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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 : Active, not recruiting
First Posted : May 9, 2018
Last Update Posted : April 30, 2019
Sponsor:
Information provided by (Responsible Party):
Mitsubishi Tanabe Pharma Development America, Inc.

Tracking Information
First Submitted Date  ICMJE April 23, 2018
First Posted Date  ICMJE May 9, 2018
Last Update Posted Date April 30, 2019
Actual Study Start Date  ICMJE July 5, 2018
Estimated Primary Completion Date September 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 30, 2019)
Change from baseline in average daily duration of sunlight exposure without symptoms [ Time Frame: Baseline (Week 0) and Week 16 ]
Duration in minutes, of sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset
Original Primary Outcome Measures  ICMJE
 (submitted: May 8, 2018)
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
Change History Complete list of historical versions of study NCT03520036 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures
 (submitted: May 8, 2018)
  • Total number of sunlight exposure episodes [ Time Frame: Baseline (Week 0) and Week 16 ]
  • Change in pigmentation as measured by melanin density [ Time Frame: Baseline (Week 0) and Week 16 ]
  • The quality of life as measured by the Patient Reported Outcomes Measurement Information System (PROMIS) 57 [ Time Frame: Baseline (Week 0) and Week 16 ]
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Study to Evaluate Efficacy, Safety, and Tolerability of MT‑7117 in Subjects With Erythropoietic Protoporphyria
Official Title  ICMJE A Phase II, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT‑7117 in Subjects With Erythropoietic Protoporphyria
Brief Summary The purpose of this study is to investigate the efficacy and safety of MT-7117 on sunlight exposure duration without symptoms and tolerance in subjects with EPP.
Detailed Description This is a Phase 2, 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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Erythropoietic Protoporphyria (EPP)
Intervention  ICMJE
  • Drug: MT-7117 low dose
    MT-7117 low dose QD, oral, 16 weeks
  • Drug: MT-7117 high dose
    MT-7117 high dose QD, oral, 16 weeks
  • Drug: Placebo
    Placebo QD, oral, 16 weeks
Study Arms  ICMJE
  • Experimental: MT-7117 low dose
    Intervention: Drug: MT-7117 low dose
  • Experimental: MT-7117 high dose
    Intervention: Drug: MT-7117 high dose
  • Placebo Comparator: Placebo
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: May 8, 2018)
102
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2019
Estimated Primary Completion Date September 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Additional screening criteria check may apply for qualification.

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 75 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 LFT values at Screening.
  • 4. Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin >1.5 × 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 pre-malignant skin lesion squamous cell carcinoma, basal cell carcinoma, or other 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 prescription-based analgesic agents including but not limited to opioids and opioid derivatives such as morphine, hydrocodone, oxycodone or their combination with other analgesics or non-steroidal anti-inflammatory drug (NSAID, as Percocet and Vicodin-like prescription drugs) 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).
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03520036
Other Study ID Numbers  ICMJE MT-7117-A01
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Mitsubishi Tanabe Pharma Development America, Inc.
Study Sponsor  ICMJE Mitsubishi Tanabe Pharma Development America, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Head of Clinical Development, Mitsubishi Tanabe Pharma Development America, Inc.
PRS Account Mitsubishi Tanabe Pharma Development America, Inc.
Verification Date April 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP