≤ 1000 IU/mL) must receive prophylactic antiviral treatment (e.g., entecavir, tenofovir, lamivudine) according to local country guidelines.
Known or suspected hereditary fructose intolerance.
Hypersensitivity to pegcetacoplan or any of its excipients.
Inability to cooperate with study procedures or any condition that, in the opinion of the investigator, could increase the patient's risk by participating in the study or confound the outcome of the study.

Trial locations and contacts

14 sites in 5 countries.

Country

City, postcode or hospital

14 sites in 5 countries

Italy5 sites

Hospital San Giuseppe Moscati
Avellino, 83100
Big Metropolitan Hospital Niguarda Regional Health Authority
Milan, 20162
ASST Monza - S. Gerardo Hospital
Monza, 20900
United Hospitals Villa Sofia Cervello
Palermo, 90146
University Polyclinic Foundation "Agostino Gemelli" - IRCCS
Roma, 00168

Other trials in Italy

France3 sites

Archet 1 hospital, Department of Clinical Hematology
Nice, 06200
Saint-Louis Hospital
Paris, Paris, 75010
CHU de Saint-Etienne
Saint-Priest-en-Jarez, 42270

Other trials in France

Greece3 sites

University General Hospital "Attikon"
Athens, Chaidari, 12462
General Hospital of Athens "Evangelismos"
Athens, 10676
General Hospital of Thessaloniki "G. Papanikolaou", Hematology Department - BMT Unit
Thessaloniki, 57010

Other trials in Greece

United States2 sites

City of Hope
Duarte, California, 91010
Mayo Clinic - Rochester
Rochester, Minnesota, 55905

Other trials in United States

Spain1 site

University Hospital Puerta de Hierro Majadahonda
Madrid, 28222

Other trials in Spain

About this study

Official title: An Open-label, Single-arm, Multicenter Pilot Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy and Safety of Pegcetacoplan in Patients With Transplant-associated Thrombotic Microangiopathy (TA-TMA) After Hematopoietic Stem Cell Transplantation (HSCT)

The purpose of the study was to assess pharmacokinetics (PK), pharmacodynamics (PD), efficacy, and safety of pegcetacoplan in patients with TA-TMA after HSCT.

Read the detailed description

This was a pilot study, and the sample size was based on practical rather than statistical aspects.

A total of 12 patients were to be included and treated in the study. With 12 patients included, it was estimated that 9 patients would complete at least 4 weeks of treatment, which is deemed sufficient to characterize the PK of pegcetacoplan in patients with TA-TMA to an appropriate precision. In addition, 12 patients would provide a 72 % probability to observe a response rate of at least 8 responders of the 12 patients recruited (assuming the true response rate is 70 %).

Conditions: Transplant-Associated Thrombotic Microangiopathy
Being tested: Pegcetacoplan
Keywords: TA-TMA

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

PegcetacoplanDrug
20-cc glass vials-1080 mg

Study groups in NCT05148299
Study group	Role	What they receive
Pegcetacoplan	Experimental	sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks. Receives: Drug: Pegcetacoplan

What is being measured

The primary outcome is the main question the study is designed to answer.

Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau)
Area under the concentration-time curve limited to the end of the dosing interval. The samples included in the calculation of AUC0-tau were collected at the following times: on dosing Days 1, 3 and 5, PK samples were taken up to 30 minutes pre-dose and at 15 minutes (± 5 min), 30 minutes (± 5 min), 1 hour (± 10 min), 4 hours (± 10 min), 8 hours (± 30 min), and 24 hours (± 30 min) post-dose as well as on Day 8 pre-dose.
Measured: Week 1
Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax)
Maximum observed serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5.
Measured: Week 1
Pegcetacoplan PK Parameter Time to Cmax (Tmax)
Time of maximum measured serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5.
Measured: Week 1
Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough)
Observed serum concentration pre-dose. From Day 8 (Week 1) and onwards, PK samples were taken pre-dose at each visit.
Measured: Week 1 up to Week 14

Secondary outcomes (18)

