Cerebral PalsyPhase 1/2NCT04873752
A Study to Investigate the Safety and Efficacy of UC-MSCs in Pediatric Patients With Cerebral Palsy
A Phase 1/2 study testing UDI-001 in people with Cerebral Palsy and Periventricular Leukomalacia, run by Rohto Pharmaceutical Co., Ltd. at 1 site in Japan.
Trial summary
UDI-001 is administered to pediatric patients with cerebral palsy attributed to periventricular leukomalacia (PVL) multiple times to investigate its safety and efficacy. Read more about this study
- Who is it for?
- Ages 12 to 24 months
- Conditions the study is for
- Cerebral Palsy, Periventricular Leukomalacia
- What is being tested?
- UDI-001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteJapan
- Study dates
- 2 Jun 2021to 15 Feb 2024
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 24 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age >= 12 months and < 24 months corrected age at the time of informed consent
- Diagnosed with cerebral palsy
- Diagnosed with PVL
- GMFCS level between II and IV
- Able to obtain written informed consent from parents (legal representative)
Who cannot join
- Presence of progressive neurological disease
- Presence of congenital anomaly
- Diagnosed with Grade 3 or more severe intraventricular hemorrhage
- Body weight < 5kg
- Profound intellectual disorder
- Complication of serious infection such as sepsis
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- Requirement of mechanical ventilation
- Complication of serious organ failure such as kidney, liver or heart diseases or others and considered to be inappropriate
- Diagnosed with or suspected of hypsarrhythmia
- Positive for HBV, HCV, HIV or HTLV-1
- Patients who have received cell therapy
- Patients who have undergone selective dorsal rhizotomy or received botulinum toxin products within a certain period of time
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Exploratory Study to Investigate the Safety and Efficacy of UC-MSCs in Pediatric Patients With Cerebral Palsy
UDI-001 is administered to pediatric patients with cerebral palsy attributed to periventricular leukomalacia (PVL) multiple times to investigate its safety and efficacy.
Read the detailed description
This is the first study of UDI-001 to be administered to children. Pediatric patients with cerebral palsy attributed to PVL are enrolled to the study and umbilical cord derived mesenchymal stromal cells (UC-MSCs) are administered to those patients multiple times intravenously. Safety and efficacy of UC-MSCs are evaluated for 52 weeks after the first administration.
- Conditions
- Cerebral Palsy, Periventricular Leukomalacia
- Being tested
- UDI-001
- Medical subject headings
- Cerebral Palsy, Leukomalacia, Periventricular
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UDI-001Biological
2.5 x 10^6 cells/kg of UDI-001 are administered intravenously. One cycle consists of twice a week administrations.
| Study group | Role | What they receive |
|---|---|---|
| UDI-001 | Experimental | Four cycles with 8 administrations Receives: Biological: UDI-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Adverse Event
Adverse events which appear in the participants after the treatment
Measured: until Week 52
Secondary outcomes (4)
Gross Motor Function Measure Score (GMFM Score)
Difference and change in GMFM score
Measured: baseline to Week 52
Gross Motor Function Classification System (GMFCS)
Improvement rate of GMFCS
Measured: baseline to Week 52
Function Independence Measure for Children (WeeFIM)
Change in WeeFIM score
Measured: baseline to Week 52
Kyoto Scale of Psychological Development Test 2001 (KSPD)
Change in and Improvement rate of KSPD
Measured: baseline to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 May 2021
- Study start2 Jun 2021
- Primary completion12 Jul 2023
- Study completion15 Feb 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cerebral PalsyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rohto Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sumito Okawa, Study director, Rohto Pharmaceutical Co., Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04873752
- Sponsor's protocol ID
- UDI-001-0555
- Status last verified
- Apr 2025
- First submitted
- 30 Apr 2021
- Last updated
- 6 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Study to Investigate the Safety and Efficacy of UC-MSCs in Pediatric Patients With Cerebral Palsy
- ClinicalTrials.gov ID
- NCT04873752
- Acronym / sponsor ID
- UDI-001-0555
- Conditions
- Cerebral Palsy, Periventricular Leukomalacia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Rohto Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 2 Jun 2021 (actual)
- Primary completion
- 12 Jul 2023 (actual)
- Study completion
- 15 Feb 2024 (actual)
- First posted
- 5 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
UDI-001 is administered to pediatric patients with cerebral palsy attributed to periventricular leukomalacia (PVL) multiple times to investigate its safety and efficacy.
Detailed description
This is the first study of UDI-001 to be administered to children. Pediatric patients with cerebral palsy attributed to PVL are enrolled to the study and umbilical cord derived mesenchymal stromal cells (UC-MSCs) are administered to those patients multiple times intravenously. Safety and efficacy of UC-MSCs are evaluated for 52 weeks after the first administration.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| UDI-001 | Experimental | Biological: UDI-001Four cycles with 8 administrations |
Who may qualify
- Ages
- 12 to 24 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Age >= 12 months and < 24 months corrected age at the time of informed consent
- Diagnosed with cerebral palsy
- Diagnosed with PVL
- GMFCS level between II and IV
- Able to obtain written informed consent from parents (legal representative)
Key exclusion criteria (12 listed)
- Presence of progressive neurological disease
- Presence of congenital anomaly
- Diagnosed with Grade 3 or more severe intraventricular hemorrhage
- Body weight < 5kg
- Profound intellectual disorder
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| UDI-0012.5 x 10^6 cells/kg of UDI-001 are administered intravenously. One cycle consists of twice a week administrations. | Biological | UDI-001 |
Primary outcome
Safety: Adverse Event
Time frame: until Week 52
Adverse events which appear in the participants after the treatment
4 secondary outcomes
Gross Motor Function Measure Score (GMFM Score)
Time frame: baseline to Week 52
Gross Motor Function Classification System (GMFCS)
Time frame: baseline to Week 52
Function Independence Measure for Children (WeeFIM)
Time frame: baseline to Week 52
Kyoto Scale of Psychological Development Test 2001 (KSPD)
Time frame: baseline to Week 52
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Apr 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sumito Okawa study director
Rohto Pharmaceutical Co., Ltd.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing UDI-001 in people with Cerebral Palsy and Periventricular Leukomalacia, run by Rohto Pharmaceutical Co., Ltd. at 1 site in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety: Adverse Event (time frame: until Week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Apr 2025.