Geranylgeraniol Supplementation in Patients With Mevalonate Kinase Deficiency

ConditionMevalonate Kinase Deficiency

Trial statusRecruiting

Trial phaseNot applicable

Trial typeInterventional

Biological sexAll

Age12+

SponsorUniversity Hospital, Motol

About this trial

Mevalonate kinase deficiency (HyperIgD syndrome, HIDS) is an inborn error of immunity caused by a block in the mevalonate pathway. The subsequent lack of isoprenoids with antiinflammatory properties might contribute to the autoinflammatory nature of the disease.

A pilot study aims to verify the safety and efficacy of the dietary supplement Geranylgeraniol, aiming at a mitigation of isoprenoids deficiency caused by the above-mentioned block in the MVK pathway.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

Patients older than 12 years with genetically confirmed MVK deficiency

Overall good clinical status apart from the clinical presentation of the MVK deficiency

Normal liver tests

Compliance with the study protocol

Disqualifiers

None

Trial design

Design model

Single group

Treatments tested in this trial

geranylgeraniol
Dietary supplement
GG Pure (Extendlife Natural Products) containing GG gold®30 Annatto Extract, 500 mg capsule, use for 3 months, once daily

Treatment groups

6 Participants

are divided into 1 treatment group

Group A: patients with MVK deficiencyExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

Change in inflammatory parameters

Monitoring inflammatory markers before and after intervention - CRP (C reactive protein) and SAA (serum amyloid A) levels will be measured before and after the intervention (time 0 and time 3 months of GG use). These values tend to be elevated in patients with MVK deficiency due to their inflammatory status compared to normal values defined in clinical laboratories (UHMotol CRP \< 8 mg/l, SAA \< 10 mg/l). The expected outcome would result in a decrease in inflammatory markers.

Time frame

3 monhts of intervention

Secondary outcomes

Change in the clinical status of patients

Monitoring of general clinical status and defined parameters - general condition, fatigue, fever, skin manifestations, abdominal pain, and headache using a questionnaire. The questionnaire is designed for daily monitoring of the parameters mentioned above. The scale of the questionnaire is from 1 to 10, with 1 being the best, normal condition, and 10 being the worst degree of disability.

Time frame

3 months

Sponsors and contacts

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University Hospital, Motol

Lead sponsor

Washington University School of Medicine

Collaborator

Source ClinicalTrials.gov

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