← Back to Search

Anti-epileptic Drug

Fenfluramine for Dravet Syndrome (ORCHID Trial)

Phase 3

Recruiting

Research Sponsored by UCB BIOSCIENCES, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participant is ≥1 to <2 years of age as of the day of the first administration of study drug

Participants must have ≥4 countable motor seizures (CMS) during the Baseline Period. The CMS include distinct seizures of generalized tonic clonic, bilateral clonic, bilateral tonic, atonic (drop), bilateral tonic/atonic, or focal to bilateral tonic-clonic type. If the participant fails to have ≥4 qualifying seizures in 28 days, the Baseline Period may be extended by an additional 14 days with Sponsor approval. Participants with an extended Baseline Period must still have ≥4 CMS in the 28 days immediately prior to the day of the first administration of study drug.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 1 (visit 3), week 4 (visit 5), week 8 (visit 7), week 12 (visit 8), week 16 (visit 9), week 20 (visit 10), week 26 (visit 11), week 39 (visit 12), week 52 (visit 13), postdose safety follow-up (visit 14), compared to baseline

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial examines the effects of a drug on infants 1-2 years old w/ Dravet Syndrome to see if it's safe & tolerable.

Who is the study for?

This trial is for infants aged 1 to less than 2 years with Dravet syndrome, weighing at least 8 kg. They must have a diagnosis according to ILAE criteria and be on stable epilepsy medication for at least 4 weeks. Participants need to have had at least four motor seizures in the month before starting the study.

What is being tested?

The study tests the safety and tolerability of fenfluramine hydrochloride, given daily in doses ranging from 0.2 to 0.8 mg/kg, in young children with Dravet syndrome.

What are the potential side effects?

While not explicitly listed here, common side effects of fenfluramine may include decreased appetite, drowsiness, fatigue, diarrhea, and potential heart-related issues.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am between 1 and 2 years old.

Select...

I have had 4 or more seizures in the last month.

Select...

My body weight is at least 8 kg.

Select...

I have been diagnosed with Dravet syndrome by a specialist.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 1 (visit 3), week 4 (visit 5), week 8 (visit 7), week 12 (visit 8), week 16 (visit 9), week 20 (visit 10), week 26 (visit 11), week 39 (visit 12), week 52 (visit 13), postdose safety follow-up (visit 14), compared to baseline

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 1 (visit 3), week 4 (visit 5), week 8 (visit 7), week 12 (visit 8), week 16 (visit 9), week 20 (visit 10), week 26 (visit 11), week 39 (visit 12), week 52 (visit 13), postdose safety follow-up (visit 14), compared to baseline

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 1 (visit 3), week 4 (visit 5), week 8 (visit 7), week 12 (visit 8), week 16 (visit 9), week 20 (visit 10), week 26 (visit 11), week 39 (visit 12), week 52 (visit 13), postdose safety follow-up (visit 14), compared to baseline for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change from Baseline in QT interval corrected by Fridericia (QTcF) at Visit 13 (End of Treatment/Early Termination (EOT/ET))

Change from Baseline in body weight (Z-score) at each visit

Change from Baseline in recumbent length (Z-score) at each visit

+3 more

Secondary study objectives

Achievement of a CGI-I rating of "much improved" or "very much improved" as assessed by the parent/caregiver at Week 20

Achievement of a Clinical Global Impression - Improvement (CGI-I) rating of "much improved" or "very much improved" as assessed by the Principal Investigator at Week 20

Area under the plasma concentration time curve from time zero to 24 hours (AUC0 24) for steady-state fenfluramine and norfenfluramine at Week 12

+5 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Fenfluramine Open-labelExperimental Treatment1 Intervention

All study participants will initiate fenfluramine hydrochloride (HCl) treatment at 0.2 mg/kg/day in the Dose-Finding Period and may be up-titrated to a maximum of 0.8 mg/kg/day based on the Investigators discretion. The dose of fenfluramine HCl can be flexibly titrated during the Maintenance Period. Study participants, who discontinue early will participate in the Taper Period. All participants will complete an End of Treatment (EOT) Visit.

0 / 4Eligibility criteria met