Trial Outcomes & Findings for Hydroxychloroquine (HCQ) in Pediatric Interstitial Lung Disease (ILD) (NCT NCT02615938)

NCT ID: NCT02615938

Last Updated: 2025-12-03

Results Overview

O2 saturation measured after 5 min at rest and after withdrawal of oxygen if supplied. Change in Oxygenation (presence or absence of response to treatment) defined as change in O2 saturation \>=5%, or change in respiratory rate \>=20%, or change in respiratory support necessary

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 35 participants
• Primary outcome timeframe: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
• Results posted on: 2025-12-03

Participant Flow

Participant milestones

Measure	Start Placebo, Then HCQ Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group A). Followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg. (Group C).	Start HCQ, Then HCQ Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B). Followed by HCQ for 4 weeks (Group D)	Stop HCQ, Then no Medication Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped. (Group E) The patient will be followed up for additional 3 months on no medication. (Group G)	Stop Placebo, Then no Medication PLACEBO (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the PLACEBO will be stopped. (Group F) The patient will be followed up for additional 3 months on no medication. (Group H)
First 4 Wks Start, First 3 Mon Stop STARTED	17	9	4	5
First 4 Wks Start, First 3 Mon Stop COMPLETED	13	7	3	5
First 4 Wks Start, First 3 Mon Stop NOT COMPLETED	4	2	1	0
Second 4 Wks Start, 2nd 3 Mon Stop STARTED	13	7	3	5
Second 4 Wks Start, 2nd 3 Mon Stop COMPLETED	12	7	3	5
Second 4 Wks Start, 2nd 3 Mon Stop NOT COMPLETED	1	0	0	0

Reasons for withdrawal

Measure	Start Placebo, Then HCQ Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group A). Followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg. (Group C).	Start HCQ, Then HCQ Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B). Followed by HCQ for 4 weeks (Group D)	Stop HCQ, Then no Medication Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped. (Group E) The patient will be followed up for additional 3 months on no medication. (Group G)	Stop Placebo, Then no Medication PLACEBO (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the PLACEBO will be stopped. (Group F) The patient will be followed up for additional 3 months on no medication. (Group H)
First 4 Wks Start, First 3 Mon Stop Physician Decision	0	0	1	0
First 4 Wks Start, First 3 Mon Stop Withdrawal by Subject	1	0	0	0

Baseline Characteristics

Hydroxychloroquine (HCQ) in Pediatric Interstitial Lung Disease (ILD)

Baseline characteristics by cohort

Measure	Start Placebo (Group A), Then Continue HCQ (Group C) n=17 Participants Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group A). Followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg. (Group C).	Start HCQ (Group B), Then Continue HCQ (Group D) n=9 Participants Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B). Followed by HCQ for 4 weeks (Group D)	Stop HCQ (Group E), Then Continued no Medication (Group G) n=4 Participants Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped. (Group E) The patient will be followed up for additional 3 months on no medication. (Group G)	Stop Placebo (Group F), Then Continued no Medication (Group H) n=5 Participants PLACEBO (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the PLACEBO will be stopped. (Group F) The patient will be followed up for additional 3 months on no medication. (Group H)	Total n=35 Participants Total of all reporting groups
Age, Customized	9 n amount of patients n=3 Participants	7.8 n amount of patients n=3 Participants	8.2 n amount of patients n=6 Participants	9.2 n amount of patients n=24 Participants	0 n amount of patients n=15 Participants
Sex: Female, Male Female	10 Participants n=3 Participants	7 Participants n=3 Participants	2 Participants n=6 Participants	3 Participants n=24 Participants	22 Participants n=15 Participants
Sex: Female, Male Male	7 Participants n=3 Participants	2 Participants n=3 Participants	2 Participants n=6 Participants	2 Participants n=24 Participants	13 Participants n=15 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=3 Participants	0 Participants n=3 Participants	0 Participants n=6 Participants	0 Participants n=24 Participants	0 Participants n=15 Participants
Race (NIH/OMB) Asian	1 Participants n=3 Participants	0 Participants n=3 Participants	0 Participants n=6 Participants	0 Participants n=24 Participants	1 Participants n=15 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=3 Participants	0 Participants n=3 Participants	0 Participants n=6 Participants	0 Participants n=24 Participants	0 Participants n=15 Participants
Race (NIH/OMB) Black or African American	0 Participants n=3 Participants	0 Participants n=3 Participants	0 Participants n=6 Participants	0 Participants n=24 Participants	0 Participants n=15 Participants
Race (NIH/OMB) White	16 Participants n=3 Participants	9 Participants n=3 Participants	4 Participants n=6 Participants	5 Participants n=24 Participants	34 Participants n=15 Participants
Race (NIH/OMB) More than one race	0 Participants n=3 Participants	0 Participants n=3 Participants	0 Participants n=6 Participants	0 Participants n=24 Participants	0 Participants n=15 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=3 Participants	0 Participants n=3 Participants	0 Participants n=6 Participants	0 Participants n=24 Participants	0 Participants n=15 Participants
Region of Enrollment Germany	17 participants n=3 Participants	9 participants n=3 Participants	4 participants n=6 Participants	5 participants n=24 Participants	35 participants n=15 Participants
O2 sat [%]	92.6 % 02 saturation n=3 Participants	93.9 % 02 saturation n=3 Participants	93.7 % 02 saturation n=6 Participants	94.8 % 02 saturation n=24 Participants	0 % 02 saturation n=15 Participants

