Trial · NCT07123363
Effect of Bitopertin on the Liver and on Levels of Protoporphyrin IX in Bile, Blood, Liver, and Stool in Patients With Erythropoietic Protoporphyria/X-linked Protoporphyria and Increased Liver Stiffness and/or Liver Enzymes at Baseline
NCT07123363, Phase 1, Withdrawn, Wake Forest University Health Sciences
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Effect of Bitopertin on the Liver and on Levels of Protoporphyrin IX in Bile, Blood, Liver, and Stool in Patients With Erythropoietic Protoporphyria/X-linked Protoporphyria and Increased Liver Stiffness and/or Liver Enzymes at Baseline. Trials, record NCT07123363. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07123363
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
Effect of Bitopertin on the Liver and on Levels of Protoporphyrin IX in Bile, Blood, Liver, and Stool in Patients With Erythropoietic Protoporphyria/X-linked Protoporphyria and Increased Liver Stiffness and/or Liver Enzymes at Baseline
- NCT number
- NCT07123363
- Status
- Withdrawn
- Phases
- Phase 1
- Enrollment
- 0 participants, actual
- Lead sponsor
- Wake Forest University Health Sciences
- Conditions
- Protoporphyria
- Interventions
- bitopertin
- First posted
- 2025-08-14
- Last updated
- 2026-01-29
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of treatment emergent adverse events (TEAE) | Week 104 | Number of treatment emergent adverse events (TEAE). Adverse events that begin after the first administration of study drug, or existing adverse events that worsen after the first dose of study drug will be considered TEAEs. |
| Secondary | Percent Change in Protoporphyrin IX (PP) Serum Levels | baseline to month 12, month 12 to month 24 | To assess effects of bitopertin on levels of protoporphyrin IX (PP) and other porphyrins in the whole blood, plasma, and red blood cells |
| Secondary | Number of Participants with Change in Histopathology | baseline to month 24 | To assess effects of bitopertin on hepatic histopathology the number of participants with change in histopathologic evidence will be tracked. Histopathology consists of histopathologic evidence of hepatic fibrosis, necroinflammation, fat, cholestasis, bile plugs, birefringent brown deposits in the liver, or other histopathology, as estimated by an experienced hepato-pathologist. |
| Secondary | Percent Change in Protoporphyrin IX (PP) Bile Levels | baseline to month 24 | the percent change of Protoporphyrin IX (PP) in bile will be used to assess effects of bitopertin on PP levels |
| Secondary | Percent Change in Protoporphyrin IX (PP) Liver Levels | baseline to month 12, baseline to month 24 | the percent change in Protoporphyrin IX (PP) in the liver will be measured by change in liver stiffness measured by vibration controlled elastography with use of dedicated instruments designed for this purpose (Fibroscan, Velocur) |
| Secondary | Percent Change in Protoporphyrin IX (PP) Stool Levels | baseline to month 24 | the percent change in Protoporphyrin IX (PP) in stool will be used to assess effects of bitopertin on PP levels |
| Other | Patient Global Impression of Severity | baseline, week 26, week 52, week 78, week 104 | Three items with total score range 3-15. Higher score indicates higher level of erythropoietic protoporphyria severity |
| Other | Patient Global Impression of Change | baseline, week 26, week 52, week 78, week 104 | Five items with total score range 5-25. Higher score indicates erythropoietic protoporphyria have gotten worse since start of the study. |
| Other | Patient-Reported Outcomes Measurement Information System - Social Isolation | baseline, week 26, week 52, week 78, week 104 | Seven items with total score range 3-15. Higher score indicates poor quality of life. |
| Other | Serum of Level of Bitopertin | baseline, week 26, week 52, week 78, week 104 | Trough concentrations of bitopertin will measured by using HPLC-mass spectroscopic procedures |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT07123363
GET https://conductscience.org/api/trials/v1/NCT07123363/registered