Miglustat on Gaucher Disease Type IIIB

NCT02520934 | Unknown

• 1 other identifier: 201503076MIPD
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 1

Brief Summary

evaluate the combination therapy with Miglustat and enzyme replacement therapy (ERT) on Gaucher disease

Conditions

Gaucher Disease

Keywords

Enzyme Replacement Therapy; Miglustat; Gaucher Disease

Outcome Measures

Primary Outcomes (1)

• Improve in Purdue Pegboard test speed

  24 months

Study Arms (2)

Case_Miglustat

EXPERIMENTAL

Besides regular ERT, patients in this group also need to take Miglustat for 24 months.

Drug: Miglustat; Drug: ERT

Control

NO INTERVENTION

Patients will be tested for their pupil cycle time.

Interventions

Miglustat DRUG

combine miglustat and ERT to see if neurologic manifestations can be improved

Also known as: Zavesca

Case_Miglustat

ERT DRUG

enzyme replacement therapy

Case_Miglustat

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Confirmed diagnosis of Gaucher Disease: blood test shown lack of beta- glucocerebrosidase, and found L444P homozygous on GBA gene.
• Aged 6 years old or above.
• Already have regular ERT (30-120 IU/kg/ every 2 weeks) at least a year; dosage and frequency of ERT had not been changed in recent 3 months.

You may not qualify if:

• History of tremor and abnormal extremities perception ( pain, numbness, tingle etc.)
• Abnormal kidney function.
• Pregnant or plan to have a baby ( potentially pregnant patient need to be transferred to gynecologist for the test and promise to have proper contraception measures).
• Allergic to Miglustat.
• Age 6-18 years
• No significant physical, mental, or psychiatric problems
• \. Children with eye disease (not include myopia, hyperopia, Astigmatism)

Sponsors & Collaborators

• National Taiwan University Hospital: lead
• Actelion: collaborator

Study Sites (1)

National Taiwan University Hospital

Taipei, 10041, Taiwan

Location

MeSH Terms

Conditions

Gaucher Disease

Interventions

miglustat

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Yin-Hsiu Chien, M.D., Ph.D.

  National Taiwan University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 6, 2015
• First Posted: August 13, 2015
• Study Start: July 1, 2015
• Primary Completion: December 1, 2017
• Study Completion: December 1, 2019
• Last Updated: March 29, 2019

Record last verified: 2019-03

Locations

TW (1)