Clinical Trials for Urea Cycle Disorders
In addition to the studies being conducted by the Urea Cycle Disorders Consortium, the following is list of clinical trials for urea cycle disorders. For more trial information and specific eligibility criteria, click on the link for each study. For information about how clinical trials are conducted, see About Clinical Trials below.
CLINICAL TRIALS FOR INVESTIGATIONAL DRUG, HPN-100 (Sponsored by Hyperion Therapeutics)
BACKGROUND: Many UCD families and patients have experienced major difficulties with administration (giving the drug), compliance (tolerating the daily regimen) or tolerance of side effects of Buphenyl® (sodium phenylbutyrate). An investigational drug, related to Buphenyl® in chemical structure, is being studied for use in urea cycle disorders. This investigational drug, called HPN-100, is a nearly tasteless, odorless liquid formulation which is intended to work similarly to Buphenyl in removing ammonia, but without the pill burden or need to mask taste. Three teaspoons of HPN-100 equals approximately 40 tablets of sodium phenylbutyrate. The sponsor of the trial, Hyperion Therapeutics, will provide HPN-100 free of charge to trial participants and cover the costs of laboratory tests and physician visits associated with the trial, as well as reasonable travel expenses to a trial location.
UPDATE! PHASE 2 Study of the Safety and Tolerability of HPN-100 compared to Sodium Phenylbutyrate in Children Aged 6-17 Years with Urea Cycle Disorders, with Long Term Safety Extension. NOW RECRUITING INTO THE LONG-TERM SAFETY EXTENSION STUDY WITH NO OVERNIGHT STAYS.
Sponsor: Hyperion Therapeutics
Purpose: This protocol (HPN-100-005) is the first study of HPN-100 in pediatric subjects with urea cycle disorders. The initial study was designed to assess the safety of HPN-100 and its ability to control blood ammonia as compared with sodium phenylbutyrate. After a two week controlled period, all participants received HPN-100 in a 12-month safety study. This study is now open to additional patients for the 12-month extension to evaluate the long-term safety of HPN-100.
Trial Locations:
UCLA: Contact Naghmeh Dorrani (310)825-8084. Principal Investigator: Stephen Cederbaum, MD
Children's National Medical Center: Contact Kara Lord (202)476-6216, Principal Investigator: Uta Lichter, MD
Mount Sinai School of Medicine NY: Contact Christina Guzman, (212)241-680, Principal Investigator: George Diaz, MD
Seattle Children's Hospital: Contact Linnea Brody, (206)987-3694, Principal Investigator: Lawrence Merritt, MD Children's Hospital of Wisconsin, Milwaukee: Contact Abby Donovan (414)266-3289, Principal Investigator: William Rhead, MD
The Hospital for Sick Children, Toronto, Ontario, Canada: Contact Mohammed Hussain (416)813-5340 ext 2,
Principal Investigator: Annette Feigenbaum, MD
UPDATE! PHASE 3 Study of the Efficacy and Safety of HPN-100 for the Treatment of Adults with Urea Cycle Disorders. NEW SITES RECRUITING
Sponsor: Hyperion Therapeutics
Purpose: This protocol (HPN-100-006) is a randomized, active-controlled, double-blind, cross-over study in subjects with UCD who are currently being treated with sodium phenylbutyrate. Subjects will be randomly assigned to receive either HPN-100 plus sodium phenylbutyrate placebo, or sodium phenylbutyrate and a placebo of HPN-100 for two weeks, and then crossed over to receive the other treatment for two weeks. Subjects who complete this study and meet entry criteria will be offered the opportunity to enroll in the twelve-month open-label safety study of HPN-100.