PRIMARY outcome

Timeframe: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks

Population: Group D received HCQ for 4 weeks. These patients had also previously as group B 4 weeks of HCQ, thus an exposure of 8 weeks. This was not deemed comparable to 4 weeks of placebo. Group H received no medication for 3 months. These patients had already withdrawal of HCQ for 3 months as group F, thus no HCQ exposure for 6 months. This was not deemed comparable to 3 months on HCQ. The data of groups D and H were thus not used for the indicated statistical comparisons.

O2 saturation measured after 5 min at rest and after withdrawal of oxygen if supplied. Change in Oxygenation (presence or absence of response to treatment) defined as change in O2 saturation >=5%, or change in respiratory rate >=20%, or change in respiratory support necessary

Outcome measures

Measure	Start Placebo, Then HCQ (Group A) n=13 Participants Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group B) n=7 Participants Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B).	Start Placebo, Then HCQ (Group C) n=12 Participants (After 4 weeks of Placebo) now followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group D) n=7 Participants (After 4 weeks of HCQ) now followed by (further) HCQ for 4 weeks	Stop HCQ, Then no Medication (Group E) n=3 Participants Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped.	Stop Placebo, Then no Medication (Group F) n=5 Participants PLACEBO (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the PLACEBO will be stopped. The patient will be followed up for additional 3 months on no medication. (Group H)	Stop HCQ, Then no Medication (Group G) n=3 Participants Now the patient was followed for additional 3 months on no medication.	Stop Placebo, Then no Medication (Group H) n=5 Participants Then the patient was followed for additional 3 months on no medication. (Group H)
Change in Oxygenation (Presence or Absence of Response to Treatment) Defined as Change in O2 Saturation >=5%, or Change in Respiratory Rate >=20%, or Change in Respiratory Support Necessary Responder	0 Participants	0 Participants	3 Participants	0 Participants	0 Participants	0 Participants	1 Participants	1 Participants
Change in Oxygenation (Presence or Absence of Response to Treatment) Defined as Change in O2 Saturation >=5%, or Change in Respiratory Rate >=20%, or Change in Respiratory Support Necessary Non-responder	13 Participants	7 Participants	9 Participants	7 Participants	3 Participants	5 Participants	2 Participants	4 Participants

SECONDARY outcome

Timeframe: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks

Population: Group D received HCQ for 4 weeks. These patients had also previously as group B 4 weeks of HCQ, thus an exposure of 8 weeks. This was not deemed comparable to 4 weeks of placebo. Group H received no medication for 3 months. These patients had already withdrawal of HCQ for 3 months as group F, thus no HCQ exposure for 6 months. This was not deemed comparable to 3 months on HCQ. The data of groups D and H were thus not used for the indicated statistical comparisons.

