Follow-up Study Using the Rare Disease Platform Registry for Inherited Metabolic Diseases: RADDAR-J [30]

Follow-up Study Using the Rare Disease Platform Registry for Inherited Metabolic Diseases: RADDAR-J [30] - Follow-up Study Using the Rare Disease Platform Registry for Inherited Metabolic Diseases: RADDAR-J [30]

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-UMIN000056715

Enrollment

1000

Registered

2025-04-01

Start date

2021-07-20

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

inherited metabolic diseases

Interventions

None listed

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1) Patients diagnosed with an inherited metabolic disease that meets the diagnostic criteria for either Pediatric Chronic Specific Diseases or Designated Intractable Diseases. 2) Patients who have provided written consent to participate in the study.

Exclusion criteria

Exclusion criteria: Patients deemed unsuitable for inclusion based on the judgment of the researchers or related personnel.

Design outcomes

Primary

Measure	Time frame
The following will be evaluated every 1-2 years: 1) Development (developmental index or intelligence quotient) 2) Growth (height, weight) 3) Presence of metabolic events (such as episodes of acidosis or hyperammonemia)	—

Countries

Japan

Contacts

Public ContactHideo Sasai

GIfu University Department of Pediatrics,Graduate School of Medicine

sasai-gif@umin.net058-230-6386

Outcome results