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, contact the Research and Clinical Trials team: 713-500-7164

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The CTPND research and clinical trials team supports the planning and implementation of projects to take the administrative and logistical burdens off clinicians.

 

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.