O2 saturation measured after 5 min at rest and after withdrawal of oxygen if supplied. Change in Oxygenation (presence or absence of response to treatment) defined as change in O2 saturation >=3%, or change in respiratory rate >=20%, or change in respiratory support necessary

Outcome measures

Measure	Start Placebo, Then HCQ (Group A) n=13 Participants Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group B) n=6 Participants Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B).	Start Placebo, Then HCQ (Group C) n=12 Participants (After 4 weeks of Placebo) now followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group D) n=6 Participants (After 4 weeks of HCQ) now followed by (further) HCQ for 4 weeks	Stop HCQ, Then no Medication (Group E) n=3 Participants Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped.	Stop Placebo, Then no Medication (Group F) n=5 Participants PLACEBO (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the PLACEBO will be stopped. The patient will be followed up for additional 3 months on no medication. (Group H)	Stop HCQ, Then no Medication (Group G) n=3 Participants Now the patient was followed for additional 3 months on no medication.	Stop Placebo, Then no Medication (Group H) n=5 Participants Then the patient was followed for additional 3 months on no medication. (Group H)
Change in Oxygenation (Presence or Absence of Response to Treatment) Defined as Change in O2 Saturation >=3%, or Change in Respiratory Rate >=20%, or Change in Respiratory Support Necessary Responder	2 Participants	1 Participants	2 Participants	0 Participants	0 Participants	3 Participants	1 Participants	1 Participants
Change in Oxygenation (Presence or Absence of Response to Treatment) Defined as Change in O2 Saturation >=3%, or Change in Respiratory Rate >=20%, or Change in Respiratory Support Necessary Non-responder	11 Participants	5 Participants	10 Participants	6 Participants	3 Participants	2 Participants	2 Participants	4 Participants

SECONDARY outcome

Timeframe: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks

Population: Group D received HCQ for 4 weeks. These patients had also previously as group B 4 weeks of HCQ, thus an exposure of 8 weeks. This was not deemed comparable to 4 weeks of placebo. Group H received no medication for 3 months. These patients had already withdrawal of HCQ for 3 months as group F, thus no HCQ exposure for 6 months. This was not deemed comparable to 3 months on HCQ. The data of groups D and H were thus not used for the indicated statistical comparisons.

Oxygen saturation in room air at rest

Outcome measures

Measure	Start Placebo, Then HCQ (Group A) n=13 Participants Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group B) n=7 Participants Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B).	Start Placebo, Then HCQ (Group C) n=12 Participants (After 4 weeks of Placebo) now followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group D) n=6 Participants (After 4 weeks of HCQ) now followed by (further) HCQ for 4 weeks	Stop HCQ, Then no Medication (Group E) n=3 Participants Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped.	Stop Placebo, Then no Medication (Group F) n=5 Participants PLACEBO (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the PLACEBO will be stopped. The patient will be followed up for additional 3 months on no medication. (Group H)	Stop HCQ, Then no Medication (Group G) n=3 Participants Now the patient was followed for additional 3 months on no medication.	Stop Placebo, Then no Medication (Group H) n=5 Participants Then the patient was followed for additional 3 months on no medication. (Group H)
Change of O2-sat in Room Air (%)	-0.1 percentage of O2 Sat Standard Deviation 2.4	-0.6 percentage of O2 Sat Standard Deviation 3.3	1.8 percentage of O2 Sat Standard Deviation 3.5	-1.7 percentage of O2 Sat Standard Deviation 2.6	0.3 percentage of O2 Sat Standard Deviation 1.2	-2.4 percentage of O2 Sat Standard Deviation 2.7	-3.7 percentage of O2 Sat Standard Deviation 6.4	0.2 percentage of O2 Sat Standard Deviation 4.8

SECONDARY outcome

Timeframe: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks

Population: Group D received HCQ for 4 weeks. These patients had also previously as group B 4 weeks of HCQ, thus an exposure of 8 weeks. This was not deemed comparable to 4 weeks of placebo. Group H received no medication for 3 months. These patients had already withdrawal of HCQ for 3 months as group F, thus no HCQ exposure for 6 months. This was not deemed comparable to 3 months on HCQ. The data of groups D and H were thus not used for the indicated statistical comparisons.

