Trial · NCT05308472
Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP
NCT05308472, Phase 2, Completed, Disc Medicine, Inc; results first posted 2026-01-08
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP. Trials, record NCT05308472. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05308472
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP
- NCT number
- NCT05308472
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 75 participants, actual
- Lead sponsor
- Disc Medicine, Inc
- Conditions
- Erythropoietic Protoporphyria
- Interventions
- DISC-1459, DISC-1459, Placebo
- First posted
- 2022-04-04
- Last updated
- 2026-01-08
- Results first posted
- 2026-01-08
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change From Baseline in Whole Blood Metal-free PPIX Levels | 121 days | Percent change from baseline in PPIX concentration was analyzed using a mixed model repeated measures analysis in the ITT population. |
| Secondary | Total Hours of Sunlight Exposure to Skin on Days With no Pain From 1000 to 1800 Hours (10:00am to 6:00pm) | 121 days | Cumulative total hours of sunlight exposure on days with no pain from 1000 to 1800 hours from baseline to Day 121 (EOS) was analyzed using analysis of variance in the ITT population. |
| Secondary | Daily Sunlight Exposure Time (Minutes) to First Prodromal Symptom (Burning, Tingling, Itching, or Stinging) Associated With Sunlight Exposure Between 1 Hour Post-sunrise and 1 Hour Pre-sunset | 121 days | Participants exposed their skin to sunlight once a week and measured the time it takes to experience a prodrome. The time (minutes) to first prodromal symptom (e.g., burning, tingling, itching, or stinging) associated with sunlight exposure was recorded in a diary. This sun exposure challenge was performed weekly. The average time (minutes) to first prodromal symptom (e.g., burning, tingling, itching, or stinging) associated with sunlight exposure was averaged over two-week intervals through Study Day 121. |
| Secondary | Total Pain Intensity | Sum of Day 1 to Day 121 | The maximum total daily pain intensity scores of phototoxic reactions over the entire treatment period (D1-D121). The maximum pain score of a phototoxic reaction was measured on a Likert Scale (0-10). Total scores range from 0-1210. A score of "0" is the best outcome; and higher scores are a worse outcome. The maximum pain values on a scale of 0-10 in a day were summed across 121 days. |
| Secondary | Incidence of Treatment-emergent Adverse Events | 121 days | Incidence of treatment-emergent adverse events |
| Secondary | Erythrocyte Total PPIX Concentrations | 121 days | Percent change from baseline in erythrocyte total PPIX concentration was summarized by analysis visit in the ITT population. |
| Secondary | Plasma Total PPIX Concentrations | 121 days | Percent change from baseline in plasma total PPIX concentration was summarized by analysis visit in the ITT population. |
| Secondary | Whole Blood Total PPIX Concentrations | 121 days | Percent change from baseline in whole blood total PPIX concentration was summarized by analysis visit in the ITT population. |
| Secondary | Plasma Bitopertin Concentrations | Day 29 | Day 29 plasma bitopertin concentrations, 4 hours post-dose |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change From Baseline in Whole Blood Metal-free PPIX Levels | 121 days | Percent Change | MEAN | |
| SECONDARY | Total Hours of Sunlight Exposure to Skin on Days With no Pain From 1000 to 1800 Hours (10:00am to 6:00pm) | 121 days | Hours | MEAN | |
| SECONDARY | Daily Sunlight Exposure Time (Minutes) to First Prodromal Symptom (Burning, Tingling, Itching, or Stinging) Associated With Sunlight Exposure Between 1 Hour Post-sunrise and 1 Hour Pre-sunset | 121 days | Minutes/Two Weeks | MEAN | |
| SECONDARY | Total Pain Intensity | Sum of Day 1 to Day 121 | Scores on a scale | MEAN | |
| SECONDARY | Incidence of Treatment-emergent Adverse Events | 121 days | Number and Proportion of Participants with at Least One TEAE | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Erythrocyte Total PPIX Concentrations | 121 days | Intention-to-treat population analyzed | Percent Change | MEAN |
| SECONDARY | Plasma Total PPIX Concentrations | 121 days | Intention-to-treat population analyzed | Percent Change | MEAN |
| SECONDARY | Whole Blood Total PPIX Concentrations | 121 days | Intention-to-treat population analyzed | Percent Change | MEAN |
