A Phase 3, Multicenter, Open-label, Long-term, Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

Conditions

Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

Brief summary

Treatment-emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs])., Physical examination., Vital signs (blood pressure, respiratory rate, pulse rate, and body temperature)., Clinical laboratory examinations (hematology, coagulation, biochemistry, and urinalysis), including liver function markers (aspartate aminotransferase [AST], alanine aminotransferase [ALT], gamma glutamyl transpeptidase [GGT], alkaline phosphatase [ALP], direct and total bilirubin)., 12-lead electrocardiogram (ECG) parameters., Nevi appearance (assessed by a dermatologist or other qualified site staff). Any nevi undergoing change of clinical concern during active treatment will be biopsied for follow-up and evaluated by the central pathology lab.

Related papers

No related papers found yet.

Interventions

DRUGMT-7117 Formulation Code C

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Treatment-emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs])., Physical examination., Vital signs (blood pressure, respiratory rate, pulse rate, and body temperature)., Clinical laboratory examinations (hematology, coagulation, biochemistry, and urinalysis), including liver function markers (aspartate aminotransferase [AST], alanine aminotransferase [ALT], gamma glutamyl transpeptidase [GGT], alkaline phosphatase [ALP], direct and total bilirubin)., 12-lead electrocardiogram (ECG) parameters., Nevi appearance (assessed by a dermatologist or other qualified site staff). Any nevi undergoing change of clinical concern during active treatment will be biopsied for follow-up and evaluated by the central pathology lab.	—

Measure

Time frame

Treatment-emergent adverse events (TEAEs) (including serious adverse events [SAEs] and adverse events of special interest [AESIs])., Physical examination., Vital signs (blood pressure, respiratory rate, pulse rate, and body temperature)., Clinical laboratory examinations (hematology, coagulation, biochemistry, and urinalysis), including liver function markers (aspartate aminotransferase [AST], alanine aminotransferase [ALT], gamma glutamyl transpeptidase [GGT], alkaline phosphatase [ALP], direct and total bilirubin)., 12-lead electrocardiogram (ECG) parameters., Nevi appearance (assessed by a dermatologist or other qualified site staff). Any nevi undergoing change of clinical concern during active treatment will be biopsied for follow-up and evaluated by the central pathology lab.

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Countries

Bulgaria, Czechia, France, Germany, Italy, Netherlands, Norway, Poland, Spain, Sweden

Outcome results

None listed