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Trial record 4 of 13 for:    "Protoporphyria"

Study to Evaluate Efficacy, Safety, and Tolerability of MT‑7117 in Subjects With Erythropoietic Protoporphyria

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ClinicalTrials.gov Identifier: NCT03520036
Recruitment Status : Recruiting
First Posted : May 9, 2018
Last Update Posted : July 11, 2018
Sponsor:
Information provided by (Responsible Party):
Mitsubishi Tanabe Pharma Development America, Inc.

Brief Summary:
The purpose of this study is to investigate the efficacy and safety of MT-7117 on sunlight exposure duration and tolerance in subjects with EPP.

Condition or disease Intervention/treatment Phase
Erythropoietic Protoporphyria (EPP) Drug: MT-7117 low dose Drug: MT-7117 high dose Drug: Placebo Phase 2

Detailed Description:
This is a Phase II, randomized, double-blind, placebo controlled study to assess the efficacy, tolerability, and safety of MT-7117 in subjects with EPP. The study consists of a 2 week screening period, a 16 week double-blind treatment period, and a 6 week follow-up period at Week 22. The total participation period is approximately 24 weeks.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 102 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase II, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT‑7117 in Subjects With Erythropoietic Protoporphyria
Actual Study Start Date : July 5, 2018
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : March 2019


Arm Intervention/treatment
Experimental: MT-7117 low dose Drug: MT-7117 low dose
MT-7117 low dose QD, oral, 16 weeks

Experimental: MT-7117 high dose Drug: MT-7117 high dose
MT-7117 high dose QD, oral, 16 weeks

Placebo Comparator: Placebo Drug: Placebo
Placebo QD, oral, 16 weeks




Primary Outcome Measures :
  1. Change from baseline in average daily duration of sunlight exposure [ Time Frame: Baseline (Week 0) and Week 16 ]
    Duration in minutes, of sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset


Other Outcome Measures:
  1. Total number of sunlight exposure episodes [ Time Frame: Baseline (Week 0) and Week 16 ]
  2. Change in pigmentation as measured by melanin density [ Time Frame: Baseline (Week 0) and Week 16 ]
  3. The quality of life as measured by the Patient Reported Outcomes Measurement Information System (PROMIS) 57 [ Time Frame: Baseline (Week 0) and Week 16 ]


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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 1. Subjects provided written informed consent to participate.
  • 2. Male and female subjects with a confirmed diagnosis of EPP based on medical history, aged 18 years to 70 years, inclusive, at Screening.
  • 3. Subjects are willing and able to travel to the study sites for all scheduled visits.
  • 4. In the Investigator's opinion, subject is able to 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).

Exclusion Criteria:

  • 1. History or presence of photodermatoses other than EPP.
  • 2. Subjects who are unwilling or unable to go outside during daylight hours (e.g., between 1 hour post sunrise and 1 hour pre-sunset) during the study.
  • 3. Presence of clinically significant hepatobiliary disease based on medical history or LFT values at Screening.
  • 4. Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin ≥1.0 × ULN at Screening.
  • 5. Subjects with or having a history (in the last 2 years) of excessive alcohol intake in the opinion of the Investigator.
  • 6. History or presence of melanoma and/or atypical nevus at Screening.
  • 7. History of familial melanoma (defined as having 2 or more first-degree relatives, such as parents, sibling and/or child).
  • 8. History or presence of squamous cell carcinoma, basal cell carcinoma, or other pre malignant or malignant skin lesions.
  • 9. 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.
  • 10. Presence of clinically significant acute or chronic renal disease based upon the subject's medical records including hemodialysis; and a serum creatinine level of greater than 1.2 mg/dL or a glomerular filtration rate (GFR) <60 ml/min.
  • 11. 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.
  • 12. Pregnancy or lactation.
  • 13. Females of child bearing potential and male subjects with partners of child-bearing potential unwilling to use adequate contraception measures as described in the protocol.
  • 14. Treatment with phototherapy within 3 months before Randomization (Visit 2).
  • 15. Treatment with afamelanotide within 3 months before Randomization (Visit 2).
  • 16. Treatment with cimetidine within 4 weeks before Randomization (Visit 2).
  • 17. Treatment with antioxidant agents at doses which, in the opinion of the Investigator, may affect study endpoints (including but not limited to beta-carotene, cysteine, pyridoxine) within 4 weeks before Randomization (Visit 2).
  • 18. Chronic treatment with centrally acting analgesic agents including but not limited to opioids and opioid derivatives within 4 weeks before Randomization (Visit 2).
  • 19. Treatment 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.
  • 20. Previous exposure to MT-7117.
  • 21. Previous treatment with any investigational agent within 12 weeks before Screening OR 5 half-lives of the investigational product (whichever is longer).
  • 22. Use of following drugs (including but not limited to) within 1 week of Randomization (Visit 2):