| SECONDARY | Plasma Bitopertin Concentrations | Day 29 | Intention-to-treat population analyzed. There is no measurable bitopertin concentrations in placebo patients, since they did not receive drug. | ng/mL | MEAN |
Source ClinicalTrials.gov
Results by group26
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change From Baseline in Whole Blood Metal-free PPIX Levels | Placebo | 8.07 | 95% Confidence Interval -6.200 to 22.338 | Percent Change | |
| Percent Change From Baseline in Whole Blood Metal-free PPIX Levels | DISC-1459 Oral Low Dose | -21.48 | 95% Confidence Interval -35.842 to -7.125 | Percent Change | |
| Percent Change From Baseline in Whole Blood Metal-free PPIX Levels | DISC-1459 Oral High Dose l | -41.74 | 95% Confidence Interval -56.531 to -26.950 | Percent Change | |
| Total Hours of Sunlight Exposure to Skin on Days With no Pain From 1000 to 1800 Hours (10:00am to 6:00pm) | Placebo | 133.91 | 95% Confidence Interval 92.180 to 175.643 | Hours | |
| Total Hours of Sunlight Exposure to Skin on Days With no Pain From 1000 to 1800 Hours (10:00am to 6:00pm) | DISC-1459 Oral Low Dose Level | 175.11 | 95% Confidence Interval 134.723 to 215.489 | Hours | |
| Total Hours of Sunlight Exposure to Skin on Days With no Pain From 1000 to 1800 Hours (10:00am to 6:00pm) | DISC-1459 Oral High Dose Level | 153.14 | 95% Confidence Interval 111.699 to 194.585 | Hours | |
| Daily Sunlight Exposure Time (Minutes) to First Prodromal Symptom (Burning, Tingling, Itching, or Stinging) Associated With Sunlight Exposure Between 1 Hour Post-sunrise and 1 Hour Pre-sunset | Placebo | 149.05 | Standard Deviation 116.434 | Minutes/Two Weeks | |
| Daily Sunlight Exposure Time (Minutes) to First Prodromal Symptom (Burning, Tingling, Itching, or Stinging) Associated With Sunlight Exposure Between 1 Hour Post-sunrise and 1 Hour Pre-sunset | DISC-1459 Oral Low Dose | 155.51 | Standard Deviation 99.903 | Minutes/Two Weeks | |
| Daily Sunlight Exposure Time (Minutes) to First Prodromal Symptom (Burning, Tingling, Itching, or Stinging) Associated With Sunlight Exposure Between 1 Hour Post-sunrise and 1 Hour Pre-sunset | DISC-1459 Oral High Dose | 176.14 | Standard Deviation 134.952 | Minutes/Two Weeks | |
| Total Pain Intensity | Placebo | 13.0 | Standard Deviation 9.38 | Scores on a scale |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo | 24 | |
| Overall Study | STARTED | DISC-1459 Oral Low Dose | 26 | |
| Overall Study | STARTED | DISC-1459 Oral High Dose | 25 | |
| Overall Study | COMPLETED | Placebo | 24 | |
| Overall Study | COMPLETED | DISC-1459 Oral Low Dose | 26 | |
| Overall Study | COMPLETED | DISC-1459 Oral High Dose | 22 | |
| Overall Study | NOT COMPLETED | Placebo | 0 | |
| Overall Study | NOT COMPLETED | DISC-1459 Oral Low Dose | 0 | |
| Overall Study | NOT COMPLETED | DISC-1459 Oral High Dose | 3 |
Source ClinicalTrials.gov
Adverse events33
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 | Dizziness | Nervous system disorders | Placebo | 3 | 24 | 3 |
| other | Dizziness | Nervous system disorders | DISC-1459 Oral Low Dose Level | 4 | 26 | 4 |
| other | Dizziness | Nervous system disorders | DISC-1459 Oral High Dose Level | 11 | 25 | 11 |
| other | Headache | Nervous system disorders | Placebo | 3 | 24 | 3 |
| other | Headache | Nervous system disorders | DISC-1459 Oral Low Dose Level | 3 | 26 | 3 |
| other | Headache | Nervous system disorders | DISC-1459 Oral High Dose Level | 2 | 25 | 2 |
| other | Nausea | Gastrointestinal disorders | Placebo | 4 | 24 | 4 |
| other | Nausea | Gastrointestinal disorders | DISC-1459 Oral Low Dose Level | 1 | 26 | 1 |
| other | Nausea | Gastrointestinal disorders | DISC-1459 Oral High Dose Level | 4 | 25 | 4 |
| other | Alanine aminotransferase increased | Investigations | Placebo | 3 | 24 | 3 |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 0 | 24 |
| Total, all-cause mortality | DISC-1459 Oral Low Dose Level | 0 | 26 |
| Total, all-cause mortality | DISC-1459 Oral High Dose Level | 0 | 25 |
| Total, other adverse events | Placebo | 19 | 24 |
| Total, other adverse events | DISC-1459 Oral Low Dose Level | 19 | 26 |
| Total, other adverse events | DISC-1459 Oral High Dose Level | 22 | 25 |
| Total, serious adverse events | Placebo | 1 | 24 |
| Total, serious adverse events | DISC-1459 Oral Low Dose Level | 0 | 26 |
| Total, serious adverse events | DISC-1459 Oral High Dose Level | 0 | 25 |
Source ClinicalTrials.gov
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/NCT05308472
GET https://conductscience.org/api/trials/v1/NCT05308472/results?page=2