Interstitial Lung DiseasePhase 2HCQ-chILD-EUNCT02615938

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

A Phase 2 study testing Hydroxychloroquine sulfate in people with Interstitial Lung Disease and Diffuse Parenchymal Lung Disease, run by Matthias Griese at 9 sites in Germany.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

This is an exploratory Phase 2, randomized, double-blind, placebo-controlled, parallel-group, multinational study investigating the initiation or withdrawal of hydroxychloroquine in subjects with chILD. Read more about this study

Who is it for?: Ages 3 weeks to 99 years
Conditions the study is for: Interstitial Lung Disease, Diffuse Parenchymal Lung Disease, Children´s Interstitial Lung Disease
What is being tested?: Hydroxychloroquine sulfate
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 35 participants
Where: 9 sitesGermany
Study dates: 21 Aug 2015to 9 Sep 2020
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 3 weeks to 99 years
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Patients should be clinically stable during baseline (between Visit 1 and 2) for inclusion into the study
To determine this, attending physicians can use SpO2 in room air for patients on room air or on O2-supplement; the absolute difference on SpO2 is expected not to be ≥ 5% between Visit 1 and 2. For patients on respiratory support, the summary key parameters should not change ≥ 20% between Visit 1 and 2 and
No major changes in other medications between Visit 1 and 2
Mature newborn ≥ 37 weeks of gestation, age ≥ 3 wks and <2y or Infants and children (≥2y and < 18y) or Adults (≥18 and ≤30y) or Previously preterm (≤ 37 weeks of gestation) babies or children and adults of all ages if chILD genetically diagnosed (see inclusion criterion 3.)
Diagnosis of chronic (≥ 3 wks of duration) diffuse parenchymal lung disease (DPLD = chILD), defined in at least one of the following ways:
chILD genetically diagnosed surfactant dysfunction disorders including patients with mutations in SFTPC, SFTPB, ABCA3, TTF1 (Nkx2-1), further extremely rare entities with specific mutations, for example in TBX4, NPC2, NPC1, NPB, COPA, LRBA and other genes. In this case, also previously preterm (≤ 37 weeks of gestation) babies or children and adults of all ages can be included into the study.

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chILD histologically diagnosed
Chronic pneumonitis of infancy (CPI)
Desquamative interstitial pneumonia (DIP)
Lipoid pneumonitis / Cholesterol pneumonia
Nonspecific interstitial pneumonia (NSIP)
PAP after the exclusion of mutations in GMCSF-Ra/b and GMCSF autoantibodies*
Usual interstitial pneumonia (UIP)
Follicular bronchitis/bronchiolitis/Lymphocytic interstitial pneumonia (LIP)
Storage disease with primary pulmonary involvement (e.g. Niemann Pick)
Hermansky-Pudlak Syndrome
Idiopathic pulmonary haemorrhage (haemosiderosis)*
Other histology diagnosing chILD, in particular combination of the above pattern, but not exclusively
Start block: no HCQ treatment in the last 12 weeks Stop block: stable HCQ treatment for at least the last 12 weeks
Ability of subject or/and legal representatives to understand character and individual consequences of clinical trial.
Signed and dated informed consent of the subject (if subject has the ability) and the representatives (of underaged children) must be available before start of any specific trial procedures.
(may be diagnosed in the absence of a lung biopsy by characteristic lung lavage cytology (PAS stain, Fe stain), CT pattern or autoantibodies (gliadin, endomysium; cANCA) and clinical course.)

Who cannot join

Subjects presenting with any of the following criteria will not be included in the trial:
chILD primarily related to developmental disorders
chILD primarily related to growth abnormalities reflecting deficient alveolarisation
chILD related to chronic aspiration
chILD related to immunodeficiency
chILD related to abnormalities in lung vessel structure

