Surfactant DysfunctionNCT03822780
Hydroxychloroquin (HCQ) in chILD of Genetic Defect
A study testing Hydroxychloroquin in people with Surfactant Dysfunction, run by Children's Hospital of Fudan University at 1 site in China.
Trial summary
The purpose of this proposed research is to investigate the efficacy and safety of hydroxychloroquine sulfate (HCQ, Quensyl) for pediatric ILD(chILD) caused by pulmonary surfactant-associated genes mutations. Read more about this study
- Who is it for?
- Ages 1 month to 18 years
- Conditions the study is for
- Surfactant Dysfunction
- What is being tested?
- Hydroxychloroquin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2017to 15 Mar 2026
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 1 month to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients should be clinically stable for inclusion into the study
- Mature newborn ≥ 37 weeks of gestation, Infants and children (≥2month and < 18y) or previously preterm (≤ 37 weeks of gestation) babies or children(≥2month and <18y) if chILD genetically diagnosed
- chILD genetically diagnosed surfactant dysfunction disorders including patients with mutations in SFTPC, SFTPB, ABCA3, TTF1 (Nkx2-1), FOXF1 further extremely rare entities with specific mutations, for example in TBX4, NPC2, NPC1, NPB, COPA, LRBA and other genes
- no HCQ treatment in the last 3 months
- 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
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 alveolarization
- chILD related to chronic aspiration
- chILD related to immunodeficiency
- chILD related to abnormalities in lung vessel structure
Show all 17Show fewer
- 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
- Proven retinopathy or maculopathy
- Glucose-6-phosphate-dehydrogenase deficiency resulting in favism or hemolytic anemia
- Myasthenia gravis
- Hematopoetic disorders
- 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
- Simultaneous prescription of other potentially nephrotoxic or hepatotoxic medication at the discretion of the treating physician
Trial locations and contacts
1 site in 1 country.
China1 site
Children's hospital of Fudan University
Shanghai, Shanghai Municipality, 201102
About this study
Official title: Hydroxychloroquine in Pediatric ILD With Genetic Surfactant Dysfunction Disorders: Cross-control, Prospective Study
The purpose of this proposed research is to investigate the efficacy and safety of hydroxychloroquine sulfate (HCQ, Quensyl) for pediatric ILD(chILD) caused by pulmonary surfactant-associated genes mutations.
Read the detailed description
Children Interstitial lung disease (chILD) is a heterogeneous group of rare respiratory disorders of known and unknown etiologies that are mostly chronic and associated with high morbidity and mortality. ILD are characterized by inflammatory and fibrotic changes of the lung parenchyma structure that typically result in the presence of diffuse infiltrates on lung imaging, and abnormal pulmonary function tests with evidence of a restrictive ventilatory defect and/or impaired gas exchange.
Genetic factors are important contributors to chILD. Genetic variations have been mainly described in genes encoding (or interacting with) the surfactant proteins (SP): SP-C (SFTPC) and the ATP-binding cassette-family A-member 3 (ABCA3) (ABCA3), and less frequently in the genes encoding NKX homeobox 2 (NKX2)-1 (NKX2-1), SP-B (SFTPB), SP-A (SFTPA) and other genes.
To date, the therapeutic managements of such chILD remain limited and are mainly based of the use of corticosteroids, however, their efficacy is highly variable. An alternative approach to treatment was originally described by Tooley who reported a good response to treatment with chloroquine in a girl with ILD, and several case reports have shown a positive response to hydroxychloroquine(HCQ) alone or in combination with systemic steroids of the children with ILD.
The exact mechanism of action of HCQ is unknown, but is probably due to its anti-inflammatory properties, HCQ have lysosomal activities such as diminished vesicle fusion, diminished exocytosis, decreased digestive efficiency of phagolysosomes and reversible "lysosomal storage disease. This may be the mechanism by which HCQ tend to help in chILD, especially in those cases related to surfactant protein deficiency. SP-B and SP-C are synthesized in the endoplasmic reticulum (ER) of alveolar type II cells as large precursor proteins, are cleaved by proteolytic enzymes and transported through Golgi apparatus to multivesicular bodies that fuse with lamellar bodies. In chILD related to SP-C gene mutations, there is misfolding of proSP-C that accumulates within ER and Golgi apparatus in alveolar type II cells, resulting in cellular injury and apoptosis. Treatment with HCQ may interfere with this accumulation of pro-surfactant proteins within alveolar cells.
