BTD-001 for Idiopathic Hypersomnia

(ARISE2 Trial)

Not currently recruiting at 31 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Balance Therapeutics

Must not be taking: Benzodiazepines, Barbiturates, Alcohol

Disqualifiers: Sleep apnea, Obesity, Seizure, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new oral treatment called BTD-001 for adults with idiopathic hypersomnia, a condition causing excessive daytime sleepiness with no known cause. The study compares BTD-001 to a placebo (a pill with no active medicine) to determine its effectiveness in reducing sleepiness. Participants will receive both the treatment and the placebo at different times to assess effectiveness. The trial seeks individuals who have experienced hypersomnia since they were between 10 and 30 years old and often feel very sleepy during the day. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people, offering participants a chance to contribute to the development of a potential new therapy.

Will I have to stop taking my current medications?

The trial requires participants to stop taking medications for idiopathic hypersomnia or any other prohibited medications before joining. If you are on stimulants, you must keep your regimen stable throughout the trial.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that BTD-001, a drug known as pentylenetetrazole, has been used in treatments worldwide for many years. It has been applied to conditions like dementia and to assist with breathing, suggesting that the drug is generally well-tolerated. However, some individuals have reported mental fogginess while taking BTD-001. This side effect is important to consider, especially for those with idiopathic hypersomnia, as it might affect daily activities.

Overall, BTD-001 has been used safely in humans for various conditions. However, since this study specifically tests the drug for idiopathic hypersomnia, its safety for this condition is still under careful examination. Prospective participants should discuss possible side effects with the research team to fully understand the risks involved. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Idiopathic Hypersomnia?

Unlike the standard treatments for idiopathic hypersomnia, which mainly focus on managing sleepiness through stimulating the nervous system or adjusting neurotransmitter levels, BTD-001 offers a potentially novel approach. Researchers are excited about BTD-001 because it could work through a different mechanism, possibly targeting the underlying causes of excessive daytime sleepiness rather than just alleviating symptoms. This new mechanism of action might provide more effective and lasting relief for patients who have not responded well to existing therapies.

What evidence suggests that BTD-001 might be an effective treatment for Idiopathic Hypersomnia?

Research has shown that BTD-001, which participants in this trial may receive, is being tested for its potential to help people with idiopathic hypersomnia, a condition characterized by excessive daytime sleepiness. Early results suggest that BTD-001 might improve wakefulness and daily functioning. The treatment blocks certain brain signals that may cause sleepiness. Although detailed information is still being gathered, initial studies have shown promise in managing symptoms of this disorder. ^{3 6 7 8 9}

Who Is on the Research Team?

Morgan Lam

Principal Investigator

Chief Operating Officer

Are You a Good Fit for This Trial?

Inclusion Criteria

Onset of hypersomnia between age 10 and 30 years of age

An Epworth Sleepiness Scale score of greater than or equal to 11

Able to comply with requirements for concomitant medications and consumption of caffeine, alcohol, tobacco and nicotine (if on stimulants, agree to adhere to stable regimen throughout the trial)

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either BTD-001 or placebo in a crossover design to evaluate efficacy and safety

8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• BTD-001
• Pentetrazol (PTZ)
• Placebo oral capsule

How Is the Trial Designed?

Group I: Subject Receives Placebo first Experimental Treatment 2 Interventions

Group II: Subject Receives BTD001 first Experimental Treatment 2 Interventions

Who Is Running the Clinical Trial?

Balance Therapeutics

Lead Sponsor

Trials

4

Recruited

220+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03542851

A Study of Oral BTD-001 in Adults With Idiopathic ... The objectives of this study are two-fold: 1) To evaluate the efficacy of BTD-001 in subjects with IH as reflected by changes in cognitive, sleep, functional, ...

2. mayo.edu mayo.edu/research/clinical-trials/cls-20441971

Study to Assess the Safety and Effectiveness of Oral BTD- ... The purpose of this study is to evaluate the safety, tolerability and effectiveness of BTD-001 in subjects with idiopathic hypersomnia (IH)

3. trial.medpath.com trial.medpath.com/clinical-trial/7851bb1119e8339d/nct02512588-oral-btd-001-idiopathic-hypersomnia

A Study of Safety and Efficacy of BTD-001 in Treatment of ... This is a randomized, placebo-controlled, double-blind, multiple cohort, fixed-dose multiple crossover, dose-finding study of oral BTD-001 in adult patients ...

4. withpower.com withpower.com/trial/phase-3-disorders-of-excessive-somnolence-4-2018-3aad4

A Study of Oral BTD-001 in Adults With Idiopathic ... The study aims to see if BTD-001 can help these individuals stay awake and improve their daily lives. Researchers will also check if the medication is safe and ...

5. hypersomniafoundation.org hypersomniafoundation.org/document/ih-brain-fog-and-objective-sleep-measures/

are reports of mental fog from patients with idiopathic ... This is a Phase II, double blind, randomized 2-period crossover study in evaluating the safety and efficacy of an oral GABA antagonist (BTD-001). Patients ...

6. io.nihr.ac.uk io.nihr.ac.uk/wp-content/uploads/2022/01/11738-Pentetrazol-for-IH-or-Narcolepsy-Type-2-V1.0-FEB2018-NON-CONF.pdf

Pentetrazol for idiopathic hypersomnia and narcolepsy type 2 Pentetrazol is a medicinal product being developed for idiopathic hypersomnia and narcolepsy type 2. It acts by blocking GABA, a substance that ...

7. adisinsight.springer.com adisinsight.springer.com/drugs/800038901

Balance Therapeutics - Pentylenetetrazole - AdisInsight BTD 001, a pentylenetetrazole for the oral treatment of Down syndrome, idiopathic hypersomnia, narcolepsy, and potentially neurodegenerative conditions such ...

8. app.trialscreen.org app.trialscreen.org/trials/phase-1-down-syndrome-drug-btd-young-adults-adolescents-trial-actrn12612000652875

Study of the Drug BTD-001 in Young Adults and ... BTD-001, has been used for several decades around the world as a treatment for dementia, a respiratory stimulant and as an ingredient of a cough medication.

9. sigma.larvol.com sigma.larvol.com/product.php?e1=698031&tab=newstrac

pentylenetetrazole (BTD-001) News These data provide a basis for using zebrafish to study the etiology of TS abnormalities and suggest t hat fever may be a particular risk for TS patients. Here, ...