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chILD related to organ transplantation/organ rejection/GvHD
chILD related to recurrent infections
Acute severe infectious exacerbations
Known hypersensitivity to HCQ, or other ingredients of the tablets (lactose-monohydrate, povidone, maize starch, magnesium stearate, hypromellose, macrogol or titanium dioxide (E 171), silicon dioxide or mannitol), to sucrose-octaacetate or sodium saccharine.
Proven retinopathy or maculopathy
Glucose-6-phosphate-dehydrogenase deficiency resulting in favism or hemolytic anemia
Myasthenia gravis
Hematopoetic disorders
Pregnancy and lactation (Women with childbearing potential have to practice a medically accepted contraception during trial and till three months after the end of the treatment with HCQ, and a negative pregnancy test (serum or urine) should be existent on Visit 1, if girls of childbearing age and only if sexual relations are known or probable. It is at the discretion and responsibility of the attending physician to decide, whether a pregnancy test is necessary or not. Reliable contraception are systematic contraceptives (oral, implant, injection). Women that are sterile by surgery can participate in the trial. At the discretion of the investigator, sexual abstinence is also accepted as contraceptive method. Girls after menarche have to receive a counselling about birth control methods in presence of at least one parent, which has to be documented in the patient notes.
Participation in other clinical trials during the present clinical trial or not beyond the time of 4 half-lives of the medication used, at least one week.
Hereditary galactose intolerance, lactase deficiency or glucose-galactose- malabsorption
Renal insufficiency at screening, defined as glomerular filtration rate (GFR)
< 40 mL/min/1.73 m2 in patients age 3 to 8 weeks
< 60 mL/min/1.73 m2 in patients ≥ 8 weeks of age (KDIGO guideline 2012, K/DOQI guideline 2002)
Liver disease, gastrointestinal disorder, haematological disorder, epilepsy or other neurological disorder, psoriasis, porphyria at the discretion of the treating physician
Simultaneous prescription of other potentially nephrotoxic or hepatotoxic medication at the discretion of the treating physician

Trial locations and contacts

9 sites in 1 country.

City, postcode or hospital

9 sites in 1 country

Germany9 sites

Charité Berlin, Klinik für Pädiatrie
Berlin, 13353
St. Joseph- und St. Elisabeth Hospital gGmbH
Bochum, North Rhine-Westphalia, 44791
Uniklinikum Essen, Pädiatrische Pneumologie
Essen, North Rhine-Westphalia, 45122
Universitätsklinikum Frankfurt, Pneumologie, Allergologie, Mukoviszidose
Frankfurt am Main, Hesse, 60590
Justus-Liebig-Universität, Allgemeine Pädiatrie u. Neonatologie
Giessen, Hesse, 35385
Medizinische Hochschule Hannover
Hanover, Lower Saxony, 30625
Klinik u. Poliklinik für Kinder- u. Jugendmedizin der Universität Leipzig
Leipzig, Saxony, 04103
Klinikum der Universität München, Haunersches Kinderspital
München, Bavaria, 80337
Universitätsklinik für Kinder- und Jugendmedizin Tübingen
Tübingen, Baden-Wurttemberg, 72076

Other trials in Germany

About this study

Official title: Hydroxychloroquine in Pediatric ILD: START Randomized Controlled in Parallel-group, Then Switch Placebo to Active Drug, and STOP Randomized Controlled in Parallel-Group to Evaluate the Efficacy and Safety of Hydroxychloroquine (HCQ)

Read the detailed description

This study is an explorative, prospective, randomized, double-blind, placebo controlled investigation of hydroxychloroquine (HCQ) in pediatric ILD. The treatments are organized in START and STOP blocks, which can be initiated in sequence, as needed by the subjects. Each patient can participate in each block only once. In the START block subjects are randomized to parallel-groups, then the placebo group is switched to active drug. In the STOP block, subjects on HCQ are randomized into parallel-groups treated with placebo or HCQ to investigate the withdrawal of HCQ for assessment of its efficacy.

Conditions: Interstitial Lung Disease, Diffuse Parenchymal Lung Disease, Children´s Interstitial Lung Disease
Being tested: Hydroxychloroquine sulfate, Placebo
Keywords: Childhood ILD, Fibrosing lung disease
Medical subject headings: Lung Diseases, Interstitial

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Hydroxychloroquine sulfateDrug
Also called Quensyl
Apply drug to modify lysosomal pH
PlaceboOther
Also called no other name
Apply Placebo not to modify lysosomal pH

Study groups in NCT02615938
Study group	Role	What they receive
Start HCQ block Verum	Experimental	4 weeks of Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., once 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. Then 4 weeks Hydroxychloroquine Sulfate (HCQ, Quensyl). First week loading dose 10 mg/kg bw/d, p.o., once daily in the evening, followed by 6.5 mg/kg bw/d for 3 weeks. Receives: Drug: Hydroxychloroquine sulfate
Start HCQ block Placebo	Placebo comparator	4 weeks of Placebo 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. Then 4 weeks Hydroxychloroquine Sulfate (HCQ, Quensyl). First week loading dose 10 mg/kg bw/d, p.o., once daily in the evening, followed by 6.5 mg/kg bw/d for 3 weeks. Receives: Other: Placebo
Stop HCQ block Verum	Experimental	Individual dose of Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., once daily in the evening; the maximum daily dose is 400 mg. The dose on which the patient was included into the trial was continued for 3 months. Then the medication was stopped and the patient followed for additional 3 months. Receives: Drug: Hydroxychloroquine sulfate
Stop HCQ block Placebo	Placebo comparator	Individual dose of Placebo 6-10 mg/kg bw/d, p.o., once daily in the evening; the maximum daily dose is 400 mg. The dose on which the patient was included into the trial was continued for 3 months. Then the medication was stopped and the patient wo received HCQ will be followed up for additional 3 months with no medication. Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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