The investigators propose to study the efficacy and safety of the therapy with HCQ for children with chILD suffered with genetic mutations, and its long-term effects. Through this study the investigators hope to confirm the benefits of HCQ in the treatment of this rare disease.
- Conditions
- Surfactant Dysfunction
- Being tested
- Hydroxychloroquin
- Keywords
- Interstitial lung disease, Hydroxychloroquine
- Medical subject headings
- Surfactant Dysfunction, Lung Diseases, Interstitial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HydroxychloroquinDrug
Also called Hydroxychloroquine Sulfate; Plaquenil
Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg*d, p.o., bid. After the illness gradually alleviate to maintain dose between 5mg/kg*d to 10mg/kg*d, p.o., bid ; the maximum daily dose is 400mg.
| Study group | Role | What they receive |
|---|---|---|
| HCQ Therapy | Experimental | Hydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg*d, p.o., bid. After the illness gradually alleviate to maintain dose between 5mg/kg*d to 10mg/kg*d, p.o., bid ; the maximum daily dose is 400mg.
Assess the efficacy and safety of HCQ after 6 months treatment compared with any other routine therapy before HCQ therapy (such as inhaling oxygen, corticosteroid, anti-infection therapy, nutritional support) Receives: Drug: Hydroxychloroquin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Oxygenation change
Clinical judgment of oxygenation condition at 6 months compared with trial day 1 (demand of oxygen supplement while transcutaneous oxygen saturation no less than 92% and with no clinical manifestations of hypoxia)
Measured: 6 months
Secondary outcomes (50)
Oxygen flow rate
O2 supplement(L/min) at 1 months compared with trial day 1
Measured: 1 month
Oxygen flow rate
O2 supplement(L/min) at 3 months compared with trial day 1
Measured: 3 month
Oxygen flow rate
O2 supplement(L/min) at 12 months compared with trial day 1
Measured: 12 month
Oxygen flow rate
O2 supplement(L/min) at 18 months compared with trial day 1
Measured: 18 month
Oxygen flow rate
O2 supplement(L/min) at 24 months compared with trial day 1
Measured: 24 month
Fraction of inspired oxygen(FiO2)
Fraction of inspired oxygen(FiO2) at 1 months compared with trial day 1
Measured: 1 month
Fraction of inspired oxygen
FiO2 at 3 months compared with trial day 1
Measured: 3 month
Fraction of inspired oxygen
FiO2 at 6 months compared with trial day 1
Measured: 6 month
Fraction of inspired oxygen
FiO2 at 12 months compared with trial day 1
Measured: 12 month
Fraction of inspired oxygen
FiO2 at 18 months compared with trial day 1
Measured: 18 month
Fraction of inspired oxygen
FiO2 at 24 months compared with trial day 1
Measured: 24 month
Number of subjects with oxygen inhalation
Number of subjects at 6 months compared with trial day 1
Measured: 6 months
Number of subjects with oxygen inhalation
Number of subjects at 12 months compared with trial day 1
Measured: 12 months
Number of subjects with oxygen inhalation
Number of subjects at 24 months compared with trial day 1
Measured: 24 months
Transcutaneous oxygen saturation
O2-sat at 1 months compared with trial day 1
Measured: 1 months
Transcutaneous oxygen saturation
O2-sat at 3 months compared with trial day 1
Measured: 3 months
Transcutaneous oxygen saturation
O2-sat at 12 months compared with trial day 1
Measured: 12 months
Transcutaneous oxygen saturation
O2-sat at 18 months compared with trial day 1
Measured: 18 months
Transcutaneous oxygen saturation
O2-sat at 24 months compared with trial day 1
Measured: 24 months
Respiratory rate
Respiratory rate(RR) at 1 months compared with trial day 1
Measured: 1 months
Respiratory rate
RR at 3 months compared with trial day 1
Measured: 3 months
Respiratory rate
RR at 6 months compared with trial day 1
Measured: 6 months
Respiratory rate
RR at 12 months compared with trial day 1
Measured: 12 months
Chronic cough
(yes/no)
Measured: 6 months
Chronic cough
(yes/no)
Measured: 12 months
Chronic cough
(yes/no)
Measured: 24 months
Clubbing finger
(yes/no)
Measured: 6 months
Clubbing finger
(yes/no)
Measured: 12 months
Clubbing finger
(yes/no)
Measured: 24 months
Functional lesion of liver and kidney
