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Study of Ataluren (PTC124) in Hemophilia A and B
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Trial · NCT00947193

Results by group17

Each measurement the trial posted, group by group, in the words and numbers it posted.

1 to 17 of 17

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OutcomeGroupCategoryValueSpreadUnits
Number of Participants With a Plasma FVIII/FIX Activity Response at Day 1410 mg/kg, 10 mg/kg, and 20 mg/kg Ataluren0Participants
Number of Participants With a Change From Baseline in Plasma Anti-FVIII/FIX Inhibitor Titers at Day 1410 mg/kg, 10 mg/kg, and 20 mg/kg Ataluren1Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) by Severity and Relationship to Study DrugAtaluren Overall StudyTEAEs8Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) by Severity and Relationship to Study DrugAtaluren Overall StudyTEAEs Related to Study Drug2Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) by Severity and Relationship to Study DrugAtaluren Overall StudySevere TEAEs0Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) by Severity and Relationship to Study DrugAtaluren Overall StudySAEs0Participants
Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Hematology, Adrenal Assays, Biochemistry, and Urinalysis) ParametersAtaluren Overall StudyHematology Assays0Participants
Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Hematology, Adrenal Assays, Biochemistry, and Urinalysis) ParametersAtaluren Overall StudyBiochemistry Assays0Participants
Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Hematology, Adrenal Assays, Biochemistry, and Urinalysis) ParametersAtaluren Overall StudyAdrenal Assays0Participants
Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Hematology, Adrenal Assays, Biochemistry, and Urinalysis) ParametersAtaluren Overall StudyUrinalysis1Participants
Ataluren Plasma Exposure10 mg/kg, 10 mg/kg, and 20 mg/kg AtalurenPre-Dose14.7Full Range 5.94 to 28.7microgram/milliliters (μg/mL)
Ataluren Plasma Exposure10 mg/kg, 10 mg/kg, and 20 mg/kg AtalurenPost-Dose6.66Full Range 1.04 to 28.3microgram/milliliters (μg/mL)
Occurrence of Bleeding EpisodesAtaluren Overall StudyParticipants Who Experienced a Bleeding Episode3Participants
Occurrence of Bleeding EpisodesAtaluren Overall StudySpontaneous Right Hip Bleed1Participants
Occurrence of Bleeding EpisodesAtaluren Overall StudySoft Tissue Bleed1Participants
Occurrence of Bleeding EpisodesAtaluren Overall StudyRight Knee Bleed1Participants
Occurrence of Bleeding EpisodesAtaluren Overall StudyUpper Shoulder Bleed1Participants

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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