Absolute Levels and Change From Baseline in sC5b-9
Absolute levels and change from baseline to Week 24 in biomarker of complement activation sC5b-9
Measured: Week 24
Absolute Levels and Change From Baseline in C3a
Absolute levels and change from baseline to Week 24 in biomarker of complement activation C3a
Measured: Week 24
Absolute Levels and Change From Baseline in C3
Absolute levels and change from baseline to Week 24 in biomarker of complement activation C3
Measured: Week 24
Absolute Levels and Change From Baseline in Bb
Absolute levels and change from baseline to Week 24 in biomarker of complement activation Bb
Measured: Week 24
Absolute Levels and Change From Baseline in C4a
Absolute levels and change from baseline to Week 24 in biomarker of complement activation C4a
Measured: Week 24
Absolute Levels and Change From Baseline in Classical Pathway (CH50)
Absolute levels and change from baseline to Week 24 in biomarker of complement activation CH50
Measured: Week 24
Absolute Levels and Change From Baseline in Alternative Pathway (AH50)
Absolute levels and change from baseline to Week 24 in biomarker of complement activation AH50
Measured: Week 24
Number of Participants Reaching Clinical Response at Week 24
A participant will be declared as reaching a clinical response upon improvement in laboratory markers of TMA and resolution of TMA clinical symptoms
Measured: Week 24
Number of Participants Reaching TMA Response at Week 24
A participant will be declared as reaching TMA response upon improvement in laboratory markers of TMA
Measured: Week 24
Overall Survival at Day 100
Survival
Measured: Day 100 from diagnosis
Overall Survival at Week 24
Survival
Measured: Week 24 from treatment start
Time to Clinical Response
Both clinical response sustained at week 24 and clinical response at any time during the study will be assessed.
Measured: From treatment start to first documentation of attainment of a clinical response, up to 24 weeks
Time to TMA Response
Both TMA response sustained at week 24 and TMA response at any time during the study will be assessed.
Measured: From treatment start to first documentation of attainment of a TMA response, up to 24 weeks
Duration of Clinical Response
Duration of clinical response sustained at week 24
Measured: From the first observed clinical response until the response criteria is no longer fulfilled or until end of study, up to 24 weeks
Duration of TMA Response
Duration of TMA response sustained at week 24
Measured: From the first observed TMA response until the response criteria is no longer fulfilled or until end of study, up to 24 weeks
TA-TMA Relapse at Week 24
A participant will be declared as relapsing upon appearance of laboratory markers of TMA
Measured: Week 24
Number of Participants Reaching Clinical Response at Week 12
Number of participants reaching clinical response at week 12
Measured: Week 12
Number of Participants Reaching TMA Response at Week 12
Number of participants reaching TMA response at week 12
Measured: Week 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 Feb 20228 Dec 2024

First posted8 Dec 2021
Study start1 Feb 2022
Primary completion8 Dec 2024
Study completion8 Dec 2024

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Transplant-Associated Thrombotic MicroangiopathyStudies of the same condition, open and under way.
More trials run by Swedish Orphan BiovitrumThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Model description: open label, single arm, multicenter
Masking: None (open label)
Enrolment: 12 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05148299
Sponsor's protocol ID: Sobi.PEGCET-201
Other IDs: 2023-510443-37-00, 2021-003157-27, 2023
Status last verified: Sep 2025
First submitted: 21 Oct 2021
Last updated: 28 Nov 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Recruiting
The Role of interferOn and Complement in SecondAry thRombotic micrioangiOpathy
Thrombotic Microangiopathies
Not yet recruitingObservational study
Assessing the Incidence of Transplant Associated Thrombotic Microangiopathy (TA-TMA) in Adult Patients Undergoing Allogeneic Stem Cell Transplant (SCT)
Transplant Associated Microangiopathy TAM, Transplant Complication
Recruiting
Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy
Thrombotic Microangiopathy, Hematopoietic Stem Cell Transplantation (HSCT)
RecruitingPhase 2
Safety and Efficacy of Eculizumab in High-risk TA-TMA
Transplant-Associated Thrombotic Microangiopathy (TA-TMA)
Active, not recruitingPhase 2/3
N-Acetylcysteine as Therapy for Transplantation- Associated Thrombotic Microangiopathy
Thrombotic Microangiopathy