(yes/no)
Measured: 3 months
Functional lesion of liver and kidney
(yes/no)
Measured: 6 months
Functional lesion of liver and kidney
(yes/no)
Measured: 12 months
Functional lesion of liver and kidney
(yes/no)
Measured: 24 months
Malnutrition
(yes/no) at 3 months compared with trial day 1, based on Reference criteria for growth and development of children under 7 years old in China
Measured: 3 months
Malnutrition
(yes/no) at 6 months compared with trial day 1, based on Reference criteria for growth and development of children under 7 years old in China
Measured: 6 months
Malnutrition
(yes/no) at 12 months compared with trial day 1, based on Reference criteria for growth and development of children under 7 years old in China
Measured: 12 months
Malnutrition
(yes/no) at 24 months compared with trial day 1, based on Reference criteria for growth and development of children under 7 years old in China
Measured: 24 months
Deterioration of pulmonary imaging
(yes/no) Clinical judgment of pulmonary imaging compared with that of last visit if X-ray or CT were done at 6 months
Measured: 6 months
Deterioration of pulmonary imaging
Clinical judgment of pulmonary imaging compared with that of last visit if X-ray or CT were done at 12 months
Measured: 12 months
Deterioration of pulmonary imaging
Clinical judgment of pulmonary imaging compared with that of last visit if X-ray or CT were done at 24 months
Measured: 24 months
Lung function decline
(yes/no) Clinical judgment of Lung-function compared with that of last visit if lung-function testing were done at 3 years
Measured: 3 years
Lung function decline
(yes/no) Clinical judgment of Lung-function compared with that of last visit if lung-function testing were done at 6 years
Measured: 6 years
Abnormal myocardial zymogram
(yes/no)
Measured: 3 months
Abnormal myocardial zymogram
(yes/no)
Measured: 6 months
Abnormal myocardial zymogram
(yes/no)
Measured: 12 months
Duration of oxygen inhalation
The time last from HCQ treatment to withdrawal of oxygen (months)
Measured: 36 months
Mortality
Number of deaths at 3 months
Measured: 3 months
Mortality
Number of deaths at 12 months
Measured: 12 months
Mortality
Number of deaths at 24 months
Measured: 24 months
Number of Treatment related adverse events
Measured on each visit
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2019
- Study start1 Jul 2017
- Primary completion15 Mar 2026
- Study completion15 Mar 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (10)Papers the sponsor has linked to this study.
- Other trials for Surfactant DysfunctionStudies of the same condition, open and under way.
- More trials run by Children's Hospital of Fudan UniversityThe sponsor's other registered studies.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- cross-control
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Fudan University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03822780
- Sponsor's protocol ID
- jzxfb-qlv
- Status last verified
- Mar 2026
- First submitted
- 15 Jan 2019
- Last updated
- 19 Mar 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Clement A, Nathan N, Epaud R, Fauroux B, Corvol H. Interstitial lung diseases in children. Orphanet J Rare Dis. 2010 Aug 20;5:22. doi: 10.1186/1750-1172-5-22.
PubMed 20727133Nathan N, Borensztajn K, Clement A. Genetic causes and clinical management of pediatric interstitial lung diseases. Curr Opin Pulm Med. 2018 May;24(3):253-259. doi: 10.1097/MCP.0000000000000471.
PubMed 29517585Barnett HL. editor. Pediatrics. 15th edition. New York: Appleton Century-Crofts; 1972. pp 1315-1316.
Rosen DM, Waltz DA. Hydroxychloroquine and surfactant protein C deficiency. N Engl J Med. 2005 Jan 13;352(2):207-8. doi: 10.1056/NEJM200501133520223. No abstract available.
PubMed 15647591Kroner C, Reu S, Teusch V, Schams A, Grimmelt AC, Barker M, Brand J, Gappa M, Kitz R, Kramer BW, Lange L, Lau S, Pfannenstiel C, Proesmans M, Seidenberg J, Sismanlar T, Aslan AT, Werner C, Zielen S, Zarbock R, Brasch F, Lohse P, Griese M. Genotype alone does not predict the clinical course of SFTPC deficiency in paediatric patients. Eur Respir J. 2015 Jul;46(1):197-206. doi: 10.1183/09031936.00129414. Epub 2015 Feb 5.
