Trial · NCT02268552
Outcomes21
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
1 to 20 of 21
Download this page as JSON| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Dose Limiting Toxicities (DLT) in Part 1 - Safety Analysis Set (SAS) | Baseline up to 2 weeks for Part 1 | Safety analysis set. No DLTs were observed, therefore maximum tolerated dose could not be determined. | Participants | NUMBER |
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events and Deaths- SAS | Baseline up to approximately 83 months | Safety analysis set, all enrolled participants who received at least one dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Area Under the Curve (AUCinf) After a Single Dose - Part 1 - Pharmacokinetics Analysis Set (PAS) | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | h*ng/mL | MEAN |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Area Under the Curve (AUCinf) for All Observation Periods - Part 1 - PAS | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | h*ng/mL | MEAN |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Cmax After a Single Dose - Part 1 - PAS | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | ng/mL | MEAN |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Cmax for All Observation Periods - Part 1 - PAS | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | ng/mL | MEAN |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Area Under the Curve (AUC) After a Single Dose - Part 2 - PAS | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | h*ng/mL | MEAN |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Area Under the Curve (AUC) for All Observation Periods - Part 2 - PAS | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | h*ng/mL | MEAN |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Cmax for a Single Dose - Part 2 - PAS | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | ng/mL | MEAN |
| SECONDARY | Summary of Plasma Pharmacokinetic (PK) Parameter Cmax for All Observation Periods - Part 2 - PAS | from 0 h to 168 h after first/single dose | Pharmacokinetic analysis set | ng/mL | MEAN |
| SECONDARY | Change From Baseline in Growth Parameters: Chest Circumference, Head Circumference and Body Length - Full Analysis Set (FAS) | Baseline, Week 52 and Month 6 of Part 3 | Full analysis set - Number of patients who have an assessment available at Baseline and the relevant post-baseline visit. | cm | MEAN |
| SECONDARY | Change From Baseline in Growth Parameter: Body Weight - FAS | Baseline, Week 52 and Month 6 of Part 3 | Full analysis set - Number of patients who have an assessment available at Baseline and the relevant post-baseline visit. | kg | MEAN |
| SECONDARY | Change From Baseline in Respiratory Function: Pulse Oximetry - FAS | Baseline, Week 52 and Month 6 of Part 3 | Full analysis set - Number of patients who have an assessment available at Baseline and the relevant post-baseline visit. | percentage | MEAN |
| SECONDARY | Change From Baseline in Respiratory Function: Respiratory Rate - FAS | Baseline, Week 52 and Month 6 of Part 3 | Full analysis set - Number of patients who have an assessment available at Baseline and the relevant post-baseline visit. | breaths per minute | MEAN |
| SECONDARY | Number of Participants With Presence of Paradoxical Breathing - FAS | Baseline, Week 52 and Month 6 of Part 3 | Full analysis set - Number of patients who have an assessment available at Baseline and the relevant post-baseline visit. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Respiratory Function: Chest Circumference During Quiet Breathing - End of Inspiration and Expiration - FAS | Baseline, Week 52 and Month 6 of Part 3 | Full analysis set - Number of patients who have an assessment available at Baseline and the relevant post-baseline visit. | cm | MEAN |
| SECONDARY | Summary of CHOP INTEND Total Score - Parts 1 and 3 and Parts 2 and 3 - FAS | Baseline, Week 52 and Month 6 of Part 3 | Full analysis set - Number of patients who have an assessment available at Baseline and the relevant post-baseline visit. | scores on a scale | MEAN |
| SECONDARY | Number of Participants Fed Orally or by Feeding Tube for Parts 1 and 3 and Parts 2 and 3 - FAS | Baseline up Week 78 | Full analysis set - Number of patients who have an assessment available at Baseline and the relevant post-baseline visit. | participants | NUMBER |
| SECONDARY | Number of Participants and HINE Motor Subscale Milestones (Ability to Sit, Stand or Walk Without Support) - FAS | Week 52 and Month 6 of Part 3 | Full analysis set - Number of patients who have an assessment available at Baseline and the relevant post-baseline visit. | participants | NUMBER |
| SECONDARY | Summary of Hammersmith Infant Neurologic Examination Section 2 (HINE-2) - FAS | Week 52 and Month 6 of Part 3 | Full analysis set - Number of patients who have an assessment available at Baseline and the relevant post-baseline visit. | total scores | MEAN |
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.
For software
Read this list from software.
Twenty rows a page, as JSON. No key.
GET https://conductscience.org/api/trials/v1/NCT02268552/outcomes