#sample size now uses latest diagnosis. It is based on the LAST visit and SECOND TO LAST

Restricted to complete data across all outcomes.

Sample size

Not_CU

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

Avg. Time Interval

Min Time Interval

Max Time Interval

FTLDCDR_SB

304

1.806

2.721

30% reduction

1.265

395.718

0.995

0.504

1.493

CBS_TOTAL_SCORE

304

6.899

12.988

30% reduction

4.83

618.057

0.995

0.504

1.493

PSPRS_TOTAL

304

4.013

7.784

30% reduction

2.809

656.177

0.995

0.504

1.493

CBS_MOTOR_SCORE

304

2.934

6.154

30% reduction

2.054

767.032

0.995

0.504

1.493

CBS_LCB_SCORE

304

3.059

6.724

30% reduction

2.142

842.594

0.995

0.504

1.493

UPDRS_SCORE

304

3.482

8.319

30% reduction

2.437

995.642

0.995

0.504

1.493

CBS_NONMOTOR_SCORE

304

0.906

3.155

30% reduction

0.634

2116.689

0.995

0.504

1.493


CBSorPSPorNFVPPSorALS

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

Avg. Time Interval

Min Time Interval

Max Time Interval

FTLDCDR_SB

66

1.273

1.789

30% reduction

0.891

344.413

1.022

0.518

1.493

CBS_MOTOR_SCORE

66

3.484

6.025

30% reduction

2.439

521.543

1.022

0.518

1.493

PSPRS_TOTAL

66

4.035

7.726

30% reduction

2.824

639.555

1.022

0.518

1.493

CBS_TOTAL_SCORE

66

6.563

12.746

30% reduction

4.594

657.8

1.022

0.518

1.493

UPDRS_SCORE

66

4.144

9.246

30% reduction

2.901

868.499

1.022

0.518

1.493

CBS_NONMOTOR_SCORE

66

0.9

2.65

30% reduction

0.63

1512.737

1.022

0.518

1.493

CBS_LCB_SCORE

66

2.18

6.748

30% reduction

1.526

1671.79

1.022

0.518

1.493


CBS

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

Avg. Time Interval

Min Time Interval

Max Time Interval

PSPRS_TOTAL

18

9.518

8.874

30% reduction

6.663

151.617

0.889

0.518

1.208

CBS_MOTOR_SCORE

18

6.485

6.115

30% reduction

4.539

155.095

0.889

0.518

1.208

FTLDCDR_SB

18

2.187

2.29

30% reduction

1.531

191.316

0.889

0.518

1.208

CBS_TOTAL_SCORE

18

8.925

10.58

30% reduction

6.248

245.109

0.889

0.518

1.208

UPDRS_SCORE

18

9.923

12.492

30% reduction

6.946

276.451

0.889

0.518

1.208

CBS_NONMOTOR_SCORE

18

1.144

3.476

30% reduction

0.801

1610.451

0.889

0.518

1.208

CBS_LCB_SCORE

18

1.297

4.964

30% reduction

0.908

2556.469

0.889

0.518

1.208


PSP

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

Avg. Time Interval

Min Time Interval

Max Time Interval

CBS_NONMOTOR_SCORE

14

2.424

2.973

30% reduction

1.697

262.39

0.868

0.562

1.115

CBS_MOTOR_SCORE

14

7.509

9.498

30% reduction

5.257

279.032

0.868

0.562

1.115

CBS_TOTAL_SCORE

14

14.691

20.146

30% reduction

10.284

328.014

0.868

0.562

1.115

UPDRS_SCORE

14

7.128

13.166

30% reduction

4.99

595.046

0.868

0.562

1.115

CBS_LCB_SCORE

14

4.757

9.453

30% reduction

3.33

688.666

0.868

0.562

1.115

PSPRS_TOTAL

14

6.666

13.755

30% reduction

4.666

742.559

0.868

0.562

1.115

FTLDCDR_SB

14

0.788

2.749

30% reduction

0.551

2123.99

0.868

0.562

1.115


bvFTD

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

Avg. Time Interval

Min Time Interval

Max Time Interval

FTLDCDR_SB

44

0.909

1.262

30% reduction

0.636

336.285

1.056

0.77

1.493

CBS_TOTAL_SCORE

44

4.3

8.747

30% reduction

3.01

721.886

1.056

0.77

1.493

CBS_LCB_SCORE

44

2.664

5.54

30% reduction

1.865

754.106

1.056

