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Aprepitant for the Treatment of CLIFAHDD

StatusRecruiting
PhaseEarly Phase 1
Started
View on ClinicalTrials.gov ↗

Record History

  1. Recruitment opened
    • Start date pushed from Oct 1, 2026 to Oct 31, 2026
    • Site details revised
  2. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
There are no drug treatments for individuals with Congenital Contractures of the Limb and Face, Hypotonia, and Developmental Delay (CLIFAHDD), an ultra-rare and severe neurodevelopmental disease. The investigators have learned more about the cause of the disease and researched possible treatments based on both the cause of the disease and the way certain drugs work. The investigators are interested in understanding the safety of using aprepitant (a drug already approved by the United States Food \& Drug Administration \[FDA\] to treat nausea and vomiting for kids and adults who are receiving chemotherapy) on people diagnosed with CLIFAHDD. The overall goal of this Phase 1 study is to study whether this drug is safe for daily use in individuals with CLIFAHDD and to find more information on how it impacts daily functioning and quality of life. The total study will take around 8 months total. This includes the 90 days before the drug starts and the 21 days after the drug ends.
Trial Details
NCT Number NCT07845825
Lead Sponsor The University of Texas Health Science Center at San Antonio
Collaborators: Channeling Hope Foundation, Cures Within Reach
Conditions
(from sponsor)		 CLIFAHDD, Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay, NALCN Channelopathy, Ataxia - Other, Neurodevelopmental Disorder (Diagnosis)
Related terms Ataxia, Channelopathies, Disease, Facies, Learning Disabilities, Muscle Hypotonia, Neurodevelopmental Disorders
Enrollment 5 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov