CompletedPhase 2

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

United States, France, Greece12 participantsStarted 2022-02-01

Plain-language 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.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. 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.
. 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.

Exclusion criteria

. Positive direct Coombs test.
. Known familial or acquired ADAMTS13 deficiency.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Timeframe: Week 1

Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax)

Timeframe: Week 1

Pegcetacoplan PK Parameter Time to Cmax (Tmax)

Timeframe: Week 1

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

Timeframe: Week 1 up to Week 14

Trial details

NCT IDNCT05148299

SponsorSwedish Orphan Biovitrum

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-12-08

Results submitted2025-09-29

View on ClinicalTrials.gov More Transplant-Associated Thrombotic Microangiopathy trials

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. 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.
. 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.

Exclusion criteria

. Positive direct Coombs test.
. Known familial or acquired ADAMTS13 deficiency.

What they're measuring

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

Timeframe: Week 1

Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax)

Timeframe: Week 1

Pegcetacoplan PK Parameter Time to Cmax (Tmax)

Timeframe: Week 1

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

Timeframe: Week 1 up to Week 14