Respiratory rate in room air at rest measured in breaths per minute

Outcome measures

Measure	Start Placebo, Then HCQ (Group A) n=13 Participants Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group B) n=7 Participants Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B).	Start Placebo, Then HCQ (Group C) n=12 Participants (After 4 weeks of Placebo) now followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group D) n=5 Participants (After 4 weeks of HCQ) now followed by (further) HCQ for 4 weeks	Stop HCQ, Then no Medication (Group E) n=3 Participants Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped.	Stop Placebo, Then no Medication (Group F) n=5 Participants PLACEBO (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the PLACEBO will be stopped. The patient will be followed up for additional 3 months on no medication. (Group H)	Stop HCQ, Then no Medication (Group G) n=3 Participants Now the patient was followed for additional 3 months on no medication.	Stop Placebo, Then no Medication (Group H) n=5 Participants Then the patient was followed for additional 3 months on no medication. (Group H)
Change of Respiratory Rate in Room Air (Breaths/Min)	1.2 breaths/min Standard Deviation 5.4	0.2 breaths/min Standard Deviation 0.4	-1.3 breaths/min Standard Deviation 5.6	13.8 breaths/min Standard Deviation 33.1	-2.7 breaths/min Standard Deviation 1.2	-5.4 breaths/min Standard Deviation 9.4	7.3 breaths/min Standard Deviation 5	2.5 breaths/min Standard Deviation 7.9

SECONDARY outcome

Timeframe: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks

Population: Group D received HCQ for 4 weeks. These patients had also previously as group B 4 weeks of HCQ, thus an exposure of 8 weeks. This was not deemed comparable to 4 weeks of placebo. Group H received no medication for 3 months. These patients had already withdrawal of HCQ for 3 months as group F, thus no HCQ exposure for 6 months. This was not deemed comparable to 3 months on HCQ. The data of groups D and H were thus not used for the indicated statistical comparisons.

Quality of life (chILD specific) questionaire. A 5-point response scale was utilized (0 = never a problem; 1=almost never a problem; 2=sometimes a problem; 3=often a problem; 4=almost always a problem). Items were reverse-scored and linearly transformed to a 0-100 scale (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). Higher scores indicate better HrQoL.

Outcome measures

Measure	Start Placebo, Then HCQ (Group A) n=13 Participants Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group B) n=7 Participants Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B).	Start Placebo, Then HCQ (Group C) n=12 Participants (After 4 weeks of Placebo) now followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group D) n=2 Participants (After 4 weeks of HCQ) now followed by (further) HCQ for 4 weeks	Stop HCQ, Then no Medication (Group E) n=3 Participants Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped.	Stop Placebo, Then no Medication (Group F) n=5 Participants PLACEBO (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the PLACEBO will be stopped. The patient will be followed up for additional 3 months on no medication. (Group H)	Stop HCQ, Then no Medication (Group G) n=3 Participants Now the patient was followed for additional 3 months on no medication.	Stop Placebo, Then no Medication (Group H) n=3 Participants Then the patient was followed for additional 3 months on no medication. (Group H)
Change of Quality of Life (chILD Specific)	6.9 score on a scale Standard Deviation 9.6	0.1 score on a scale Standard Deviation 3.18	-3.2 score on a scale Standard Deviation 4.8	-9.1 score on a scale Standard Deviation 3.25	-5.7 score on a scale Standard Deviation 11.2	7.5 score on a scale Standard Deviation 2.3	2.3 score on a scale Standard Deviation 3.2	-2.0 score on a scale Standard Deviation 12.3

SECONDARY outcome

Timeframe: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks

Population: Group D received HCQ for 4 weeks. These patients had also previously as group B 4 weeks of HCQ, thus an exposure of 8 weeks. This was not deemed comparable to 4 weeks of placebo. Group H received no medication for 3 months. These patients had already withdrawal of HCQ for 3 months as group F, thus no HCQ exposure for 6 months. This was not deemed comparable to 3 months on HCQ. The data of groups D and H were thus not used for the indicated statistical comparisons.

Quality of life (total score) questionaire. A 5-point response scale was utilized (0 = never a problem; 1=almost never a problem; 2=sometimes a problem; 3=often a problem; 4=almost always a problem). Items were reverse-scored and linearly transformed to a 0-100 scale (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0). Higher scores indicate better HrQoL.

Outcome measures

Measure	Start Placebo, Then HCQ (Group A) n=13 Participants Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group B) n=7 Participants Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B).	Start Placebo, Then HCQ (Group C) n=12 Participants (After 4 weeks of Placebo) now followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group D) n=3 Participants (After 4 weeks of HCQ) now followed by (further) HCQ for 4 weeks	Stop HCQ, Then no Medication (Group E) n=3 Participants Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped.	Stop Placebo, Then no Medication (Group F) n=5 Participants PLACEBO (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the PLACEBO will be stopped. The patient will be followed up for additional 3 months on no medication. (Group H)	Stop HCQ, Then no Medication (Group G) n=3 Participants Now the patient was followed for additional 3 months on no medication.	Stop Placebo, Then no Medication (Group H) n=3 Participants Then the patient was followed for additional 3 months on no medication. (Group H)
Change of Quality of Life (Total Score)	0.4 score on a scale Standard Deviation 6.9	0.3 score on a scale Standard Deviation 1.2	4.8 score on a scale Standard Deviation 4.3	0.4 score on a scale Standard Deviation 7.0	9.2 score on a scale Standard Deviation 5.4	-9.5 score on a scale Standard Deviation 8.8	-1.7 score on a scale Standard Deviation 2.3	-5.5 score on a scale Standard Deviation 4.3

