Trial · NCT03338816
Outcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
1 to 12 of 12
Download this page as JSON| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Annualized Rate of Porphyria Attacks in Participants With Acute Intermittent Porphyria (AIP) | 6 months | AIP participants in the Full Analysis Set (FASAIP): All randomized AIP participants (with identified mutation in the hydroxymethylbilane synthase \[HMBS\] gene) who received at least one dose of study drug. | annualized attack rate | MEAN |
| SECONDARY | The Pharmacodynamic (PD) Effect of Givosiran on Urine Levels of Delta-aminolevulinic Acid (ALA) in Participants With AIP | 3 and 6 months | FASAIP: All randomized AIP participants (with identified mutation in the HMBS gene) who received at least one dose of study drug. | mmol/mol creatinine (Cr) | LEAST_SQUARES_MEAN |
| SECONDARY | The PD Effect of Givosiran on Urine Levels of Porphobilinogen (PBG) in Participants With AIP | 6 months | FASAIP: All randomized AIP participants (with identified mutation in the HMBS gene) who received at least one dose of study drug. | mmol/mol Cr | LEAST_SQUARES_MEAN |
| SECONDARY | Annualized Rate of Hemin Administration in Participants With AIP | 6 months | FASAIP: All randomized AIP participants (with identified mutation in the HMBS gene) who received at least one dose of study drug. | annualized rate of use | MEAN |
| SECONDARY | Annualized Rate of Porphyria Attacks in Participants With AHP | 6 months | Full Analysis Set (FAS): All randomized patients who received at least one dose of study drug. | annualized attack rate | MEAN |
| SECONDARY | Area Under the Curve (AUC) of the Change From Baseline in Weekly Mean Score of Daily Worst Pain as Measured by the Brief Pain Inventory-Short Form (BPI-SF) Numeric Rating Scale (NRS) in Participants With AIP | Baseline and 6 months | FASAIP: All randomized AIP participants (with identified mutation in the HMBS gene) who received at least one dose of study drug. | score on a scale*week | MEDIAN |
| SECONDARY | Average Change From Baseline in Weekly Mean Score of Daily Worst Pain as Measured by the Brief Pain Inventory-Short Form (BPI-SF) Numeric Rating Scale (NRS) in Participants With AIP | Baseline and 6 months | FASAIP: All randomized AIP participants (with identified mutation in the HMBS gene) who received at least one dose of study drug. | score on a scale | MEDIAN |
| SECONDARY | AUC of the Change From Baseline in Weekly Mean Score of Daily Worst Fatigue Score as Measured by the Brief Fatigue Inventory-Short Form (BFI-SF) NRS in Participants With AIP | Baseline and 6 months | FASAIP: All randomized AIP participants (with identified mutation in the HMBS gene) who received at least one dose of study drug. | score on a scale*week | LEAST_SQUARES_MEAN |
| SECONDARY | Average Change From Baseline in Weekly Mean Score of Daily Worst Fatigue Score as Measured by the Brief Fatigue Inventory-Short Form (BFI-SF) NRS in Participants With AIP | Baseline and 6 months | FASAIP: All randomized AIP participants (with identified mutation in the HMBS gene) who received at least one dose of study drug. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | AUC of the Change From Baseline in Weekly Mean Score Daily Worst Nausea Score as Measured by NRS in Participants With AIP | Baseline and 6 months | FASAIP: All randomized AIP participants (with identified mutation in the HMBS gene) who received at least one dose of study drug. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Average Change From Baseline in Weekly Mean Score Daily Worst Nausea Score as Measured by NRS in Participants With AIP | Baseline and 6 months | FASAIP: All randomized AIP participants (with identified mutation in the HMBS gene) who received at least one dose of study drug. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in the Physical Component Summary (PCS) of the 12-Item Short Form Survey (SF-12) in Participants With AIP | Baseline and 6 months | FASAIP: All randomized AIP participants (with identified mutation in the HMBS gene) who received at least one dose of study drug. | score on a scale | LEAST_SQUARES_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.
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GET https://conductscience.org/api/trials/v1/NCT03338816/outcomes