The National Institutes of Health is Recruiting for Clinical Trial Phase 1 with ManNac

The National Institutes of Health (NIH) are now recruiting for phase 1 the clinical trial using a substrate called ManNac for HIBM. This would be a good trial for those of us not particicipating in the Sialic Acid trial to consider participating in. I have participated in the NIH - Natural History study for HIBM, this study is still ongoing. I would absolutely recommend the NIH team working with HIBM patients. They are the best in the field for our disorder. I copied the important parts of the study and have also attached the link for those who would like to participate.
Hope continues to strive.
Tara


A Phase 1 Study to Evaluate the Safety and Tolerability of ManNAc in Subjects With Hereditary Inclusion Body Myopathy (HIBM)
This study is not yet open for participant recruitment.
Verified June 2012 by National Institutes of Health Clinical Center (CC)
First Received on July 3, 2012. Last Updated on September 12, 2012 History of Changes
Experimental: ManNAc
Drug: ManNAc
Single dose
Placebo Comparator: Placebo
Drug: ManNAc
Single dose

Detailed Description:

Hereditary inclusion body myopathy (HIBM) is an autosomal recessive, neuromuscular disorder characterized by progressive muscle weakness with onset in early adulthood. The causative gene, GNE, codes for the bifunctional enzyme uridine diphospho (UDP) N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase (GNE/MNK), which catalyzes the first 2 steps in the biosynthesis of sialic acid. The subsequent paucity of sialic acid production is presumed to cause decreased sialylation of HIBM muscle glycoproteins, resulting in muscle deterioration. In this Phase 1, randomized, placebo-controlled, double-blind, escalating single-dose study, we propose to provide ManNAc (N-acetyl-D-mannosamine monohydrate) orally as a liquid solution to 3 cohorts of 6 subjects (Cohorts A, B, C) at doses of 3,000 mg, 6,000 mg, and 10,000 mg ManNAc, respectively, or up to the maximum tolerated dose (MTD). The objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics (PK) of a single dose of orally administered ManNAc to HIBM subjects, to identify the MTD of a single dose of orally administered ManNAc to HIBM subjects, and to explore the effect of a single dose of ManNAc on potential pharmacodynamic (PD) markers of HIBM. All subjects will be randomly assigned in a 2:1 ratio to receive ManNAc (n=4) or placebo (n=2) and the decision to dose-escalate will be the responsibility of the Safety Review Committee (SRC). Safety will be assessed by adverse events (AEs), clinical laboratory tests, vital signs, physical examinations, and electrocardiograms (ECGs). PK will be assessed for both ManNAc and sialic acid. PD will be assessed by some of the following exploratory biomarkers: serum transferrin sialylation status; plasma glycan profiles; and plasma, white cell, platelet, and urine sialylation status (free and bound sialic acid [N-acetylneuraminic acid] (Neu5Ac)] and cytidine 5'-monophosphate [CMP]-Neu5Ac).
Contacts Contacts and Locations Please refer to this study by its ClinicalTrials.gov identifier: NCT01634750 Contacts Contact: Lea B. Latham, R.N. (301) 827-9235 llatham@mail.nih.gov Contact: Nuria Carrillo-Carrasco, M.D. (301) 402-2324 carrilln@mail.nih.gov http://clinicaltrials.gov/ct2/show/NCT01634750?term=hibm&rank=2