SECONDARY outcome

Timeframe: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks

Population: Group D received HCQ for 4 weeks. These patients had also previously as group B 4 weeks of HCQ, thus an exposure of 8 weeks. This was not deemed comparable to 4 weeks of placebo. Group H received no medication for 3 months. These patients had already withdrawal of HCQ for 3 months as group F, thus no HCQ exposure for 6 months. This was not deemed comparable to 3 months on HCQ. The data of groups D and H were thus not used for the indicated statistical comparisons.

BMI percentile to adapt for age and sex related differences

Outcome measures

Measure	Start Placebo, Then HCQ (Group A) n=13 Participants Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group B) n=7 Participants Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B).	Start Placebo, Then HCQ (Group C) n=12 Participants (After 4 weeks of Placebo) now followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group D) n=7 Participants (After 4 weeks of HCQ) now followed by (further) HCQ for 4 weeks	Stop HCQ, Then no Medication (Group E) n=3 Participants Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped.	Stop Placebo, Then no Medication (Group F) n=5 Participants PLACEBO (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the PLACEBO will be stopped. The patient will be followed up for additional 3 months on no medication. (Group H)	Stop HCQ, Then no Medication (Group G) n=3 Participants Now the patient was followed for additional 3 months on no medication.	Stop Placebo, Then no Medication (Group H) n=5 Participants Then the patient was followed for additional 3 months on no medication. (Group H)
Change of BMI Percentile	7.5 percentile Standard Deviation 11.7	-1.8 percentile Standard Deviation 5.1	-1.6 percentile Standard Deviation 8.1	22.6 percentile Standard Deviation 43.7	-3.3 percentile Standard Deviation 7.9	2.2 percentile Standard Deviation 11.4	13.3 percentile Standard Deviation 20.9	3.9 percentile Standard Deviation 9.2

SECONDARY outcome

Timeframe: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks

Population: There was no blood sampled at the visit at the end of group G and H, thus no values available. Group D received HCQ for 4 weeks. These patients had also previously as group B 4 weeks of HCQ, thus an exposure of 8 weeks. This was not deemed comparable to 4 weeks of placebo. This was not deemed comparable to 3 months on HCQ. The data of groups D and H were thus not used for the indicated statistical comparisons.

Lactate dehydrogenase

Outcome measures

Measure	Start Placebo, Then HCQ (Group A) n=9 Participants Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group B) n=5 Participants Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B).	Start Placebo, Then HCQ (Group C) n=10 Participants (After 4 weeks of Placebo) now followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group D) n=6 Participants (After 4 weeks of HCQ) now followed by (further) HCQ for 4 weeks	Stop HCQ, Then no Medication (Group E) n=2 Participants Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped.	Stop Placebo, Then no Medication (Group F) n=3 Participants PLACEBO (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the PLACEBO will be stopped. The patient will be followed up for additional 3 months on no medication. (Group H)	Stop HCQ, Then no Medication (Group G) Now the patient was followed for additional 3 months on no medication.	Stop Placebo, Then no Medication (Group H) Then the patient was followed for additional 3 months on no medication. (Group H)
Change of LDH (U/ml)	-28.2 Enzyme activity units per ml Standard Deviation 53.7	43.0 Enzyme activity units per ml Standard Deviation 61	11.3 Enzyme activity units per ml Standard Deviation 67	-32.2 Enzyme activity units per ml Standard Deviation 55.3	34.5 Enzyme activity units per ml Standard Deviation 0.7	9.3 Enzyme activity units per ml Standard Deviation 65.5	—	—

SECONDARY outcome

Timeframe: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks

Population: Group D received HCQ for 4 weeks. These patients had also previously as group B 4 weeks of HCQ, thus an exposure of 8 weeks. This was not deemed comparable to 4 weeks of placebo. Group H received no medication for 3 months. These patients had already withdrawal of HCQ for 3 months as group F, thus no HCQ exposure for 6 months. This was not deemed comparable to 3 months on HCQ. The data of groups D and H were thus not used for the indicated statistical comparisons.

