Intracerebral HemorrhagePhase 1/2RAINBOW-HxNCT06361433
Intracerebral Transplantation of Autologous MSC Combined With Scaffold Product for Chronic Intracerebral Hemorrhage
A Phase 1/2 study testing HUFF-01 in people with Intracerebral Hemorrhage, run by Hokkaido University Hospital at 1 site in Japan.
Trial summary
The goal of this clinical trial is to test intracerebral transplantation of stem cell product in patient with chronic intracerebral hemorrhage. The main questions aims to answer are: The safety of the product The efficacy of the product Read more about this study
- Who is it for?
- Ages 20 to 70 years
- Conditions the study is for
- Intracerebral Hemorrhage
- What is being tested?
- HUFF-01
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteJapan
- Study dates
- 1 Dec 2023to Sep 2027
- Phase
- Phase 1/2
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 20 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 1. Age between 20 and 70 years
- 2. Clinical diagnosis of intracerebral hemorrhage 12 month or more from onset
- 3. Hemorrhagic location of unilateral basal ganglia or thalamus
- 4. Moderate to severe neurological symptoms; mRS 3 or 4, and Brunnstrom stage Ⅲ or IV
- 5. No significant neurological impairment before hemorrhage (Pre-hemorrhagic mRS of 0 or 1)
- 6. Subjects who can give informed consent by its self
Who cannot join
- 1. Severe microbleeds found by T2*
- 2. Hemorrhagic cavity less than 2 mL
- 3. The hemorrhage is due to arteriovenous malformation, cerebral aneurysm, and Moyamoya disease
- 4. Subject showing obvious communication between the hemorrhagic cavity and lateral ventricle on MRI
- 5. Subject showing severe lower extremity contracture (Maximum knee extension less than -15 degrees, Maximum ankle flexion less than 0 degree)
- 6. Subject's body weight less than 45 kg for male and 40kg for female
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- 7. Anaemia (Hg < 10·0 g/dL)
- 8. Thrombocytopaenia (platelet count < 100,000/mm3)
- 9. Severe heart disease (ischaemic heart disease, heart failure)
- 10. Uncontrolled hypertension, despite antihypertensive therapy
- 11. Carriers of infectious disease: syphilis, HBV, HCV, HIV-1/HIV-2, HTLV-1, parvovirus B19
- 12. Pregnant or lactating or expecting to become pregnant during the study
- 13. History of malignancy
- 14. Systemic organ failure ALT ≤ 3·0× upper limit of normal Total bilirubin ≤ 1·5× upper limit of normal Serum creatinine ≤ 1·5× upper limit of normal
- 15. Known serious allergy to any agents used in the study
- 16. Contraindication for magnetic resonance imaging
- 17. Any condition that in the judgement of the investigator would place the patient at undue risk
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase I/II Trial of Intracerebral Transplantation of Autologous Bone Marrow Stromal Cells Combined With Recombinant Peptide Scaffold for Patients With Chronic Intracerebral Hemorrhage
The goal of this clinical trial is to test intracerebral transplantation of stem cell product in patient with chronic intracerebral hemorrhage. The main questions aims to answer are:
- The safety of the product
- The efficacy of the product
Read the detailed description
Introduction:
Intracerebral hemorrhage stands as a leading global cause of death and disability, posing a significant challenge with limited treatment options, especially for chronic patients. Recent advances of stem cell therapies have opened new avenues for therapeutic potential. The investigators' previous preclinical research has demonstrated that intracerebral transplantation of bone marrow stromal cells (BMSCs) combined with a recombinant human collagen type I scaffold enables higher cell survival and engraftment, holds promising potential. The investigators present the protocol for a novel clinical trial, named "Research on Advanced Intervention using Novel Bone Marrow Stem Cells for Chronic Intracerebral Hemorrhage" (RAINBOW-Hx).
Methods and Analysis:
RAINBOW-Hx is a phase I/II, open-label, uncontrolled study with the primary objective of assessing the safety and efficacy of intracerebral transplantation of autologous BMSCs combined with the scaffold (HUFF-01) in patients with chronic intracerebral hemorrhage. Eight patients, experiencing moderate to severe neurological deficits for 12 months or longer, will be enrolled. The hemorrhage location will be limited to the basal ganglia and thalamus. Approximately 50 mL of bone marrow will be extracted from the iliac bone of each patient, and BMSCs will be cultured using autologous platelet lysate. Three days before transplantation, BMSCs will be combined with the scaffold to generate HUFF-01. Each patient will receive a 50,000 HUFF-01 dose, containing approximately 50 million BMSCs, through stereotactic transplantation into the hemorrhagic cavity. Neurological assessments, magnetic resonance imaging, 18F-fluorodeoxyglucose positron emission tomography, and 123I-Iomazenil single-photon emission computed tomography will be performed for one year after administration.
- Conditions
- Intracerebral Hemorrhage
- Being tested
- HUFF-01
- Keywords
- Mesenchymal stromal cell, scaffold, chronic, intracerebral hemorrhage, intraparenchymal transplantation
- Medical subject headings
- Cerebral Hemorrhage, Bronchiolitis Obliterans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HUFF-01Drug
autologous MSC combined with scaffold
| Study group | Role | What they receive |
|---|---|---|
| Cell product transplantation | Experimental | Autologous MSC with scaffold containing approximately 5 x 10^7 MSC cells. Receives: Drug: HUFF-01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0
Listing unexpected CTCAE grade changes in physiological (physical examination/blood test) and Radiographical (MRI/CT) examination
Measured: one year
Secondary outcomes (7)
Change in mRS examination
Percentage of the patients number who showed improvement of mRS (0-6, higher score means worse outcome) of more than 1 point
Measured: One year
Change in NIHSS examination
Mean improvement of NIHSS (0-42, higher score means worse outcome)
Measured: One year
Change in FIM examination
Mean improvement of FIM (18-126, higher score means better outcome)
Measured: One year
Change in Fugl-Myer examination
Mean improvement of Fugl-Myer (0-226, higher score means better outcome)
Measured: One year
Change in Barthal index examination
Mean improvement of Barthal index (0-100, higher score means better outcome)
Measured: One year
Change in FDG-PET examination
Mean improvement of FDG-PET set for ipsilateral motor cortex
Measured: One year
Change in IMZ-SPECT examination
Mean improvement of IMZ-SPECT set for ipsilateral motor cortex
Measured: One year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2024
- Study start1 Dec 2023
- Primary completionSep 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Intracerebral HemorrhageStudies 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
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hokkaido University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Japan Agency for Medical Research and Development
- Responsible party
- Principal investigator, Masahito Kawabori, Hokkaido University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06361433
- Sponsor's protocol ID
- R5-11
- Acronym
- RAINBOW-Hx
- Status last verified
- Oct 2026
- First submitted
- 25 Jan 2024
- Last updated
- 15 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No