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Inhibition of Reverse Transcription in Aicardi-Goutières Syndrome

NCT04731103 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 13

Last updated 2024-05-09

No results posted yet for this study

Summary

Aicardi-Goutières syndrome (AGS) is a disease of children, particularly affecting the brain and the skin. There is a close link between AGS and increased amounts of a chemical called interferon. Normally humans only produce interferon when they are infected with a virus. In AGS, there is no viral infection. Instead, the cells in the cells of affected patients are confused into thinking that their own genetic material is coming from a virus. As a result they produce interferon all the time, which acts as a poison that damages the cells. The Investigators wish to treat AGS patients with drugs called reverse transcriptase inhibitors (RTIs), used to fight the HIV-1 virus that causes AIDS. The investigators will monitor the effect of treatment on interferon levels, and look at other markers which might give us clues to how the drugs are working. The trial is funded by the Medical Research Council, and involves experts based in Edinburgh, Birmingham, Manchester and Great Ormond Street Hospital.

Conditions

  • Aicardi-Goutières Syndrome

Interventions

DRUG

Abacavir (ABC)

Tablets or oral solution

DRUG

Lamivudine (3TC)

Tablet or oral solution

DRUG

Abacavir (ABC)+Lamivudine (3TC)+Zidovudine (AZT)

Tablet or oral solution

Sponsors & Collaborators

Principal Investigators

  • Yanick Crow · University of Edinburgh

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
CROSSOVER

Eligibility

Min Age
3 Months
Max Age
15 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-08-24
Primary Completion
2024-03-11
Completion
2024-03-11

Countries

  • United Kingdom

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04731103 on ClinicalTrials.gov