Peripheral Artery Disease, Intermittent Claudication
Conditions
Keywords
Peripheral Artery Disease, Intermittent Claudication, Autologous Stem Cells, ALDH cells, Claudicants, PAD, Peak Walking Time, MRI, Vascular Flow, Anatomy, Perfusion
Brief summary
The purpose of this study is to find out if aldehyde dehydrogenase bright (ALDHbr) cells taken from a patient's bone marrow can be placed safely, via intramuscular injections, into their affected calf and lower thigh muscles and improve blood flow and/or peak walking time in patients experiencing pain associated with blocked blood vessels in the leg.
Detailed description
Peripheral Artery Disease (PAD) occurs when arteries in the arms and legs (most often the legs) become narrowed by plaque. Because of this plaque, patients with PAD are also at increased risk for heart attacks and strokes. Those with PAD often have intermittent claudication (blockage of blood vessels in the leg). This blockage decreases blood flow to the leg muscles, which can cause pain in one or both legs during exercise (such as during walking). Intermittent means the pain comes and goes. Because PAD interferes with circulation, worsening of this condition can increase pain in the leg; sometimes even during periods of rest. Bone marrow contains special stem cells that may promote blood vessel growth, prevent cell death, and transform themselves into a number of tissues including new muscle. There is a small subpopulation of bone marrow mononuclear cells, called aldehyde dehydrogenase-bright (ALDHbr) cells, that is highly enriched in these types of stem cells. The enzyme in ALDHbr cells responds to damage signals and may play an important role in tissue repair. In this study we investigate the safety and efficacy of bone marrow derived stem cells with particular characteristics in PAD patients with intermittent claudication and explore new end-points to evaluate therapeutic effects using novel MRI imaging modalities as well as traditional endpoints.
Interventions
BIOLOGICALALD-301
Ten 1ml injections of ALD-301 in the index calf and posterior, lower thigh
BIOLOGICALPlacebo (vehicle)
Ten 1ml injections of placebo in the index calf and posterior, lower thigh
Sponsors
National Heart, Lung, and Blood Institute (NHLBI)
Aldagen
Center for Cell and Gene Therapy, Baylor College of Medicine
The University of Texas Health Science Center, Houston
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
40 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Patients with atherosclerotic peripheral artery disease with classic claudication (exercise-induced pain, cramps, fatigue, or other equivalent discomfort involving large muscle groups of the leg(s) that is consistently relieved by rest) or atypical leg pain (exertional leg pain that does not begin at rest or does not resolve consistently with rest) as defined by the San Diego Claudication Questionnaire. 2. Age ≥40 years 3. Resting ankle-brachial index \<0.90 or a resting toe-brachial index of \<0.70 at baseline testing 4. Presence of significant stenosis or occlusion of infrainguinal arteries including the superficial femoral artery, popliteal artery and/or infrapopliteal arteries as determined by: Duplex ultrasound imaging (occlusion or focal doubling of peak systolic velocity of one or more affected segments) OR lower extremity computed Tomography Angiography (CTA) OR lower extremity magnetic resonance angiography (MRA) OR lower extremity catheter-based contrast arteriography. Each of these noninvasive and invasive anatomic assessments will identify patients with at least a 50% stenosis in the affected segment.
