Boost to Brittle Bones - Stem Cell Transplantation for Treatment of Brittle Bones (BOOST2B)

Christian Medical College, Vellore, India

Status and phase: Unknown; Phase 2; Phase 1

Conditions: Osteogenesis Imperfecta

Treatments: Biological: BOOST cells

Study type: Interventional

Funder types: Other

Identifiers

NCT04623606

CMCB2B0X

Details and patient eligibility

About

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.

Enrollment: 15 estimated patients

Sex: All

Ages: 1 to 8 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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15 participants in 2 patient groups

Prospective Control (Untreated) and historical controls

No Intervention group

Description:

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

Treatment

Experimental group

Description:

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.

Treatment:

Biological: BOOST cells