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Name:
UMIN ID:

Recruitment status Preinitiation
Unique ID issued by UMIN UMIN000045717
Receipt No. R000052188
Scientific Title Prospective Observational Study on the Method to Assess the Efficacy of Risdiplam on Manual Dexterity in Patients with Spinal Muscular Atrophy
Date of disclosure of the study information 2021/10/12
Last modified on 2021/10/11

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Basic information
Public title Prospective Observational Study on the Method to Assess the Efficacy of Risdiplam on Manual Dexterity in Patients with Spinal Muscular Atrophy
Acronym Study on the Efficacy of Risdiplam on Hand Function in Patients with Spinal Muscular Atrophy
Scientific Title Prospective Observational Study on the Method to Assess the Efficacy of Risdiplam on Manual Dexterity in Patients with Spinal Muscular Atrophy
Scientific Title:Acronym PRIME-SMA
(Prospective Observational Study on the Method to Assess the Efficacy of Risdiplam on Manual Dexterity in Patients with Spinal Muscular Atrophy)
Region
Japan

Condition
Condition Spinal Muscular Atrophy
Classification by specialty
Neurology Pediatrics
Classification by malignancy Others
Genomic information NO

Objectives
Narrative objectives1 1.To examine whether the efficacy of Risdiplam on manual dexterity can be assessed by the 9-hole peg test and the box and block test

2.To explore the alterations in activities of daily living and the quality of daily life after the initiation of administration of Risdiplam.
Basic objectives2 Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes 9-hole peg test

Fatigue assessed in the 9-hole peg test
Key secondary outcomes Box and block test, Upper limb function, ADL and QoDL etc

Base
Study type Observational

Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms
Purpose of intervention
Type of intervention
Interventions/Control_1
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit
6 years-old <=
Age-upper limit

Not applicable
Gender Male and Female
Key inclusion criteria Type 2, 3 or 4 SMA patients who meet all the following criteria
(1)Patients who provide written informed consent/assent for participation in the study, whose ages are 6 years and older at the time of enrollment
(2)Patients who have deletions or mutations in the SMN1 gene that are expected to cause loss of function and clinical symptoms due to Type 2, 3 or 4 SMA are observed
(3)Patients who can keep a sitting position without assistance of others and perform the 9-hole peg test at least once
(4)Patients who are to receive Risdiplam according to the package insert
Key exclusion criteria <Cohort A>
(1) Patients who have received Nusinersen

<Cohort A and Cohort B>
(1) Patients who have received Onasemnogene abeparvovec, Risdiplam or other SMN2 modifiers
(2) Patients who are receiving valproic acid.
(3) Patients who have undergone surgery for scoliosis or hip arthrodesis within 6 months before screening or are scheduled to undergo within 18 months after screening
(4) Patients who require invasive ventilation or tracheostomy
(5) Patients who are judged by the investigator to have a disease other than SMA that causes functional decline in the upper limbs
(6) Patients who are judged by the investigator not to be suitable for this study for any reason
Target sample size 30

Research contact person
Name of lead principal investigator
1st name Masahisa
Middle name
Last name Katsuno
Organization Nagoya University Graduate School of Medicine
Division name Department of Neurology
Zip code 466-8550
Address 65 Tsurumai-cho Showa, Nagoya Aichi
TEL 052-744-2111
Email ka2no@med.nagoya-u.ac.jp

Public contact
Name of contact person
1st name Koji
Middle name
Last name Tanaka
Organization Linical Co., Ltd.
Division name Contract Medical Affairs Unit
Zip code 532-0003
Address 6-1 Miyahara 1-chome, Yodogawa-ku, Osaka
TEL 06-6150-2478
Homepage URL
Email sma_study@linical.co.jp

Sponsor
Institute CHUGAI PHARMACEUTICAL CO.LTD.
Institute
Department

Funding Source
Organization CHUGAI PHARMACEUTICAL CO.LTD.
Organization
Division
Category of Funding Organization Profit organization
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization Specified Nonprofit Corporation MINS
Address 1 -15 -14 Dogenzaka, Shibuya-ku, Tokyo
Tel 03-6416-1868
Email npo-mins@j-rib.com

Secondary IDs
Secondary IDs NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information
2021 Year 10 Month 12 Day

Related information
URL releasing protocol
Publication of results Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status Preinitiation
Date of protocol fixation
2021 Year 08 Month 18 Day
Date of IRB
Anticipated trial start date
2022 Year 01 Month 01 Day
Last follow-up date
2024 Year 06 Month 30 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information Spinal muscular atrophy (SMA) is a single-gene neuromuscular disorder that causes severe weakness of the limbs, trunk, swallowing and respiratory muscles. Currently, motor function assessment scales such as MFM, HFMSE and RULM are used as a method to assess the efficacy of therapeutic agents for SMA. However, these scales have problems in capturing changes in manual dexterity, which affect detailed tasks such as eating and changing clothes, and in assessing changes between scores. Therefore, there is a need to develop a more quantitative and fine motor function evaluation method.
In this study, we will examine whether or not we can assess the efficacy of Risdiplam on manual dexterity in patients with SMA using the 9-hole peg test and the box and block test, which are capable of quantitative evaluation and can be easily introduced into clinical practice.

Management information
Registered date
2021 Year 10 Month 11 Day
Last modified on
2021 Year 10 Month 11 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000052188

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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