Disease · MONDO_0010315

T-B+ severe combined immunodeficiency due to gamma chain deficiency

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. T-B+ severe combined immunodeficiency due to gamma chain deficiency. Diseases, record MONDO_0010315. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010315

On this page

Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: T-B+ severe combined immunodeficiency due to gamma chain deficiency; Open Targets
Open Targets ID: MONDO_0010315; Open Targets
Other names: SCIDX1, T-B+ SCID due to gamma chain deficiency, T-B+ severe combined immunodeficiency, X-linked, X-linked severe combined immunodeficiency, XSCID, gamma chain deficiency, severe combined immunodeficiency, X-linked, X-linked recessive, thymic epithelial hypoplasia; Open Targets
Disease Ontology: DOID:0060013; Disease Ontology
NCI Thesaurus: C4682; NCI Thesaurus
Orphanet: ORPHA:276; Orphanet
Highest stage of its drugs: Phase 1/2, for 3 of its 3 drugs, from ClinicalTrials.gov; Open Targets

Drugs3

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
BUSULFAN	CHEMBL820	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
MECASERMIN	CHEMBL1201716	Protein	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
PALIFERMIN	CHEMBL1201821	Protein	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets2

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
FGFR2	Fibroblast growth factor receptor 2	1	Fibroblast growth factor receptor 2 agonist	Phase 1/2	https://conductscience.org/library/genes/2263	Drugs and targets
IGF1R	Insulin-like growth factor 1 receptor	1	Insulin-like growth factor I receptor agonist	Phase 1/2	https://conductscience.org/library/genes/3480	Drugs and targets

Trials2

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00490100	Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1	Phase 1/2	6	2015-07-03	Growth Failure, X-linked Severe Combined Immunodeficiency (XSCID), Growth Hormone Resistence	MECASERMIN	Its record names the disease	ClinicalTrials.gov
NCT01306019	Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)	Phase 1/2	40		X-linked Severe Combined Immunodeficiency (XSCID)	BUSULFAN, PALIFERMIN	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials0

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

None in this release.

Papers from its trials1

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it
35764638	DERIVED	1

Source PubMed

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0010315

GET https://conductscience.org/api/diseases/v1/MONDO_0010315/drugs

API reference For AI assistants