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MucopolysaccharidosesNCT07173010

Pediatric Arthropathy Beyond Inflammation: Clinical Spectrum and Diagnostic Approach at Assiut University Children Hospital

An observational study of Mucopolysaccharidoses and Progressive Pseudorheumatoid Dysplasia, run by Assiut University.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
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Trial summary

To identify the most common underlying causes of non-inflammatory arthropathy in children presenting to Assiut University Children Hospital for through clinical evaluation, laboratory testing, and imaging, in order to improve diagnostic precision, guide appropriate management, and distinguish these conditions from… Read more about this study

Who is it for?
Ages 1 day to 18 years
How many people
35 participants
Where
No sites listed yet
Study dates
Sep 2025to Oct 2026
Phase
Observational
Are there results?
Not yet: the study is under way

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 day to 18 years
  • All sexes
  • Child, Adult

Study population: The study will include all pediatric patients presented with non inflammatory arthropathy based on clinical manifestations, laboratory investigations and imaging up to age of 18 years in the last 3 years (retrospective) and those who will present newly through a duration of one year (preospective). They will be recruited from the inpatient and outpatient clinic of Allergy, Immunology and Rheumatology department at Assuit University Children's hospital.

Who may be able to join

  • Infants and children up to 18 years at diagnosis.
  • Patients presents with picture of non inflammatory arthropathy as joint deformity, stiffness and skeletal abnormality (arthropathy) with normal inflammatory markers

Who cannot join

  • Patients more than 18years.
  • Patients presents with painful,hot, tender or red joints (Arthritis) associated with elevated inflammatory markers .

Trial locations and contacts

No sites are listed yet. Contact the study team or a site near you to ask about taking part.

Nora Ammar Shawket contact

+201129859299

nrnr87355@gmail.com

About this study

To identify the most common underlying causes of non-inflammatory arthropathy in children presenting to Assiut University Children Hospital for through clinical evaluation, laboratory testing, and imaging, in order to improve diagnostic precision, guide appropriate management, and distinguish these conditions from inflammatory joint diseases.

Read the detailed description

Arthropathy refers to any disease or abnormal condition of a joint. It includes both inflammatory ( arthritis) and non-inflammatory types. Arthritis involves joint inflammation with symptoms such as pain, swelling, and stiffness, along with elevated inflammatory markers. In contrast, non-inflammatory arthropathy may mimic arthritis but lacks signs of inflammation and typically shows normal inflammatory markers.

Non inflammatory arthropathy can closely mimic juvenile idiopathic arthritis (JIA), leading to frequent misdiagnosis and inappropriate treatment.

These disorders are sometimes incorrectly diagnosed and treated as JIA, patients often do not respond to immunosuppressive treatment, leading to poor compliance, disease progression, and unnecessary exposure to potentially harmful medications.

A high index of suspicion, detailed clinical evaluation, and appropriate imaging are essential to distinguish these mimics from true inflammatory arthritis.

Here's a breakdown of some conditions categorized as non-inflammatory arthropathies:

Mucopolysaccharidoses (MPS) are rare inherited metabolic disorders caused by enzyme deficiencies that impair the breakdown of glycosaminoglycans (GAGs), leading to their accumulation and progressive tissue and organ damage. Patients often present with arthropathy ,joint stiffness alongside hallmark features such as skeletal abnormalities, developmental delays, intellectual disability, cardiac issues, corneal clouding, hearing loss, short stature, dysostosis multiplex, and coarse facial features.However Some attenuated forms of mucopolysaccharidoses (MPS), particularly MPS I (Scheie syndrome), MPS II (attenuated Hunter syndrome), and MPS VI (Maroteaux-Lamy syndrome), may present predominantly with joint stiffness and contractures without significant systemic or cognitive involvement.

Progressive Pseudorheumatoid Dysplasia (PPRD) is a rare autosomal recessive skeletal disorder caused by mutations in the WISP3 gene, crucial for joint cartilage development and function. It leads to progressive degeneration of articular cartilage, primarily affecting the interphalangeal joints. Patients typically present with painless, progressive joint stiffness and enlargement without inflammation, joint contractures, gait abnormalities, and spinal deformities such as scoliosis or kyphosis, resulting in abnormal posture and muscle weakness-related morbidities.