FVC predicted expressed in % of the normal reference population (Quanjer PH, Brazzale DJ, Boros PW, et al. Implications of adopting the Global Lungs Initiative 2012 all-age reference equations for spirometry. Eur Respir J. 2013;42:1046-54.) The absolute change in FVC % predicited between two measurements is reported.

Outcome measures

Measure	Start Placebo, Then HCQ (Group A) n=13 Participants Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group B) n=7 Participants Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B).	Start Placebo, Then HCQ (Group C) n=12 Participants (After 4 weeks of Placebo) now followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group D) n=4 Participants (After 4 weeks of HCQ) now followed by (further) HCQ for 4 weeks	Stop HCQ, Then no Medication (Group E) n=3 Participants Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped.	Stop Placebo, Then no Medication (Group F) n=5 Participants PLACEBO (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the PLACEBO will be stopped. The patient will be followed up for additional 3 months on no medication. (Group H)	Stop HCQ, Then no Medication (Group G) n=3 Participants Now the patient was followed for additional 3 months on no medication.	Stop Placebo, Then no Medication (Group H) n=2 Participants Then the patient was followed for additional 3 months on no medication. (Group H)
Absolute Change of Forced Vital Capacity (FVC) (% Predicted of the Reference Population)	0.0 percentage of reference population Standard Deviation 3.7	2.5 percentage of reference population Standard Deviation 13	10.6 percentage of reference population Standard Deviation 19.7	-1.0 percentage of reference population Standard Deviation 4.2	2.3 percentage of reference population Standard Deviation 7.6	0.5 percentage of reference population Standard Deviation 2.1	-1.3 percentage of reference population Standard Deviation 5.1	4.5 percentage of reference population Standard Deviation 4.9

SECONDARY outcome

Timeframe: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks

Population: Group D received HCQ for 4 weeks. These patients had also previously as group B 4 weeks of HCQ, thus an exposure of 8 weeks. This was not deemed comparable to 4 weeks of placebo. Group H received no medication for 3 months. These patients had already withdrawal of HCQ for 3 months as group F, thus no HCQ exposure for 6 months. This was not deemed comparable to 3 months on HCQ. The data of groups D and H were thus not used for the indicated statistical comparisons.

FEV1 predicted expressed in % of the normal reference population (Quanjer PH, Brazzale DJ, Boros PW, et al. Implications of adopting the Global Lungs Initiative 2012 all-age reference equations for spirometry. Eur Respir J. 2013;42:1046-54.). The absolute change in FEV1 % predicited between two measurements is reported.

Outcome measures

Measure	Start Placebo, Then HCQ (Group A) n=13 Participants Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group B) n=7 Participants Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B).	Start Placebo, Then HCQ (Group C) n=12 Participants (After 4 weeks of Placebo) now followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group D) n=4 Participants (After 4 weeks of HCQ) now followed by (further) HCQ for 4 weeks	Stop HCQ, Then no Medication (Group E) n=3 Participants Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped.	Stop Placebo, Then no Medication (Group F) n=5 Participants PLACEBO (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the PLACEBO will be stopped. The patient will be followed up for additional 3 months on no medication. (Group H)	Stop HCQ, Then no Medication (Group G) n=3 Participants Now the patient was followed for additional 3 months on no medication.	Stop Placebo, Then no Medication (Group H) n=2 Participants Then the patient was followed for additional 3 months on no medication. (Group H)
Absolute Change of Forced Expiratory Volume in One Second (FEV1) (% Predicted of the Reference Population)	0.6 percentage of reference population Standard Deviation 4.1	2.0 percentage of reference population Standard Deviation 13.1	9.0 percentage of reference population Standard Deviation 18.6	0.5 percentage of reference population Standard Deviation 5.1	0.0 percentage of reference population Standard Deviation 4.4	-0.5 percentage of reference population Standard Deviation 0.7	-1.0 percentage of reference population Standard Deviation 4.4	5.5 percentage of reference population Standard Deviation 4.9

SECONDARY outcome

Timeframe: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks

Population: Group D received HCQ for 4 weeks. These patients had also previously as group B 4 weeks of HCQ, thus an exposure of 8 weeks. This was not deemed comparable to 4 weeks of placebo. Group H received no medication for 3 months. These patients had already withdrawal of HCQ for 3 months as group F, thus no HCQ exposure for 6 months. This was not deemed comparable to 3 months on HCQ. The data of groups D and H were thus not used for the indicated statistical comparisons.