Trial Locations:
Stanford University, Stanford CA: Contact Vivian de Leon (650)736-8166
Denver Children's Hospital, Aurora CO: Contact Hazel Senz, RN (720)777-5380
Maine Medical Center, Portland Maine: Contact Susan Mortenson, RN (207)396-8078
SNBL Clinical Pharmacology Center, Baltimore MD: Contact Rob Casper (410)706-8818
Mount Sinai School of Medicine, New York NY: Contact Christina Guzman (212)241-6805
Nationwide Children's Hospital, Columbus OH: Contact Kimberly Regis (614)722-3565
Oregon Health & Science University, Portland OR: Contact Tina Marrone (503)418-3620
University of Pittsburgh, PA: Contact Judith Henry, RN (412)692-7530
Baylor College of Medicine, Houston TX: Contact Mary Mullins (832)822-4263
Hospital for Sick Children, Toronto, Canada: Contact Mohammed Hussain (416)813-7654 ext 2636
Case Western Reserve, Cleveland OH: Contact Christine Heggie (216)844-7124
Milwaukee Regional Medical Center, Milwaukee WI: Contact Jeff Crawford (414)266-7254
Long Beach Memorial Medical Center, Long Beach CA: Contact Nan O'Donnell (562)492-6383
University of Florida, Gainesville: Contact Christel Gross (352)265-0032
University of Minnesota: Contact Teri Carlson (612)625-0673
Children's National Medical Center, DC: Contact Kara Simpson (202)476-6216
UCLA, Los Angeles CA: Contact Nagmeh Dorrani (310)825-8084
University of Utah, Salt Lake City: Contact Carrie Bailey (801)587-3605
Yale School of Medicine, New Haven CT: Contact Kristin Defrancesco (203)737-2585
Tufts-New England Medical Center, Boston MA: Ellen Santo (978)884-5464
Additional sites will be added April/May 2010
PHASE 3 Study of the Safety of HPN-100 for the Long-Term Treatment of Urea Cycle Disorders.
Sponsor: Hyperion Therapeutics
Purpose: This protocol (HPN-100-007)is a long-term safety study of HPN-100 in patients 18 years and older with urea cycle disorder. Subjects will be assessed regularly for safety and control of blood ammonia. Hyperammonemic events will be characterized with respect to contributing factors such as intercurrent illness, diet, and noncompliance with medication.
Trial Locations: See Locations for Phase 3 Study of the Efficacy and Safety of HPN-100 for the Treatment of Adults with Urea Cycle Disorders
About Clinical Trials
What is a clinical trial?
A clinical trial is a study to determine whether a treatment improves a clinical condition or a disease. A clinical trial is an "experiment" which is the most rigorous type of study. The protocol for the study is designed to test the specific idea that a treatment is effective and safe. Each study must be approved and monitored by an independent committee of physicians, statisticians and members of the community called an Institutional Review Board (IRB). The IRB ensures that risks are acceptably small and in relationship to potential benefits.
Participating in clinical trials offers many opportunities to learn more about treatments and disease. However, it is important to have a good understanding of the risks and benefits of any study when making a decision to participate.
Many individuals question why they should consider participating in a clinical trial, what it is like to participate in a clinical trial and what they should expect. An individual’s decision to enter a clinical trial is a very personal one and there are a variety of reasons for considering taking part in a study. Having hope for a future cure, greater understanding of their own illness, feeling better and contributing to research and society are some personal reasons participants choose to enter trials. Participants in clinical trials may also receive new treatments before they are readily available to nonparticipants.
What are the phases of clinical trials?
Clinical trials are conducted in phases. In orphan diseases like urea cycle disorders, trial size is smaller than the average numbers presented below due to the limited patient population. Each phase has a specific purpose and answers different questions:
Phase I Trials: An experimental drug or treatment is tested in a very small group of people (20-80) to evaluate its safety, determine a safe dosage range, and identify side effects. This is a safety study.
Phase II Trials: The experimental drug or treatment is now given to a larger group of people (100-300) to determine its efficacy (whether it does what it is supposed to do) and to further evaluate its safety.
Phase III Trials: The experimental drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, and to collect information that will allow the drug or treatment to be used safely.
Phase IV Trials: These are post-marketing trials, usually conducted by the pharmaceutical company after the drug receives FDA approval and has been made available to the public. These studies collect additional information on the drug's risks, benefits and optimal uses.
Who can participate in a clinical trial?
All clinical trials have specific guidelines about who is eligible to participate. Some studies seek participants with illnesses or conditions, while others require healthy volunteers.
Who sponsors clinical trials?
Clinical trials are often funded by pharmaceutical companies to test new drugs. However, other funders of clinical trials are researchers, individuals, medical institutions, nonprofit organizations, foundations, voluntary health organizations, as well as federal agencies like the National Institutes of Health (NIH).
Where can I ask questions or find more information about the clinical trials for urea cycle disorders?
Contact Cindy Le Mons, Executive Director of the National Urea Cycle Disorders Foundation (626)578-0833. The Foundation is here to support you and answer any questions you may have about the trials or about how to participate in a trial.
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