All Transplant-Associated Thrombotic Microangiopathy trials

Study at a glance

From the registry record, last updated by the sponsor on 28 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-label, Single-arm, Multicenter Pilot Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy and Safety of Pegcetacoplan in Patients With Transplant-associated Thrombotic Microangiopathy (TA-TMA) After Hematopoietic Stem Cell Transplantation (HSCT)
ClinicalTrials.gov ID: NCT05148299
Acronym / sponsor ID: Sobi.PEGCET-201
Conditions: Transplant-Associated Thrombotic Microangiopathy
Keywords: TA-TMA
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 12 participants (actual)
Lead sponsor: Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
Collaborators: Apellis Pharmaceuticals, Inc.
Study start: 1 Feb 2022 (actual)
Primary completion: 8 Dec 2024 (actual)
Study completion: 8 Dec 2024 (actual)
First posted: 8 Dec 2021 (actual)
Results: Posted 28 Nov 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The purpose of the study was to assess pharmacokinetics (PK), pharmacodynamics (PD), efficacy, and safety of pegcetacoplan in patients with TA-TMA after HSCT.

Detailed description

This was a pilot study, and the sample size was based on practical rather than statistical aspects.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: open label, single arm, multicenter

Arm	Type	Receives
Pegcetacoplan	Experimental	Drug: Pegcetacoplansterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (12 listed)

Male and female patients aged ≥ 18 years at the time of informed consent form (ICF) signature.
Received allogeneic HSCT.
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.
Have random urine protein/creatinine ratio (rUPCR) ≥ 1 mg/mg.

Key exclusion criteria (17 listed)

Positive direct Coombs test.
Known familial or acquired ADAMTS13 deficiency.
Known Shiga toxin-related hemolytic uremic syndrome.
Known bone marrow or graft failure.
Diagnosis of disseminated intravascular coagulation.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Pegcetacoplan20-cc glass vials-1080 mg	Drug	Pegcetacoplan

Primary outcome

Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau)
Time frame: Week 1
Area under the concentration-time curve limited to the end of the dosing interval. The samples included in the calculation of AUC0-tau were collected at the following times: on dosing Days 1, 3 and 5, PK samples were taken up to 30 minutes pre-dose and at 15 minutes (± 5 min), 30 minutes (± 5 min), 1 hour (± 10 min), 4 hours (± 10 min), 8 hours (± 30 min), and 24 hours (± 30 min) post-dose as well as on Day 8 pre-dose.
Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax)
Time frame: Week 1
Maximum observed serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5.
Pegcetacoplan PK Parameter Time to Cmax (Tmax)
Time frame: Week 1
Time of maximum measured serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5.
Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough)
Time frame: Week 1 up to Week 14
Observed serum concentration pre-dose. From Day 8 (Week 1) and onwards, PK samples were taken pre-dose at each visit.

18 secondary outcomes

Absolute Levels and Change From Baseline in sC5b-9
Time frame: Week 24
Absolute Levels and Change From Baseline in C3a
Time frame: Week 24
Absolute Levels and Change From Baseline in C3
Time frame: Week 24
Absolute Levels and Change From Baseline in Bb
Time frame: Week 24
Absolute Levels and Change From Baseline in C4a
Time frame: Week 24
Absolute Levels and Change From Baseline in Classical Pathway (CH50)
Time frame: Week 24
Absolute Levels and Change From Baseline in Alternative Pathway (AH50)
Time frame: Week 24
Number of Participants Reaching Clinical Response at Week 24
Time frame: Week 24
Number of Participants Reaching TMA Response at Week 24
Time frame: Week 24
Overall Survival at Day 100
Time frame: Day 100 from diagnosis
Overall Survival at Week 24
Time frame: Week 24 from treatment start
Time to Clinical Response
Time frame: From treatment start to first documentation of attainment of a clinical response, up to 24 weeks
Time to TMA Response
Time frame: From treatment start to first documentation of attainment of a TMA response, up to 24 weeks
Duration of Clinical Response
Time frame: From the first observed clinical response until the response criteria is no longer fulfilled or until end of study, up to 24 weeks
Duration of TMA Response
Time frame: From the first observed TMA response until the response criteria is no longer fulfilled or until end of study, up to 24 weeks
TA-TMA Relapse at Week 24
Time frame: Week 24
Number of Participants Reaching Clinical Response at Week 12
Time frame: Week 12
Number of Participants Reaching TMA Response at Week 12
Time frame: Week 12

