Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy

• ClinicalTrials.gov Identifier: NCT01594853
• Recruitment Status: Completed
• First Posted: May 9, 2012
• Results First Posted: August 29, 2018
• Last Update Posted: August 29, 2018
• Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
• Collaborator: European Leukodystrophy Association
• Information provided by (Responsible Party): Kathleen Zackowski, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: X-linked Adrenoleukodystrophy
• Intervention: Behavioral: exercise training
• Enrollment: 31

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Female Carriers	Healthy Controls
Arm/Group Description	Female carriers will participate in an exercise paradigm. exercise training: The exercise program uses a local Curves® gym to provide an individualized program of strength and cardiovascular training. The program is expected to be performed for 45 minutes 4-6 days per week.	Healthy age and gender matched individuals will participate in an exercise paradigm. exercise training: The exercise program uses a local Curves® gym to provide an individualized program of strength and cardiovascular training. The program is expected to be performed for 45 minutes 4-6 days per week.
Period Title: Overall Study
Started	16	15
Completed	14	12
Not Completed	2	3
Reason Not Completed
Withdrawal by Subject	1	1
Lost to Follow-up	1	1
noncompliance with exercise	0	1

Baseline Characteristics

Arm/Group Title		Female Carriers	Healthy Controls	Total
Arm/Group Description		Female carriers will participate in an exercise paradigm. exercise training: The exercise program uses a local Curves® gym to provide an individualized program of strength and cardiovascular training. The program is expected to be performed for 45 minutes 4-6 days per week.	Healthy age and gender matched individuals will participate in an exercise paradigm. exercise training: The exercise program uses a local Curves® gym to provide an individualized program of strength and cardiovascular training. The program is expected to be performed for 45 minutes 4-6 days per week.	Total of all reporting groups
Overall Number of Baseline Participants		14	12	26
Baseline Analysis Population Description		[Not Specified]
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	14 participants	12 participants	26 participants
		52.1 (8.4)	51.3 (12.5)	51.7 (10.5)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	14 participants	12 participants	26 participants
	Female	14 100.0%	12 100.0%	26 100.0%
	Male	0 0.0%	0 0.0%	0 0.0%
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	14 participants	12 participants	26 participants
	American Indian or Alaska Native	0 0.0%	0 0.0%	0 0.0%
	Asian	0 0.0%	0 0.0%	0 0.0%
	Native Hawaiian or Other Pacific Islander	0 0.0%	0 0.0%	0 0.0%
	Black or African American	1 7.1%	3 25.0%	4 15.4%
	White	13 92.9%	9 75.0%	22 84.6%
	More than one race	0 0.0%	0 0.0%	0 0.0%
	Unknown or Not Reported	0 0.0%	0 0.0%	0 0.0%
Region of Enrollment; Measure Type: Count of Participants; Unit of measure: Participants
United States	Number Analyzed	14 participants	12 participants	26 participants
		14 100.0%	12 100.0%	26 100.0%

Outcome Measures

1. Primary Outcome

Title	Change in Maximal Voluntary Contraction of the Hip Flexors From Baseline to End of Training and to Post-training
Description	Here, we will assess whether the degree of a person's white matter integrity (i.e. for tracts of interest as measured from MRI) predicts their ability to benefit from exercise. Our prediction is that those individuals with preserved white matter integrity will show the greatest improvement in hip flexor strength.
Time Frame	Participants are assessed at baseline (visit 1, enrollment), end of training (visit 2, week 12) and post-training (visit 3, week 18).

Outcome Measure Data

Analysis Population Description

For the follow up visit, there are fewer participants in the analysis because 5 female carriers and 1 control were unable to return for the third follow up visit due to burden to travel costs.

Arm/Group Title		Female Carriers	Healthy Controls
Arm/Group Description:		Female carriers will participate in an exercise paradigm. exercise training: The exercise program uses a local Curves® gym to provide an individualized program of strength and cardiovascular training. The program is expected to be performed for 45 minutes 4-6 days per week.	Healthy age and gender matched individuals will participate in an exercise paradigm. exercise training: The exercise program uses a local Curves® gym to provide an individualized program of strength and cardiovascular training. The program is expected to be performed for 45 minutes 4-6 days per week.
Overall Number of Participants Analyzed		14	12
Mean (Standard Deviation); Unit of Measure: kg
Change post-training	Number Analyzed	14 participants	12 participants
		0.94 (1.14)	0.98 (2.01)
Change at follow up	Number Analyzed	11 participants	9 participants
		1.32 (1.28)	1.37 (1.68)

Adverse Events

Time Frame	[Not Specified]
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Female Carriers	Healthy Controls
Arm/Group Description	Female carriers will participate in an exercise paradigm. exercise training: The exercise program uses a local Curves® gym to provide an individualized program of strength and cardiovascular training. The program is expected to be performed for 45 minutes 4-6 days per week.	Healthy age and gender matched individuals will participate in an exercise paradigm. exercise training: The exercise program uses a local Curves® gym to provide an individualized program of strength and cardiovascular training. The program is expected to be performed for 45 minutes 4-6 days per week.
All-Cause Mortality
	Female Carriers	Healthy Controls
	Affected / at Risk (%)	Affected / at Risk (%)
Total	0/14 (0.00%)	0/12 (0.00%)
Serious Adverse Events
	Female Carriers	Healthy Controls
	Affected / at Risk (%)	Affected / at Risk (%)
Total	0/14 (0.00%)	0/12 (0.00%)
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	Female Carriers	Healthy Controls
	Affected / at Risk (%)	Affected / at Risk (%)
Total	0/14 (0.00%)	0/12 (0.00%)

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: Kathleen Zackowski
• Organization: Kennedy Krieger Institute
• Phone: 443-923-2716
• EMail: zackowski@kennedykrieger.org

• Responsible Party: Kathleen Zackowski, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
• ClinicalTrials.gov Identifier: NCT01594853
• Other Study ID Numbers: NA_00045673
• First Submitted: May 7, 2012
• First Posted: May 9, 2012
• Results First Submitted: December 14, 2017
• Results First Posted: August 29, 2018
• Last Update Posted: August 29, 2018