Camptodactyly-Arthropathy-Coxa Vara-Pericarditis (CACP) syndrome is a rare autosomal recessive disorder characterized by congenital or early-onset camptodactyly, non-inflammatory arthropathy with synovial hyperplasia, progressive coxa vara deformity of the hip, and non-inflammatory pericarditis. Joint effusions are typically cool and unresponsive to anti-inflammatory treatments.

Idiopathic Multicentric Osteolysis is a rare autosomal dominant skeletal disorder characterized by progressive osteolysis primarily affecting the carpal and tarsal bones, leading to joint deformities, pain, and mobility loss. Typically presenting in childhood, patients experience arthritic-like episodes, progressive deformities, osteolytic changes on imaging, and varying degrees of disability. Associated features may include short stature, muscle weakness, cardiovascular abnormalities, chronic renal failure, as well as occasional mental retardation and minor facial anomalies.

Infantile Systemic Hyalinosis (ISH) is a rare autosomal recessive fibromatosis caused by mutations in the ANTXR2 (CMG2) gene, resulting in widespread hyaline material deposition in multiple tissues including skin, joints, and internal organs. Clinically, ISH presents with painful joint contractures which may lead to arthrogryposis-like appearance (multiple joint contractures), Progressive stiffness and fixed flexion deformities develop especially in knees, elbows, hips, and fingers ,Limited range of motion,subcutaneous nodules, gingival hypertrophy, osteopenia, failure to thrive, protein-losing enteropathy with diarrhea, and recurrent infections.

Farber disease is a rare, fatal autosomal recessive metabolic disorder caused by mutations in the ASAH1 gene, leading to deficient acid ceramidase enzyme activity and subsequent ceramide accumulation in tissues. Affected children typically present with progressive painful joint stiffness, limited motion, contractures, subcutaneous nodules, and hoarseness due to laryngeal involvement, which can result in breathing difficulties.

Osteogenesis imperfecta (OI) is a genetic connective tissue disorder caused by mutations in the COL1A1 and COL1A2 genes, leading to defective type I collagen synthesis. Characterized by brittle bones with increased fracture risk and low bone density,Skeletal deformities and recurrent fractures contribute to abnormal joint mechanics, resulting in arthropathy, stiffness, reduced mobility, and functional impairment over time. OI also presents with blue sclerae, dentinogenesis imperfecta, short stature, and adult-onset hearing loss. Cardiovascular manifestations such as valvular insufficiencies and aortic root dilation have been reported. Milder symptoms include joint laxity, easy bruising, hernias, and hyperhidrosis.

Medical subject headings
Mucopolysaccharidoses, Arthropathy, progressive pseudorheumatoid, of childhood, Farber Lipogranulomatosis, Osteogenesis Imperfecta, Hyaline Fibromatosis Syndrome, Osteolysis, Essential

What is being measured

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

  1. To diagnose most common causes of non inflammatory arthropathy

    To identify the most common underlying causes of non-inflammatory arthropathy in children presenting to Assiut University Children Hospital for through clinical evaluation, laboratory testing, and imaging, in order to improve diagnostic precision, guide appropriate management, and distinguish these conditions from inflammatory joint diseases

    Measured: 1year

Secondary outcomes (1)
  1. To avoid misdiagnosis as inflammatory arthropathy

    To Avoid use of unnecessary treatment in patients with non inflammatory arthropathy who wrongly diagnosed as inflammatory arthropathy

    Measured: 1year

Timeline

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

  1. First posted15 Sep 2025
  2. Study startSep 2025 (estimated)
  3. Primary completionSep 2026 (estimated)
  4. Study completionOct 2026 (estimated)

Resources

More details

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

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Cross-sectional
Enrolment
35 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07173010
Sponsor's protocol ID
Non inflammatory arthropathy
Status last verified
Sep 2025
First submitted
6 Sep 2025
Last updated
15 Sep 2025
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • Alfadhli F, Alrehaili L, Bindekhayel JN, Alzamil L, Alrehaili A, Hussain Z. A Rare Presentation of a 12-Year-Old With Systemic Infantile Hyalinosis: A Case Report and Review of the Literature. Cureus. 2024 Aug 9;16(8):e66495. doi: 10.7759/cureus.66495. eCollection 2024 Aug.

