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Trial · NCT03205995

Outcomes16

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

TypeOutcomeTime framePopulationUnitsMeasured as
PRIMARYPlatelet Count (10^9 Platelets/L) Change From Baseline at Week 26Week 26Any patent who received drug10^9 platelets/LMEDIAN
SECONDARYSafety as Measured by Incidences of Adverse Events, Vital Signs, ECG, and Clinical Laboratory TestsPre-dose and up to 771 days post-doseThe safety population includes all participants who received any amount of study drug.ParticipantsCOUNT_OF_PARTICIPANTS
SECONDARYThrombotic Microangiopathies (TMA) Response26 weeksAny patient who received drugParticipantsCOUNT_OF_PARTICIPANTS
SECONDARYTMA Event-free Status26 weeksAny patient who received drugParticipantsCOUNT_OF_PARTICIPANTS
SECONDARYIncrease in Estimated Glomerular Filtration Rate (eGFR)26 weeksAny patient who received drugParticipantsCOUNT_OF_PARTICIPANTS
SECONDARYHematological Normalization26 weeksAny patient who received drugParticipantsCOUNT_OF_PARTICIPANTS
SECONDARYTMA Remission26 weeksAny patient who received drugParticipantsCOUNT_OF_PARTICIPANTS
SECONDARYIncidence of Antidrug Antibodies (ADA)771 days post-doseAny patient who received drugParticipantsCOUNT_OF_PARTICIPANTS
SECONDARYChange From Baseline in Serum Creatinine (mg/dL)26 weeksAny patient who received drugmg/dLMEDIAN
SECONDARYChange From Baseline in Serum LDH (U/L)26 weeksAny patient who received drug.U/LMEDIAN
SECONDARYChange From Baseline in Haptoglobin (mg/dL)26 weeksAny patient who received drug.mg/dLMEDIAN
SECONDARYPharmacokinetics (PK): Trough Plasma Concentration, Lower Limit of Quantification (LLOQ)Days 1-4; Treatment Maintenance (103 weeks): 17 visits; Rescue Therapy (RT) (if occurs): RT Days 1-4; Follow-Up at Day 771Pharmacokinetics trough plasma concentrations are provided on the pre-dose levels for the six patients enrolled.ng/mLMEAN
SECONDARYPharmacokinetics (PK): Maximum Plasma Concentrations (Cmax)Days 1-4; Treatment Maintenance (103 weeks): 17 visits; Rescue Therapy (if occurs): RT Days 1-4; Follow-Up at Day 771The observed Cmax values on the 6 patients have been provided.ng/mLMEDIAN
SECONDARYPharmacokinetics (PK): Area Under Time-concentration Curve (AUC)Days 1-4; Treatment Maintenance (103 weeks): 17 visits; Rescue Therapy (if occurs): RT Days 1-4; Follow-Up at Day 771PK modeling for calculating the AUC was not performed because the study was not completed.
SECONDARYPharmacodynamics (PD): Inhibition of C3 Activity (%)Days 1-4; Treatment Maintenance (103 weeks): 17 visits; Rescue Therapy (if occurs): RT Days 1-4; Follow-Up at Day 771Any patient who received drug and measurable C3 activity.Inhibition of C3 activity (%)MEDIAN
SECONDARYPharmacodynamics (PD): Inhibition of C4 Activity (%)Days 1-4; Treatment Maintenance (103 weeks): 17 visits; Rescue Therapy (if occurs): RT Days 1-4; Follow-Up at Day 771Any patient who received drug and measurable C4 activity.Inhibition of C4 activity (%)MEDIAN

Source ClinicalTrials.govtrials-2026-11-12-0.5.0

Sources 3

  • ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
  • ClinicalTrials.govNo license stated
  • NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
  • ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
  • ClinicalTrials.govnone stated
  • NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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