    1. Drugs known to be predominantly metabolized by cytochrome P450 (CYP) 3A4. (e.g., aprepitant, cyclosporine, pimozide, quinidine, and tacrolimus), for which elevated plasma concentrations are associated with significant medical events.
    2. Drugs that are known substrates of P-glycoprotein (P-gp), breast cancer resistance protein (BCRP), organic anion transporter 3, organic anion transporting polypeptide (OATP)1B1, or OATP1B3 (e.g., rosuvastatin, pitavastatin, atorvastatin, telmisartan, valsartan, olmesartan; for which elevated plasma concentrations are associated with significant medical events)
    3. Drugs known to inhibit P gp, UGT, OATP1B1 or OATP1B3. (e.g. probenecid, valproic acid, amiodarone, captopril, clarithromycin, felodipine, verapamil, rifampin, gemfibrozil)
    4. Drugs known to increase the gastric pH (e.g., omeprazole, lansoprazole, sodium hydrogen carbonate, aluminum hydroxide)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03520036


Contacts
Contact: Clinical Trials Information Desk, to prevent miscommunication, please email: information@mt-pharma-us.com

Locations
United States, Alabama
University of Alabama at Birmingham Not yet recruiting
Birmingham, Alabama, United States, 35294
Contact: Lisa D. Williams    205-975-9564    liwilliams@uabmc.edu   
Principal Investigator: Singal Ashwani         
United States, California
University of California at San Francisco Not yet recruiting
San Francisco, California, United States, 94143
Contact: Raaj Kapoor    415-476-8405    Yuvraaj.Kapoor@ucsf.edu   
Contact: Sasan Zenhari Abharrodi    415-476-5352    Sasan.ZenhariAbharrodi@ucsf.edu   
Principal Investigator: Montgomery Bissell         
United States, Florida
University of Miami Miller School of Medicine Recruiting
Miami, Florida, United States, 33136
Contact: Tiffannia Grant    305-243-4649    t.grant11@med.miami.edu   
Principal Investigator: Cynthia Levy         
United States, New York
Ichan School of Medicine at Mount Sinai Recruiting
New York, New York, United States, 10029
Contact: Hentashi Naik    212-241-7699    hetanshi.naik@mssm.edu   
Contact: Natasha Zeid    212-241-9196    natasha.zeid@mssm.edu   
Principal Investigator: Manisha Balwani         
United States, North Carolina
Wake Forest Baptist Medical Center Recruiting
Winston-Salem, North Carolina, United States, 27157
Contact: Dee Faust    336-713-1442    delannin@wakehealth.edu   
Principal Investigator: Herbert Bonkovsky         
United States, Texas
University of Texas Medical Branch Porphyria Center Recruiting
Galveston, Texas, United States, 77555
Contact: Csilla Hallberg    409-772-1128    ckhallbe@utmb.edu   
Contact: Linda Ede       lcede@utmb.edu   
Principal Investigator: Karl Anderson         
United States, Utah
University of Utah Not yet recruiting
Salt Lake City, Utah, United States, 84108
Contact: Sharada Dixit    801-587-7525    porphyria.center@hsc.utah.edu, Sharada.Dixit@hsc.utah.edu   
Contact: Hina Yazdani    801-587-2506    hina.yazdani@hsc.utah.edu   
Principal Investigator: Charles Parker         
Sponsors and Collaborators
Mitsubishi Tanabe Pharma Development America, Inc.
Investigators
Study Director: Head of Clinical Development, Mitsubishi Tanabe Pharma Development America, Inc.

Responsible Party: Mitsubishi Tanabe Pharma Development America, Inc.
ClinicalTrials.gov Identifier: NCT03520036     History of Changes
Other Study ID Numbers: MT-7117-A01
First Posted: May 9, 2018    Key Record Dates
Last Update Posted: July 11, 2018
Last Verified: July 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Protoporphyria, Erythropoietic
Porphyrias, Hepatic
Liver Diseases
Digestive System Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Porphyrias
Metabolic Diseases