臨床研究等提出・公開システム

Evaluation of the safety and efficacy of triheptanoin for citrin deficiency.

CD triheptanoin Clinical Trials

Oishi Kimihiko

The Jikei University Hospital

3-19-18 Nishi-Shinbashi, Minato-ku, Tokyo

kimihiko.oishi@jikei.ac.jp

Nishida Hikaru

The Jikei University Hospital

3-19-18 Nishi-Shinbashi, Minato-ku, Tokyo

+81-3-3433-1111

hikaru-nishida@jikei.ac.jp

Recruiting

Mar. 29, 2024

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

Patients who meet all of the following selection criteria.
1) Patients with biallelic pathogenic variants in the SLC25A13 gene
2) Patients who are capable of using CGM according to the instruction manual
3) Age: Patients who are at least 6 years old and younger than 60 years old at the time of consent.
4) Gender: Any gender
5) Patients who are able to make outpatient visits to the research institution according to the research schedule during the period of participation in the study.
6) Patients who have given written consent of their own or their surrogate's decision based on a thorough understanding of the study upon thorough explanations of their participation in the study.

Patients who meet at least one of the following exclusion criteria will be excluded.
1) Patients with severe symptoms (liver tumor, acute pancreatitis, altered mental status, psychiatric symptoms, etc.) due to the underlying disease and who are considered to be in critical condition
2) Patients who have undergone liver transplantation
3) Patients with pancreatic insufficiency
4) Patients with a history of serious drug or food allergy such as anaphylactic shock or a history of serious adverse reactions
5) Those who are pregnant or may become pregnant
6) Those who are breast-feeding.
7) Other persons who are judged by the principal investigator (or subinvestigator) to be inappropriate to participate in this study.

Both

citrin deficiency

Oral administration of triheptanoin (5-35% of estimated daily energy requirements)

D006111

Frequency (percentage), nature and extent of adverse events during 14 weeks of treatment

(1) Percentage change in time below target glucose range (TBR) over a 24-hour period ((value after 10-14 weeks of treatment - value before the start of treatment (week 0)) / value before the start of treatment (week 0) x 100)
(2) Percentage change in area under the glucose curve (AUC) for 6 hours during the night (from 0:00 to 6:00) ((value after 10-14 weeks of treatment - value before the start of treatment (week 0))/value before the start of treatment (week 0) x 100)
(3) Change in QOL scores
(4) Frequency of hypoglycemia symptoms (cold sweat, palpitations, dizziness, fatigue, nausea, tremors in arms and legs)
(5) Changes in the patient's subjective evaluation of the treatment before and after administration of the study drug

+81-3-3433-1111

Mar. 05, 2024

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