DRPLA

Clinical trial pipeline · Data from ClinicalTrials.gov

See which DRPLA trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 2 trials

RecruitingNot Applicable

NCT05489393

CureDRPLA Global Patient Registry

The objective of the CureDRPLA Global Patient Registry is to establish a longitudinal database of patient-reported data on individuals affected with Dentatorubr…

United States100 participantsStarted 2021-03-01

RecruitingNot Applicable

NCT06273150

Dentatorubral-pallidoluysian Atrophy Natural History and Biomarkers Study

DRPLA Natural History and Biomarkers Study (DRPLA NHBS) is a prospective observational study that will lay the foundation for clinical trials in DRPLA. The aims…

United States, United Kingdom225 participantsStarted 2022-05-01

Treatment: Positive genetic test for pathological expansion in ATN1

All recent trials (5 shown)

Active — Not RecruitingPhase 1/2

NCT06706388

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with dentatorubral-pallidoluysia…

United States1 participantsStarted 2024-02-21

Treatment: nL-ATN1-002

Active — Not RecruitingPhase 1/2

NCT07221760

Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with dentatorubral-pallidoluysia…

United States1 participantsStarted 2025-11-06

Treatment: nL-ATN1-001

DRPLA

Trial phase breakdown

Trial status

Actively recruiting — 2 trials

CureDRPLA Global Patient Registry

Dentatorubral-pallidoluysian Atrophy Natural History and Biomarkers Study

All recent trials (5 shown)

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation

Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation

Trial phase breakdown

Trial status

Actively recruiting — 2 trials

CureDRPLA Global Patient Registry

Dentatorubral-pallidoluysian Atrophy Natural History and Biomarkers Study

All recent trials (5 shown)

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation

Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation

Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation