Erythropoietic Protoporphyria (EPP)Phase 3INSPIRENCT06144840
INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP
A Phase 3 study testing Dersimelagon in people with Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP), run by Tanabe Pharma America, Inc. at 35 sites in 11 countries.
Trial summary
To investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and adolescents with EPP or XLP. Read more about this study
- Who is it for?
- Ages 12 to 75 years
- Conditions the study is for
- Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
- What is being tested?
- Dersimelagon
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 165 participants
- Where
- 35 sitesin 11 countries
- Study dates
- 11 Dec 2023to 19 May 2026
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subjects provided written informed consent to participate. For minor subjects, both minor's assent and parental consent will be required.
- Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical history.
- Subjects aged 12 years to 75 years, inclusive, at Screening.
- Subjects are willing and able to travel to the study sites for all scheduled visits.
- In the Investigator's opinion, subject can understand the nature of the study and any risks involved in participation, and willing to cooperate and comply with the protocol restrictions and requirements (including travel and receiving direct sunlight exposure as much as possible).
- Female subjects who are non-lactating and have a negative urine pregnancy test at baseline visit prior to receiving the first dose of study drug.
- Female subjects of childbearing potential and male subjects with partner of child-bearing potential currently using/willing to use 2 effective methods of contraception.
- Additional screening criteria check may apply for qualification.
Who cannot join
- History or presence of photodermatoses other than EPP or XLP.
- Subjects who are unwilling or unable to go outside in sunlight during daylight hours most days (e.g., between 1-hour post-sunrise and 1 hour pre-sunset) during the study.
- Presence or history of any hepatobiliary disease, including druginduced liver injury at screening, determined as clinically significant by the Investigator after the discussion with the Sponsor Medical Monitor.
- Subjects with aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP) ≥ 2.0 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN at Screening.
- History (in the last 2 years) or presence of alcohol abuse, or abuse of illicit drugs in the opinion of the Investigator.
- History of melanoma.
Show all 26Show fewer
- Presence of squamous cell carcinoma, basal cell carcinoma, or other malignant skin lesions. Any suspicious lesions or nevi will be evaluated. If the suspicious lesion or nevi cannot be resolved through biopsy or excision, the subject will be excluded from the study.
- History or presence of psychiatric disease judged to be clinically significant by the Investigator and which may interfere with the study evaluation and/or safety of the subjects.
- Presence of clinically significant acute or chronic renal disease or subjects with an estimated glomerular filtration rate (eGFR) <60 mL/min as calculated by the Chronic Kidney DiseaseEpidemiology Collaboration (CKD-EPI) creatinine equation (2021) for adults and by the Schwartz creatinine equation for adolescents (2009). Modification of Diet in Renal Disease can be used for adults per local recommendations.
- Presence of any clinically significant disease or laboratory abnormality which, in the opinion of the Investigator, can interfere with the study objectives and/or safety of the subjects.
- Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
- Treatment with any of the following medications or therapy within each period before Randomization (Visit 2);
- Afamelanotide within 3 months
- Phototherapy within 3 months
- Cimetidine within 4 weeks
- Antioxidant agents within 4 weeks, at doses which, in the opinion of the Investigator, may affect study endpoints (including but not limited to beta-carotene, cysteine, pyridoxine).
- Chronic treatment with any scheduled analgesic agents including, but not limited to, opioids and opioid derivatives such as morphine, hydrocodone, oxycodone, fentanyl, or their combination with other unscheduled analgesics or non-steroidal anti-inflammatory drug (Percocet and Vicodin-like prescription drugs) within 4 weeks.
- Note: Acute use of scheduled narcotics more than 3 months prior to randomization are allowed. Non-steroidal anti-inflammatory drug, aspirin for analgesia, or prior temporary use of scheduled agents within 3 months of screening is allowed.
- Dermatological treatments with any drugs or supplements which, in the opinion of the Investigator, can interfere with the objectives of the study or safety of the subjects at screening, such as, for example, tanning agents.
- Subjects who participated in any previous MT-7117 clinical studies.
- Previous treatment with any investigational agent such as bitopertin, within 12 weeks before Screening or 5 half-lives of the investigational product (whichever is longer).
- Use of sunscreens with zinc oxide. Note: Sunscreens without zinc oxide are allowed, however their use, in frequency, quantity and body surface area should be maintained relatively stable throughout the duration of the study.