Exclusion criteria
1. Presence of any musculoskeletal disease, cardiac or pulmonary disease, or neurological disease that limits the patient's ability to walk to fulfill protocol requirements (claudication must be the consistent primary exercise limitation) 2. Inability to complete treadmill testing per protocol requirements. 3. Ability to walk for more than 12 minutes on the treadmill during treadmill testing. 4. Patients who identify both legs as equivocally symptomatic or alternate between symptomatic legs on the baseline treadmill tests. 5. Patients with critical limb ischemia (ischemic rest pain or ischemia-related non healing wounds or tissue loss (Rutherford categories 4-6). 6. Recent (\<3 months) infrainguinal revascularization (surgery or endovascular revascularization) or revascularization planned during study period 7. Patients with a patent infrainguinal bypass graft in the index limb, with or without evidence of a hemodynamically significant stenosis or other defect (kinking, pseudoaneurysm, or fistula). Patients with an occluded infrainguinal bypass graft or a patent aortobifemoral or femoral-femoral bypass graft are NOT excluded. 8. Patients with \>2+ lower extremity pitting edema 9. Patients with myelodysplastic syndrome (MDS) 10. Patients who are pregnant or lactating, planning to become pregnant in the next 12 months, or are unwilling to use acceptable forms of birth control during study participation. 11. Congestive Heart Failure hospitalization within the last 1 month prior to enrollment 12. Acute coronary syndrome in the last 1 month prior to enrollment 13. Human Immunodeficiency Virus positive, active Hepatitis B Virus or Hepatitis C Virus Disease 14. History of cancer within the last 5 years, except basal cell skin carcinoma 15. Any bleeding diathesis defined as an International Normalized Ratio ≥ 2.0 (off anticoagulation therapy) or history of platelet count less than 100,000 or hemophilia 16. Contraindication to magnetic resonance imaging (MRI) (including knee/tibial/fibular replacement hardware in the index leg) or known allergy to MRI contrast media 17. Chronic kidney disease \[effective Glomerular Filtration Rate \<30 by modification of diet in renal disease (MDRD) or Mayo or Cockcroft-Gault formula\] 18. Uncontrolled diabetes \[Hemoglobin A1C (HbA1C)\>8.5\] 19. Planned change to (initiate or terminate) active involvement in a supervised exercise program (e.g., with a trainer, exercise protocol, and goals, such as in a peripheral arterial disease, cardiac or pulmonary rehabilitation program) during study participation 20. Plans to change medical therapy during the duration of the study, (i.e. patients who use cilostazol should remain on a stable dose for four weeks prior to enrollment and should not change doses for the 6 months of the study duration.) As always, cilostazol can be discontinued if new heart failure or intolerance occurs during study participation. 21. Any condition requiring immunosuppressant medications (e.g., for treatment of organ transplants, psoriasis, Crohn's disease, alopecia areata). 22. History of inflammatory or progressively fibrotic conditions (e.g. rheumatoid arthritis, systemic lupus erythematosis, vasculitic disorders, idiopathic pulmonary fibrosis, retroperitoneal fibrosis). 23. Patients with any untreated stenosis \> 70% of the distal aorta, common iliac, or external iliac arteries by CT, Angiography or MRI imaging will be excluded from enrollment (patients with previously successfully revascularized inflow stenoses may enroll in PACE). Subjects who were screen failures for a flow-limiting proximal lesion may be rescreened 3 months after successful angioplasty/stenting. 24. Inability to provide written informed consent due to cognitive or language barriers (interpreter permitted) 25. Concurrent enrollment in another clinical interventional investigative trial. 26. Presence of any clinical condition that in the opinion of the principal Investigator or the sponsor makes the patient not suitable to participate in the trial
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Peak Walking Time (PWT) | Assessed at baseline and 6 months | The placebo adjusted average change over time in the maximum time (in minutes) walked by a patient on a treadmill under standardized conditions. The patient continues the test until walking can no longer be tolerated because of claudication symptoms. |
| Leg Collateral Count (Via Contrast Enhanced-MR) | Assessed at baseline and 6 months | The placebo adjusted average change in the number of collateral vessels over time. |
| Peak Hyperemic Popliteal Flow (Phase Contrast MRA) | Assessed at baseline and 6 months | The placebo adjusted average change in peak hyperemic popliteal flow (mL/s) over time. |
