Assessment of the safety and cost of using the medication Pamidronate when used in a one-day or three-day treatment regimen in patients with the rare disease called Fibrous Dysplasia of Bone

Assessment of the safety and cost of using Pamidronate in a single-dose regimen rather than a fractional dose, in patients with Fibrous Dysplasia of Bone

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

REBEC

Registry ID

RBR-7t8qnj8

Enrollment

Unknown

Registered

2023-04-03

Start date

2022-05-01

Completion date

Unknown

Last updated

2025-10-27

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

Conditions

Fibrous dysplasia of bone

Interventions

This prospective, crossover clinical study will evaluate patients with bone fibrous dysplasia through the collection of laboratory tests and application of satisfaction and pain questionnaires before

Group 2 will receive the new therapy (T) first, followed by the standard therapy (R), so each subject is randomly allocated to the RT or TR sequence in two dosing periods, with medication use separate

in short, each patient will undergo a treatment at the beginning (time 0) and another treatment after 6 months (time 1), with data collection being carried out at these times, as well as, between (wit

the standard treatment (R) consists of the application of pamidronate at a dosage of 60mg or 1mg/kg (for patients weighing less than 60kg) diluted in 250ml of saline solution infused intravenously in

the test treatment (T) consists of the application of pamidronate 90mg or 1.5mg/kg (for patients weighing less than 60kg) diluted in 250ml of saline solution infused intravenously in 2 hours and 30 mi

randomization will be done using sealed envelopes, 10

D02.705.429.500.858

Eligibility

Inclusion criteria

Inclusion criteria: Bone fibrous dysplasia; bone pain; expanding bone lesion

Exclusion criteria

Exclusion criteria: Creatinine clearance below 30mg/dL

Design outcomes

Primary

Measure	Time frame
We seek to evaluate the safety of the new therapeutic regimen with pamidronate in a single dose through the comparative method of routinely collected laboratory tests, so we will observe creatinine variations by the change score method and we expect to find variations smaller than 0.3 in the serum creatinine value. , with variations below 15% of pre and post infusion values. We believe that the proposed new treatment is non-inferior to the standard treatment and does not result in increased toxicity.	—

Measure

Time frame

We seek to evaluate the safety of the new therapeutic regimen with pamidronate in a single dose through the comparative method of routinely collected laboratory tests, so we will observe creatinine variations by the change score method and we expect to find variations smaller than 0.3 in the serum creatinine value. , with variations below 15% of pre and post infusion values. We believe that the proposed new treatment is non-inferior to the standard treatment and does not result in increased toxicity.

—

Secondary

Measure	Time frame
Evaluate through the comparative method of laboratory tests and observe the variations of urea, corrected calcium and lymphocytes by the change score method and we expect to find variations below 15% for all these parameters as well as we intend to evaluate by the comparative method of bone pain questionnaires , we expect to find an improvement in bone pain above 30% after each infusion, with no difference between the groups, showing the non-inferiority of the new proposed treatment	—

Measure

Time frame

Evaluate through the comparative method of laboratory tests and observe the variations of urea, corrected calcium and lymphocytes by the change score method and we expect to find variations below 15% for all these parameters as well as we intend to evaluate by the comparative method of bone pain questionnaires , we expect to find an improvement in bone pain above 30% after each infusion, with no difference between the groups, showing the non-inferiority of the new proposed treatment

—

Countries

Brazil

Contacts

Public ContactMonise Pechutti

Universidade Estadual Paulista - UNESP / Faculdade de Medicina de Botucatu - FMB

monise.pechutti@unesp.br+55(14)38801171

Outcome results

None listed

Source: REBEC (via WHO ICTRP)