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Study to evaluate Pegcetacoplan in patients with TA-TMA after hematopoietic stem cell transplantation
EU CTIS ID: 2023-510443-37-00
What this study is testing
To evaluate the pharmacokinetics (PK), safety and tolerability of pegcetacoplan in patients with TA-TMA.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male and female patients aged ≥ 18 years at the time of informed consent form (ICF) signature.
- Received allogeneic HSCT from a related or unrelated, human leukocyte antigen-matched or mismatched donor. Patients having received any of the following stem cell sources are eligible: granulocyte colony stimulating factor mobilized peripheral blood stem cells, bone marrow, umbilical cord blood.
- Diagnosis of TA-TMA established as per the laboratory markers below, indicating TMA: a. De novo or progressing thrombocytopenia (platelet count < 50 x 109 /L or > 50 % decrease in platelet count from the highest value achieved after transplantation). AND b. Elevated LDH (> 1.5 x ULN). AND at least 2 additional laboratory/clinical criteria among the following: c. Presence of schistocytes on the peripheral blood smear (≥ 2 per hpf) or histologic evidence of microangiopathy in any biopsied organ. OR d. De novo anemia (hemoglobin < LLN or anemia requiring PRBC transfusion support as per local institutional standard). OR e. Proteinuria (random urinalysis protein concentration ≥ 30 mg/dL). OR f. Elevated plasma concentration of sC5b-9 above ULN. OR g. Arterial hypertension, defined by systolic blood pressure (BP) ≥ 140 mmHg and/or diastolic BP ≥ 90 mmHg.
- Have a diagnosis of TA-TMA that persists despite initial management of any triggering condition.
- Have 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 IMP dose. Note: Postmenopausal is defined as having had 12 consecutive months with no menses without an alternative medical cause.
You likely can't join if
- Positive direct Coombs test.
- 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
See the full eligibility criteria
Who can join
- Male and female patients aged ≥ 18 years at the time of informed consent form (ICF) signature.
- Received allogeneic HSCT from a related or unrelated, human leukocyte antigen-matched or mismatched donor. Patients having received any of the following stem cell sources are eligible: granulocyte colony stimulating factor mobilized peripheral blood stem cells, bone marrow, umbilical cord blood.
- Diagnosis of TA-TMA established as per the laboratory markers below, indicating TMA: a. De novo or progressing thrombocytopenia (platelet count < 50 x 109 /L or > 50 % decrease in platelet count from the highest value achieved after transplantation). AND b. Elevated LDH (> 1.5 x ULN). AND at least 2 additional laboratory/clinical criteria among the following: c. Presence of schistocytes on the peripheral blood smear (≥ 2 per hpf) or histologic evidence of microangiopathy in any biopsied organ. OR d. De novo anemia (hemoglobin < LLN or anemia requiring PRBC transfusion support as per local institutional standard). OR e. Proteinuria (random urinalysis protein concentration ≥ 30 mg/dL). OR f. Elevated plasma concentration of sC5b-9 above ULN. OR g. Arterial hypertension, defined by systolic blood pressure (BP) ≥ 140 mmHg and/or diastolic BP ≥ 90 mmHg.
- Have a diagnosis of TA-TMA that persists despite initial management of any triggering condition.
- Have 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 IMP dose. 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: a. Avoid fathering a child. b. Use protocol-defined methods of contraception. c. 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.
What rules you out
- Positive direct Coombs test.
- 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.
- 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).
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.