INSPIRE (INcreased Sun exposure without Pain In Research subjects with EPP).

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Tanabe Pharma America Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

Trial duration

5 Apr 2024 → ongoing

Decision date (initial)

2024-03-20

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Tanabe Pharma America, Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Pharmacodynamic, Pharmacokinetic

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 Erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP).

Secondary objectives 2

1. To investigate the effect on Patient Global Impression of Change (PGIC) in adults and adolescents with EPP or XLP.
2. To investigate the effect on number of sunlight-induced pain events (prodrome and phototoxic reactions) in adults and adolescents with EPP or XLP.

Conditions and MedDRA coding

Erythropoietic Protoporphyria or X-Linked Protoporphyria

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Subjects provided written informed consent to participate. For minor subjects, both minor’s assent and legal representative´s consent will be required.
2. Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical history.
3. Subjects aged 12 years to 75 years, inclusive, at Screening.
4. Subjects are willing and able to travel to the study sites for all scheduled visits.
5. 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).
6. 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.
7. Female subjects of childbearing potential and male subjects with partner of childbearing potential currently using/willing to use 2 effective methods of contraception

Exclusion criteria 16

1. History or presence of photodermatoses other than EPP or XLP.
2. 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.
3. 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.
4. 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 Disease-Epidemiology 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.
5. 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.
6. Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
7. 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; - Chronic treatment with any scheduled analgesic agents.
8. 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.
9. Subjects who participated in any previous MT-7117 clinical studies.
10. 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).
11. 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.
12. Use of sunscreens with zinc oxide.
13. Presence or history of any hepatobiliary disease, including drug-induced liver injury at screening, determined as clinically significant by the Investigator after the discussion with the Sponsor Medical Monitor.
14. 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.
15. History (in the last 2 years) or presence of alcohol abuse, or abuse of illicit drugs in the opinion of the Investigator.
16. History of melanoma.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline in average daily sunlight exposure time (in 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.

Secondary endpoints 3

1. Patient Global Impression of Change (PGIC) at Week 16.
2. 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.
3. Total number of sunlight-induced non-prodrome, phototoxic reactions during the 16-week double-blind treatment period.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Tanabe Pharma America Inc.

Sponsor organisation

Tanabe Pharma America Inc.

Address

525 Washington Boulevard Suite 1100

City

Jersey City

Postcode

07310-2604

Country

United States

Scientific contact point

Organisation

Tanabe Pharma America Inc.

Contact name

General Information, Tanabe Pharma Europe Ltd.

Public contact point

Organisation

Tanabe Pharma America Inc.

Contact name

General Information, Tanabe Pharma Europe Ltd.

Third parties 11

Locations

7 EU/EEA countries · 17 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 74 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

16 submissions — initial application plus substantial / non-substantial modifications