Research

The Center actively participates in a variety of clinical studies including expanded-access programs, patient registries, natural history studies, and investigational clinical trials. This work investigates groundbreaking therapies to find treatment — and ultimately cures — for devastating genetic neurodegenerative diseases of childhood.

For more information: 713-500-7164

 

Center for the Treatment of Pediatric Neurodegenerative Disease Studies

    • An interventional, randomized, double-blind, parallel-group, placebo-controlled, flexible-dose, adaptive study of the efficacy of KL1333 in adult patients with primary mitochondrial disease (PMD). PI: Mary Kay Koenig, MD NCT05650229
    • A long-term safety study to assess the potential for chronic liver injury in participants treated with Epidiolex (Cannabidiol) Oral Solution. PI: Mary Kay Koenig. NCT05044819
    • Child to adult neurodevelopment in gene-expanded Huntington’s Disease (ChANGE HD). Site PI: Erin Furr-Stimming. NCT01860339
    • A phase 4, interventional, multicenter, open-label, single-arm study to assess behavioral and other co-occurring outcomes following treatment with EPID(I/Y)OLEX as add-on therapy in participants (Aged 1 to 65 Years Old) with seizures associated with Tuberous Sclerosis Complex (EpiCom). PI: Sarah Wilson, MD. NCT05864846
    • Effects of N-Acetyl-L-Leucine on ataxia-telangiectasia (A-T): a phase III, randomized, placebo-controlled, double-blind, crossover study. PI: Nivedita Thakur, MD. NCT06673056
    • A phase 3, randomized, double-blind, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with developmental and epileptic encephalopathies (DEE). PI: Shelley Varnado. NCT06719141
    • A phase 3, randomized, double-blind, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with Dravet Syndrome. PI: Shelley Varnado. NCT06660394
    • A phase 2/3 multicenter, randomized, double-blind, placebo-controlled, study to evaluate the efficacy, safety, and tolerability of BHV-7000 in subjects with refractory focal onset epilepsy. PI: Jeremy Lankford. NCT06132893
    • Multicenter, prospective, double-blinded, parallel group, randomized phase III study to evaluate safety and efficacy of different PANZYGA dose regimens in pediatric chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) patients. PI: Shadé Moody. NCT04929236
    • Phase 3, multicenter, randomized, double-blind, group sequential, placebo-controlled study to assess efficacy and safety of Rimegepant for the treatment of migraine (with or without aura) in children and adolescents ≥6 to <18 Years of Age. PI: Mary Kay Koenig. NCT04649242
    • An interventional, efficacy, and safety, phase 3 randomized, double-blind, placebo-controlled study with an open-label extension to investigate Rimegepant in migraine prevention in adolescents 12 to less than 18 years of age with chronic migraine. PI: Mary Kay Koenig. NCT06616194
    • Phase 2b randomized, double-blind, placebo-controlled crossover study evaluating the efficacy and safety of Zagociguat in participants with MELAS (PRIZM). PI: Mary Kay Koenig. NCT06402123
    • A double-blind, randomized, placebo-controlled, multicenter, parallel-group study to evaluate the efficacy, safety, and tolerability of SPN-8178 in adults with focal onset seizures. PI: Mary Kay Koenig. NCT06798896
    • A randomized, double-blind, placebo-controlled, multicenter, phase 3 study to evaluate the safety, tolerability, and efficacy of XEN1101 as adjunctive therapy in primary generalized tonic-clonic seizures. PI: Mary Kay Koenig. NCT05667142
    • Tolerability of transcranial direct current stimulation in pediatric stroke survivors (TOPSS). PI: Stuart Fraser. NCT05812794
    • Focal cerebral arteriopathy steroid (FOCAS) trial. PI: Stuart Fraser. NCT06040255

  • Elamipratide expanded access for patients with mitochondrial myopathy. SPIES-006. Site PI: Mary Kay Koenig. NCT04689360
  • MT-1621 Expanded access for patient with TK2 Deficiency. PI: S. Nick Russo, MD. NCT03845712
  • Expanded access use of Intra-Erythrocyte Dexamethasone Sodium Phosphate for patients diagnosed with ataxia telangiectasia (AT) who completed the phase III IEDAT-02-2015 (ATTeST) study and its open-label extension (IEDAT-03-2018). PI: Mary Kay Koenig. NCT06664853
  • Intermediate size expanded access program (EAP) protocol for patients previously treated with Vatiquinone (PTC 743) with inherited mitochondrial risease. PI: Mary Kay Koenig. NCT05218655

 

  • Predisposition to weight in mitochondrial patients. PI: Mary Kay Koenig
  • Leigh Syndrome roadmap project: a natural history study. PI: S. Nick Russo
  • International pediatric stroke study (IPSS). PI: Stuart Fraser.
  • A novel wearable system for monitoring and alerting for seizures and cardiorespiratory functions. PI: Samden Lhatoo
  • Mitochondrial specimen banking. PI: Mary Kay Koenig
  • Mitochondrial database. PI: Mary Kay Koenig
  • The myelin disorders biorepository project and global leukodystrophy initiative clinical trials network (GLIA-CTN). PI: Nivedita Thakur. NCT03047369

 

Patents/Licenses

Dr. Koenig is listed as an inventor on a patent application held by the Board of Regents of The University of Texas System for a topical composition of rapamycin for the treatment of facial angiofibromas. This IP is covered under a license agreement with AI Therapeutics.