PubMed 25657025Hevroni A, Goldman A, Springer C. Infant pulmonary function testing in chronic pneumonitis of infancy due to surfactant protein C mutation. Pediatr Pulmonol. 2015 Jun;50(6):E17-23. doi: 10.1002/ppul.23166. Epub 2015 Mar 9.
PubMed 25755194Avital A, Hevroni A, Godfrey S, Cohen S, Maayan C, Nusair S, Nogee LM, Springer C. Natural history of five children with surfactant protein C mutations and interstitial lung disease. Pediatr Pulmonol. 2014 Nov;49(11):1097-105. doi: 10.1002/ppul.22971. Epub 2013 Dec 17.
PubMed 24347114Thouvenin G, Abou Taam R, Flamein F, Guillot L, Le Bourgeois M, Reix P, Fayon M, Counil F, Depontbriand U, Feldmann D, Pointe HD, de Blic J, Clement A, Epaud R. Characteristics of disorders associated with genetic mutations of surfactant protein C. Arch Dis Child. 2010 Jun;95(6):449-54. doi: 10.1136/adc.2009.171553. Epub 2010 Apr 19.
PubMed 20403820Hepping N, Griese M, Lohse P, Garbe W, Lange L. Successful treatment of neonatal respiratory failure caused by a novel surfactant protein C p.Cys121Gly mutation with hydroxychloroquine. J Perinatol. 2013 Jun;33(6):492-4. doi: 10.1038/jp.2012.131.
PubMed 23719253Arikan-Ayyildiz Z, Caglayan-Sozmen S, Isik S, Deterding R, Dishop MK, Couderc R, Epaud R, Louha M, Uzuner N. Survival of an infant with homozygous surfactant protein C (SFTPC) mutation. Pediatr Pulmonol. 2014 Mar;49(3):E112-5. doi: 10.1002/ppul.22976. Epub 2013 Dec 17.
PubMed 24347240
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hydroxychloroquine in Pediatric ILD With Genetic Surfactant Dysfunction Disorders: Cross-control, Prospective Study
- ClinicalTrials.gov ID
- NCT03822780
- Acronym / sponsor ID
- jzxfb-qlv
- Conditions
- Surfactant Dysfunction
- Keywords
- Interstitial lung disease, Hydroxychloroquine
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Children's Hospital of Fudan UniversityUniversity, hospital or other
- Study start
- 1 Jul 2017 (actual)
- Primary completion
- 15 Mar 2026 (actual)
- Study completion
- 15 Mar 2026 (actual)
- First posted
- 30 Jan 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this proposed research is to investigate the efficacy and safety of hydroxychloroquine sulfate (HCQ, Quensyl) for pediatric ILD(chILD) caused by pulmonary surfactant-associated genes mutations.
Detailed description
Children Interstitial lung disease (chILD) is a heterogeneous group of rare respiratory disorders of known and unknown etiologies that are mostly chronic and associated with high morbidity and mortality. ILD are characterized by inflammatory and fibrotic changes of the lung parenchyma structure that typically result in the presence of diffuse infiltrates on lung imaging, and abnormal pulmonary function tests with evidence of a restrictive ventilatory defect and/or impaired gas exchange.
Genetic factors are important contributors to chILD. Genetic variations have been mainly described in genes encoding (or interacting with) the surfactant proteins (SP): SP-C (SFTPC) and the ATP-binding cassette-family A-member 3 (ABCA3) (ABCA3), and less frequently in the genes encoding NKX homeobox 2 (NKX2)-1 (NKX2-1), SP-B (SFTPB), SP-A (SFTPA) and other genes.
To date, the therapeutic managements of such chILD remain limited and are mainly based of the use of corticosteroids, however, their efficacy is highly variable. An alternative approach to treatment was originally described by Tooley who reported a good response to treatment with chloroquine in a girl with ILD, and several case reports have shown a positive response to hydroxychloroquine(HCQ) alone or in combination with systemic steroids of the children with ILD.
The exact mechanism of action of HCQ is unknown, but is probably due to its anti-inflammatory properties, HCQ have lysosomal activities such as diminished vesicle fusion, diminished exocytosis, decreased digestive efficiency of phagolysosomes and reversible "lysosomal storage disease. This may be the mechanism by which HCQ tend to help in chILD, especially in those cases related to surfactant protein deficiency. SP-B and SP-C are synthesized in the endoplasmic reticulum (ER) of alveolar type II cells as large precursor proteins, are cleaved by proteolytic enzymes and transported through Golgi apparatus to multivesicular bodies that fuse with lamellar bodies. In chILD related to SP-C gene mutations, there is misfolding of proSP-C that accumulates within ER and Golgi apparatus in alveolar type II cells, resulting in cellular injury and apoptosis. Treatment with HCQ may interfere with this accumulation of pro-surfactant proteins within alveolar cells.