- History of any hypersensitivity to the active ingredient and/or excipients (lactose monohydrate, hydroxypropylcellulose, carmellose calcium, magnesium stearate, hypromellose, titanium dioxide, talc, polyethylene glycol, iron oxide yellow, iron oxide red, and iron oxide black). (EU ONLY)
- Subjects who are unable to swallow tablets or have diseases significantly affecting the gastrointestinal function such as malabsorption syndrome, resection of the stomach or small bowel, bariatric surgery procedures, symptomatic inflammatory bowel disease, or partial or complete bowel obstruction.(EU ONLY)
- History of any hypersensitivity to the active ingredient and/or excipients contained in MT-7117 IMP (lactose monohydrate, hydroxypropyl cellulose, carmellose calcium, magnesium stearate, hypromellose, titanium dioxide, talc, polyethylene glycol, iron oxide yellow, iron oxide red, and iron oxide black). (UK ONLY)
- Additional screening criteria check may apply for qualification.
Trial locations and contacts
35 sites in 11 countries.
35 sites in 11 countries
United States13 sites
MGH
Boston, Massachusetts, 02129
MetroBoston Clinical Partners, LLC
Brighton, Massachusetts, 02135-3511
Remington-Davis Clinical Research
Columbus, Ohio, 43215
Henry Ford Health System
Detroit, Michigan, 48202
The University of Texas Medical Branch (UTMB)
Galveston, Texas, 77555-0342
Marvel Clinical Research, LLC
Huntington Beach, California, 92647
Kansas City Research Institute
Kansas City, Missouri, 64131
University Of Miami School Of Medicine, Center For Liver Diseases
Miami, Florida, 33136
Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)
New York, New York, 10029-0311
Einstein Medical Center (EMC)
Philadelphia, Pennsylvania, 19141
University of California at San Francisco
San Francisco, California, 94143
University of Washington
Seattle, Washington, 98195
Wake Forest University Baptist Health
Winston-Salem, North Carolina, 27103
Italy7 sites
Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia
Brescia, 25123
Azienda Sanitaria Ospedaliera Santa Croce E Carle - Cuneo
Cuneo, 12100
Ospedalle Galliera
Genova, 16128
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
Milan, 20122
U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena
Modena, 41124
IFO-San Gallicano IRCCS
Rome, 00144
Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Materno-Infantile - Burlo Garofolo - Clinica Pediatrica
Trieste, 34137
France4 sites
Centre Hospitalier Universitaire de Bordeaux - Hopital Saint - Andre
Bordeaux, 33000
CHU Nantes
Nantes, 44000
Hôpital Bichat - Hospital Bichat-Hopitaux Universitaires Paris Nord Val de Seine
Paris, 75018
Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Louis-Mourier
Paris, 92701
Japan3 sites
Mazda Hospital
Aki-gun, Hiroshima, 735-8585
Hamamatsu University Hospital
Hamamatsu, Shizuoka, 431-3194
Tokyo Saiseikai Central Hospital
Tokyo, 108-0073
Spain2 sites
Hospital Universitario 12 de Octubre
Madrid, 28041
Hospital General Universitario De Valencia
Valencia, 46014
Bulgaria1 site
University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski - Porphyria Center
Sofia, 01000
Czechia1 site
Institute for Clinical and Experimental Medicine - IKEM
Prague, 01958
About this study
Official title: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents With Erythropoietic Protoporphyria or X-Linked Protoporphyria
To investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and adolescents with EPP or XLP.
- Being tested
- Dersimelagon, Placebo
- Medical subject headings
- Protoporphyria, Erythropoietic, Protoporphyria, Erythropoietic, X-Linked Dominant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DersimelagonDrug
MT-7117
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| MT-7117 | Experimental | Not described Receives: Drug: Dersimelagon |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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 16
The comparison between MT-7117 treatment group and placebo group will be performed.
Measured: Week 16
Secondary outcomes (3)
Patient Global Impression of Change (PGIC) at Week 16.
The comparison between MT-7117 treatment group and placebo group will be performed.
Measured: Week 16
Total number of sunlight-induced pain events defined as prodromal symptoms (burning, tingling, itching, or stinging) with pain rating of 1-10 on the Likert scale during the 16-week double-blind treatment period.