| Capillary Perfusion | Assessed at baseline and 6 months | The placebo adjusted average change in capillary perfusion over time. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Peripheral Artery Questionnaire (PAQ) | Assessed as a trajectory (baseline, 1mos, 3mos, and 6 mos) | The Peripheral Artery Questionnaire (PAQ) assesses subjective physical limitations, leg symptoms, social function, treatment satisfaction, and quality of life. It is administered as a self report. Higher scores are indicative of better outcome. The summary scores is compiled by taking the mean of five subscales generated from the original questions. Range: Minimum score is 11.1, maximum 85. The measure represents placebo adjusted average change in Peripheral Artery Questionnaire (PAQ) summary score assessed over time. The reported value is the estimate from regression analysis of the slope of the placebo adjusted measure over the time course of the trial adjusted for baseline weight. |
| Walking Impairment Questionnaire (WIQ)-Walking Distance Score | Assessed as a trajectory (baseline, 1mos, 3mos, and 6 mos) | The Walking Impairment Questionnaire (WIQ) assesses the severity of the subjective walking impairment on distance, speed, and stair climbing scales. It is administered as a self report. Range: Minimum score is 0.2, maximum 100. The measure represents the placebo adjusted average change in WIQ walking distance score assessed over time. The reported value is the estimate from regression analysis of the slope of the placebo adjusted measure over the time course of the trial adjusted for baseline weight. |
| Pre-exercise Ankle-Brachial Index (ABI) | Assessed as a trajectory (baseline, 3mos, and 6 mos) | ABI is the ratio of the blood pressure at the ankle to the blood pressure of the upper arm. Pre-exercise ABI is collected routinely with the patient supine immediately prior to a treadmill test. This measure represents the placebo adjusted average change over time in arm and pedal (ankle) blood pressure. The reported value is the estimate from regression analysis of the slope of the placebo adjusted measure over the time course of the trial adjusted for baseline weight. |
| Walking Impairment Questionnaire (WIQ)-Ability to Climb Stairs Score | Assessed as a trajectory (baseline, 1mos, 3mos, and 6 mos) | The Walking Impairment Questionnaire (WIQ) assesses the severity of the subjective walking impairment on distance, speed, and stair climbing scales. It is administered as a self report. Range: Minimum score is 0, maximum 100. The measure represents the placebo adjusted average change in WIQ ability to climb stairs score assessed over time. The reported value is the estimate from regression analysis of the slope of the placebo adjusted measure over the time course of the trial adjusted for baseline weight. |
| Walking Impairment Questionnaire (WIQ)- Walking Speed Score | Assessed as a trajectory (baseline, 1mos, 3mos, and 6 mos) | The Walking Impairment Questionnaire (WIQ) assesses the severity of the subjective walking impairment on distance, speed, and stair climbing scales. It is administered as a self report. Range: Minimum score is 0, maximum 87. The measure represents the placebo adjusted average change in WIQ walking speed score assessed over time. The reported value is the estimate from regression analysis of the slope of the placebo adjusted measure over the time course of the trial adjusted for baseline weight. |
| Post-exercise Ankle-Brachial Index (ABI) | Assessed as a trajectory (baseline, 3mos, and 6 mos) | ABI is the ratio of the blood pressure at the ankle to the blood pressure of the upper arm. Post-exercise ABI is collected routinely with the patient supine immediately following a treadmill test. This measure represents the placebo adjusted average change over time in arm and pedal (ankle) blood pressure. The reported value is the estimate from regression analysis of the slope of the placebo adjusted measure over the time course of the trial adjusted for baseline weight. |
| Claudication Onset Time (COT) | Assessed as a trajectory (baseline, 3mos, and 6 mos) | Claudication Onset Time (COT) is the walking time at which patients first experience leg pain during a treadmill test. The measure represents placebo adjusted average change over time (in minutes) in the time walked by a patient on a treadmill under standardized conditions before the onset of claudication symptoms, regardless of whether this is manifested or characterized as muscle pain, ache, cramp, numbness or fatigue. This does not include joint pain or other pain not associated with claudication. The reported value is the estimate from regression analysis of the slope of the placebo adjusted measure over the time course of the trial adjusted for baseline weight. |
| Peak Walking Time (PWT) | Assessed at baseline and 3 months | The average change in maximum time (in minutes) walked by a patient on a treadmill under standardized conditions. The patient continues the test until walking can no longer be tolerated because of claudication symptoms. |
Countries
United States
Participant flow
Recruitment details
Enrollment took place at seven Network centers and their associated satellite facilities between 6/13/2013 and 12/8/2015. The main centers are located in Texas, Florida (2 locations), Minnesota, Kentucky, Indiana, and California. Study brochures, patient informational DVDs, and clinicaltrials.gov were among the tools used for recruitment.
