Experimental Study Protocol on Treatment with plasma transfusions in Patients affected by type VI Osteogenesis Imperfecta

Experimental Study Protocol on Treatment with plasma transfusions in Patients affected by type VI Osteogenesis Imperfecta - Experimental Study Protocol on Treatment with plasma transfusions in Patients affected by type VI Os

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2015-002622-39-IT

Enrollment

Registered

2021-05-27

Start date

2015-11-18

Completion date

Unknown

Last updated

2022-03-14

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

Conditions

type VI Osteogenesis Imperfecta MedDRA version: 20.0 Level: PT Classification code 10031243 Term: Osteogenesis imperfecta System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Trade Name: PLASMASAFE - SOLUZIONE PER INFUSIONE 1 SACCA DA 200 ML Product Name: PLASMASAFE Pharmaceutical Form: Solution for infusion INN or Proposed INN: 00244301 Current Sponsor code: plasmasafe C

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Children between 1 and 18 years of age, affected by type VI OI, genetically and biochemically confirmed. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: - Previous transfusional adverse events. - Need of transfusions for other reasons

Design outcomes

Primary

Measure	Time frame
Main Objective: To study the efficacy and safety of PEDF administration, using pharmaceutical grade plasma (Plasmasafe), in Patients affected by type VI OI, genetically and biochemically confirmed.;Secondary Objective: NA;Primary end point(s): a) to measure the concentration of PEDF in subjects suffering from OI type VI at the end of the transfusion of a standard dose of plasma (12-15 ml / kg in single infusion), after 1 day, 7 days, 1 month plasma transfusion and before each transfusion, and to evaluate the time interval in which remains a quantity of PEDF equal to the levels of heterozygous subjects for the genetic defect (estimated about 1/5 of the normal values, about 1 mg / ml) establishing, at the end of the transfusions, the best dose of plasma to be administered and the best interval between any subsequent infusions of plasma. b) to evaluate at 1 month after the sixth plasma transfusion, the markers of bone apposition and reabsorption, the bone histomorphometry and densitometric parameters. c) to monitor the occurrence of any adverse events, particularly hemodynamic overload and transfusion reactions or allergic-type reactions febrile non hemolytic transfusion evaluating vital signs before, during and at the end of plasma transfusions, or within the following three hours. d) to monitor the possible occurrence of infectious diseases through the repetition of serology and NAT for HBV, HCV and HIV and serology for LUE at 6 months after the last transfusion;Timepoint(s) of evaluation of this end point: a) 1 day, 7 days, 1 month plasma transfusion and before each transfusion b) 1 month after the sixth plasma transfusion c) before, during and at the end of plasma transfusions, or within the following three hours. d) 6 months after the last transfusion	—

Measure

Time frame

Main Objective: To study the efficacy and safety of PEDF administration, using pharmaceutical grade plasma (Plasmasafe), in Patients affected by type VI OI, genetically and biochemically confirmed.;Secondary Objective: NA;Primary end point(s): a) to measure the concentration of PEDF in subjects suffering from OI type VI at the end of the transfusion of a standard dose of plasma (12-15 ml / kg in single infusion), after 1 day, 7 days, 1 month plasma transfusion and before each transfusion, and to evaluate the time interval in which remains a quantity of PEDF equal to the levels of heterozygous subjects for the genetic defect (estimated about 1/5 of the normal values, about 1 mg / ml) establishing, at the end of the transfusions, the best dose of plasma to be administered and the best interval between any subsequent infusions of plasma. b) to evaluate at 1 month after the sixth plasma transfusion, the markers of bone apposition and reabsorption, the bone histomorphometry and densitometric parameters. c) to monitor the occurrence of any adverse events, particularly hemodynamic overload and transfusion reactions or allergic-type reactions febrile non hemolytic transfusion evaluating vital signs before, during and at the end of plasma transfusions, or within the following three hours. d) to monitor the possible occurrence of infectious diseases through the repetition of serology and NAT for HBV, HCV and HIV and serology for LUE at 6 months after the last transfusion;Timepoint(s) of evaluation of this end point: a) 1 day, 7 days, 1 month plasma transfusion and before each transfusion b) 1 month after the sixth plasma transfusion c) before, during and at the end of plasma transfusions, or within the following three hours. d) 6 months after the last transfusion

—

Secondary

Measure	Time frame
Secondary end point(s): NA;Timepoint(s) of evaluation of this end point: NA	—

Countries

Italy

Contacts

Public ContactUnit¿ Supporto alla Ricerca e Biost

Azienda Ospedaliera Universitaria Integrata Verona

supporto.noprofit@ospedaleuniverona.it0458127043

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026