Ignite Creation Date:
2024-05-05 @ 10:25 PM
Last Modification Date:
2024-10-26 @ 10:18 AM
Study NCT ID:
NCT01108068
Status:
COMPLETED
Last Update Posted:
2019-11-04
First Post:
2010-04-20
Brief Title:
Trial of Lithium Carbonate for Treatment of Osteoporosis-pseudoglioma Syndrome
Sponsor:
University of Maryland Baltimore
Organization:
University of Maryland Baltimore
Study Overview
Official Title:
Trial of Lithium Carbonate for Treatment of Osteoporosis Pseudoglioma Syndrome
Status:
COMPLETED
Status Verified Date:
2018-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This was a pilot study of 10 patients with Osteoporosis-pseudoglioma syndrome OPPG from the Old Order Mennonite community and 16 controls who did not have OPPG Five of the 10 OPPG patient elected to participate in the Lithium trial and 5 participated only in baseline data labs pQCT The 5 with OPPG who were given lithium for 6 months had both dual energy xray absorptiometry DXA peripheral quantitative computerized tomography pQCT and lab assessment at baseline and 6 months Studies in the mouse model of OPPG showed that lithium normalized their bone strength Controls n16 were recruited from the Old Order Mennonite community to minimize the effects of environmental and lifestyle factors The controls were not be given lithium The age range of participants was 4-64 years
Detailed Description:
Osteoporosis-pseudoglioma OPPG syndrome is a very rare genetic disorder approximately 50 cases have been reported worldwide due to mutations in the LRP5 gene causing blindness from birth and fragile bones osteoporosisin early childhood The bony fragility can lead to recurrent fractures of major bones such as the hip femur and spine leaving some children in wheelchairs
Treatment to strengthen the bones in OPPG has primarily been with osteoporosis medications used in other fragile bone disorders of childhood and in adults namely the bisphosphonates eg pamidronate alendronate These drugs have helped the bone strength in OPPG somewhat but have not prevented all fractures We have observed fractures of the hip in 3 children with OPPG who we have treated in spite of their attaining normal bone density determined by DXA dual xray absorptiometry with bisphosphonates Therefore new treatments for OPPG are greatly needed and new methods besides DXA are needed to monitor bone strength on treatment
A mouse model of OPPG has been created In the mouse model of OPPG lithium dramatically improved their bones returning them to normal strength and preventing fractures Lithium which is used for people with psychiatric disease is known to lead to higher bone strength and reduced fractures in people who are on it for psychiatric disease Lithium has been used safely and is approved for children 12 and above The theory is that lithium will improve bone strength in OPPG in humans as it has in the mouse by stimulating bone production bypassing the genetic defect in OPPG
In this study we recruited 10 patients with OPPG and treated those who agreed n5 with lithium for 6 months monitoring the response of the bones by both DXA and pQCT peripheral quantitative computed tomography the latter which gives information about bone quality An IND was obtained to use lithium in this study
Treatment to strengthen the bones in OPPG has primarily been with osteoporosis medications used in other fragile bone disorders of childhood and in adults namely the bisphosphonates eg pamidronate alendronate These drugs have helped the bone strength in OPPG somewhat but have not prevented all fractures We have observed fractures of the hip in 3 children with OPPG who we have treated in spite of their attaining normal bone density determined by DXA dual xray absorptiometry with bisphosphonates Therefore new treatments for OPPG are greatly needed and new methods besides DXA are needed to monitor bone strength on treatment
A mouse model of OPPG has been created In the mouse model of OPPG lithium dramatically improved their bones returning them to normal strength and preventing fractures Lithium which is used for people with psychiatric disease is known to lead to higher bone strength and reduced fractures in people who are on it for psychiatric disease Lithium has been used safely and is approved for children 12 and above The theory is that lithium will improve bone strength in OPPG in humans as it has in the mouse by stimulating bone production bypassing the genetic defect in OPPG
In this study we recruited 10 patients with OPPG and treated those who agreed n5 with lithium for 6 months monitoring the response of the bones by both DXA and pQCT peripheral quantitative computed tomography the latter which gives information about bone quality An IND was obtained to use lithium in this study
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None