Secondary outcomes (11)

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
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
Measured: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Change of O2-sat in Room Air (%)
Oxygen saturation in room air at rest
Measured: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Change of Respiratory Rate in Room Air (Breaths/Min)
Respiratory rate in room air at rest measured in breaths per minute
Measured: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Change of Quality of Life (chILD Specific)
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.
Measured: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Change of Quality of Life (Total Score)
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.
Measured: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Change of BMI Percentile
BMI percentile to adapt for age and sex related differences
Measured: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Change of LDH (U/ml)
Lactate dehydrogenase
Measured: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Absolute Change of Forced Vital Capacity (FVC) (% Predicted of the Reference Population)
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.
Measured: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Absolute Change of Forced Expiratory Volume in One Second (FEV1) (% Predicted of the Reference Population)
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.
Measured: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Absolute Change of Mean Expiratory Flow Between 25 and 75% of Vital Capacity (MEF25-75) (% Predicted of the Reference Population)
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.
Measured: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Absolute Change of Six Minute Walking Test (6MWT) Distance (% Predicted of the Reference Population)
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.
Measured: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

21 Aug 20159 Sep 2020

First posted26 Nov 2015 (estimated)
Study start21 Aug 2015
Primary completion27 Jun 2019
Study completion9 Sep 2020

Why it stopped: Lack of ressources after inspection and appropriate correcting actions to be taken

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (3)Papers the sponsor has linked to this study.
Other trials for Interstitial Lung DiseaseStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Model description: Goal of the study was to include all chILD patients who are planned to be, or are actively being, treated with HCQ. The character of this study is explorative. It was designed to closely accommodate the current clinical care situation.The study was an explorative, prospective, randomized, doubleblind, placebo-controlled investigation of HCQ in chILD and was one of the first investigator-initiated trials in this condition. The study contained two different study blocks, a START and a STOP block, which could be initiated in any sequence. Each patient can participate in each block only once. In the START block, subjects were randomized to parallel groups for 4 weeks treatment, then the placebo group switched to the active drug. In the STOP block, subjects taking HCQ were randomized into parallel groups (placebo or HCQ).
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 35 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02615938
Sponsor's protocol ID: EudratCT:2013-003714-40
Acronym: HCQ-chILD-EU
Status last verified: Nov 2025
First submitted: 8 Nov 2015
Last updated: 3 Dec 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Individual data sharing: No

Publications

Griese M, Kohler M, Witt S, Sebah D, Kappler M, Wetzke M, Schwerk N, Emiralioglu N, Kiper N, Kronfeld K, Ruckes C, Rock H, Anthony G, Seidl E. Prospective evaluation of hydroxychloroquine in pediatric interstitial lung diseases: Study protocol for an investigator-initiated, randomized controlled, parallel-group clinical trial. Trials. 2020 Apr 3;21(1):307. doi: 10.1186/s13063-020-4188-4.
PubMed 32245508
Braun S, Ferner M, Kronfeld K, Griese M. Hydroxychloroquine in children with interstitial (diffuse parenchymal) lung diseases. Pediatr Pulmonol. 2015 Apr;50(4):410-9. doi: 10.1002/ppul.23133. Epub 2014 Dec 9.
PubMed 25491573
Griese M, Kappler M, Stehling F, Schulze J, Baden W, Koerner-Rettberg C, Carlens J, Prenzel F, Nahrlich L, Thalmeier A, Sebah D, Kronfeld K, Rock H, Ruckes C; HCQ-study group; Wetzke M, Seidl E, Schwerk N. Randomized controlled phase 2 trial of hydroxychloroquine in childhood interstitial lung disease. Orphanet J Rare Dis. 2022 Jul 23;17(1):289. doi: 10.1186/s13023-022-02399-2.
PubMed 35871071

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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Study at a glance

