Phase 2 of the Extension Clinical Trial of Sialic Acid - Extended Release Tablet Recruiting Participants

For those who are interested Ultragenyx has now  opened the extension of Phase 2 study or SA-ER. Seems like you have had to participate in Phase 2 to qualify for this study.  For those interested, please contact the  sites listed.

An Open Label Phase 2 Extension Study of(SA-ER)Tablet

This study is currently recruiting participants.

Verified July 2013 by Ultragenyx Pharmaceutical Inc

Sponsor:

Ultragenyx Pharmaceutical Inc

Information provided by (Responsible Party):

Ultragenyx Pharmaceutical Inc

ClinicalTrials.gov Identifier:

NCT01830972

First received: April 10, 2013

Last updated: July 3, 2013

Last verified: July 2013

History of Changes

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  Purpose

GNE myopathy or hereditary inclusion body myopathy (HIBM) is a severe progressive metabolic myopathy caused by a defect in the biosynthetic pathway for sialic acid(SA). The purpose of the study is to measure long term safety and the effects of Sialic Acid-Extended Release (SA-ER) pills.

Condition	Intervention	Phase
GNE Myopathy HIBM	Drug: SA-ER tablets	Phase 2

Study Type:	Interventional
Study Design:	Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	An Open-label Phase 2 Extension Study to Evaluate the Long Term Safety and Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

Further study details as provided by Ultragenyx Pharmaceutical Inc:

Primary Outcome Measures:

• Assess long-term safety of 6000 mg/day SA-ER in HIBM subjects [ Time Frame: approximately 3 years ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	45
Study Start Date:	June 2013
Estimated Primary Completion Date:	June 2016 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: open label, 6000 mg/day	Drug: SA-ER tablets

Detailed Description:

GNE myopathy or hereditary inclusion body myopathy (HIBM) is a severe progressive metabolic myopathy caused by a defect in the biosynthetic pathway for sialic acid (SA). Substrate replacement therapy is a potential therapeutic strategy based on the success of replacing missing SA and reducing muscle disease in a relevant mouse model of the human disease (Malicdan et al., 2009). Successful use of SA replacement therapy in humans is believed to depend upon providing steady long-term exposure to the compound in an extended release form (such as Sialic Acid-Extended Release [SA-ER]), given SA's short half-life. Following a Phase 1 study to establish the pharmacokinetics for SA-ER and an ongoing Phase 2 study to assess the pharmacodynamic effect of restoring sialylation of muscle by treatment over 48 weeks, Ultragenyx is conducting this study to evaluate the long term safety and efficacy of SA-ER treatment for up to 36 additional months.

  Eligibility

Ages Eligible for Study:	18 Years to 65 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	Yes

Criteria

Inclusion Criteria:

• Enrollment in and successful completion of the UX001-CL201 protocol. Must be willing and able to provide written, signed informed consent after the nature of the study has been explained, and prior to any research-related procedures.
• Must be willing and able to comply with all study procedures
• Sexually active subjects must be willing to use an acceptable method of contraception while participating in the study.
• Females of childbearing potential must have a negative pregnancy test at Baseline and be willing to have additional pregnancy tests during the study.
• Females considered not of childbearing potential include those who have been in menopause for at least two years, or have had tubal ligation at least one year prior to Baseline, or who have had total hysterectomy.

Exclusion Criteria:

• Pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the study
• Use of any investigational product (other than SA-ER tablets) to treat HIBM
• Ingestion of ManNAc or similar SA producing compounds
• Has had any hypersensitivity to SA or its excipients that, in the judgment of the investigator, places the subject at increased risk for adverse effects
• Have any co-morbid conditions, including unstable major organ-system disease(s) that in the opinion of the Investigator, places the subject at increased risk of complications, interferes with study participation or compliance, or confounds study objectives.

  Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01830972

Locations

United States, California
UCLA Medical Center	Recruiting
Los Angeles, California, United States
Contact: Perry Shieh, MD     310-994-5142     pshieh@mednet.ucla.edu
Principal Investigator: Perry Shieh, MD
United States, New York
NYU Medical Center	Recruiting
New York, New York, United States, 10016
Contact: Heather Lau, MD     212-263-8344     Heather.Lau@nyumc.org
Contact: Pankaj Patel     212-263-0139     pankaj.patel@nyumc.org
Principal Investigator: Heather Lau, MD
Israel
Hadassah Univeristy Hospital	Recruiting
Jerusalem, Israel
Contact: Yoseph Caraco, MD     972-2-6778584     caraco@hadassah.org.il
Principal Investigator: Yoseph Caraco, MD

Sponsors and Collaborators

Ultragenyx Pharmaceutical Inc

  More Information

No publications provided

Responsible Party:	Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier:	NCT01830972     History of Changes
Other Study ID Numbers:	UX001-CL202
Study First Received:	April 10, 2013
Last Updated:	July 3, 2013
Health Authority:	United States: Food and Drug Administration

ClinicalTrials.gov processed this record on July 04, 2013

http://clinicaltrials.gov/ct2/show/NCT01830972?term=hibm&rank=5