The investigators propose to study the efficacy and safety of the therapy with HCQ for children with chILD suffered with genetic mutations, and its long-term effects. Through this study the investigators hope to confirm the benefits of HCQ in the treatment of this rare disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- cross-control
| Arm | Type | Receives |
|---|---|---|
| HCQ Therapy | Experimental | Drug: HydroxychloroquinHydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg*d, p.o., bid. After the illness gradually alleviate to maintain dose between 5mg/kg*d to 10mg/kg*d, p.o., bid ; the maximum daily dose is 400mg. Assess the efficacy and safety of HCQ after 6 months treatment compared with any other routine therapy before HCQ therapy (such as inhaling oxygen, corticosteroid, anti-infection therapy, nutritional support) |
Who may qualify
- Ages
- 1 month to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Patients should be clinically stable for inclusion into the study
- Mature newborn ≥ 37 weeks of gestation, Infants and children (≥2month and < 18y) or previously preterm (≤ 37 weeks of gestation) babies or children(≥2month and <18y) if chILD genetically diagnosed
- chILD genetically diagnosed surfactant dysfunction disorders including patients with mutations in SFTPC, SFTPB, ABCA3, TTF1 (Nkx2-1), FOXF1 further extremely rare entities with specific mutations, for example in TBX4, NPC2, NPC1, NPB, COPA, LRBA and other genes
- no HCQ treatment in the last 3 months
- Ability of subject or/and legal representatives to understand character and individual consequences of clinical trial
Key exclusion criteria (17 listed)
- chILD primarily related to developmental disorders
- chILD primarily related to growth abnormalities reflecting deficient alveolarization
- 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 |
|---|---|---|
| HydroxychloroquinAlso: Hydroxychloroquine Sulfate; PlaquenilHydroxychloroquine Sulfate (HCQ, Quensyl) in a loading dose of 10 mg/kg*d, p.o., bid. After the illness gradually alleviate to maintain dose between 5mg/kg*d to 10mg/kg*d, p.o., bid ; the maximum daily dose is 400mg. | Drug | HCQ Therapy |
Primary outcome
Oxygenation change
Time frame: 6 months
Clinical judgment of oxygenation condition at 6 months compared with trial day 1 (demand of oxygen supplement while transcutaneous oxygen saturation no less than 92% and with no clinical manifestations of hypoxia)
50 secondary outcomes
Oxygen flow rate
Time frame: 1 month
Oxygen flow rate
Time frame: 3 month
Oxygen flow rate
Time frame: 12 month
Oxygen flow rate
Time frame: 18 month
Oxygen flow rate
Time frame: 24 month
Fraction of inspired oxygen(FiO2)
Time frame: 1 month
Fraction of inspired oxygen
Time frame: 3 month
Fraction of inspired oxygen
Time frame: 6 month
Fraction of inspired oxygen
Time frame: 12 month
Fraction of inspired oxygen
Time frame: 18 month
Fraction of inspired oxygen
Time frame: 24 month
Number of subjects with oxygen inhalation
Time frame: 6 months
Number of subjects with oxygen inhalation
Time frame: 12 months
Number of subjects with oxygen inhalation
Time frame: 24 months
Transcutaneous oxygen saturation
Time frame: 1 months
Transcutaneous oxygen saturation
Time frame: 3 months
Transcutaneous oxygen saturation
Time frame: 12 months
Transcutaneous oxygen saturation
Time frame: 18 months
Transcutaneous oxygen saturation
Time frame: 24 months
Respiratory rate
Time frame: 1 months
Respiratory rate
Time frame: 3 months
Respiratory rate
Time frame: 6 months
Respiratory rate
Time frame: 12 months
Chronic cough
Time frame: 6 months
Chronic cough
Time frame: 12 months
Chronic cough
Time frame: 24 months
Clubbing finger
Time frame: 6 months
Clubbing finger
Time frame: 12 months
Clubbing finger
Time frame: 24 months
Functional lesion of liver and kidney
Time frame: 3 months
Functional lesion of liver and kidney
Time frame: 6 months
Functional lesion of liver and kidney
Time frame: 12 months
Functional lesion of liver and kidney
Time frame: 24 months
Malnutrition
Time frame: 3 months
Malnutrition
Time frame: 6 months
Malnutrition
Time frame: 12 months
Malnutrition
Time frame: 24 months
Deterioration of pulmonary imaging
Time frame: 6 months
Deterioration of pulmonary imaging
Time frame: 12 months
Deterioration of pulmonary imaging
Time frame: 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2026.