From the registry record, last updated by the sponsor on 3 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Hydroxychloroquine in Pediatric ILD: START Randomized Controlled in Parallel-group, Then Switch Placebo to Active Drug, and STOP Randomized Controlled in Parallel-Group to Evaluate the Efficacy and Safety of Hydroxychloroquine (HCQ)
ClinicalTrials.gov ID: NCT02615938
Acronym / sponsor ID: HCQ-chILD-EU / EudratCT:2013-003714-40
Conditions: Interstitial Lung Disease, Diffuse Parenchymal Lung Disease, Children´s Interstitial Lung Disease
Keywords: Childhood ILD, Fibrosing lung disease
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Lack of ressources after inspection and appropriate correcting actions to be taken
Enrolment: 35 participants (actual)
Lead sponsor: Matthias GrieseUniversity, hospital or other
Study start: 21 Aug 2015 (actual)
Primary completion: 27 Jun 2019 (actual)
Study completion: 9 Sep 2020 (actual)
First posted: 26 Nov 2015 (estimated)
Results: Posted 3 Dec 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 4
Model description: Goal of the study was to include all chILD patients who are planned to be, or are actively being, treated with HCQ. The character of this study is explorative. It was designed to closely accommodate the current clinical care situation.The study was an explorative, prospective, randomized, doubleblind, placebo-controlled investigation of HCQ in chILD and was one of the first investigator-initiated trials in this condition. The study contained two different study blocks, a START and a STOP block, which could be initiated in any sequence. Each patient can participate in each block only once. In the START block, subjects were randomized to parallel groups for 4 weeks treatment, then the placebo group switched to the active drug. In the STOP block, subjects taking HCQ were randomized into parallel groups (placebo or HCQ).

Arm	Type	Receives
Start HCQ block Verum	Experimental	Drug: Hydroxychloroquine sulfate4 weeks of Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg bw/d, p.o., once 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. Then 4 weeks Hydroxychloroquine Sulfate (HCQ, Quensyl). First week loading dose 10 mg/kg bw/d, p.o., once daily in the evening, followed by 6.5 mg/kg bw/d for 3 weeks.
Start HCQ block Placebo	Placebo comparator	Other: Placebo4 weeks of Placebo 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. Then 4 weeks Hydroxychloroquine Sulfate (HCQ, Quensyl). First week loading dose 10 mg/kg bw/d, p.o., once daily in the evening, followed by 6.5 mg/kg bw/d for 3 weeks.
Stop HCQ block Verum	Experimental	Drug: Hydroxychloroquine sulfateIndividual dose of Hydroxychloroquine Sulfate (HCQ, Quensyl) 6-10 mg/kg bw/d, p.o., once daily in the evening; the maximum daily dose is 400 mg. The dose on which the patient was included into the trial was continued for 3 months. Then the medication was stopped and the patient followed for additional 3 months.
Stop HCQ block Placebo	Placebo comparator	Other: PlaceboIndividual dose of Placebo 6-10 mg/kg bw/d, p.o., once daily in the evening; the maximum daily dose is 400 mg. The dose on which the patient was included into the trial was continued for 3 months. Then the medication was stopped and the patient wo received HCQ will be followed up for additional 3 months with no medication.

Who may qualify

Ages: 3 weeks to 99 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (22 listed)

Patients should be clinically stable during baseline (between Visit 1 and 2) for inclusion into the study
Mature newborn ≥ 37 weeks of gestation, age ≥ 3 wks and <2y or Infants and children (≥2y and < 18y) or Adults (≥18 and ≤30y) or Previously preterm (≤ 37 weeks of gestation) babies or children and adults of all ages if chILD genetically diagnosed (see inclusion criterion 3.)
Diagnosis of chronic (≥ 3 wks of duration) diffuse parenchymal lung disease (DPLD = chILD), defined in at least one of the following ways:
Start block: no HCQ treatment in the last 12 weeks Stop block: stable HCQ treatment for at least the last 12 weeks
Ability of subject or/and legal representatives to understand character and individual consequences of clinical trial.

Key exclusion criteria (22 listed)

chILD primarily related to developmental disorders
chILD primarily related to growth abnormalities reflecting deficient alveolarisation
chILD related to chronic aspiration
chILD related to immunodeficiency
chILD related to abnormalities in lung vessel structure

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Hydroxychloroquine sulfateAlso: QuensylApply drug to modify lysosomal pH	Drug	Start HCQ block Verum, Stop HCQ block Verum
PlaceboAlso: no other nameApply Placebo not to modify lysosomal pH	Other	Start HCQ block Placebo, Stop HCQ block Placebo

Primary outcome

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

11 secondary outcomes

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
Time frame: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Change of O2-sat in Room Air (%)
Time frame: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Change of Respiratory Rate in Room Air (Breaths/Min)
Time frame: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Change of Quality of Life (chILD Specific)
Time frame: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Change of Quality of Life (Total Score)
Time frame: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Change of BMI Percentile
Time frame: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Change of LDH (U/ml)
Time frame: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Absolute Change of Forced Vital Capacity (FVC) (% Predicted of the Reference Population)
Time frame: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Absolute Change of Forced Expiratory Volume in One Second (FEV1) (% Predicted of the Reference Population)
Time frame: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Absolute Change of Mean Expiratory Flow Between 25 and 75% of Vital Capacity (MEF25-75) (% Predicted of the Reference Population)
Time frame: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks
Absolute Change of Six Minute Walking Test (6MWT) Distance (% Predicted of the Reference Population)
Time frame: 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 and safety

