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triheptanoin for Rett Syndrome

Phase 2
Waitlist Available
Led By Daniel Tarquinio, DO
Research Sponsored by Center for Rare Neurological Diseases, Norcross, GA
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up visit 2, visit 9 (up to 8.5 months)
Awards & highlights

Study Summary

This study is evaluating whether a new drug may help reduce seizures and improve movement for individuals with Rett syndrome.

Eligible Conditions
  • Rett Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~visit 2, visit 9 (up to 8.5 months)
This trial's timeline: 3 weeks for screening, Varies for treatment, and visit 2, visit 9 (up to 8.5 months) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change in Clinical Seizure Frequency
Change in Dystonia frequency
Secondary outcome measures
Change in Burke-Fahn-Marsden Dystonia Rating Scale (BFM) Score
Change in Child Health Questionnaire-50 (CHQ) Score
Change in Daily 3-axis accelerometry
+24 more

Trial Design

6Treatment groups
Experimental Treatment
Group I: Participants aged 7 to 9 yearsExperimental Treatment1 Intervention
Participants will receive 2.5 grams of triheptanoin per kilogram of body weight daily.
Group II: Participants aged 4 to 6 yearsExperimental Treatment1 Intervention
Participants will receive 3 grams of triheptanoin per kilogram of body weight daily.
Group III: Participants aged 21 years and olderExperimental Treatment1 Intervention
Participants will receive 1.2 grams of triheptanoin per kilogram of body weight daily.
Group IV: Participants aged 15 to 20 yearsExperimental Treatment1 Intervention
Participants will receive 1.5 grams of triheptanoin per kilogram of body weight daily.
Group V: Participants aged 10 to 14 yearsExperimental Treatment1 Intervention
Participants will receive 2 grams of triheptanoin per kilogram of body weight daily.
Group VI: Participants aged 0 months to 3 yearsExperimental Treatment1 Intervention
Participants will receive 4 grams of triheptanoin per kilogram of body weight daily.

Find a Location

Who is running the clinical trial?

Center for Rare Neurological Diseases, Norcross, GALead Sponsor
Ultragenyx Pharmaceutical IncIndustry Sponsor
89 Previous Clinical Trials
179,062 Total Patients Enrolled
1 Trials studying Rett Syndrome
10 Patients Enrolled for Rett Syndrome
Rett Syndrome Research TrustOTHER
7 Previous Clinical Trials
5,376 Total Patients Enrolled
7 Trials studying Rett Syndrome
5,376 Patients Enrolled for Rett Syndrome

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin
Daniel C. Tarquinio 2016. "Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02696044.
All > Rett Syndrome
~1 spots leftby Apr 2025
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