MEF25-75 predicted expressed in % of the normal reference population (Quanjer PH, Brazzale DJ, Boros PW, et al. Implications of adopting the Global Lungs Initiative 2012 all-age reference equations for spirometry. Eur Respir J. 2013;42:1046-54.). The absolute change in MEF25-75 % predicited between two measurements is reported.

Outcome measures

Measure	Start Placebo, Then HCQ (Group A) n=13 Participants Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group B) n=7 Participants Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B).	Start Placebo, Then HCQ (Group C) n=12 Participants (After 4 weeks of Placebo) now followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group D) n=4 Participants (After 4 weeks of HCQ) now followed by (further) HCQ for 4 weeks	Stop HCQ, Then no Medication (Group E) n=3 Participants Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped.	Stop Placebo, Then no Medication (Group F) n=5 Participants PLACEBO (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the PLACEBO will be stopped. The patient will be followed up for additional 3 months on no medication. (Group H)	Stop HCQ, Then no Medication (Group G) n=3 Participants Now the patient was followed for additional 3 months on no medication.	Stop Placebo, Then no Medication (Group H) n=2 Participants Then the patient was followed for additional 3 months on no medication. (Group H)
Absolute Change of Mean Expiratory Flow Between 25 and 75% of Vital Capacity (MEF25-75) (% Predicted of the Reference Population)	3.0 percentage of reference population Standard Deviation 13.1	-14.8 percentage of reference population Standard Deviation 15.2	-2.6 percentage of reference population Standard Deviation 8.6	18.5 percentage of reference population Standard Deviation 17.4	-8.7 percentage of reference population Standard Deviation 14.2	-7.0 percentage of reference population Standard Deviation 1.4	-11.3 percentage of reference population Standard Deviation 7.8	15.5 percentage of reference population Standard Deviation 6.4

SECONDARY outcome

Timeframe: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks

Population: Group D received HCQ for 4 weeks. These patients had also previously as group B 4 weeks of HCQ, thus an exposure of 8 weeks. This was not deemed comparable to 4 weeks of placebo. Group H received no medication for 3 months. These patients had already withdrawal of HCQ for 3 months as group F, thus no HCQ exposure for 6 months. This was not deemed comparable to 3 months on HCQ. The data of groups D and H were thus not used for the indicated statistical comparisons.

6 Minute walking distance expressed as % of the predicted distance for age and sex. Ulrich et al, Reference values for the 6-minute walk test. BMC Pulm Med. 2013 Aug 5:13:49. doi: 10.1186/1471-2466-13-49. The absolute difference of two measuremnts between two time points is reported.

Outcome measures

Measure	Start Placebo, Then HCQ (Group A) n=4 Participants Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group B) n=1 Participants Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B).	Start Placebo, Then HCQ (Group C) n=4 Participants (After 4 weeks of Placebo) now followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg.	Start HCQ, Then HCQ (Group D) n=1 Participants (After 4 weeks of HCQ) now followed by (further) HCQ for 4 weeks	Stop HCQ, Then no Medication (Group E) n=3 Participants Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped.	Stop Placebo, Then no Medication (Group F) n=2 Participants PLACEBO (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the PLACEBO will be stopped. The patient will be followed up for additional 3 months on no medication. (Group H)	Stop HCQ, Then no Medication (Group G) n=3 Participants Now the patient was followed for additional 3 months on no medication.	Stop Placebo, Then no Medication (Group H) n=2 Participants Then the patient was followed for additional 3 months on no medication. (Group H)
Absolute Change of Six Minute Walking Test (6MWT) Distance (% Predicted of the Reference Population)	-0.8 percentage of reference population Standard Deviation 3.6	-31.0 percentage of reference population Standard Deviation NA n.a. because with n=1 you cannot calculate SD	0.8 percentage of reference population Standard Deviation 2.1	44 percentage of reference population Standard Deviation NA n.a. because with n=1 you cannot calculate SD	0.3 percentage of reference population Standard Deviation 8.5	-0.5 percentage of reference population Standard Deviation 2.1	8.7 percentage of reference population Standard Deviation 12.6	-3.0 percentage of reference population Standard Deviation 2.8