Participants by arm
| Arm | Count |
|---|---|
| ALDHbr Participants will receive ALDHbr cells via intramuscular injection
ALDHbr: Ten 1ml injections of ALDHbr cells in the index calf and posterior, lower thigh | 38 |
| Placebo (Vehicle) Participants will receive placebo (vehicle) via intramuscular injection
Placebo (vehicle): Ten 1ml injections of placebo in the index calf and posterior, lower thigh | 40 |
| Total | 78 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 1 | 1 |
| Overall Study | Withdrawal by Subject | 1 | 1 |
Baseline characteristics
| Characteristic | ALDHbr | Total | Placebo (Vehicle) |
|---|---|---|---|
| Age, Continuous | 66.3 Years STANDARD_DEVIATION 8.6 | 66.2 Years STANDARD_DEVIATION 8.65 | 66.2 Years STANDARD_DEVIATION 8.7 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 3 Participants | 9 Participants | 6 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 34 Participants | 68 Participants | 34 Participants |
| Region of Enrollment United States | 38 participants | 78 participants | 40 participants |
| Sex: Female, Male Female | 12 Participants | 21 Participants | 9 Participants |
| Sex: Female, Male Male | 26 Participants | 57 Participants | 31 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 1 / 40 | 6 / 42 |
| serious Total, serious adverse events | 4 / 40 | 8 / 42 |
Outcome results
Primary
Capillary Perfusion
The placebo adjusted average change in capillary perfusion over time.
Time frame: Assessed at baseline and 6 months
Population: Participants with available analyzable MRI imaging at baseline and 6 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| ALDHbr | Capillary Perfusion | -0.42 percent | Standard Deviation 2.4 |
| Placebo (Vehicle) | Capillary Perfusion | -0.25 percent | Standard Deviation 2.36 |
p-value: 0.75295% CI: [-1.26, 0.91]t-test, 2 sided
Primary
Leg Collateral Count (Via Contrast Enhanced-MR)
The placebo adjusted average change in the number of collateral vessels over time.
Time frame: Assessed at baseline and 6 months
Population: Participants who had available analyzable baseline and 6 month MRI imaging
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| ALDHbr | Leg Collateral Count (Via Contrast Enhanced-MR) | 1.5 vessel count | Standard Deviation 2.7 |
| Placebo (Vehicle) | Leg Collateral Count (Via Contrast Enhanced-MR) | 0.6 vessel count | Standard Deviation 2.2 |
p-value: 0.11695% CI: [-0.2, 2.1]t-test, 2 sided
Primary
Peak Hyperemic Popliteal Flow (Phase Contrast MRA)
The placebo adjusted average change in peak hyperemic popliteal flow (mL/s) over time.
Time frame: Assessed at baseline and 6 months
Population: Participants who had available analyzable baseline and 6 month MRI imaging
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| ALDHbr | Peak Hyperemic Popliteal Flow (Phase Contrast MRA) | 0.2 ml/sec | Standard Deviation 1.5 |
| Placebo (Vehicle) | Peak Hyperemic Popliteal Flow (Phase Contrast MRA) | 0.2 ml/sec | Standard Deviation 1.9 |
p-value: 0.97895% CI: [-0.8, 0.8]t-test, 2 sided
Primary
Peak Walking Time (PWT)
The placebo adjusted average change over time in the maximum time (in minutes) walked by a patient on a treadmill under standardized conditions. The patient continues the test until walking can no longer be tolerated because of claudication symptoms.
