Effect of Nilotinib in Cerebellar Ataxia Patients

NCT03932669 | Completed Phase 2 DMC

• 1 other identifier: 1811-074-985
• Study Type: interventional
• Target: 70
• Locations: 1 country
• Sites: 1

Brief Summary

This is an institutional cohort study. Patients confirmed with spinocerebellar ataxia (SCA) and taking or planning to take Nilotinib (Tasigna®) are enrolled in this study. The daily dose of Nilotinib is 150mg-300mg and the patients will be followed up at 1, 3, 6, and 12 months. Rating scale for Friedreich's ataxia I and II and Barthel index are used as general function and daily living performance index. Scale for assessment and rating of ataxia (SARA) are used as an objective measure of cerebellar function. Adverse drug reactions are evaluated based on CTCAE version 4.0.

Conditions

Ataxia, Cerebellar; Ataxia, Progressive

Keywords

spinocerebellar ataxia; treatment; nilotinib; bcr-abl; receptor-tyrosine-kinase-inhibitor

Outcome Measures

Primary Outcomes (1)

• Activity of daily living

  Barthel index: score range 0-6

  12 month

Secondary Outcomes (3)

• Cerebellar function

  12 month

• General function

  12 month

• Activity of daily living

  12 month

Other Outcomes (1)

• Occurrence of adverse events

  Baseline, 1 month, 3 month, 6 month, 12 month

Study Arms (1)

Nilotinib group

EXPERIMENTAL

Patients with SCA and taking nilotinib treatment

Drug: Nilotinib

Interventions

Nilotinib DRUG

150-300mg daily dose of nilotinib

Nilotinib group

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosed as chronic cerebellar ataxia
• Confirmed as spinocerebellar ataxia by gene test

You may not qualify if:

• Laboratory abnormalities that could interfere with the proper use of Nilotinib QTc interval \>450ms on initial electrocardiograph, Hb \<8.0, WBC\<2000, ANC \<1600, PLT \<140,000, AST \>200, ALT\>200, ALP\>575, Positive HIV serology, active hepatitis B
• Unstable mental or physical status that could interfere with precise evaluation and proper management of SCA Heart failure (NYHA Grade III or IV), history of major heart disease Pregnancy, on breast feeding

Sponsors & Collaborators

• Seoul National University Hospital: lead

Study Sites (1)

Seoul National University Hospital

Seoul, 03080, South Korea

Location

Related Publications (2)

• Lee WJ, Moon J, Kim TJ, Jun JS, Lee HS, Ryu YJ, Lee ST, Jung KH, Park KI, Jung KY, Kim M, Lee SK, Chu K. The c-Abl inhibitor, nilotinib, as a potential therapeutic agent for chronic cerebellar ataxia. J Neuroimmunol. 2017 Aug 15;309:82-87. doi: 10.1016/j.jneuroim.2017.05.015. Epub 2017 May 24.

  PMID: 28601294 RESULT

• Lee WJ, Moon J, Jang Y, Shin YW, Son H, Shin S, Jeon D, Han D, Lee ST, Park KI, Jung KH, Lee SK, Chu K. Nilotinib treatment outcomes in autosomal dominant spinocerebellar ataxia over one year. Sci Rep. 2024 Jul 15;14(1):16303. doi: 10.1038/s41598-024-67072-z.

  PMID: 39009709 DERIVED

MeSH Terms

Conditions

Cerebellar Ataxia; Ataxia; Spinocerebellar Ataxias

Interventions

nilotinib

Condition Hierarchy (Ancestors)

Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dyskinesias; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Spinocerebellar Degenerations; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Kon Chu, MD, PhD

  Seoul National University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: April 25, 2019
• First Posted: May 1, 2019
• Study Start: November 19, 2018
• Primary Completion: August 13, 2020
• Study Completion: August 13, 2020
• Last Updated: September 29, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)