Boost to Brittle Bones - Stem Cell Transplantation for Treatment of Brittle Bones
NCT ID: NCT04623606
Last Updated: 2020-11-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
PHASE1/PHASE2
15 participants
INTERVENTIONAL
2019-05-20
2021-12-31
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Prospective Control (Untreated) and historical controls
Subjects eligible for the trial but not willing/able to participate in any of the experimental arms Matched historical controls. Subjects will be identified in historical registries and data will be retrieved from OI database
No interventions assigned to this group
Treatment
Administration of four doses of BOOST cells with the first dose between 1-4 years of age and the three additional doses at +4, +8 and +12 months after the first dose. Each dose is 3x10\^6 MSC/kg body weight.
BOOST cells
Four doses of expanded human 1st trimester fetal liver-derived mesenchymal stem cells.
Interventions
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BOOST cells
Four doses of expanded human 1st trimester fetal liver-derived mesenchymal stem cells.
Eligibility Criteria
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Inclusion Criteria
2. Clinical diagnosis of OI type III or IV AND
3. Molecular diagnosis of OI (Glycine substitution in the collagen triple-helix encoding region of either the COL1A1 or COL1A2 gene)
4. Age between 1 to 4 years
5. BP treatment initiated before inclusion
6. Parent/legal guardian over 18 years of age
1. Parent's/legal guardian's signed informed-consent form
2. Clinical and molecular diagnosis of OI (Glycine substitution in the collagen triple-helix encoding region of either the COL1A1 or COL1A2 gene)
3. Age between 4 to 8 years
4. Parent/legal guardian over 18 years of age
Exclusion Criteria
2. Any contraindication for invasive procedures such as a moderate/severe bleeding tendency or contagious infections
3. Abnormal karyotype or other confirmed genetic syndromes
4. Oncologic disease
5. Inability to comply with the trial protocol and evaluation and follow-up schedule
6. Inability to understand the information and to provide informed consent
1 Year
8 Years
ALL
No
Sponsors
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Ministry of Science and Technology, India
OTHER_GOV
Vinnova
OTHER_GOV
Karolinska Institutet
OTHER
Christian Medical College, Vellore, India
OTHER
Responsible Party
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Vrisha Madhuri
Professor
Principal Investigators
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Vrisha Madhuri, MS Orth
Role: PRINCIPAL_INVESTIGATOR
Christian Medical College, Vellore, India
Locations
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Christian Medical College
Vellore, Tamil Nadu, India
Countries
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Central Contacts
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Facility Contacts
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Suhasini Ganesh, M.Pharm
Role: primary
Suhasini Ganesh, M Pharm
Role: backup
Other Identifiers
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CMCB2B0X
Identifier Type: -
Identifier Source: org_study_id