UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000045717 |
|---|---|
| Receipt number | 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 | 2022/11/17 11:14:14 |
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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 | Rehabilitation medicine |
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 whose RULM score are less than or equal to 36 points
(5)Patients who are to receive or are already receiving 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 Risdiplam
<Cohort A, Cohort B and Cohort C>
(1) Patients who have received Onasemnogene abeparvovecor 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
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
sma_study@linical.co.jp
Sponsor or person
Institute
CHUGAI PHARMACEUTICAL CO.LTD.
Institute
Department
Personal name
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
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
独立行政法人国立病院機構 大阪刀根山医療センター（大阪府）、国立精神・神経医療研究センター 病院（東京都）、久留米大学病院（福岡県）、宮城県立こども病院（宮城県）、国立研究開発法人 国立国際医療研究センター病院（東京都）、名古屋大学医学部附属病院（愛知県）、横浜市立大学附属市民総合医療センター（神奈川県）、独立行政法人国立病院機構北海道医療センター（北海道）、神戸大学医学部附属病院（兵庫県）、兵庫医科大学病院（兵庫県）、鹿児島大学病院（鹿児島県）、京都大学医学部附属病院（京都府）、地方独立行政法人大阪府立病院機構大阪母子医療センター（大阪府）
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
Open public recruiting
Date of protocol fixation
| 2021 | Year | 08 | Month | 18 | Day |
Date of IRB
| 2021 | Year | 08 | Month | 19 | Day |
Anticipated trial start date
| 2021 | Year | 11 | Month | 22 | 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
| 2022 | Year | 11 | Month | 17 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000052188
