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Anti-epileptic Drug

Fenfluramine for Dravet Syndrome (ORCHID Trial)

Q: Is enrollment available for this clinical study at the present time?

Indeed, as evidenced by the clinicaltrials.gov website, this medical trial is recruiting for 20 participants from 1 site. The study was initially posted on November 13th 2023 and its most recent update was made on November 9th of the same year.

Q: Who would be considered a qualified participant in this medical research project?

To qualify for this investigation, applicants must be diagnosed with dravet syndrome and between 1 year old and 23 months of age. The study is aiming to enrol 20 eligible individuals.

Q: Are members of the younger demographic eligible for this research study?

Eligibility for this trial is restricted to individuals between 1 year and 23 months of age.

Q: What is the number of participants enrolled in this experiment?

Affirmative. Clinicaltrials.gov's data reveals that this trial, which was initially uploaded on November 13th 2023, is actively recruiting patients. The study seeks to enrol 20 individuals from a single site.

Q: To what extent can Fenfluramine Open-label be detrimental to individuals?

The safety of Fenfluramine Open-label has been evaluated by the Power team and scored as a 3. This reflects that there is clinical data which shows its efficacy, along with multiple rounds of testing to ensure its safety.

Q: What is the primary goal of this experiment?

This clinical trial spans up to 52 weeks, and its primary outcome is the incidence of VHD following treatment. Additional secondary objectives include monitoring changes in monthly motor seizure frequency (CMSF) from Baseline during Weeks 1-20, achieving a CGI-I rating of "much improved" or better at Week 20 as rated by caregivers, and assessing AUC0 24 for fenfluramine/norfenfluramine steady state values at Week 12.

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

ORCHID 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.Check my eligibility

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.See study design

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.

ORCHID Trial Eligibility Criteria

Inclusion Criteria

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

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I am between 1 and 2 years old.

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

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My body weight is at least 8 kg.

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I have been diagnosed with Dravet syndrome by a specialist.

ORCHID Trial 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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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 outcome measures

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

Side effects data

From 2020 Phase 3 trial • 262 Patients • NCT02682927

38%

Decreased appetite

21%

Somnolence

19%

Pyrexia

17%

Blood glucose decreased

17%

Echocardiogram abnormal

15%

Diarrhoea

13%

Tremor

10%

Fatigue

Upper respiratory tract infection

Platelet count decreased

Weight decreased

Rash

Cough

Vomiting

Lethargy

Asthenia

Hypoglycaemia

Drooling

Viral infection

Rhinitis

Blood pressure increased

Ataxia

Balance disorder

Abnormal behaviour

Irritability

Urinary tract infection

Varicella

Toxicity to various agents

Hepatic enzyme increased

Nasopharyngitis

Blood pressure diastolic increased

Constipation

Gait disturbance

Influenza

Sinusitis

Fall

Hypotonia

Seizure

Urinary incontinence

Rhinorrhoea

100%

80%

60%

40%

20%

Study treatment Arm

Study 3: ZX008 0.8 mg/kg/Day

Study 1: Placebo

Study 1: ZX008 0.2 mg/kg/Day

Study 1: ZX008 0.8 mg/kg/Day

Study 3: Placebo

Study 3: ZX008 0.2 mg/kg/Day

ORCHID 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

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Who is running the clinical trial?

UCB BIOSCIENCES, Inc.Lead Sponsor

25 Previous Clinical Trials

7,180 Total Patients Enrolled

UCB CaresStudy Director001 844 599 2273

205 Previous Clinical Trials

44,943 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is enrollment available for this clinical study at the present time?

"Indeed, as evidenced by the clinicaltrials.gov website, this medical trial is recruiting for 20 participants from 1 site. The study was initially posted on November 13th 2023 and its most recent update was made on November 9th of the same year."

Answered by AI

Who would be considered a qualified participant in this medical research project?

"To qualify for this investigation, applicants must be diagnosed with dravet syndrome and between 1 year old and 23 months of age. The study is aiming to enrol 20 eligible individuals."

Answered by AI

Are members of the younger demographic eligible for this research study?

"Eligibility for this trial is restricted to individuals between 1 year and 23 months of age."

Answered by AI

What is the number of participants enrolled in this experiment?

"Affirmative. Clinicaltrials.gov's data reveals that this trial, which was initially uploaded on November 13th 2023, is actively recruiting patients. The study seeks to enrol 20 individuals from a single site."

Answered by AI

To what extent can Fenfluramine Open-label be detrimental to individuals?

"The safety of Fenfluramine Open-label has been evaluated by the Power team and scored as a 3. This reflects that there is clinical data which shows its efficacy, along with multiple rounds of testing to ensure its safety."

Answered by AI

What is the primary goal of this experiment?

"This clinical trial spans up to 52 weeks, and its primary outcome is the incidence of VHD following treatment. Additional secondary objectives include monitoring changes in monthly motor seizure frequency (CMSF) from Baseline during Weeks 1-20, achieving a CGI-I rating of "much improved" or better at Week 20 as rated by caregivers, and assessing AUC0 24 for fenfluramine/norfenfluramine steady state values at Week 12."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome

2024. "A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06118255.

All > Dravet Syndrome