Clinical study of NPC-15 in patients with xeroderma pigmentosum (XP) sunburn enhancement.

JRCT ID: jRCT2051210181

Registered date:23/02/2022

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Xeroderma pigmentosum (XP) sunburn-enhanced
• Date of first enrollment: 20/04/2022
• Target sample size: 20
• Study type: Interventional
• Intervention(s): NPC-15 once daily 0.5-4 mg (0.067 mg / kg in terms of body weight) for 62 weeks (Crossover study: 10 weeks , Open study: 52 weeks), when orally administered before bedtime.

Outcome(s)

• Primary Outcome: Minimum Erythema Dose (MED) 72 hours (+/- 6 hours) after UV irradiation on the 15th day (stage I and stage II) of investigational drug administration

Secondary Outcome

Efficasy
1) Minimum Erythema Dose (MED) at 24 hours, 48 hours, and 96 hours (+/- 6 hours) after UV irradiation on the 15th day (stage I and stage II) of investigational drug administration.
2) Evaluation of Melanin index regarding pigmented area in MED judgment area
3) Pigment spot inspection (number, area, color tone)
4) Neurological symptoms (severity scale nerve score, hearing test, 5m gait test)
5) Presence or absence of onset of acute skin symptoms
6) Presence or absence of skin cancer
Safety
Adverse events, Side effects, Intensity of drowsiness and dizziness (VAS scale)

Key inclusion & exclusion criteria

• Age minimum: >= 1age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1. Sunburn-enhanced type (XP-A, XP-B, XP-D, XP-F, XP-G) patients diagnosed by the genetic test among patients diagnosed with xeroderma pigmentosum based on the XP diagnosis flowchart of the Xeroderma pigmentosum medical treatment guideline (created in 2015 by the Japan Dermatology Society)
2. Patients who are 1 year old or older (weight 7.5 kg or more) at the time of consent acquisition. However, after confirming the safety of up to the 10th case of the crossover study stage I in the subjects aged 6 years or older by the Efficacy Safety Evaluation Committee, patients aged 1 to under 6 years will be enrolled.
3. Patients who have been fully informed about the conduct of this study and / or their substitute have been fully informed and consented to participate in this study in writing. If even patients aged 20 years or older does not consent to participate in this study in writing due to intellectual disability, obtain consent from the substitute

Exclude criteria

1. Patients with a history of allergies to melatonin or ramelteon
2. Patients receiving other investigational drugs (including placebo) within 4 months prior to obtaining consent
3. Patients who have been using the following drugs for 4 weeks before the start of study drug administration
1) Melatonin (including health foods containing melatonin as the principal component)
2) Fluvoxamine maleate (Lubox, Depmerol, etc.)
4. Patients who are pregnant or may become pregnant
5. Patients judged by the investigator to be ineligible for this study

Related Information

• Primary Sponsor: Ono Ryusuke
• Secondary Sponsor: Moriwaki Shinichi