Trial · NCT00947193
Study of Ataluren (PTC124) in Hemophilia A and B
NCT00947193, Phase 2, Terminated, PTC Therapeutics; results first posted 2020-06-16
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. Study of Ataluren (PTC124) in Hemophilia A and B. Trials, record NCT00947193. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00947193
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Ataluren (PTC124) in Hemophilia A and B
- NCT number
- NCT00947193
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 13 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Hemophilia A, Hemophilia B
- Interventions
- Ataluren
- First posted
- 2009-07-27
- Last updated
- 2020-06-16
- Results first posted
- 2020-06-16
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With a Plasma FVIII/FIX Activity Response at Day 14 | Baseline up to Day 14 | A plasma FVIII/FIX activity response was defined as an end-of-treatment (Day 14) activity of ≥1%. |
| Secondary | Number of Participants With a Change From Baseline in Plasma Anti-FVIII/FIX Inhibitor Titers at Day 14 | Baseline and Day 14 | To assess the change from Baseline in plasma anti-FVIII/FIX inhibitor titers, it was determined if any potential antibodies were neutralizing using the Bethesda assay. The Bethesda assay demonstrates antibodies that are neutralizing by quantifying residual FVIII/FIX activity in normal plasma after serial dilutions with participant plasma. For this assay, the neutralizing antibody threshold value was 0.6 Bethesda units (BU). |
| Secondary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) by Severity and Relationship to Study Drug | Baseline up to Day 28 | The relationship of TEAEs and SAEs to the study drugs was assessed as: probable related, possible related, unlikely related, and unrelated. The severity of TEAEs were graded using the Common Terminology Criteria for Adverse Events, Version 3.0, as: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe), Grade 4 (life-threatening), or Grade 5 (fatal). A summary of other non-serious adverse events (AEs) and all serious AEs, regardless of causality is located in Reported AE section. |
| Secondary | Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Hematology, Adrenal Assays, Biochemistry, and Urinalysis) Parameters | Baseline up to Day 28 | The Investigator used his/her judgment in determining whether an abnormality was clinically significant, diagnostic evaluation was warranted, and potential interruption of ataluren was appropriate. Life-threatening (Grade 4) or severe (Grade 3) laboratory abnormalities were considered dose-limiting, although recurrent or persistent moderate (Grade 2) events were also considered dose-limiting in certain circumstances. Values considered abnormal included -Hematology: Serum total bilirubin Grade 2 (\>1.5-3.0\*upper limit of normal \[ULN\]) and Serum alanine aminotransferase, Serum aspartate aminotransferase, and Serum gamma glutamyl transferase Grade 2 (\>2.5-3.0\*ULN); -Adrenal: Plasma adrenocorticotropic hormone \>ULN (and cortisol within normal limits); and -Renal: serum creatinine Grade 1 (\>ULN-1.5\*ULN) and Serum blood urea nitrogen ≥1.5-3.0\*ULN. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section. |
| Secondary | Compliance With Ataluren Administration | Baseline up to Day 28 | Ataluren compliance as assessed by quantification of used and unused drug. Data were not collected or analyzed for this measure because participants were terminated early from the study. |
| Secondary | Ataluren Plasma Exposure | Day 10 (pre-dose) and Day 14 (post-dose) | The ataluren plasma concentrations before and 2 hours after the first morning dose at end of treatment was measured. |
| Secondary | Occurrence of Bleeding Episodes | Baseline up to Day 28 | Frequency, timing, anatomic location, and severity of any bleeding episodes were recorded. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With a Plasma FVIII/FIX Activity Response at Day 14 | Baseline up to Day 14 | All enrolled participants who received at least 1 dose of study drug and completed the study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With a Change From Baseline in Plasma Anti-FVIII/FIX Inhibitor Titers at Day 14 | Baseline and Day 14 | All enrolled participants who received at least 1 dose of study drug and completed the study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) by Severity and Relationship to Study Drug | Baseline up to Day 28 | All enrolled participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Hematology, Adrenal Assays, Biochemistry, and Urinalysis) Parameters | Baseline up to Day 28 | All enrolled participants who received at least 1 dose of study drug and had evaluable clinical laboratory data. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Compliance With Ataluren Administration | Baseline up to Day 28 | All enrolled participants who received at least 1 dose of study drug, completed the study, and had evaluable compliance with ataluren administration data. | ||
