Trial · NCT04402489

Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria

NCT04402489, Phase 3, Completed, Tanabe Pharma America, Inc.; results first posted 2025-03-21

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria. Trials, record NCT04402489. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04402489

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria
NCT number: NCT04402489
Status: Completed
Phases: Phase 3
Enrollment: 184 participants, actual
Lead sponsor: Tanabe Pharma America, Inc.
Conditions: EPP, XLP
Interventions: Placebo, MT-7117 Low Dose, MT-7117 High Dose
First posted: 2020-05-26
Last updated: 2025-12-30
Other registries' IDs: eudract:2019-004226-16
Results first posted: 2025-03-21

Source ClinicalTrials.gov

Registered outcomes6

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change From Baseline in Average 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 at Week 26 (Visit 7)	From 1 hour post-sunrise to 1 hour pre-sunset at Week 26 (Visit 7)
Secondary	Patient Global Impression of Change (PGIC) at Week 26	Week 26	PGIC: Scale from 1 to 7, where 7 is worse.
Secondary	Total Number of Sunlight-induced Pain Events Defined as Prodrome Symptoms (Burning, Tingling, Itching, or Stinging) With Pain Rating of 1-10 on the Likert Scale During the 26-week Double-blind Treatment Period.	During the 26-week double-blind treatment period	The Likert scale used ranges from 0 to 10, where 0 indicates the lowest pain rating and 10 indicates the highest pain rating. Likewise, 0 indicates to best outcome and 10 indicates the worst outcome. The sum of the number of pain events with pain rating of 1 to 10 for the day is used as the number of sunlight-induced pain events in the day. The sum of the number of the pain events with pain rating of 1 to 10 in each day during the 26-week Double-blind Treatment Period is calculated as this endpoint.
Secondary	Change From Baseline for Total Score in the Domain of Pain Intensity in the PROMIS-57 at Week 26	Baseline (Week 0) and Week 26	Pain intensity: 0 to 10, where 10 is worst pain imaginable.
Secondary	The Percentage of Subjects Who Are Responders	Week 26	The percentage of subjects who are responders based on average daily sunlight exposure time to first prodromal symptom associated with sunlight exposure between 1 hour post-sunrise and 1 hour presunset defined by within-subject meaningful change of 66 minutes increase from baseline to Week 26
Secondary	Change From Baseline for Total Score in the Domain of Physical Function in the PROMIS-57 at Week 26	Baseline (Week 0) and Week 26	Physical function: 1-5, where 5 is without any difficulty.

Source ClinicalTrials.gov

Outcomes6

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline in Average 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 at Week 26 (Visit 7)	From 1 hour post-sunrise to 1 hour pre-sunset at Week 26 (Visit 7)	Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.	minute	LEAST_SQUARES_MEAN
SECONDARY	Patient Global Impression of Change (PGIC) at Week 26	Week 26	Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.	point	LEAST_SQUARES_MEAN
SECONDARY	Total Number of Sunlight-induced Pain Events Defined as Prodrome Symptoms (Burning, Tingling, Itching, or Stinging) With Pain Rating of 1-10 on the Likert Scale During the 26-week Double-blind Treatment Period.	During the 26-week double-blind treatment period	Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.	events	GEOMETRIC_MEAN
SECONDARY	Change From Baseline for Total Score in the Domain of Pain Intensity in the PROMIS-57 at Week 26	Baseline (Week 0) and Week 26	Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.	total score	LEAST_SQUARES_MEAN
SECONDARY	The Percentage of Subjects Who Are Responders	Week 26	Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.	percentage of participants	NUMBER
SECONDARY	Change From Baseline for Total Score in the Domain of Physical Function in the PROMIS-57 at Week 26	Baseline (Week 0) and Week 26	Included all randomized subjects who received at least 1 dose of study medication with Baseline and post randomization- sunlight exposure diary assessments during the double-blind treatment period.	total score	LEAST_SQUARES_MEAN

