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Osteogenesis ImperfectaPhase 1/2BOOST2BNCT04623606

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

A Phase 1/2 study testing BOOST cells in people with Osteogenesis Imperfecta, run by Christian Medical College, Vellore, India at 1 site in India.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 1 year to 8 years
Conditions the study is for
Osteogenesis Imperfecta
What is being tested?
BOOST cells
Could I receive a placebo?
No, everyone receives an active treatment
How many people
15 participants
Where
1 siteIndia
Study dates
20 May 2019to Dec 2021
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 1 year to 8 years
  • All sexes
  • No healthy volunteers
  • Child

(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:
  7. Parent's/legal guardian's signed informed-consent form
  8. Clinical and molecular diagnosis of OI (Glycine substitution in the collagen triple-helix encoding region of either the COL1A1 or COL1A2 gene)
  9. Age between 4 to 8 years
  10. Parent/legal guardian over 18 years of age (iii)Exclusion Criteria Treatment group Prospective and historical control group:
  11. 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
  12. Any contraindication for invasive procedures such as a moderate/severe bleeding tendency or contagious infections
  13. Abnormal karyotype or other confirmed genetic syndromes
  14. Oncologic disease
  15. Inability to comply with the trial protocol and evaluation and follow-up schedule
  16. Inability to understand the information and to provide informed consent

Trial locations and contacts

1 site in 1 country.

Vrisha Madhuri, MS Orth contact

91-416-2282172

madhuriwalter@cmcvellore.ac.in

Suhasini Ganesh, M.Pharm contact

91-416-2285117

brittlebonekids@cmcvellore.ac.in

India1 site, 1 recruiting

  • Christian Medical College

    Vellore, Tamil Nadu, 632004

    Suhasini Ganesh, M.Pharm, 91-416-2285117, brittlebonekids@cmcvellore.ac.in

    Suhasini Ganesh, M Pharm, 91-416-2285117, brittlebonekids@cmcvellore.ac.in

    Recruiting

Other trials in India

About this study

Official title: 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

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.

Being tested
BOOST cells
Keywords
OI, Brittle bone disease, Hereditary bone fragility
Medical subject headings
Osteogenesis Imperfecta

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BOOST cellsBiological

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

Study groups in NCT04623606
Study groupRoleWhat they receive
Prospective Control (Untreated) and historical controlsNo interventionSubjects 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
TreatmentExperimentalAdministration 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.
Receives: Biological: BOOST cells

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety and tolerability measured as seriousness, severity and frequency of treatment related adverse events (AEs)

    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

    Measured: From baseline to 16 months follow up

Secondary outcomes (16)

  1. Number of fractures [ Time Frame: From baseline to 16 months follow-up ]

    Number of fractures.

    Measured: From baseline to 16 months follow up

  2. 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.

    Time (days) to first fracture after each stem cell administration. Number of fractures

    Measured: From baseline to 16 months follow up

  3. Change in bone-marrow density (g/cm2). [ Time Frame: From baseline to the primary follow-up (From baseline to 16 months follow up)

    Change in bone-marrow density (g/cm2).

    Measured: From baseline to 16 months follow up

  4. Growth (cm). [ Time Frame: From baseline to16 months follow up]

    Growth (cm) as assessed by clinician

    Measured: From baseline to 16 months follow up

  5. Weight (kg). [ Time Frame: From baseline to 16 months follow up]

    Growth (kg) as assessed by clinician.

    Measured: From baseline to 16 months follow up

  6. Change in clinical status of OI. [ Time Frame: 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.

    Measured: From baseline to 16 months follow up

  7. Assessment of biochemical bone turnover by analysis of the markers P-Calcium (mg %) in blood samples.

    Assessment of biochemical bone turnover marker P-Calcium (mg %)

    Measured: From at baseline to 16 months follow up

  8. Assessment of biochemical bone turnover by analysis of the markers P-Phosphate (mg %) in blood samples.

    Assessment of biochemical bone turnover marker P-Phosphate (mg %)

    Measured: From baseline to 16 months follow up

  9. Assessment of biochemical bone turnover by analysis of the markers P-Albumin (g/dL)

    Assessment of biochemical bone turnover marker P-Albumin (g/dL)

    Measured: From baseline to 16 months follow up

  10. Assessment of biochemical bone turnover by analysis of the markers S-ALP (IU/L) in blood samples.

    Assessment of biochemical bone turnover marker S-ALP (IU/L)

    Measured: From baseline to 16 months follow up

  11. Assessment of biochemical bone turnover by analysis of the markers S-CTx (mg %) in blood samples.

    Assessment of biochemical bone turnover marker S-CTx (mg %)

    Measured: From baseline to 16 months follow up

  12. Assessment of biochemical bone turnover by analysis of the markers fP-PTH (pg/mL)in blood samples.

    Assessment of biochemical bone turnover marker fP-PTH (pg/mL)

    Measured: From baseline to 16 months follow up

  13. Assessment of biochemical bone turnover by analysis of the markers Vitamin D (nmol/L) in blood samples.

    Assessment of biochemical bone turnover marker Vitamin D (nmol/L)

    Measured: From baseline to 16 months follow up

  14. Assessment of biochemical bone turnover by analysis of the markers Bone specific S-ALP (μg/L) in blood samples.

    Assessment of biochemical bone turnover marker Bone specific S-ALP (μg/L)

    Measured: From baseline to 16 months follow up

  15. Assessment of biochemical bone turnover by analysis of the markers S-Osteocalcin (ng/mL) in blood samples.

    Assessment of biochemical bone turnover marker S-Osteocalcin (ng/mL)

    Measured: From baseline to 16 months follow up

  16. Assessment of biochemical bone turnover by analysis of the markers U-DPD/Krea and U-NTx/Krea (mg %) in blood samples.

    Assessment of biochemical bone turnover marker U-DPD/Krea and U-NTx/Krea (mg %)

    Measured: From baseline to 16 months follow up

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Nov 2020
  2. Study start20 May 2019
  3. Primary completionNov 2021 (estimated)
  4. Study completionDec 2021 (estimated)

Resources

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
Non-randomised
Intervention model
Parallel groups
Model description
Historical and Untreated prospective control and Treatment group
Masking
None (open label)
Enrolment
15 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04623606
Sponsor's protocol ID
CMCB2B0X
Acronym
BOOST2B
Status last verified
Nov 2020
First submitted
18 Sep 2020
Last updated
10 Nov 2020
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing BOOST cells in people with Osteogenesis Imperfecta, run by Christian Medical College, Vellore, India at 1 site in India.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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: status not confirmed, as of the sponsor's last update on 10 Nov 2020. 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 India. 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?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Vrisha Madhuri, MS Orth (91-416-2282172, madhuriwalter@cmcvellore.ac.in), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety and tolerability measured as seriousness, severity and frequency of treatment related adverse events (AEs) (time frame: From baseline to 16 months follow up). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04623606.

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 10 Nov 2020.

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