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Recruiting NCT05848271

NCT05848271 Natural History Study of Patients with HPDL Mutations

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Clinical Trial Summary
NCT ID NCT05848271
Status Recruiting
Phase
Sponsor University of California, San Diego
Condition Mitochondrial Encephalomyopathies
Study Type OBSERVATIONAL
Enrollment 50 participants
Start Date 2023-05-18
Primary Completion 2026-12-31

Eligibility & Interventions

Sex All sexes
Min Age N/A
Max Age N/A
Study Type OBSERVATIONAL
Interventions
Patient RegistryDry blood spots sampling

Eligibility Fast-Check

Enter your details for a quick preliminary check. This does not replace medical advice.

What to Expect as a Participant

This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.

This trial targets 50 participants in total. It began in 2023-05-18 with a primary completion date of 2026-12-31.

⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.

Brief Summary

This study uses medical records that allow retrospective data extraction of clinical manifestation to assess the natural history of HPDL mutations

Eligibility Criteria

Inclusion Criteria: * Any individuals diagnosed with HPDL variants * Clinical diagnosis can include: * HPDL-related hereditary spastic paraplegia (HSP) * HPDL-related neonatal mitochondrial encephalopathy * Spastic paraplegia -83 (SPG83) * Neurodevelopmental disorder with progressive spasticity and brain white matter abnormalities (NEDSWMA) Exclusion Criteria: * Any known genetic abnormality (other than HPDL mutation) * Any condition that, in the opinion of the Site Investigator, could put the participant at undue risk and/or would ultimately prevent the completion of study procedures

Contact & Investigator

Central Contact

Eun Hae Lee

✉ gleesonlab@health.ucsd.edu

📞 8582460547

Principal Investigator

Joseph Gleeson

PRINCIPAL INVESTIGATOR

UCSD

Frequently Asked Questions

Who can join the NCT05848271 clinical trial?

This trial is open to participants of all sexes, studying Mitochondrial Encephalomyopathies. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.

Is NCT05848271 currently recruiting?

Yes, NCT05848271 is actively recruiting participants. Contact the research team at gleesonlab@health.ucsd.edu for enrollment information.

Where is the NCT05848271 trial being conducted?

This trial is being conducted at San Diego, United States.

Who is sponsoring the NCT05848271 clinical trial?

NCT05848271 is sponsored by University of California, San Diego. The principal investigator is Joseph Gleeson at UCSD. The trial plans to enroll 50 participants.

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ClinicalMetric — Independent clinical trial intelligence platform. Not affiliated with NIH, ClinicalTrials.gov, the U.S. FDA, or any pharmaceutical company, hospital, or clinical research organization. Trial data is sourced from ClinicalTrials.gov for informational purposes only and does not constitute medical advice. Do not make any treatment, enrollment, or health decisions based solely on information found here — always consult a qualified healthcare professional. Full Disclaimer  ·  Last Reviewed: April 2026  ·  Data Methodology