IRB #: 3378-B FB

                                                                                                 Approved:(1/22/2001)

 

 

                                                                              PATIENT NAME:______________________

                                                                                 PATIENT DOB:   ______________________

                                                                                 OHSU MED REC #:____________________

                                                                                 TODAY’S DATE:______________________

 

 

                                     OREGON HEALTH SCIENCES UNIVERSITY

                                                           CONSENT FORM

 

 Treatment of Congenital Urea Cycle Disorders with IV Sodium Benzoate & Sodium Phenylacetate                 

PRINCIPAL INVESTIGATOR: Robert D. Steiner, MD, (503) 494-7859.          

Co-Investigators:  Markus Grompe MD; Cary Harding, MD; Susan Hayflick; MD; David Koeller,MD      

 

PURPOSE:   [“You” refers to you or your child throughout this consent form] You have been invited to participate in this research because you have been diagnosed with an enzyme deficiency that results in abnormal ammonia metabolism.  Due to this enzyme deficiency, you may develop high levels of ammonia in the blood which causes severe toxicity including vomiting, tiredness and coma.  Dr. Robert Steiner and other physicians, who are listed above as coinvestigators and who are on the staff of the Metabolic Clinic, are participating in a national collaborative research study with Ucyclyd Pharma Inc. to help determine how to best treat people with urea cycle disorders who develop very high ammonia levels.  In the event of an acute crisis, we would use an intravenous (IV) dosage form of Sodium Benzoate and Sodium Phenyl acetate to lower the elevated ammonia levels rapidly.  The IV drug becomes useful when you are very ill and are not capable of taking medicine by mouth because it is delivered right into the blood stream.  This IV treatment has been approved for research purposes by the US Food and Drug Administration in order to establish whether or not ammonia levels can be controlled by using this medication.  The focus of this study is to show whether IV Sodium Benzoate and Sodium Phenylacetate actually can regulate the ammonia level.  Your participation is expected to last until this medication is approved for general use the FDA and/or the study is terminated.

 

PROCEDURE:In the event that the your ammonia level rises markedly above the normal level and is associated with severe symptoms you will be hospitalized and IV Sodium Benzoate and Sodium Phenyl acetate will be given until you improve and the blood ammonia level returns to normal. The medication will be dosed as directed by Ucyclyd Pharmaceuticals.  It will be mixed into intravenous fluids and given continuously until the ammonia level has fallen.  This medication would be given along with other standard therapy for high ammonia levels in urea cycle disorders including intravenous fluids, intravenous glucose and often arginine. This IV medication would be particularly useful if you are too ill to tolerate oral medications.  However, if this therapy is not effective in lowering the ammonia levels within 12 hours, it may be necessary to treat the ammonia accumulation with some form of dialysis, a procedure used to filter the ammonia out of your blood.  If this is necessary,  your permission will be required.

 

RISK AND DISCOMFORT: As with all patients with these disorders, blood  samples (about 1 tsp) will be drawn to monitor effectiveness of treatment and metabolic stability every 4 to 12 hours while receiving the medication.  These tests are no more and no different from those used in normal management of urea cycle disorders and are considered standard of care. Blood drawing may cause some pain and carries a small risk of bleeding and/or discomfort.

 

This IV preparation consisting of Sodium Benzoate and Sodium Phenylacetate may cause nausea and vomiting.  If you have ulcers, these may be worsened with this medication.  Mild hyperventilation has been noted with these medications.  The medication may not work.  If it does not work to bring the ammonia down, brain damage or even death may occur.

 

BENEFITS: You may or may not personally benefit by participating in this study.  However, by serving as a research subject you may contribute new information which may benefit patients like you in the future.  Treatment with IV Sodium Benzoate and Sodium Phenylacetate can be used in patients too ill to tolerate oral medication and may speed the lowering of toxic ammonia in acute crisis management.