| SECONDARY | Ataluren Plasma Exposure | Day 10 (pre-dose) and Day 14 (post-dose) | All enrolled participants who received at least 1 dose of study drug, completed the study, and had evaluable plasma data. | microgram/milliliters (μg/mL) | MEDIAN |
| SECONDARY | Occurrence of Bleeding Episodes | Baseline up to Day 28 | All enrolled participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group17
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With a Plasma FVIII/FIX Activity Response at Day 14 | 10 mg/kg, 10 mg/kg, and 20 mg/kg Ataluren | 0 | Participants | ||
| Number of Participants With a Change From Baseline in Plasma Anti-FVIII/FIX Inhibitor Titers at Day 14 | 10 mg/kg, 10 mg/kg, and 20 mg/kg Ataluren | 1 | Participants | ||
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) by Severity and Relationship to Study Drug | Ataluren Overall Study | TEAEs | 8 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) by Severity and Relationship to Study Drug | Ataluren Overall Study | TEAEs Related to Study Drug | 2 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) by Severity and Relationship to Study Drug | Ataluren Overall Study | Severe TEAEs | 0 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) by Severity and Relationship to Study Drug | Ataluren Overall Study | SAEs | 0 | Participants | |
| Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Hematology, Adrenal Assays, Biochemistry, and Urinalysis) Parameters | Ataluren Overall Study | Hematology Assays | 0 | Participants | |
| Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Hematology, Adrenal Assays, Biochemistry, and Urinalysis) Parameters | Ataluren Overall Study | Biochemistry Assays | 0 | Participants | |
| Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Hematology, Adrenal Assays, Biochemistry, and Urinalysis) Parameters | Ataluren Overall Study | Adrenal Assays | 0 | Participants | |
| Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Hematology, Adrenal Assays, Biochemistry, and Urinalysis) Parameters | Ataluren Overall Study | Urinalysis | 1 | Participants |
1 to 10 of 17Source ClinicalTrials.gov
See all 17 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | 5 mg/kg, 5 mg/kg, and 10 mg/kg Ataluren | 3 | |
| Overall Study | STARTED | 10 mg/kg, 10 mg/kg, and 20 mg/kg Ataluren | 10 | |
| Overall Study | Received at Least 1 Dose of Study Drug | 5 mg/kg, 5 mg/kg, and 10 mg/kg Ataluren | 3 | |
| Overall Study | Received at Least 1 Dose of Study Drug | 10 mg/kg, 10 mg/kg, and 20 mg/kg Ataluren | 10 | |
| Overall Study | COMPLETED | 5 mg/kg, 5 mg/kg, and 10 mg/kg Ataluren | 0 | |
| Overall Study | COMPLETED | 10 mg/kg, 10 mg/kg, and 20 mg/kg Ataluren | 10 | |
| Overall Study | NOT COMPLETED | 5 mg/kg, 5 mg/kg, and 10 mg/kg Ataluren | 3 | |
| Overall Study | NOT COMPLETED | 10 mg/kg, 10 mg/kg, and 20 mg/kg Ataluren | 0 |
Source ClinicalTrials.gov
Adverse events9
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Diarrhoea | Gastrointestinal disorders | Ataluren Overall Study | 1 | 13 | |
| other | Constipation | Gastrointestinal disorders | Ataluren Overall Study | 1 | 13 | |
| other | Dyspepsia | Gastrointestinal disorders | Ataluren Overall Study | 1 | 13 | |
| other | Nausea | Gastrointestinal disorders | Ataluren Overall Study | 1 | 13 | |
| other | Conduction disorder | Cardiac disorders | Ataluren Overall Study | 1 | 13 | |
| other | Asthenia | General disorders | Ataluren Overall Study | 1 | 13 | |
| other | Haematuria | Renal and urinary disorders | Ataluren Overall Study | 1 | 13 | |
| other | Dysuria | Renal and urinary disorders | Ataluren Overall Study | 1 | 13 | |
| other | Haemorrhage | Vascular disorders | Ataluren Overall Study | 3 | 13 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ataluren Overall Study | ||
| Total, other adverse events | Ataluren Overall Study | 8 | 13 |
| Total, serious adverse events | Ataluren Overall Study | 0 | 13 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00947193
GET https://conductscience.org/api/trials/v1/NCT00947193/registered