The comparison between MT-7117 treatment group and placebo group will be performed.
Measured: Week 16
Total number of sunlight-induced non-prodrome, phototoxic reactions during the 16-week double-blind treatment period.
The comparison between MT-7117 treatment group and placebo group will be performed.
Measured: Week 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Nov 2023
- Study start11 Dec 2023
- Primary completion14 Apr 2026
- Study completion19 May 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Erythropoietic Protoporphyria (EPP)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 165 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tanabe Pharma America, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Head of Medical Science, Study director, Tanabe Pharma America, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06144840
- Sponsor's protocol ID
- MT-7117-A-302
- Other IDs
- jRCT2031230656, 2023-506735-15-00, 2023
- Acronym
- INSPIRE
- Status last verified
- Jun 2026
- First submitted
- 17 Nov 2023
- Last updated
- 4 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents With Erythropoietic Protoporphyria or X-Linked Protoporphyria
- ClinicalTrials.gov ID
- NCT06144840
- Acronym / sponsor ID
- INSPIRE / MT-7117-A-302
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 165 participants (actual)
- Lead sponsor
- Tanabe Pharma America, Inc.Industry (pharma, biotech, devices)
- Study start
- 11 Dec 2023 (actual)
- Primary completion
- 14 Apr 2026 (actual)
- Study completion
- 19 May 2026 (actual)
- First posted
- 22 Nov 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and adolescents with EPP or XLP.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MT-7117 | Experimental | Drug: Dersimelagon |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 12 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Subjects provided written informed consent to participate. For minor subjects, both minor's assent and parental consent will be required.
- Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical history.
- Subjects aged 12 years to 75 years, inclusive, at Screening.
- Subjects are willing and able to travel to the study sites for all scheduled visits.
- In the Investigator's opinion, subject can understand the nature of the study and any risks involved in participation, and willing to cooperate and comply with the protocol restrictions and requirements (including travel and receiving direct sunlight exposure as much as possible).
Key exclusion criteria (26 listed)
- History or presence of photodermatoses other than EPP or XLP.
- Subjects who are unwilling or unable to go outside in sunlight during daylight hours most days (e.g., between 1-hour post-sunrise and 1 hour pre-sunset) during the study.
- Presence or history of any hepatobiliary disease, including druginduced liver injury at screening, determined as clinically significant by the Investigator after the discussion with the Sponsor Medical Monitor.
- Subjects with aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP) ≥ 2.0 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN at Screening.
- History (in the last 2 years) or presence of alcohol abuse, or abuse of illicit drugs in the opinion of the Investigator.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DersimelagonMT-7117 | Drug | MT-7117 |
| PlaceboPlacebo | Drug | Placebo |
Primary outcome
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 16
Time frame: Week 16
The comparison between MT-7117 treatment group and placebo group will be performed.
3 secondary outcomes
Patient Global Impression of Change (PGIC) at Week 16.
Time frame: Week 16
Total number of sunlight-induced pain events defined as prodromal symptoms (burning, tingling, itching, or stinging) with pain rating of 1-10 on the Likert scale during the 16-week double-blind treatment period.
Time frame: Week 16
Total number of sunlight-induced non-prodrome, phototoxic reactions during the 16-week double-blind treatment period.
Time frame: Week 16
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 2026.
- Individual participant dataSharing planned: No
Related trials for Erythropoietic Protoporphyria (EPP)
Enrolling by invitationPhase 2/3
HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP
Erythropoietic Protoporphyria
RecruitingPhase 2/3
A Study to Investigate PORT-77 Oral Dosing Compared With Placebo in Participants With Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Active, not recruitingPhase 3
Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)
EPP, XLP
RecruitingObservational study
Longitudinal Study of the Porphyrias
Acute Porphyrias, Cutaneous Porphyrias
RecruitingObservational study
Observational Study of Adults and Adolescents With Erythropoietic Protoporphyria (EPP) and X-linked Porphyria (XLP)
Erythropoietic Protoporphyria (EPP), X-Linked Porphyria (XLP)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Head of Medical Science study director
Tanabe Pharma America, Inc.
35 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Dersimelagon in people with Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP), run by Tanabe Pharma America, Inc. at 35 sites in 11 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 35 sites in United States, Italy, France, Japan, Spain and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 16 (time frame: Week 16). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Jun 2026.