Umbilical Cord Mesenchymal Stem Cells Therapy (19#iSCLife®-SA) for Patients With Spinocerebellar Ataxia

Spinocerebellar Ataxia Type 3 Clinical Trial

Official title:

A Clinical Research on the Safety/Efficacy of Umbilical Cord Mesenchymal Stem Cells Therapy for Patients With Spinocerebellar Ataxia

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT03378414
• Other study ID #: SCLnow-XY-03
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: December 31, 2024
• Est. completion date: December 31, 2025

Study information

• Verified date: April 2024
• Source: Sclnow Biotechnology Co., Ltd.
• Contact: Lei Guo
• Phone: 861064368977
• Email: georgeguo[@]sclnow.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is verify the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) therapy for patients with Spinocerebellar Ataxia, and in addition, explore the possible mechanisms of UC-MSC therapy in Spinocerebellar Ataxia.

Description:

This is a random, open label, and parallel controled experiment. 45 patients are selected and sign consent forms, then divided into three groups. Doctors collect the basic information of patient (including age,height, mental condition, vital sign, history of disease, pharmaco-history, genotype test, and so on.) All patients receive laboratory and image examination as baseline. Then, they will give cell treatment based on the clinical protocol. Doctors have follow-up visit on 1, 2, 3, 6, and 12 months after treatment, and do efficacy evaluation.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 45
• Est. completion date: December 31, 2025
• Est. primary completion date: June 30, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years to 60 Years

Eligibility

Inclusion Criteria:

• Clinical and genomic test diagnoses as spinocerebellar ataxias (SCA) (include SCA 1, SCA 2, SCA 3, SCA 6), SARA (Scale for Assessment and Rating of Ataxia) score is 2-5, can complete 8-meter walking test(8MW)

• Do not receive stem cells treatment in 6 months

• Participants sign the consent form based on the experiment process and statement

Exclusion Criteria:

• Cardiac, renal, hepatic insufficiency; total bilirubin is 1.5 times higher than normal value, aspartate transaminase(AST)or alanine aminotransferase(ALT)is 2.5 times higher than normal value

• Hemogram: total white blood cells <3.0 * 10^9 cells/L, blood platelet <75 * 10^9/L, hemoglobin <100g/L

• pneumonia, or severe infection

• With severe allergic history

• Brain organic disorder, like brain tumor

• Serum with HIV, syphilis antibody positive

• Severe mental disease, cognitive disorder patients

• Other severe system or organ organic disease

• Pregnant, breast feeding, or planning pregnant women

• Participate other clinical experiments in 3 months

• With some other conditions that doctor propose not to participate

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Machado-Joseph Disease
• Spinocerebellar Ataxia Type 1
• Spinocerebellar Ataxia Type 2
• Spinocerebellar Ataxia Type 3
• Spinocerebellar Ataxia Type 6
• Spinocerebellar Ataxias
• Spinocerebellar Degenerations

Intervention

Procedure:
• Intravenous infusion
Intravenous infusion of mesenchymal stem cells: 2 * 10^7 cells (30ml)
• Intrathecal injection
Intrathecal injection of mesenchymal stem cells: 2 * 10^7 cells (1ml)

Biological:
• umbilical cord mesenchymal stem cell
Treat patients with umbilical cord mesenchymal stem cell (SCLnow 19#), the dosage based on different procedure

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Sclnow Biotechnology Co., Ltd.

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Scale for the assessment anf rating of ataxia (SARA)	Based on SARA score to calculate treatment efficacy. The equation is as follow, Efficacy = (patients who accepted treatment are effective/patients who accepted treatment)* 100% Effective: after 12 months, score of patient decrease 1 point or more; Ineffective: after 12 months, score decrease less than 1 point or increase.	12 months
Secondary	Image examinations	MRI plain scan of brain.	12 months
Secondary	Inventory of Non-Ataxia Symptoms (INAS) score	Using Inventory of Non-Ataxia Symptoms (INAS) score to determine the presence and severity of non-ataxia signs.	12 months
Secondary	Cerebrospinal fluid (csf) routine	Patients is observed by professionals, compare the changes of each observation point and baseline. Baseline is the data acquire from patients before stem cells treatment.	12 months