Clinical trials: Dentatorubral-Pallidoluysian Atrophy
DRPLA
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 Dentatorubral-pallidoluysian atrophy (DRPLA) from anywhere in the world.
- Trial name
- NCT05489393
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- CureDRPLA
Dentatorubral-Pallidoluysian Atrophy
Personalized Antisense Oligonucleotide 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-pallidoluysian atrophy (DRPLA) due to a heterozygous pathogenic CAG trinucleotide…
- Trial name
- NCT07084311
- Testing
- nL-ATN1-002
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- n-Lorem Foundation
Dentatorubral-Pallidoluysian Atrophy
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.
- Trial name
- DRPLA NHBS (NCT06273150)
- Testing
- Positive genetic test for pathological expansion in ATN1
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 225 people, healthy volunteers welcome
- Sponsor
- University College, London
Dentatorubral-Pallidoluysian Atrophy
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-pallidoluysian atrophy (DRPLA) due to a heterozygous pathogenic CAG trinucleotide…
- Trial name
- NCT06706388
- Testing
- nL-ATN1-002
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- n-Lorem Foundation
Dentatorubral-Pallidoluysian Atrophy
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-pallidoluysian atrophy (DRPLA) due to a heterozygous pathogenic CAG trinucleotide…
- Trial name
- NCT07221760
- Testing
- nL-ATN1-001
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- n-Lorem Foundation