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Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

NCT01634750 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 26

Last updated 2017-10-19

No results posted yet for this study

Summary

Background:

\- Hereditary inclusion body myopathy (HIBM) is a genetic disorder caused by mutations in a gene called GNE. This gene is responsible for producing a sugar called sialic acid. Low levels of sialic acid may cause muscle problems. Symptoms of HIBM include walking difficulties and muscle weakness, which usually start in a person s 20s or 30s and become worse over time.

Researchers are studying a drug called ManNAc. It may be useful for treating HIBM. However, this drug is still being tested. Researchers want to see how ManNAc is absorbed into and removed from the blood. They will not be looking specifically at whether ManNAc can stop or slow the symptoms of HIBM.

Objectives:

* \<TAB\>To study how MaNAc is absorbed into and removed from the blood in people with HIBM.
* \<TAB\>To study of safety of ManNAc in people with HIBM.

Eligibility:

\- Individuals between 18 and 70 years of age who have HIBM.

Design:

* Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected.
* Participants will have a 3 to 4-day inpatient stay for the main part of the study.
* Participants will be divided into groups of six. In each group, four will take ManNAc and two will take a placebo. Participants will not know which one they will receive.
* Participants will have a single dose of either ManNAc or placebo. They will be monitored for any possible side effects. Frequent blood samples will be collected during the 4-day stay.
* No treatment for HIBM will be provided as part of this study.

Conditions

  • Hereditary Inclusion Body Myopathy (HIBM)
  • GNE Myopathy

Interventions

DRUG

ManNAc

Single dose

Sponsors & Collaborators

  • National Center for Advancing Translational Sciences (NCATS)

    collaborator NIH

  • Therapeutics for Rare and Neglected Diseases (TRND)

    collaborator NIH

  • National Human Genome Research Institute (NHGRI)

    lead NIH

Principal Investigators

  • Nuria Carrillo-Carrasco, M.D. · National Human Genome Research Institute (NHGRI)

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-09-11
Completion
2013-05-29

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01634750 on ClinicalTrials.gov