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

Christian Medical College, Vellore, India15 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Osteogenesis Imperfecta

Interventions

BOOST cellsBIOLOGICAL

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

Eligibility

Sex: ALLMin age: 1 YearMax age: 8 Years

Medical Language ↔ Plain English

(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

Locations (1)

Christian Medical College

Vellore, Tamil Nadu, India

RECRUITING

Outcomes

Primary Outcomes

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