Could HIV drugs tame a rare immune disease in kids?
NCT ID NCT02363452
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This small pilot study tested whether drugs normally used for HIV could help children with Aicardi-Goutières syndrome (AGS), a rare genetic disorder that causes severe brain inflammation. The trial gave 11 children a combination of three reverse transcriptase inhibitors to see if it could lower their interferon levels, a key marker of inflammation. The goal was to check safety and whether the drugs could normalize the immune overreaction seen in AGS.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Reverse transcriptase inhibitors (zidovudine, lamivudine, abacavir)
- What this could lead to
- If it works, this could point toward a treatment that reduces harmful inflammation in children with Aicardi-Goutières syndrome.
- What could go wrong
- This is a very small, early-phase pilot study with only 11 participants. It is designed to check if the drug can lower certain immune markers, not yet to prove it helps symptoms or changes the course of the disease. Side effects from the drugs are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
11 people
The number who actually took part.
- Started
-
Sep 2015
- Finished
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Jun 2018
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
1 month to 17 years
- Sex
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Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * A molecular diagnosis of AGS i.e. biallelic or known dominant mutations, with pathogenicity assessed using our extensive mutation database / functional data, in any of TREX1, RNASEH2A, RNASEH2B, RNASEH2C and SAMHD1 genes * A pre-defined interferon signature (consistently present, moderate or high, on at least three occasions, over a period of 6 months prior to enrolment in the study) * Age ≥ 1 month and \< 18 years (either sex) * Patient beneficiary or affiliated to " health insurance" * Written informed consent Exclusion Criteria: * Pre-existing disease, not due to AGS, which would preclude the use of zidovudine, Lamivudine and abacavir (as currently assessed in routine clinical HIV-related practice) * HLA B57-01 positive result, which indicates a greater risk of abacavir hypersensitivity reaction * Patients with abnormally low neutrophile counts (\<0.75 x 109/l), or abnormally low haemoglobin levels (\<7.5 g/dl or 4.65 mmol/l)(zidovudine contraindication) * Positive serology for HIV, HBV * Known history of cirrhosis and history of clinically relevant hepatitis within last 6 months * Moderate to severe renal impairment * Pregnancy, breastfeeding * Patient participating to a biomedical research with drug
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Hôpital Necker - Enfants Malades
Paris, 75015, France
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