Restricted to complete data across all outcomes.

Sample size

Not_CU

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

FTLDCDR_SB

293

1.633

2.696

30% reduction

1.143

475.27

CBS_MOTOR_SCORE

293

3.497

5.836

30% reduction

2.448

485.873

CBS_TOTAL_SCORE

293

7.692

13.01

30% reduction

5.385

498.963

PSPRS_TOTAL

293

3.529

8.105

30% reduction

2.47

919.968

CBS_LCB_SCORE

293

3.013

7.524

30% reduction

2.109

1087.33

UPDRS_SCORE

293

3.511

9.761

30% reduction

2.458

1348.155

CBS_NONMOTOR_SCORE

293

1.182

3.469

30% reduction

0.827

1502.897


CBSorPSPorNFVPPSorALS

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

FTLDCDR_SB

149

1.981

2.515

30% reduction

1.386

281.34

CBS_TOTAL_SCORE

149

8.384

13.21

30% reduction

5.869

433.034

CBS_MOTOR_SCORE

149

3.944

6.374

30% reduction

2.761

455.528

PSPRS_TOTAL

149

4.749

8.82

30% reduction

3.324

601.701

UPDRS_SCORE

149

4.903

9.957

30% reduction

3.432

719.355

CBS_NONMOTOR_SCORE

149

1.438

3.602

30% reduction

1.006

1095.023

CBS_LCB_SCORE

149

3.002

7.633

30% reduction

2.102

1127.466


CBS

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

PSPRS_TOTAL

30

8.511

6.93

30% reduction

5.957

115.661

FTLDCDR_SB

30

2.211

2.206

30% reduction

1.548

173.663

CBS_TOTAL_SCORE

30

11.418

13.13

30% reduction

7.993

230.628

CBS_MOTOR_SCORE

30

5.879

7.426

30% reduction

4.115

278.307

UPDRS_SCORE

30

9.031

11.512

30% reduction

6.322

283.427

CBS_NONMOTOR_SCORE

30

1.947

3.84

30% reduction

1.363

678.425

CBS_LCB_SCORE

30

3.592

8.326

30% reduction

2.514

937.233


PSP

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

CBS_MOTOR_SCORE

29

6.821

7.783

30% reduction

4.775

227.06

CBS_TOTAL_SCORE

29

12.711

16.802

30% reduction

8.898

304.776

FTLDCDR_SB

29

2.12

2.803

30% reduction

1.484

305.067

CBS_NONMOTOR_SCORE

29

3.118

4.783

30% reduction

2.182

410.532

UPDRS_SCORE

29

7.9

13.79

30% reduction

5.53

531.521

PSPRS_TOTAL

29

7.184

13.287

30% reduction

5.029

596.711

CBS_LCB_SCORE

29

2.772

10.107

30% reduction

1.94

2318.858


bvFTD

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

FTLDCDR_SB

67

1.886

2.745

30% reduction

1.321

369.255

CBS_TOTAL_SCORE

67

6.378

11.613

30% reduction

4.465

578.202

CBS_LCB_SCORE

67

3.083

6.89

30% reduction

2.158

870.99

CBS_MOTOR_SCORE

67

2.083

4.673

30% reduction

1.458

878.207

CBS_NONMOTOR_SCORE

67

1.212

3.085

30% reduction

0.849

1129.469

PSPRS_TOTAL

67

1.97

5.555

30% reduction

1.379

1386.053

UPDRS_SCORE

67

1.428

5.09

30% reduction

1

2214.579


nfvPPA

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

FTLDCDR_SB

28

1.597

1.948

30% reduction

1.118

259.329

UPDRS_SCORE

28

5.253

9.25

30% reduction

3.677

540.825

CBS_MOTOR_SCORE

28

3.016

5.365

30% reduction

2.111

552.142

PSPRS_TOTAL

28

3.989

8.948

30% reduction

2.792

877.712

CBS_TOTAL_SCORE

28

4.52

12.166

30% reduction

3.164

1263.551

CBS_LCB_SCORE

28

1.646

6.577

30% reduction

1.152

2784.135

CBS_NONMOTOR_SCORE

28

-0.141

2.359

30% reduction

-0.099

48541.105


CU

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

FTLDCDR_SB

150

0.071

0.375

30% reduction

0.05

4894.004

CBS_LCB_SCORE

150

-0.319

2.217

30% reduction

-0.223

8448.715

CBS_TOTAL_SCORE

150

-0.379

3.754

30% reduction

-0.265

17148.707

PSPRS_TOTAL

150

-0.104

1.137

30% reduction

-0.073

20732.028

UPDRS_SCORE

150

0.064

1.074

30% reduction

0.045

48572.552

CBS_NONMOTOR_SCORE

150

-0.052

1.468

30% reduction

-0.037

137132.066

CBS_MOTOR_SCORE

150

-0.008

1.313

30% reduction

-0.005

5168753.05


PSPorCBS

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

FTLDCDR_SB

59

2.166

2.496

30% reduction

1.516

231.565

CBS_MOTOR_SCORE

59

6.342

7.553

30% reduction

4.439

247.353

CBS_TOTAL_SCORE

59

12.054

14.93

30% reduction

8.437

267.604

PSPRS_TOTAL

59

7.858

10.473

30% reduction

5.501

309.82

UPDRS_SCORE

59

8.475

12.586

30% reduction

5.933

384.654

CBS_NONMOTOR_SCORE

59

2.523

4.332

30% reduction

1.766

514.415

CBS_LCB_SCORE

59

3.189

9.173

30% reduction

2.232

1443.368


CBSorbvFTDornfvPPAorPSP

Variable

N

Mean

SD

Drug_effect

Treated_Mean

Estimated_Sample_Per_Arm

FTLDCDR_SB

153

1.967

2.502

30% reduction

1.377

281.992

CBS_TOTAL_SCORE

153

8.078

13.293

30% reduction

5.654

472.393

CBS_MOTOR_SCORE

153

3.841

6.324

30% reduction

2.689

472.726

PSPRS_TOTAL

153

4.555

8.746

30% reduction

3.189

642.991

UPDRS_SCORE

153

4.787

9.847

30% reduction

3.351

738.146

CBS_NONMOTOR_SCORE

153

1.464

3.635

30% reduction

1.025

1075.424

CBS_LCB_SCORE

153

2.772

7.712

30% reduction

1.94

1349.956


New Spaghetti plots

Red line = LOESS, Black = Linear effect

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