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Pediatric Clinical Trials at the National Institutes of Health

Pediatric Clinical Trials at the National Institutes of Health. Anne Zajicek, MD, PharmD Obstetric and Pediatric Pharmacology Branch Center for Research for Mothers and Children National Institute of Child Health and Human Development, NIH. Outline. ClinicalTrials.gov listings

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Pediatric Clinical Trials at the National Institutes of Health

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  1. Pediatric Clinical Trials at the National Institutes of Health Anne Zajicek, MD, PharmD Obstetric and Pediatric Pharmacology Branch Center for Research for Mothers and Children National Institute of Child Health and Human Development, NIH

  2. Outline • ClinicalTrials.gov listings • Pediatric Clinical Trials in the Obstetric and Pediatric Pharmacology Branch • Pediatric Pharmacology Research Units Network • Best Pharmaceuticals for Children Act • Grants

  3. ClinicalTrials.gov • Industry: 5250 total studies (adults + children), 813 enrolling children (15 %) • Other Federal Agencies: 627 total studies, 151 in children (24 %) • NIH: 4892 total studies, 1400 in children (29 %)

  4. Pediatric Clinical Trials: ongoing/all trials including closed

  5. Break-down by Institute

  6. Obstetric Pharmacology Research Units • Funded in 2004 • Define pharmacokinetic (PK), pharmacodynamic (PD), pharmacogenomic (PG) changes that occur during pregnancy • Opportunistic PK studies • PK studies of 17α hydroxyprogesterone caproate used to prevent of preterm delivery • PK/PD/PG studies in glyburide

  7. Pediatric Pharmacology Research Units • First funded in 1994 • Purpose: to improve knowledge of PK, PD, PG in children • 13 sites • Performs Phase 1, 2, 3 studies in children of all ages

  8. NIH Rapid Access to Interventional Development (RAID) • Purpose: to make available, on a competitive basis, certain critical resources needed for the development of new small molecule therapeutic agents • Projects associated with NICHD • Metastin • Friedrich ataxia • More funding available than projects- please apply • Contact information • (301) 594-4660 • nih-raid@mail.nih.gov

  9. Best Pharmaceuticals for Children Act • Legislation signed into law 2002, sunsets Oct 2007 • Purpose: improve pediatric labeling • Extends marketing exclusivity for on-patent drugs if FDA-requested studies (as described in the Written Request) are performed • For off-patent drugs: NIH prioritizes drugs for study, sponsors clinical trials if the Written Request declined

  10. Master List of all Off-Patent Drugs which lack adequate pediatric labeling N=200 • Consider for prioritizing: • Availability of S/E data • Are additional data needed? • Will new studies produce • health benefits? • Reformulation? Consultation with experts in pediatric practice and research Develop, prioritize, publish an Annual List N=5-15

  11. Azithromycin Baclofen Bumetanide Dobutamine Dopamine Furosemide Heparin Lithium Lorazepam Rifampin Sodium nitroprusside Spironolactone Drugs on the Priority List: January 2003FR 68: 13; Jan 21, 2003

  12. Ampicillin/sulbactam Diazoxide Isoflurane Lindane Meropenem Metoclopramide Piperacillin/ tazobactam Promethazine Drugs on the Priority List: August 2003FR 68: 156; Aug 13, 2003

  13. Drugs on the Priority List: February 2004FR 69:30; February 13, 2004 • Ampicillin • Ketamine • Vincristine • Dactinomycin • Metolazone

  14. Drugs on the Priority List: January 2005FR 70:17; January 27, 2005 • Ivermectin • Hydrocortisone valerate • Hydrochlorothiazide • Ethambutol • Griseofulvin • Methadone • Hydroxychloroquine • Sevelamer • Morphine

  15. Drugs on the Priority List: January 2006 FR 71:79; April 25, 2006 • Sickle Cell Disease: Hydroxyurea • Oncology: Methotrexate, Daunomycin • Poisoning: Pralidoxime • Other disease areas of continuing interest/discussion: • ADHD • Hypertension • Parasitic Diseases • Influenza

  16. Off-Patent Written Requests Issued by FDA • Lindane • Rifampin • Methicillin resistant Staph aureus endocarditis • CNS shunt infections • Meropenem • Vincristine • Actinomycin-D • Ampicillin • Griseofulvin • Methotrexate

  17. Off-Patent Written Requests Issued by FDA • Lorazepam • Sedation • Status epilepticus • Nitroprusside • Azithromycin • for Ureaplasma pneumonia • Azithromycin for Chlamydia • Baclofen* • Lithium • Morphine* • Hydroxyurea*

  18. Off-Patent Written Requests Declined by Industry • Rifampin • MRSA endocarditis • CNS shunt infections • Meropenem • Vincristine • Dactinomycin • Ampicillin • Griseofulvin • Methotrexate

  19. Off-Patent Written Requests Declined by Industry • Lorazepam • Sedation • Status epilepticus • Nitroprusside • Azithromycin • for Ureaplasma pneumonia • Azithromycin for Chlamydia • Baclofen* • Lithium • Morphine*

  20. On-Patent Written Requests Declined by Industry • Morphine* • Bupropion • Sevelamer • Zonisamide • Hydroxyurea • Baclofen* • Dexrazoxane • Eletriptan • Metoclopramide*

  21. Pediatric Studies that are on-going under BPCA

  22. Contracts • Contracts awarded • Lorazepam for sedation • Is lorazepam more effective than midazolam? • How should lorazepam be given, by intermittent bolus or by continuous infusion? • Lorazepam for status epilepticus • What is the correct dose of lorazepam to treat seizures? • Is lorazepam more effective or safer than diazepam for children in status epilepticus?

  23. BPCA Studies on-going • Nitroprusside for controlled hypotension • What is the dose-response relationship for nitroprusside? • Is there tolerance to the hypotensive effect? • Ketamine (pre-clinical) • Does ketamine cause apoptosis? • Lithium for acute mania • Is lithium safe and effective for the treatment of acute mania in children with bipolar disease? • Baclofen for spasticity in cerebral palsy • Is oral baclofen safe and effective in children with cerebral palsy?

  24. BPCA Studies On-going • Methylphenidate • Replication of cytogenetic studies in children receiving methylphenidate

  25. Intra-Agency Agreements • Vincristine • Actinomycin-D • What is the incidence of toxicity in children? • What are the toxicities, and is there a relationship to dose, diagnosis, age, weight? • Is there a relationship between systemic exposure and efficacy/toxicity? • Methotrexate and neurocognition • Is the method of dosing MTX related to neurocognitive changes in children with high-risk acute lymphocytic leukemia? • Daunomycin: relationship of disposition to body size • Hydroxyurea in sickle-cell disease

  26. Contracts under Negotiation • Meropenem for complicated intra-abdominal infections

  27. Grant • Morphine • To evaluate the relationship of developmental stage (defined by both gestational and postnatal age) to the disposition of morphine and the clinical response

  28. Coordinating Center • Purpose: • Coordinate patient enrollment • Coordinate, monitor data collection • Report adverse events and other data (usually blinded) to Data Safety Monitoring Board (DSMB) • Analyze, organize results into a supplemental NDA for submission to FDA • Contract awarded to Premier Research, Philadelphia, Pennsylvania

  29. Summary • NIH is sponsoring clinical studies in children that will produce improvements in pediatric therapeutics

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