Fibrodysplasia Ossificans Progressiva
Conditions
Interventions
Research participants form four intervention groups. Group 2: eight people with FOP (Fibrodysplasia Ossificans Progressiva), without previous specific treatment for the disease
Group 3: eight people with FOP being treated with FOPCON (ascorbic acid + propranolol)
Group 4: eight people with FOP being treated with FOPCON (ascorbic acid + propranolol) and AA+HSP (ascorbic acid and hesperidin)
Group 5: eight people with FOP being treated with FOPCON (ascorbic acid + propranolol) and AA+Epi (ascorbic acid + epigallocatechin). The proposed medication (FOPCON) is a patented pharmaceutical comp
D09.408.386
D02.033.100.624.698.711
Sponsors
Universidade Federal de Mato Grosso do Sul
Instituto de Assistência e Pesquisa em Educação e Saúde - IAPES
Eligibility
Inclusion criteria
Inclusion criteria: Individuals with clinical signs of classic FOP (and possible variants); both sexes; any age; term of consent/signed assent
Exclusion criteria
Exclusion criteria: Link to other experimental research proposals
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| It is expected to show the effectiveness of FOPCON (patent name) in the control of inflammatory signs and symptoms and recurrence of crises, through different records of the evolution of the disease, with regard to recurrence of crises, cumulative disability and quality of life. Data will be obtained using: semi-structured questionnaire prepared by the researchers; WHOQOL(World Health Organization Quality of Life)-bref questionnaire; Katz scale; CAJIS (Cumulative Analogue Joint Involvement Scale) scale and goniometry; and treatment adherence questionnaire. For analysis of anthropometric and clinical data, non-parametric tests of comparison (Mann-Whitney Test) and association (Fisher's Exact Test and Chi-square Test) will be used.;It is expected to prove the involvement of the studied target genes in the pathophysiology of FOP (Fibrodisplasia Ossificante Progressiva), as well as their participation in the control of inflammation and inhibition of heterotopic ossification, when modulated in response to treatment with ascorbic acid and propranolol (FOPCON; patent name). Data will be obtained from analysis of gene expression in in vivo and in vitro models, from samples individual samples of blood and oral mucosa scrapings, using the reverse transcription method (RT) and real-time PCR (polymerase chain reaction). For statistical analysis of the results, the comparative CT (cycle threshold) method will be used and the unpaired t-Student test and ANOVA will be applied (p<0.05) | — |
Secondary
| Measure | Time frame |
|---|---|
| To assess whether the daily ingestion of therapeutic doses of Hesperidin or Epigallocatechin, due to their antioxidant and antiangiogenic effects, can enhance the beneficial effects of FOPCON (patent name) on the inflammatory and osteogenic process, reducing episodes of flare-ups and preventing the installation of ectopic bone. Data will be obtained through clinical evaluation and from analysis of gene expression in in vivo and in vitro models, from individual samples of blood and oral mucosa scrapings, by the reverse transcription method (RT) and PCR (polymerase chain reaction) in time real. For statistical analysis of the results, the comparative CT (cylce threshold) method will be used and the unpaired t-Student test and ANOVA will be applied (p<0.05) | — |
Countries
Brazil
Contacts
Public ContactDurval Palhares
Universidade Federal de Mato Grosso do Sul
Outcome results
None listed