Trial record 2 of 20 for:    HPN-100

A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders (UCDs)

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified September 2014 by Hyperion Therapeutics, Inc.
Sponsor:
Information provided by (Responsible Party):
Hyperion Therapeutics, Inc.
ClinicalTrials.gov Identifier:
NCT02246218
First received: September 17, 2014
Last updated: September 18, 2014
Last verified: September 2014
  Purpose

This is an open-label study consisting of a transition period to RAVICTI, followed by a safety extension period for at least 6 months and up to 24 months of treatment with RAVICTI, depending on age at enrollment. It is designed to capture information important for evaluating safety, pharmacokinetics and efficacy in young children.

Subjects who are followed by or referred to the Investigator for management of their UCD or assessment of high blood ammonia may be eligible for this study. Subjects eligible for this study will include patients ranging from newborn to < 2 years of age with either a diagnosed or clinically suspected UCD.


Condition Intervention Phase
Urea Cycle Disorder
Drug: RAVICTI
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open Label Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate (GPB; RAVICTI®) in Pediatric Subjects Under Two Years of Age With Urea Cycle Disorders (UCDs)

Resource links provided by NLM:


Further study details as provided by Hyperion Therapeutics, Inc.:

Primary Outcome Measures:
  • Successful transition to RAVICTI with controlled ammonia (i.e. no clinical symptoms and ammonia < 100 µmol/L) [ Time Frame: Up to 24 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Rate of hyperammonemic crises during the first 6 months on RAVICTI [ Time Frame: Every month for the first 6 months and every 3 months thereafter up to 24 months ] [ Designated as safety issue: Yes ]
  • Rate of Adverse Events [ Time Frame: Every month for the first 6 months and every 3 months thereafter up to 24 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 24
Study Start Date: November 2014
Estimated Study Completion Date: June 2018
Estimated Primary Completion Date: May 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: RAVICTI
RAVICTI Oral Liquid should be administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
Drug: RAVICTI
Other Names:
  • HPN-100
  • Glycerol Phenylbutyrate
  • GPB
  • GT4P

  Eligibility

Ages Eligible for Study:   up to 2 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male and female subjects up to 2 years of age
  • Signed informed consent by subject's parent/legal guardian
  • UCD diagnosis or suspected diagnosis of any subtype, except N-acetyl glutamate synthetase deficiency. If UCD has not been previously confirmed by genetic testing, consent must be obtained from parent/legal guardian prior to perform genetic testing. If genetic testing is inconsistent with or excludes a UCD diagnosis, the subject will be withdrawn from the study.

Exclusion Criteria:

  • Use of any investigational drug within 30 days of Day 1
  • Uncontrolled infection (viral or bacterial) or any other condition known to precipitate hyperammonemic crises. Once these precipitating factors are medically controlled, patients presenting in crisis are eligible.
  • Any clinical or laboratory abnormality of Grade 3 or greater severity according to the Common Terminology Criteria for Adverse Events (CTCAE) v4.03, except Grade 3 elevations in ammonia and liver enzymes, defined as levels 5-20 times the upper limit of normal in alanine aminotransferase (ALT), aspartate aminotransferase (AST), or gamma glutamyl transpeptidase (GGT) in a clinically stable subject
  • Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the subject at increased risk by participating in this study
  • Known hypersensitivity to phenylacetate (PAA) or phenylbutyrate (PBA)
  • Liver transplantation, including hepatocellular transplant
  • Subjects on hemodialysis at time of initiating RAVICTI
  • Subjects on RAVICTI for UCD management
  • Currently treated with Carbaglu® (carglumic acid)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02246218

Contacts
Contact: Kathleen Tam, MPH, MBA (650) 745-3153 kathleen.tam@hyperiontx.com
Contact: Craig James, RN (650) 745-7840 craig.james@hyperiontx.com