Time frame: Assessed at baseline and 6 months
Population: Participants who had available analyzable baseline and 6 month treadmill test results.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| ALDHbr | Peak Walking Time (PWT) | 2.2 minutes | Standard Deviation 3.9 |
| Placebo (Vehicle) | Peak Walking Time (PWT) | 1.2 minutes | Standard Deviation 2.7 |
p-value: 0.23895% CI: [-0.6, 2.5]t-test, 2 sided
Secondary
Claudication Onset Time (COT)
Claudication Onset Time (COT) is the walking time at which patients first experience leg pain during a treadmill test. The measure represents placebo adjusted average change over time (in minutes) in the time walked by a patient on a treadmill under standardized conditions before the onset of claudication symptoms, regardless of whether this is manifested or characterized as muscle pain, ache, cramp, numbness or fatigue. This does not include joint pain or other pain not associated with claudication. The reported value is the estimate from regression analysis of the slope of the placebo adjusted measure over the time course of the trial adjusted for baseline weight.
Time frame: Assessed as a trajectory (baseline, 3mos, and 6 mos)
Population: Participants who had available analyzable baseline, 3 month, and 6 month treadmill test results.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| ALDHbr | Claudication Onset Time (COT) | 2.8 minutes | Standard Deviation 1.9 |
| Placebo (Vehicle) | Claudication Onset Time (COT) | 2.5 minutes | Standard Deviation 1.7 |
p-value: 0.24195% CI: [-0.2, 0.6]Regression, Linear
Secondary
Peak Walking Time (PWT)
The average change in maximum time (in minutes) walked by a patient on a treadmill under standardized conditions. The patient continues the test until walking can no longer be tolerated because of claudication symptoms.
Time frame: Assessed at baseline and 3 months
Population: Participants who had available analyzable baseline, 3 month, and 6 month treadmill test results.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| ALDHbr | Peak Walking Time (PWT) | 7.1 minutes | Standard Deviation 4.4 |
| Placebo (Vehicle) | Peak Walking Time (PWT) | 6.0 minutes | Standard Deviation 4 |
Secondary
Peripheral Artery Questionnaire (PAQ)
The Peripheral Artery Questionnaire (PAQ) assesses subjective physical limitations, leg symptoms, social function, treatment satisfaction, and quality of life. It is administered as a self report. Higher scores are indicative of better outcome. The summary scores is compiled by taking the mean of five subscales generated from the original questions. Range: Minimum score is 11.1, maximum 85. The measure represents placebo adjusted average change in Peripheral Artery Questionnaire (PAQ) summary score assessed over time. The reported value is the estimate from regression analysis of the slope of the placebo adjusted measure over the time course of the trial adjusted for baseline weight.
Time frame: Assessed as a trajectory (baseline, 1mos, 3mos, and 6 mos)
Population: Participants who had available analyzable baseline, 1 month, 3 month, and 6 month PAQs.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| ALDHbr | Peripheral Artery Questionnaire (PAQ) | 63.9 scores on a scale | Standard Deviation 21.5 |
| Placebo (Vehicle) | Peripheral Artery Questionnaire (PAQ) | 55.2 scores on a scale | Standard Deviation 20 |
p-value: 0.13195% CI: [-0.6, 4.8]Regression, Linear
Secondary
Post-exercise Ankle-Brachial Index (ABI)
ABI is the ratio of the blood pressure at the ankle to the blood pressure of the upper arm. Post-exercise ABI is collected routinely with the patient supine immediately following a treadmill test. This measure represents the placebo adjusted average change over time in arm and pedal (ankle) blood pressure. The reported value is the estimate from regression analysis of the slope of the placebo adjusted measure over the time course of the trial adjusted for baseline weight.
Time frame: Assessed as a trajectory (baseline, 3mos, and 6 mos)
Population: Participants with available analyzable ABI at baseline, 3 months, and 6 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| ALDHbr | Post-exercise Ankle-Brachial Index (ABI) | 0.30 ratio | Standard Deviation 0.15 |
| Placebo (Vehicle) | Post-exercise Ankle-Brachial Index (ABI) | 0.34 ratio | Standard Deviation 0.19 |
p-value: 0.25695% CI: [-0.06, 0.02]Regression, Linear
Secondary
Pre-exercise Ankle-Brachial Index (ABI)
ABI is the ratio of the blood pressure at the ankle to the blood pressure of the upper arm. Pre-exercise ABI is collected routinely with the patient supine immediately prior to a treadmill test. This measure represents the placebo adjusted average change over time in arm and pedal (ankle) blood pressure. The reported value is the estimate from regression analysis of the slope of the placebo adjusted measure over the time course of the trial adjusted for baseline weight.
