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diffuse scleroderma - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

diffuse scleroderma

MONDO:0005019

Also known as:diffuse systemic sclerosissystemic sclerosis, diffuse

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Overview

A variant of systemic scleroderma characterized by sclerosis of the skin, Raynaud phenomenon, and organ involvement, including pulmonary fibrosis, renal disease, and gastrointestinal tract involvement.

TreatmentsNo approved treatments · 1 in pipeline

Clinical Trials134 registered · 65 recruiting

Diagnosis

Biomarker and diagnostic research for diffuse scleroderma has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

No approved treatments are currently available for diffuse scleroderma. An additional 1 compound holds orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for diffuse scleroderma, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for diffuse scleroderma. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

134 clinical trials registered, 65 recruiting. Interventions under study include other interventions, drug therapy, biologic therapy, and procedural interventions. Pipeline includes 5 PHASE4, 5 PHASE3, 15 PHASE2. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT06733935](https://clinicaltrials.gov/study/NCT06733935)

Data assembled from 4 of 12 sources · Last updated Sep 20, 2026, 1:45 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

GARD

Genetic and Rare Diseases Info Center

GARD:7727

Questions for Your Care Team

→Are any of the 134 active clinical trials a good fit for my situation?
→What is the current development status of Type 1 native bovine skin collagen, and when might it become available?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

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Research Type	Count	% of Total
Patient case studies	21	42%
Disease patterns and progression	11	22%
Clinical study results	5	10%
Other research	3	6%
Research summaries	3	6%
Laboratory research	3	6%
New treatment approaches	3	6%
Testing and diagnosis research	1	2%

diffuse scleroderma

Overview

Diagnosis

Treatment

Orphan Drug Designations

Research Landscape

Medical References

External Resources

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

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