5-ALA study of mitochondrial diabetes mellitus

JRCT ID: jRCTs071200025

Registered date:03/08/2020

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Mitochondrial diabetes mellitus
• Date of first enrollment: 12/10/2020
• Target sample size: 5
• Study type: Interventional
• Intervention(s): Patients with mitochondrial diabetes mellitus receive an additional 24 weeks of 5-ALA/SFC.

Outcome(s)

• Primary Outcome: Changes of AUC of IRI during 75gOGTT after administration of 5-ALA/SFC for 24 weeks.
• Secondary Outcome: Changes in the following items after administration of 5-ALA/SFC for 24 weeks; fasting blood glucose, glycated hemoglobin level, glycated albumin level, some values in 75gOGTT, and required amount of daily insulin treatment.

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1.Patient aged >= 20 years old.
2.Outpatient.
3.Patients who meet all of the following main criteria and one of the sub-criteria.
<main criteria>
(1)Patients with diabetes mellitus treated with insulin or clinically required insulin therapy for glycemic control due to the insulin secretory defect.
(2)Patients complicated with neurosensory deafness.
<sub-criteria>
(1)Patients who have a family history of maternally inherited diabetes mellitus.
(2)Patients whose serum lactate level or lactate/pyruvate ratio are high at bed rest.
(3)Patients with short stature.
(4)Patients complicated with cardiomyopathy or cardiac conduction abnormality.
(5)Patients whose basal-ganglia and brainstem have bilaterally symmetric abnormal lesions on CT/MRI.
4.Patients who have not changed the drug or dose of antidiabetic agents except insulin for 8 weeks until obtaining informed consent.
5.Written informed consent.

Exclude criteria

1.Patients who have previously taken 5-ALA/SFC within 8 weeks.
2.Patients treated with metformin.
3.Patients with a history of porphyria, hemochromatosis, or viral hepatitis.
4.Women who are currently pregnant or will not be compliant with a medically approved contraceptive regimen during the study period and lactating women. Men who will not be compliant with a contraceptive regimen during the study period.
5.Patients participated in another clinical study within 4 months.
6.Patients whom the investigator considered inappropriate to participating this trial.

Related Information

• Primary Sponsor: Nakamura Yuta
• Source(s) of Monetary Support: Neopharama Japan Co.LTD
• Secondary ID(s): UMIN000040581