Locations
United States, California
Loma Linda University Health Care Not yet recruiting
Loma Linda, California, United States, 92354
Contact: Melissa Rundquist    909-651-5002    mrundquist@llu.edu   
Contact: Lila Dalton, RN    (909) 651-5002    ldalton@llu.edu   
Principal Investigator: James Bartley, MD         
Stanford University School of Medicine Not yet recruiting
Palo Alto, California, United States, 94034
Contact: Elisabeth Merkel, RN    650-736-0644    merkel@stanford.edu   
Contact: May Zepeda    (650) 724-0177    zepeda@stanford.edu   
Principal Investigator: William Berquist, MD         
United States, District of Columbia
Children's National Medical Center Not yet recruiting
Washington, District of Columbia, United States, 20010
Contact: Kara Simpson    202-476-6216    ksimpson@childrensnational.org   
Principal Investigator: Nicholas Ah Mew, MD         
United States, Florida
University of Florida Not yet recruiting
Gainesville, Florida, United States, 32610
Contact: Christel Gross, RN    352-294-5511    grosscd@peds.ufl.edu   
Principal Investigator: Roberto Zori, MD         
United States, Indiana
Indiana University School of Medicine Not yet recruiting
Indianapolis, Indiana, United States, 46202
Contact: Susan S Romie, MS    317-278-6650    sromie@iu.edu   
Principal Investigator: Bryan Hainline, MD, PhD         
United States, Maine
Maine Medical Center Not yet recruiting
Portland, Maine, United States, 04102
Contact: Susan Mortenson, RN    207-661-7646      
Contact: Tim Bilodeau    (207) 396-8074    bilodt@mmc.org   
Principal Investigator: Wendy Smith, MD         
United States, Michigan
Children's Hospital of Michigan Not yet recruiting
Detroit, Michigan, United States, 48201
Contact: Allison Bannick, RN    303-832-9261    abannick@dmc.com   
Principal Investigator: Robert Conway, MD         
United States, Minnesota
University of Minnesota Not yet recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Sara Elsbecker    612-626-5275    selsbeck10@umphysicians.umn.edu   
Contact: Lisa Hostetler    (612) 273-0691    hoste005@umn.edu   
Principal Investigator: Susan Berry, MD         
United States, New York
Mount Sinai School of Medicine Not yet recruiting
New York, New York, United States, 10029
Contact: Luca Fierro    212-659-1477    luca.fierro@mssm.edu   
Principal Investigator: George Diaz, MD, PhD         
United States, Ohio
University Hospitals Case Medical Center Not yet recruiting
Cleveland, Ohio, United States, 44106
Contact: Audrey Lynn, PhD    216-844-7124    audrey.lynn@UHhospitals.org   
Principal Investigator: Shawn McCandless, MD         
United States, Oregon
Oregon Health & Science University Not yet recruiting
Portland, Oregon, United States, 97239
Contact: Julie Martin    503-494-7608    martijul@ohsu.edu   
Contact: Tina Marrone    (503) 494-2775    marronet@ohsu.edu   
Principal Investigator: Cary Harding, MD         
United States, Pennsylvania
The Children's Hospital of Philadelphia Not yet recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Denise De Paul    267-426-6845    depaul@email.chop.edu   
Contact: Lauren Vernau    (267) 426-2586    chorneyl@email.chop.edu   
Principal Investigator: Can Ficicioglu, MD, PhD         
Children's Hospital of Pittsburgh of UPMC Not yet recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Jennifer Baker    412-692-6378    jennifer.baker@chp.edu   
Principal Investigator: Gerard Vockley, MD, PhD         
United States, Washington
Seattle Children's Hospital Not yet recruiting
Seattle, Washington, United States, 98105
Contact: Linnea Brody    206-884-1264    linnea.brody@seattlechildrens.org   
Principal Investigator: Lawrence Merritt, MD         
Canada, Alberta
Alberta Children's Hospital Not yet recruiting
Calgary, Alberta, Canada, T3B 6A8
Contact: Connie Mohan    (403) 955-7186    cmohan@ucalgary.ca   
Principal Investigator: Aneal Khan, MD         
Canada, Ontario
The Hospital for Sick Children Not yet recruiting
Toronto, Ontario, Canada, M5G 1X8
Contact: Liora Caspi    (416) 813-7654 ext 328027    liora.caspi@sickkids.ca   
Principal Investigator: Andreas Schulze, MD         
Sponsors and Collaborators
Hyperion Therapeutics, Inc.
Investigators
Principal Investigator: Susan Berry, MD University of Minnesota - Clinical and Translational Science Institute
  More Information

No publications provided

Responsible Party: Hyperion Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT02246218     History of Changes
Other Study ID Numbers: HPN-100-009
Study First Received: September 17, 2014
Last Updated: September 18, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Hyperion Therapeutics, Inc.:
Urea Cycle Disorder
UCD
GT4P
Buphenyl
Glycerol Phenylbutyrate
Ravicti
Hyperammonemia
Sodium Phenylbutyrate
GPB
HPN-100

Additional relevant MeSH terms:
Disease
Urea Cycle Disorders, Inborn
Pathologic Processes
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
4-phenylbutyric acid
Glycerol
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Cryoprotective Agents
Protective Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on September 22, 2014