    PubMed 39246942
  • Mohammed SE, Mohammed MM, Saeed M, Al Zahrani D, Alasmari BG. Infantile Systemic Hyalinosis: A Case Report and Literature Review. Cureus. 2023 Oct 5;15(10):e46519. doi: 10.7759/cureus.46519. eCollection 2023 Oct.

    PubMed 37927741
  • Sands MS. Farber disease: understanding a fatal childhood disorder and dissecting ceramide biology. EMBO Mol Med. 2013 Jun;5(6):799-801. doi: 10.1002/emmm.201302781. Epub 2013 May 13. No abstract available.

    PubMed 23666771
  • Maatallah K, Boussaa H, Lassoued Ferjani H, Kaffel D, Hamdi W. Progressive pseudorheumatoid dysplasia: A rare entity mimicking juvenile idiopathic arthritis. Clin Case Rep. 2021 Aug 16;9(8):e04670. doi: 10.1002/ccr3.4670. eCollection 2021 Aug.

    PubMed 34430024
  • Muenzer J. Overview of the mucopolysaccharidoses. Rheumatology (Oxford). 2011 Dec;50 Suppl 5:v4-12. doi: 10.1093/rheumatology/ker394.

    PubMed 22210669
  • Khan SA, Peracha H, Ballhausen D, Wiesbauer A, Rohrbach M, Gautschi M, Mason RW, Giugliani R, Suzuki Y, Orii KE, Orii T, Tomatsu S. Epidemiology of mucopolysaccharidoses. Mol Genet Metab. 2017 Jul;121(3):227-240. doi: 10.1016/j.ymgme.2017.05.016. Epub 2017 May 26.

    PubMed 28595941
  • Ablin DS. Osteogenesis imperfecta: a review. Can Assoc Radiol J. 1998 Apr;49(2):110-23.

    PubMed 9561014
  • Carnevale A, Canun S, Mendoza L, del Castillo V. Idiopathic multicentric osteolysis with facial anomalies and nephropathy. Am J Med Genet. 1987 Apr;26(4):877-86. doi: 10.1002/ajmg.1320260415.

    PubMed 3591830
  • Tasar M, Eyileten Z, Kasimzade F, Ucar T, Kendirli T, Uysalel A. Camptodactyly-arthropathy-coxa vara-pericarditis (CACP) syndrome. Turk J Pediatr. 2014 Nov-Dec;56(6):684-6.

    PubMed 26388606
  • Bhavani GS, Al Rashdi S, Shah H, Shukla A, Dalal A, Girisha KM. Progressive Pseudorheumatoid Dysplasia. 2015 Nov 25 [updated 2026 Apr 7]. In: Adam MP, Bick S, Mirzaa GM, Pagon RA, Wallace SE, Amemiya A, editors. GeneReviews(R) [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2026. Available from http://www.ncbi.nlm.nih.gov/books/NBK327267/

    PubMed 26610319
  • Al-Mayouf SM. Noninflammatory disorders mimic juvenile idiopathic arthritis. Int J Pediatr Adolesc Med. 2018 Mar;5(1):1-4. doi: 10.1016/j.ijpam.2018.01.004. Epub 2018 Feb 24.

    PubMed 30805524

Questions about this trial

What is this clinical trial studying?

An observational study of Mucopolysaccharidoses and Progressive Pseudorheumatoid Dysplasia, run by Assiut University.

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 2 inclusion and 2 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: opening soon, as of the sponsor's last update on 15 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.

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?

This study is still under way. Results are not available in the source record yet.

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

Contact Nora Ammar Shawket (+201129859299, nrnr87355@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

To diagnose most common causes of non inflammatory arthropathy (time frame: 1year). 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 NCT07173010, which also lists 11 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 15 Sep 2025.

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