Results and safety

Results posted to the registry on 28 Nov 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Pegcetacoplan	12	9

Primary outcome: Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau)

Time frame: Week 1. Mean (Standard Deviation). Unit: µg*h/mL

Measure	Pegcetacoplan n = 12
Day 1-Day 3	14934.65 (4448.067)
Day 3-Day 5	20089.79 (5162.653)
Day 5-Day 8	36208.37 (10685.626)

Population analysed: On Day 1 and 5 the AUC0-tau parameters did not meet lambda-z acceptance criteria for 2 patients, so they were not included. On Day 8 the AUC0-tau parameter did not meet lambda-z acceptance criteria for 1 patient, so they were not included.

Primary outcome: Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax)

Time frame: Week 1. Mean (Standard Deviation). Unit: µg/mL

Measure	Pegcetacoplan n = 12
Day 1	508.1 (134.86)
Day 3	579.6 (142.11)
Day 5	705.2 (203.04)

Population analysed: On Day 1, Cmax could not be estimated for 2 patients due to a) no sample provided; and b) Cmax considered unreliable due to pegcetacoplan positive pre-dose sample. On Day 3 and Day 5, Cmax could not be estimated for 1 patient due to a limited number of samples provided.

Primary outcome: Pegcetacoplan PK Parameter Time to Cmax (Tmax)

Time frame: Week 1. Median (Full Range). Unit: Hour

Measure	Pegcetacoplan n = 12
Day 1	1.0 (0.3 to 48.0)
Day 3	1.02 (0.3 to 8.5)
Day 5	1.05 (0.5 to 24.0)

Population analysed: On Day 1, Tmax could not be estimated for 2 patients due to a) no sample provided; and b) Tmax considered unreliable due to pegcetacoplan positive pre-dose sample. On Day 3 and Day 5, Tmax could not be estimated for one patient due to a limited number of samples provided.

Primary outcome: Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough)

Time frame: Week 1 up to Week 14. Geometric mean (95% Confidence Interval). Unit: µg/mL

Measure	Pegcetacoplan n = 12
Week 1	365.22 (308.37 to 432.55)
Week 2	449.86 (344.63 to 587.23)
Week 3	585.71 (456.62 to 751.30)
Week 4	665.32 (501.51 to 882.64)
Week 6	750.82 (602.41 to 935.80)
Week 8	826.94 (655.21 to 1043.69)
Week 10	761.76 (569.21 to 1019.44)
Week 12	466.29 (112.54 to 1931.99)

Population analysed: The number of patients in the analysis population for Ctrough decreased over the course of the study, from 11 at Week 1 to 7 at Week 14, reflecting the limited number of samples available due to study discontinuation of patients.

18 secondary outcomes are reported in the full results.

Adverse events

Time frame: From the first dose of IMP and up to 8 weeks after the date of administration of the last dose of IMP, up to 24 weeks. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Pegcetacoplan	8 of 12 (66.7%)	5 of 12 (41.7%)	6 of 12

Limitations and caveats (from the sponsor): The small sample size limits the generalizability of the findings.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 28 Nov 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

Related trials for Transplant-Associated Thrombotic Microangiopathy

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Assessing the Incidence of Transplant Associated Thrombotic Microangiopathy (TA-TMA) in Adult Patients Undergoing Allogeneic Stem Cell Transplant (SCT)
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Study physician study director

Swedish Orphan Biovitrum

14 sites in 5 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Pegcetacoplan in people with Transplant-Associated Thrombotic Microangiopathy, run by Swedish Orphan Biovitrum at 14 sites in 5 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 28 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 14 sites in Italy, France, Greece, United States, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 28 Nov 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau) (time frame: Week 1). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05148299.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Nov 2025.