Time frame: Assessed as a trajectory (baseline, 3mos, and 6 mos)
Population: Participants with available analyzable ABI data at baseline, 3 months, and 6 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| ALDHbr | Pre-exercise Ankle-Brachial Index (ABI) | 0.61 ratio | Standard Deviation 0.17 |
| Placebo (Vehicle) | Pre-exercise Ankle-Brachial Index (ABI) | 0.64 ratio | Standard Deviation 0.14 |
p-value: 0.87195% CI: [-0.02, 0.03]Regression, Linear
Secondary
Walking Impairment Questionnaire (WIQ)-Ability to Climb Stairs Score
The Walking Impairment Questionnaire (WIQ) assesses the severity of the subjective walking impairment on distance, speed, and stair climbing scales. It is administered as a self report. Range: Minimum score is 0, maximum 100. The measure represents the placebo adjusted average change in WIQ ability to climb stairs score assessed over time. The reported value is the estimate from regression analysis of the slope of the placebo adjusted measure over the time course of the trial adjusted for baseline weight.
Time frame: Assessed as a trajectory (baseline, 1mos, 3mos, and 6 mos)
Population: Participants who had available analyzable baseline, 1 month, 3 month, and 6 month WIQs
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| ALDHbr | Walking Impairment Questionnaire (WIQ)-Ability to Climb Stairs Score | 46.3 scores on a scale | Standard Deviation 29.9 |
| Placebo (Vehicle) | Walking Impairment Questionnaire (WIQ)-Ability to Climb Stairs Score | 43.8 scores on a scale | Standard Deviation 32 |
p-value: 0.72295% CI: [-3.3, 4.7]Regression, Linear
Secondary
Walking Impairment Questionnaire (WIQ)-Walking Distance Score
The Walking Impairment Questionnaire (WIQ) assesses the severity of the subjective walking impairment on distance, speed, and stair climbing scales. It is administered as a self report. Range: Minimum score is 0.2, maximum 100. The measure represents the placebo adjusted average change in WIQ walking distance score assessed over time. The reported value is the estimate from regression analysis of the slope of the placebo adjusted measure over the time course of the trial adjusted for baseline weight.
Time frame: Assessed as a trajectory (baseline, 1mos, 3mos, and 6 mos)
Population: Participants who had available analyzable baseline, 1 month, 3 month, and 6 month WIQs.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| ALDHbr | Walking Impairment Questionnaire (WIQ)-Walking Distance Score | 35.6 scores on a scale | Standard Deviation 30.1 |
| Placebo (Vehicle) | Walking Impairment Questionnaire (WIQ)-Walking Distance Score | 23.0 scores on a scale | Standard Deviation 18.8 |
p-value: 0.62695% CI: [-2.6, 4.2]Regression, Linear
Secondary
Walking Impairment Questionnaire (WIQ)- Walking Speed Score
The Walking Impairment Questionnaire (WIQ) assesses the severity of the subjective walking impairment on distance, speed, and stair climbing scales. It is administered as a self report. Range: Minimum score is 0, maximum 87. The measure represents the placebo adjusted average change in WIQ walking speed score assessed over time. The reported value is the estimate from regression analysis of the slope of the placebo adjusted measure over the time course of the trial adjusted for baseline weight.
Time frame: Assessed as a trajectory (baseline, 1mos, 3mos, and 6 mos)
Population: Participants who had available analyzable baseline, 1 month, 3 month, and 6 month WIQs
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| ALDHbr | Walking Impairment Questionnaire (WIQ)- Walking Speed Score | 48.9 scores on a scale | Standard Deviation 20.7 |
| Placebo (Vehicle) | Walking Impairment Questionnaire (WIQ)- Walking Speed Score | 41.8 scores on a scale | Standard Deviation 25.1 |
p-value: 0.59195% CI: [-4.1, 2.3]Regression, Linear