0.77

1.493

CBS_NONMOTOR_SCORE

44

0.646

2.096

30% reduction

0.452

1838.033

1.056

0.77

1.493

CBS_MOTOR_SCORE

44

0.99

3.403

30% reduction

0.693

2061.975

1.056

0.77

1.493

PSPRS_TOTAL

44

0.745

3.153

30% reduction

0.521

3128.495

1.056

0.77

1.493

UPDRS_SCORE

44

0.571

3.005

30% reduction

0.4

4827.139

1.056

0.77

1.493


nfvPPA

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

Avg. Time Interval

Min Time Interval

Max Time Interval

CBS_MOTOR_SCORE

18

2.264

4.41

30% reduction

1.585

661.553

1.051

0.789

1.419

FTLDCDR_SB

18

0.878

1.782

30% reduction

0.615

718.184

1.051

0.789

1.419

CBS_NONMOTOR_SCORE

18

-0.716

1.677

30% reduction

-0.501

955.59

1.051

0.789

1.419

PSPRS_TOTAL

18

2.919

6.957

30% reduction

2.044

990.614

1.051

0.789

1.419

CBS_TOTAL_SCORE

18

3.701

10.539

30% reduction

2.59

1414.713

1.051

0.789

1.419

CBS_LCB_SCORE

18

2.152

6.215

30% reduction

1.507

1454.384

1.051

0.789

1.419

UPDRS_SCORE

18

2.924

9.084

30% reduction

2.047

1682.843

1.051

0.789

1.419


CU

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

Avg. Time Interval

Min Time Interval

Max Time Interval

FTLDCDR_SB

152

-0.019

0.139

30% reduction

-0.014

8875.149

1.069

0.521

1.455

UPDRS_SCORE

152

-0.052

0.466

30% reduction

-0.036

14118.226

1.069

0.521

1.455

PSPRS_TOTAL

152

-0.073

0.746

30% reduction

-0.051

18227.784

1.069

0.521

1.455

CBS_LCB_SCORE

152

-0.14

1.676

30% reduction

-0.098

25063.897

1.069

0.521

1.455

CBS_TOTAL_SCORE

152

-0.246

3.093

30% reduction

-0.172

27517.045

1.069

0.521

1.455

CBS_MOTOR_SCORE

152

-0.082

1.281

30% reduction

-0.057

42482.686

1.069

0.521

1.455

CBS_NONMOTOR_SCORE

152

-0.024

1.277

30% reduction

-0.017

477031.954

1.069

0.521

1.455


PSPorCBS

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

Avg. Time Interval

Min Time Interval

Max Time Interval

CBS_MOTOR_SCORE

32

6.933

7.655

30% reduction

4.853

212.657

0.88

0.518

1.208

PSPRS_TOTAL

32

8.271

11.162

30% reduction

5.789

317.703

0.88

0.518

1.208

CBS_TOTAL_SCORE

32

11.448

15.493

30% reduction

8.013

319.481

0.88

0.518

1.208

UPDRS_SCORE

32

8.7

12.659

30% reduction

6.09

369.287

0.88

0.518

1.208

FTLDCDR_SB

32

1.575

2.558

30% reduction

1.102

460.199

0.88

0.518

1.208

CBS_NONMOTOR_SCORE

32

1.704

3.279

30% reduction

1.193

645.668

0.88

0.518

1.208

CBS_LCB_SCORE

32

2.811

7.351

30% reduction

1.968

1192.877

0.88

0.518

1.208


CBSorbvFTDornfvPPAorPSP

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

Avg. Time Interval

Min Time Interval

Max Time Interval

FTLDCDR_SB

94

1.13

1.898

30% reduction

0.791

492.126

0.995

0.518

1.493

CBS_MOTOR_SCORE

94

3.257

5.976

30% reduction

2.28

587.236

0.995

0.518

1.493

CBS_TOTAL_SCORE

94

6.618

12.162

30% reduction

4.633

588.982

0.995

0.518

1.493

PSPRS_TOTAL

94

3.723

8.15

30% reduction

2.606

835.718

0.995

0.518

1.493

CBS_LCB_SCORE

94

2.616

6.27

30% reduction

1.831

1001.987

0.995

0.518

1.493

UPDRS_SCORE

94

3.789

9.275

30% reduction

2.652

1045.001

0.995

0.518

1.493

CBS_NONMOTOR_SCORE

94

0.745

2.62

30% reduction

0.522

2155.611

0.995

0.518

1.493


New Spaghetti plots

Black line = LOESS, Red = Linear effect

Left plot is all data. Right is restricted to ~ 1.5 years