Trial Outcomes & Findings for Effect of Thiazolidinediones on Human Bone (NCT NCT00927355)

NCT ID: NCT00927355

Last Updated: 2016-03-29

Results Overview

To determine the effect of PIO (pioglitazone) on BMSC (bone marrow stem cell) lineage choice in vivo, a bone marrow aspiration was obtained from patients at baseline and after 6 months of treatment with PIO or placebo. The bone marrow was used for ex vivo CFU-OB (Colony forming units-Osteoblast) and CFU-AD assays using the same protocol described for the in vitro studies previously. We also analyzed the number of total colonies per patient at both baseline and final visit.

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 10 participants
• Primary outcome timeframe: 6 months
• Results posted on: 2016-03-29

Participant Flow

3/09 to 4/11, diabetes clinic and grady hospital

one participant failed to disclose that she was already on pioglitazone and was excluded

Participant milestones

Measure	Pioglitazone half of the diabetic patients will be randomized to pioglitazone treatment for 6 months starting out with 15mg qday for 4 weeks and if no adverse effects noted increased to 30mg qday for 5 more months.	Placebo The other half will be randomized to placebo, starting out with 15mg qday for 4 weeks and if no adverse effects noted increased to 30mg qday for 5 more months.
Overall Study STARTED	5	5
Overall Study COMPLETED	4	4
Overall Study NOT COMPLETED	1	1

Reasons for withdrawal

Measure	Pioglitazone half of the diabetic patients will be randomized to pioglitazone treatment for 6 months starting out with 15mg qday for 4 weeks and if no adverse effects noted increased to 30mg qday for 5 more months.	Placebo The other half will be randomized to placebo, starting out with 15mg qday for 4 weeks and if no adverse effects noted increased to 30mg qday for 5 more months.
Overall Study Adverse Event	1	1

Baseline Characteristics

Effect of Thiazolidinediones on Human Bone

Baseline characteristics by cohort

Measure	Pioglitazone n=5 Participants half of the diabetic patients will be randomized to pioglitazone treatment for 6 months.	Placebo n=5 Participants The other half will be randomized to placebo.	Total n=10 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants	3 Participants n=7 Participants	8 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Age, Continuous	52 years STANDARD_DEVIATION 10 • n=5 Participants	60 years STANDARD_DEVIATION 6 • n=7 Participants	55 years STANDARD_DEVIATION 5 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants	3 Participants n=7 Participants	7 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Region of Enrollment United States	5 participants n=5 Participants	5 participants n=7 Participants	10 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

To determine the effect of PIO (pioglitazone) on BMSC (bone marrow stem cell) lineage choice in vivo, a bone marrow aspiration was obtained from patients at baseline and after 6 months of treatment with PIO or placebo. The bone marrow was used for ex vivo CFU-OB (Colony forming units-Osteoblast) and CFU-AD assays using the same protocol described for the in vitro studies previously. We also analyzed the number of total colonies per patient at both baseline and final visit.

Outcome measures

Measure	Pioglitazone-Osteoblast CFU (Colony Forming Units) n=5 Participants Percent change in Osteoblast CFU numbers as stained with Alizarin red S at baseline and 6 months after treatment with study drug.	Placebo-OSteoblast CFU n=3 Participants Percent change in Osteoblast CFU numbers as stained with Alizarin at baseline and 6 months after treatment with study drug.	Pioglitazone-Adipocyte CFU n=5 Participants Percent change in adipocyte CFU numbers as stained with Oil red O at baseline and 6 months after treatment with study drug.	Placebo-Adipocyte CFU n=3 Participants Percent change in adipocyte CFU numbers as stained with Oil red O, at baseline and 6 months after treatment with study drug.	Pioglitazone-OSc serum Osteocalcin percent change 6 mo after pioglitazone Rx compared to baseline.	Placebo - Osc serum Osteocalcin percent change 6 mo after placebo Rx compared to baseline.
Percent Change in Number of Osteoblast and Adipocyte Colony Forming Units Cultured From Bone Marrow Stem Cells Harvested 6 Months After Treatment With Study Drug Compared to Baseline	0 percent change from baseline to 6months Standard Error 0	12 percent change from baseline to 6months Standard Error 5	4 percent change from baseline to 6months Standard Error 4	-7 percent change from baseline to 6months Standard Error 3	—	—

SECONDARY outcome

Timeframe: 6 months

Outcome measures

Measure	Pioglitazone-Osteoblast CFU (Colony Forming Units) n=5 Participants Percent change in Osteoblast CFU numbers as stained with Alizarin red S at baseline and 6 months after treatment with study drug.	Placebo-OSteoblast CFU n=3 Participants Percent change in Osteoblast CFU numbers as stained with Alizarin at baseline and 6 months after treatment with study drug.	Pioglitazone-Adipocyte CFU n=5 Participants Percent change in adipocyte CFU numbers as stained with Oil red O at baseline and 6 months after treatment with study drug.	Placebo-Adipocyte CFU n=3 Participants Percent change in adipocyte CFU numbers as stained with Oil red O, at baseline and 6 months after treatment with study drug.	Pioglitazone-OSc n=5 Participants serum Osteocalcin percent change 6 mo after pioglitazone Rx compared to baseline.	Placebo - Osc n=3 Participants serum Osteocalcin percent change 6 mo after placebo Rx compared to baseline.
βCTX (Carboxy Terminal Collagen Crosslinks), Osteocalcin, and Adiponectin.	78.7 percent change from baseline Standard Error 100.2	-0.6 percent change from baseline Standard Error 24.5	-9.2 percent change from baseline Standard Error 36.4	0.2 percent change from baseline Standard Error 25.9	3.6 percent change from baseline Standard Error 22.1	-5 percent change from baseline Standard Error 9.4

SECONDARY outcome

Timeframe: 6 months

Outcome measures

Measure	Pioglitazone-Osteoblast CFU (Colony Forming Units) n=4 Participants Percent change in Osteoblast CFU numbers as stained with Alizarin red S at baseline and 6 months after treatment with study drug.	Placebo-OSteoblast CFU n=3 Participants Percent change in Osteoblast CFU numbers as stained with Alizarin at baseline and 6 months after treatment with study drug.	Pioglitazone-Adipocyte CFU n=5 Participants Percent change in adipocyte CFU numbers as stained with Oil red O at baseline and 6 months after treatment with study drug.	Placebo-Adipocyte CFU n=3 Participants Percent change in adipocyte CFU numbers as stained with Oil red O, at baseline and 6 months after treatment with study drug.	Pioglitazone-OSc serum Osteocalcin percent change 6 mo after pioglitazone Rx compared to baseline.	Placebo - Osc serum Osteocalcin percent change 6 mo after placebo Rx compared to baseline.
Bone Mineral Density	-4 percent change from baseline to 6 months Standard Error 4.4	-2.6 percent change from baseline to 6 months Standard Error 6.3	-1.1 percent change from baseline to 6 months Standard Error 2.7	1.9 percent change from baseline to 6 months Standard Error 4.2	—	—

Adverse Events

Pioglitazone

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. George Beck

Emory

Phone: 404-686-1000

Email: gbeck@emory.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place