 

CONFIDENTIALITY:  Research records will be kept confidential to the extent provided by law. Your name, dosage of the medication, anthropometrics and biochemical data as well as any side effects to the medication will be made available to Ucyclyd Pharma, Inc. at 8125 North Hayden Road, Scottsdale, Arizona 85258-2463; Phone: (602) 667-3914.  Your records may be reviewed by the US Food and Drug Administration, Washington, D.C.. Neither your name nor your identity will be used for publication or publicity purposes.

 

ALTERNATIVES: Dialysis would be an alternative treatment.

 

COST:  OHSU Outpatient Pharmacy will charge you for these drugs. Costs will be about $975.00 per vial. Some, but not all, insurance plans will cover this expense. Laboratory analysis will be about $300 per complete set of analysis. Since these are standard tests required to monitor these disorders most insurance plans cover this expense but you will be required to pay for this expense if your insurance plan doesn’t cover it.

 

LIABILITY: 

 

1) It is not the policy of the US Department of the Health and Human Services or any federal agency funding the research project in which you are participating to compensate or provide medical treatment for human subjects in the event the research results in physical injury.  

 

2) Ucycyld Pharma, Inc. does not have any program to provide compensation to you if you experience injury or other bad effects which are not the fault of the investigators.  However, you have not waived your legal rights by signing this form.

 

3)  The Oregon Health Sciences University, as a public institution, is subject to the Oregon Tort Claims Act (ORS 30.260 through 30.300).  If you suffer any injury and damage from this research project through the fault of the University, its officers or employees, you have the right to bring legal action against the University to recover the damage done to you subject to the limitations and condition of the Oregon Tort Claims Act.  You have not waived your legal rights by signing this form.  For clarification on this subject, or if you have further questions, please call the OHSU Legal Department,(503) 494-5222.

 

PARTICIPATION.  Dr. Robert Steiner or his designee has explained this study to you and answered your questions.  If you have other questions or research related problems, you may reach Dr. Steiner at (503)494-7859 or (503)494-8311.  If you have questions about your rights as a research subject, you may contact the OHSU Institutional Review Board at (503)494-7887.  You may refuse to participate or you may withdraw from this study at any time without affecting your relationship with or your treatment at the Oregon Health Sciences University.

Your signature below indicated that you have read the foregoing and agree to participate in this study.  You will receive a copy of this consent form.

 

 

_________________________                       _________________________________________

DATE                                                                Signature of Patient/Parent

 

 

 

_________________________                       _________________________________________

DATE                                                                Signature of Supervising Physician

 

 

_________________________                       _________________________________________

DATE                                                                Signature of Witness

 

 

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                                                                                          IRB #: 3378-B FB                           

Approval Date:1/22/2001                    

 

OREGON HEALTH SCIENCES UNIVERSITY

Child Assent Form

(Use for children ages 7 to 18)

 

Title: Treatment of Congenital urea Cycle Disorders with IV Sodium Benzoate & Sodium Phenylacetate

 

Principal Investigator: Robert D. Steiner, MD, (503) 494-7859

Co-Investigators:  Markus Grompe MD; Cary Harding, MD; Susan Hayflick, MD; David Koeller,MD

 

Part I

 

Dr._________________has explained this research study to me.  I know how it may or may not help me.  I also know that this study will help doctors know more about Urea Cycle Disorders.

 

            1) The study will ask me to explain what I will do and what will happen in this

            study to be sure I understand the study.

 

2) The study doctor will ask me if I have any questions or want to know anything else about this study or Urea Cycle Disorders.

 

            3) The study doctor will ask me to explain some of the good and bad things that might

            happen to me if I enter this study.

 

Part II

 

I have thought about being a part of this study.  I have asked and received answers to my questions.  I agree to be in this study.  I know that I don’t have to agree to be in the study.  Even though I agree to be in it now, I know I may feel differently later on and can ask to stop being in the study.  I know that I may talk with my parents and/or doctor about not being in this study at any time.

 

 

 

 

_______________________________________            __________________________

Name/Signature                                                                                                Date