Results posted to the registry on 3 Dec 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Start Placebo, Then HCQ	17	13
Start HCQ, Then HCQ	9	7
Stop HCQ, Then no Medication	4	3
Stop Placebo, Then no Medication	5	5

Primary outcome: 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

Time frame: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks. Count of participants. Unit: Participants

Measure	Start Placebo, Then HCQ (Group A) n = 13	Start HCQ, Then HCQ (Group B) n = 7	Start Placebo, Then HCQ (Group C) n = 12	Start HCQ, Then HCQ (Group D) n = 7	Stop HCQ, Then no Medication (Group E) n = 3	Stop Placebo, Then no Medication (Group F) n = 5	Stop HCQ, Then no Medication (Group G) n = 3	Stop Placebo, Then no Medication (Group H) n = 5
Responder	0	0	3	0	0	0	1	1
Non-responder	13	7	9	7	3	5	2	4

Start Placebo, Then HCQ (Group A) vs Start HCQ, Then HCQ (Group B): p = 1, Fisher Exact
Stop HCQ, Then no Medication (Group E) vs Stop Placebo, Then no Medication (Group F): p = 1.0, Fisher Exact

Population analysed: 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.

11 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse events were collected separately according their occurance during Start periods (8 weeks) or Stop periods (6 months).. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Start Placebo, Then HCQ	1 of 13 (7.7%)	12 of 13 (92.3%)	0 of 13
Start HCQ, Then HCQ	0 of 7 (0%)	7 of 7 (100%)	0 of 7
Stop HCQ, Then no Medication	0 of 4 (0%)	4 of 4 (100%)	0 of 4
Stop Placebo, Then no Medication	0 of 5 (0%)	5 of 5 (100%)	0 of 5

Limitations and caveats (from the sponsor): Limitations include not reaching the target number of participants. As this was the first randomized controlled trial in fibrosing chILD performed ever, the study was exploratory and there was no formal sample size calculation. Including more patients was prevented by interference with an authority inspection, COVID19 pandemic and the ultra-rare conditions targeted. See for more details Orphanet J Rare Dis. 2022 Jul 23;17(1):289. doi: 10.1186/s13023-022-02399-2. PMID: 35871071.

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Research links

Official record on ClinicalTrials.govLast updated by the sponsor 3 Dec 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 32245508Griese M, Kohler M, Witt S, Sebah D, Kappler M, Wetzke M, Schwerk N, Emiralioglu N, Kiper N, Kronfeld K, Ruckes C, Rock H, Anthony G, Seidl E. Prospective evaluation of hydroxychloroquine in pediatric interstitial lung diseases: Study protocol for an investigator-initiated, randomized controlled, parallel-group clinical trial. Trials. 2020 Apr 3;21(1):307. doi: 10.1186/s13063-020-4188-4.
Background: PubMed 25491573Braun S, Ferner M, Kronfeld K, Griese M. Hydroxychloroquine in children with interstitial (diffuse parenchymal) lung diseases. Pediatr Pulmonol. 2015 Apr;50(4):410-9. doi: 10.1002/ppul.23133. Epub 2014 Dec 9.
Results publication: PubMed 35871071Griese M, Kappler M, Stehling F, Schulze J, Baden W, Koerner-Rettberg C, Carlens J, Prenzel F, Nahrlich L, Thalmeier A, Sebah D, Kronfeld K, Rock H, Ruckes C; HCQ-study group; Wetzke M, Seidl E, Schwerk N. Randomized controlled phase 2 trial of hydroxychloroquine in childhood interstitial lung disease. Orphanet J Rare Dis. 2022 Jul 23;17(1):289. doi: 10.1186/s13023-022-02399-2.
Individual participant dataSharing planned: No

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Matthias Griese principal investigator

Hauner Children´s Hospital, LMU

9 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Hydroxychloroquine sulfate in people with Interstitial Lung Disease and Diffuse Parenchymal Lung Disease, run by Matthias Griese at 9 sites in Germany.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 22 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 3 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 3 Dec 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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 (time frame: Start block: A,B after 4 and C,D after 8 weeks; Stop block: E,F after 12 and G,H after 24 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02615938, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Dec 2025.