- Background: PubMed 20727133Clement A, Nathan N, Epaud R, Fauroux B, Corvol H. Interstitial lung diseases in children. Orphanet J Rare Dis. 2010 Aug 20;5:22. doi: 10.1186/1750-1172-5-22.
- Background: PubMed 29517585Nathan N, Borensztajn K, Clement A. Genetic causes and clinical management of pediatric interstitial lung diseases. Curr Opin Pulm Med. 2018 May;24(3):253-259. doi: 10.1097/MCP.0000000000000471.
- BackgroundBarnett HL. editor. Pediatrics. 15th edition. New York: Appleton Century-Crofts; 1972. pp 1315-1316.
- Background: PubMed 15647591Rosen DM, Waltz DA. Hydroxychloroquine and surfactant protein C deficiency. N Engl J Med. 2005 Jan 13;352(2):207-8. doi: 10.1056/NEJM200501133520223. No abstract available.
- Background: PubMed 25657025Kroner C, Reu S, Teusch V, Schams A, Grimmelt AC, Barker M, Brand J, Gappa M, Kitz R, Kramer BW, Lange L, Lau S, Pfannenstiel C, Proesmans M, Seidenberg J, Sismanlar T, Aslan AT, Werner C, Zielen S, Zarbock R, Brasch F, Lohse P, Griese M. Genotype alone does not predict the clinical course of SFTPC deficiency in paediatric patients. Eur Respir J. 2015 Jul;46(1):197-206. doi: 10.1183/09031936.00129414. Epub 2015 Feb 5.
- Background: PubMed 25755194Hevroni A, Goldman A, Springer C. Infant pulmonary function testing in chronic pneumonitis of infancy due to surfactant protein C mutation. Pediatr Pulmonol. 2015 Jun;50(6):E17-23. doi: 10.1002/ppul.23166. Epub 2015 Mar 9.
- Background: PubMed 24347114Avital A, Hevroni A, Godfrey S, Cohen S, Maayan C, Nusair S, Nogee LM, Springer C. Natural history of five children with surfactant protein C mutations and interstitial lung disease. Pediatr Pulmonol. 2014 Nov;49(11):1097-105. doi: 10.1002/ppul.22971. Epub 2013 Dec 17.
- Background: PubMed 20403820Thouvenin G, Abou Taam R, Flamein F, Guillot L, Le Bourgeois M, Reix P, Fayon M, Counil F, Depontbriand U, Feldmann D, Pointe HD, de Blic J, Clement A, Epaud R. Characteristics of disorders associated with genetic mutations of surfactant protein C. Arch Dis Child. 2010 Jun;95(6):449-54. doi: 10.1136/adc.2009.171553. Epub 2010 Apr 19.
- Background: PubMed 23719253Hepping N, Griese M, Lohse P, Garbe W, Lange L. Successful treatment of neonatal respiratory failure caused by a novel surfactant protein C p.Cys121Gly mutation with hydroxychloroquine. J Perinatol. 2013 Jun;33(6):492-4. doi: 10.1038/jp.2012.131.
- Background: PubMed 24347240Arikan-Ayyildiz Z, Caglayan-Sozmen S, Isik S, Deterding R, Dishop MK, Couderc R, Epaud R, Louha M, Uzuner N. Survival of an infant with homozygous surfactant protein C (SFTPC) mutation. Pediatr Pulmonol. 2014 Mar;49(3):E112-5. doi: 10.1002/ppul.22976. Epub 2013 Dec 17.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Hydroxychloroquin in people with Surfactant Dysfunction, run by Children's Hospital of Fudan University at 1 site in China.
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 6 inclusion and 17 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: finished, as of the sponsor's last update on 19 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Oxygenation change (time frame: 6 months). 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 NCT03822780, which also lists 10 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 19 Mar 2026.