Source ClinicalTrials.gov

Results by group18

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Change From Baseline in Average 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 at Week 26 (Visit 7)	DBT ITT1 Placebo	20.59	Standard Error 10.29	minute
Change From Baseline in Average 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 at Week 26 (Visit 7)	DBT ITT1 MT- 7117 Low Dose	30.39	Standard Error 10.04	minute
Change From Baseline in Average 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 at Week 26 (Visit 7)	DBT ITT1 MT- 7117 High Dose	43.29	Standard Error 10.13	minute
Patient Global Impression of Change (PGIC) at Week 26	DBT ITT1 Placebo	3.25	Standard Error 1.14	point
Patient Global Impression of Change (PGIC) at Week 26	DBT ITT1 MT- 7117 Low Dose	2.41	Standard Error 0.14	point
Patient Global Impression of Change (PGIC) at Week 26	DBT ITT1 MT- 7117 High Dose	1.82	Standard Error 0.14	point
Total Number of Sunlight-induced Pain Events Defined as Prodrome Symptoms (Burning, Tingling, Itching, or Stinging) With Pain Rating of 1-10 on the Likert Scale During the 26-week Double-blind Treatment Period.	DBT ITT1 Placebo	23.9	Standard Deviation 30.93	events
Total Number of Sunlight-induced Pain Events Defined as Prodrome Symptoms (Burning, Tingling, Itching, or Stinging) With Pain Rating of 1-10 on the Likert Scale During the 26-week Double-blind Treatment Period.	DBT ITT1 MT- 7117 Low Dose	15.87	Standard Deviation 22.05	events
Total Number of Sunlight-induced Pain Events Defined as Prodrome Symptoms (Burning, Tingling, Itching, or Stinging) With Pain Rating of 1-10 on the Likert Scale During the 26-week Double-blind Treatment Period.	DBT ITT1 MT- 7117 High Dose	13.78	Standard Deviation 18.7	events
Change From Baseline for Total Score in the Domain of Pain Intensity in the PROMIS-57 at Week 26	DBT ITT1 Placebo	-1.42	Standard Error 0.18	total score

1 to 10 of 18Source ClinicalTrials.gov

See all 18 results by group

Participant flow30

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
26 Weeks Double-Blind Treatment (DBT)	STARTED	Placebo	61
26 Weeks Double-Blind Treatment (DBT)	STARTED	MT-7117 Low Dose	63
26 Weeks Double-Blind Treatment (DBT)	STARTED	MT-7117 High Dose	60
26 Weeks Double-Blind Treatment (DBT)	STARTED	Placebo -> MT-7117 Low Dose	0
26 Weeks Double-Blind Treatment (DBT)	STARTED	Placebo -> MT-7117 High Dose	0
26 Weeks Double-Blind Treatment (DBT)	COMPLETED	Placebo	57
26 Weeks Double-Blind Treatment (DBT)	COMPLETED	MT-7117 Low Dose	60
26 Weeks Double-Blind Treatment (DBT)	COMPLETED	MT-7117 High Dose	58
26 Weeks Double-Blind Treatment (DBT)	COMPLETED	Placebo -> MT-7117 Low Dose	0
26 Weeks Double-Blind Treatment (DBT)	COMPLETED	Placebo -> MT-7117 High Dose	0

1 to 10 of 30Source ClinicalTrials.gov

See all 30 participant flow

Adverse events280

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
other	Thrombocytopenia	Blood and lymphatic system disorders	DBT: Placebo	0	61
other	Thrombocytopenia	Blood and lymphatic system disorders	DBT: MT-7117 Low Dose	0	63
other	Thrombocytopenia	Blood and lymphatic system disorders	DBT: MT-7117 High Dose	0	60
other	Thrombocytopenia	Blood and lymphatic system disorders	DBE: MT-7117 Low Dose Switched From Placebo	0	28
other	Thrombocytopenia	Blood and lymphatic system disorders	DBE: MT-7117 High Dose Switched From Placebo	2	28
other	Thrombocytopenia	Blood and lymphatic system disorders	DBE: MT-7117 Low Dose	0	56
other	Thrombocytopenia	Blood and lymphatic system disorders	DBE: MT-7117 High Dose	0	54
other	Porphyria non-acute	Congenital, familial and genetic disorders	DBT: Placebo	5	61
other	Porphyria non-acute	Congenital, familial and genetic disorders	DBT: MT-7117 Low Dose	1	63
other	Porphyria non-acute	Congenital, familial and genetic disorders	DBT: MT-7117 High Dose	1	60

1 to 10 of 280Source ClinicalTrials.gov

See all 280 adverse events

Adverse event totals21

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	DBT: Placebo	0	61
Total, all-cause mortality	DBT: MT-7117 Low Dose	0	63
Total, all-cause mortality	DBT: MT-7117 High Dose	1	60
Total, all-cause mortality	DBE: MT-7117 Low Dose Switched From Placebo	0	28
Total, all-cause mortality	DBE: MT-7117 High Dose Switched From Placebo	0	28
Total, all-cause mortality	DBE: MT-7117 Low Dose	0	56
Total, all-cause mortality	DBE: MT-7117 High Dose	0	54
Total, other adverse events	DBT: Placebo	30	61
Total, other adverse events	DBT: MT-7117 Low Dose	35	63
Total, other adverse events	DBT: MT-7117 High Dose	40	60

1 to 10 of 21Source ClinicalTrials.gov

See all 21 adverse event totals

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
39887900	DERIVED

Source PubMed

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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.

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GET https://conductscience.org/api/trials/v1/NCT04402489/flow?page=2

API reference For AI assistants