N/A
N=26
Trial of Lithium Carbonate for Treatment of Osteoporosis-pseudoglioma Syndrome
Osteoporosis Pseudoglioma
Bottom Line
View on ClinicalTrials.gov: NCT01108068 ↗Enrolled (actual)
26
Serious AEs
0.0%
Results posted
Apr 2018
Primary outcome: Primary: pQCT of Lower Leg — -2.19; 0.04; -2.09; -0.30 Z-score — p=0.0003
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- Lithium (Drug)
- Age
- Pediatric, Adult · 4+ yrs
- Sex
- All
- Sponsor
- University of Maryland, Baltimore
- Primary completion
- Jul 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY pQCT of Lower Leg |
-2.19; 0.04; -2.09; -0.30 | 0.0003 sig |
| SECONDARY pQCT Z-score in OPPG Participants at Baseline and 6 Months After Lithium |
-4.96; -4.93 | — |
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 (n=16) 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.
Eligibility Criteria
Eligibility Criteria
Inclusion Criteria
- Age 4 years or greater
- Diagnosed with osteoporosis pseudoglioma syndrome (OPPG) or a first degree relative of someone with OPPG. For diagnosis of OPPG, one of the following is required: (1) congenital blindness in a child born into a family with known OPPG where at least one affected family member has had an LRP5 mutation demonstrated or (2) a child with no known family members with OPPG who has congenital blindness, DXA Z-score 1.3, known cardiovascular disease [history of myocardial infarction, heart failure], currently on diuretic or ACE inhibitor)
- Glomerular filtration rate below 80 cc/min
Data sourced from ClinicalTrials.gov (NCT01108068). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.