NCT02845609

Brief Summary

Sialic Acid-Extended Release (SA-ER, aceneuramic acid, UX001) is an extended release formulation of sialic acid (SA, also known as N-acetylneuraminic acid or NANA). The SA-ER is currently studied as a substrate replacement therapy for patients with GNE myopathy. The investigators plan to study the SA-ER compound in a cohort of five patients with GNE-related thrombocytopenia.

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
5

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Sep 2016

Shorter than P25 for phase_2

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 24, 2016

Completed
3 days until next milestone

First Posted

Study publicly available on registry

July 27, 2016

Completed
1 month until next milestone

Study Start

First participant enrolled

September 1, 2016

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2017

Completed
Last Updated

January 30, 2018

Status Verified

July 1, 2016

Enrollment Period

1 year

First QC Date

July 24, 2016

Last Update Submit

January 29, 2018

Conditions

Thrombocytopenia

Keywords

Congenital macrothrombocytopeniaSialic acidBleeding

Outcome Measures

Primary Outcomes (1)

  • Increase in platelet count from baseline

    3 month

Secondary Outcomes (1)

  • Incidence and frequency of AEs and SAEs (Safety)

    3 months

Study Arms (1)

Intervention

EXPERIMENTAL

Sialic acid-Extended release 2000 mg, three times per day (TID) for 3 months

Drug: Sialic Acid-Extended Release

Interventions

Sialic Acid-Extended ReleaseDRUG

Oral drug of Sialic acid

Also known as: aceneuramic acid, UX001
Intervention

Eligibility Criteria

Age18 Years - 45 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

  • Patients with GNE-related macrothrombocytopenia in the investigated family

You may not qualify if:

  • non consenting for this study
  • pregnancy

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

ThrombocytopeniaHemorrhage

Condition Hierarchy (Ancestors)

Blood Platelet DisordersHematologic DiseasesHemic and Lymphatic DiseasesCytopeniaPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 24, 2016

First Posted

July 27, 2016

Study Start

September 1, 2016

Primary Completion

September 1, 2017

Study Completion

September 1, 2017

Last Updated

January 30, 2018

Record last verified: 2016-07