Adverse Events

Start Placebo, Then HCQ

Serious events: 1 serious events

Other events: 12 other events

Deaths: 0 deaths

Start HCQ, Then HCQ

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Stop HCQ, Then no Medication

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Stop Placebo, Then no Medication

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Measure	Start Placebo, Then HCQ n=13 participants at risk Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group A). Followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg. (Group C).	Start HCQ, Then HCQ n=7 participants at risk Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B). Followed by HCQ for 4 weeks (Group D)	Stop HCQ, Then no Medication n=4 participants at risk Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped. (Group E) The patient will be followed up for additional 3 months on no medication. (Group G)	Stop Placebo, Then no Medication n=5 participants at risk lacebo (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the placebo will be stopped. (Group F) The patient will be followed up for additional 3 months on no medication. (Group H)
Infections and infestations Placebogroup Startarm SAE due to infection	7.7% 1/13 • Number of events 1 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/7 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/4 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/5 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.

Other adverse events

Measure	Start Placebo, Then HCQ n=13 participants at risk Placebo for 4 weeks in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group A). Followed by Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg. (Group C).	Start HCQ, Then HCQ n=7 participants at risk Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., one daily dose in the evening for 7 days, then reduction to 6.5 mg/kg bw/d for 3 weeks; the maximum daily dose is 400 mg (Group B). Followed by HCQ for 4 weeks (Group D)	Stop HCQ, Then no Medication n=4 participants at risk Individual dose, usually Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the medication will be stopped. (Group E) The patient will be followed up for additional 3 months on no medication. (Group G)	Stop Placebo, Then no Medication n=5 participants at risk lacebo (given according to the individual dose the patient used to take as usually Hydroxychloroquine Sulfate (HCQ, Quensyl) before 6-10 mg/kg bw/d, p.o., one daily dose in the evening; the maximum daily dose is 400 mg. The dose, on which the patient is included into the trial, was continued for 3 months. After therapy of 3 months the placebo will be stopped. (Group F) The patient will be followed up for additional 3 months on no medication. (Group H)
Infections and infestations AEs gastrointestinal disorder	15.4% 2/13 • Number of events 3 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	28.6% 2/7 • Number of events 4 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	50.0% 2/4 • Number of events 2 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	20.0% 1/5 • Number of events 1 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.
General disorders AE General disorders	7.7% 1/13 • Number of events 1 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	14.3% 1/7 • Number of events 1 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/4 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	20.0% 1/5 • Number of events 1 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.
Infections and infestations Infections	69.2% 9/13 • Number of events 14 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	42.9% 3/7 • Number of events 4 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	50.0% 2/4 • Number of events 6 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	80.0% 4/5 • Number of events 13 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.
Blood and lymphatic system disorders AE Blood	7.7% 1/13 • Number of events 1 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/7 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/4 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/5 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.
Psychiatric disorders AE psychiatric disorders	7.7% 1/13 • Number of events 1 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/7 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/4 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/5 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.
Nervous system disorders AE Nervouse System	7.7% 1/13 • Number of events 1 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/7 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/4 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/5 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.
Eye disorders AE Eye disorders	7.7% 1/13 • Number of events 1 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/7 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/4 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/5 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.
Musculoskeletal and connective tissue disorders AE Musculoskeletal and connective tissue	7.7% 1/13 • Number of events 2 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/7 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	0.00% 0/4 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	20.0% 1/5 • Number of events 1 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.
Respiratory, thoracic and mediastinal disorders AE respiratory disorder	15.4% 2/13 • Number of events 3 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	14.3% 1/7 • Number of events 2 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	50.0% 2/4 • Number of events 2 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.	20.0% 1/5 • Number of events 1 • Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months). Adverse events were collected based on the initial assignment in the START or STOP arms. This resulted in the START arm in an allocation of AEs under HCQ to placebo (only group C) and in the STOP arm of placebo events to HCQ (only group G). This mistake was noticed after closure of the data analysis, when entering into Clin.gov. The overall type and frequency of AEs are correct, the frequencies allocated HCQ may be underestimated and those for placebo overestimated. The SAE occurred in group A.

Additional Information

Prof. Matthias Griese

LMU/ Haunersches Kinderspital

Phone: +498944000

Email: matthias.griese@med.uni-muenchen.de

Results disclosure agreements

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