Boost to Brittle Bones - Stem Cell Transplantation for Treatment of Brittle Bones

Exploratory, Open Label, Multiple Dose, Phase I/II Trial Evaluating Safety, Efficacy of Intravenous and Intraosseous Infusion of Allogeneic Fetal Mesenchymal Stem In Treatment of Severe Osteogenesis Imperfecta Compared With Historical and Untreated Prospective Controls

Status

UNKNOWN

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04623606

Acronym

BOOST2B

Enrollment

Registered

2020-11-10

Start date

2019-05-20

Completion date

2021-12-31

Last updated

2020-11-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Osteogenesis Imperfecta

Keywords

OI, Brittle bone disease, Hereditary bone fragility

Brief summary

An exploratory, open label, multiple dose, phase I/II trial (n=15) evaluating safety and efficacy of intravenous and intraosseous infusion of allogeneic expanded fetal mesenchymal stem cells (MSC) for the treatment of severe Osteogenesis Imperfecta (OI) compared with historical and untreated prospective controls.

Interventions

BIOLOGICALBOOST cells

Four doses of expanded human 1st trimester fetal liver-derived mesenchymal stem cells.

Study design

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

NONE

Intervention model description

Historical and Untreated prospective control and Treatment group

Eligibility

Sex/Gender

ALL

Age

1 Years to 8 Years

Healthy volunteers

Inclusion criteria

(i)Inclusion Criteria Treatment group 1. Parent's/legal guardian's signed informed-consent form 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 (ii)Inclusion Criteria Prospective Untreated Control Group and Historical Control Group: 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 (iii)

Exclusion criteria

Treatment group Prospective and historical control group: 1. Existence of other known disorder that might interfere with the treatment (such as severe malformations, congenital heart defect, hypoxic encephalopathy (l-lll), neurological problems, immune deficiencies, muscle diseases, syndromes) diagnosed by clinical examination 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

Design outcomes

Primary

Measure	Time frame	Description
Safety and tolerability measured as seriousness, severity and frequency of treatment related adverse events (AEs)	From baseline to 16 months follow up	The primary endpoint is safety and tolerability measured as seriousness, severity and frequency of treatment related adverse events (AEs)/Serious AE (SAE)/Suspected Unexpected Serious Adverse Reaction (SUSAR)with specific focus on the following: 1. Vital signs in conjunction with the MSC infusion 2. Transfusion reactions (infusion toxicity, embolism, allergy, infections) 3. Immune reaction towards the cells, donor-specific antibodies, graft rejection, Graft versus Host Disease, autoimmunity) 4. Tumourigenicity 5. Mortality/morbidity

Secondary

Other

Measure	Time frame	Description
Analysis of an array of cytokines and micro vesicles to evaluate paracrine effects. [ Time Frame: From baseline to the 16 month follow up	From baseline to 16 months follow up	Paracrine effects will be analysed from plasma isolated from peripheral blood.
Incidence of donor cells engrafted into patient tissue samples assessed by histology. [ Time Frame: From baseline to the 16 month follow up	From baseline to 16 months follow up	Donor cell engraftment.
Impact on the subjects Quality of Life: Pediatric Quality of Life Questionnaire™ (PedsQOL) [ Time Frame: From baseline to the 16 month follow-up	From baseline to 16 months follow up	Quality of life assessed using the Infant Pediatric Quality of Life Questionnaire™ (PedsQOL).

Countries

India

Contacts

Primary ContactVrisha Madhuri, MS Orth

madhuriwalter@cmcvellore.ac.in91-416-2282172

Backup ContactSuhasini Ganesh, M.Pharm

brittlebonekids@cmcvellore.ac.in91-416-2285117

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026

Measure	Time frame	Description
Assessment of biochemical bone turnover by analysis of the markers U-DPD/Krea and U-NTx/Krea (mg %) in blood samples.	From baseline to 16 months follow up	Assessment of biochemical bone turnover marker U-DPD/Krea and U-NTx/Krea (mg %)
Assessment of biochemical bone turnover by analysis of the markers Vitamin D (nmol/L) in blood samples.	From baseline to 16 months follow up	Assessment of biochemical bone turnover marker Vitamin D (nmol/L)
Assessment of biochemical bone turnover by analysis of the markers Bone specific S-ALP (μg/L) in blood samples.	From baseline to 16 months follow up	Assessment of biochemical bone turnover marker Bone specific S-ALP (μg/L)
Assessment of biochemical bone turnover by analysis of the markers S-Osteocalcin (ng/mL) in blood samples.	From baseline to 16 months follow up	Assessment of biochemical bone turnover marker S-Osteocalcin (ng/mL)
Number of fractures [ Time Frame: From baseline to 16 months follow-up ]	From baseline to 16 months follow up	Number of fractures.
Time (days) to first fracture after each stem cell administration. [ Time Frame: From each dose of stem cells to the time point of the first fracture.	From baseline to 16 months follow up	Time (days) to first fracture after each stem cell administration. Number of fractures
Change in bone-marrow density (g/cm2). [ Time Frame: From baseline to the primary follow-up (From baseline to 16 months follow up)	From baseline to 16 months follow up	Change in bone-marrow density (g/cm2).
Growth (cm). [ Time Frame: From baseline to16 months follow up]	From baseline to 16 months follow up	Growth (cm) as assessed by clinician
Weight (kg). [ Time Frame: From baseline to 16 months follow up]	From baseline to 16 months follow up	Growth (kg) as assessed by clinician.
Change in clinical status of OI. [ Time Frame: From baseline to 16 months follow up]	From baseline to 16 months follow up	Change in clinical status of OI based on parameters defined under efficacy assessments described in protocol, as assessed by OI clinician.
Assessment of biochemical bone turnover by analysis of the markers P-Calcium (mg %) in blood samples.	From at baseline to 16 months follow up	Assessment of biochemical bone turnover marker P-Calcium (mg %)
Assessment of biochemical bone turnover by analysis of the markers P-Phosphate (mg %) in blood samples.	From baseline to 16 months follow up	Assessment of biochemical bone turnover marker P-Phosphate (mg %)
Assessment of biochemical bone turnover by analysis of the markers P-Albumin (g/dL)	From baseline to 16 months follow up	Assessment of biochemical bone turnover marker P-Albumin (g/dL)
Assessment of biochemical bone turnover by analysis of the markers S-ALP (IU/L) in blood samples.	From baseline to 16 months follow up	Assessment of biochemical bone turnover marker S-ALP (IU/L)
Assessment of biochemical bone turnover by analysis of the markers S-CTx (mg %) in blood samples.	From baseline to 16 months follow up	Assessment of biochemical bone turnover marker S-CTx (mg %)
Assessment of biochemical bone turnover by analysis of the markers fP-PTH (pg/mL)in blood samples.	From baseline to 16 months follow up	Assessment of biochemical bone turnover marker fP-PTH (pg/mL)