Physician-initiated clinical trial in patients with 5-aminolevulinic acid-induced ATR-X syndrome.

JRCT ID: jRCT2051220062

Registered date:14/07/2022

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: ATR-X syndrome
• Date of first enrollment: 14/07/2022
• Target sample size: 5
• Study type: Interventional

Intervention(s)

Containing 5-aminolevulinic acid (5-ALA) hydrochloride and sodium ferrous citrate (SFC)
Tablets (25 mg 5-ALA hydrochloride/39.2 mg SFC per tablet, NPJ005) for 24 weeks

Outcome(s)

Primary Outcome

1 Safety: (frequency and extent of adverse events and side effects)
2 Efficacy: assessment of development using the new version of the K-type developmental test
3 Efficacy: assessment of social competence with the S-M Social Competence Test

Secondary Outcome

1 Assessment of symptoms by M-CHAT
2 Assessment of social developmental gaze patterns using gaze measurement device, Gazefinder
3 Assessment of gastrointestinal symptoms
4 Development (body weight)
5 Blood levels of 5-ALA and PP9

Key inclusion & exclusion criteria

• Age minimum: >= 2age old
• Age maximum: Not applicable
• Gender: Male

Include criteria

1) meet the Definite diagnostic criteria for the ATR-X syndrome
2) Age > 2 years at the time of enrolment
3) Weigh 10 kg or more at the time of enrolment
4) Have a parent or reliable carer
5) Have given written consent to participate in this clinical trial. (If the patient is under 18 years of age, consent should be obtained from the individual and his/her legal guardian). If the patient's intellectual disability makes it difficult to obtain consent from the individual, consent should be obtained from the legal guardian.

Exclude criteria

1) Those with complications not directly related to the ATR-X syndrome (e.g. perinatal hypoxic encephalopathy, which may affect the assessment)
2) Those with serious complications (e.g. haematological, renal, hepatic, cardiac, cancer)
3) Those with a history of major illness or surgery in the past, which is judged to affect the assessment of central nervous system function
4) Those with no visual function that allows recognition of test indices
5) Those with concomitant or previous history of photosensitivity or porphyria
6) With a history of hypersensitivity to 5-ALA, porphyrins or iron preparations
7) Those with haemochromatosis
8) have taken supplements containing 5-ALA as a main ingredient within 3 months prior to enrolment (same date 3 months earlier)
9) Participating in another clinical study or trial involving an intervention within 3 months (same date 3 months earlier) prior to obtaining consent
10) Any other case that the investigator or sub-investigator deems inappropriate for the safe conduct of this clinical trial.

Related Information

• Primary Sponsor: Wada Takahito