Trial · NCT07877727
Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data
NCT07877727, Recruiting, Istituto Giannina Gaslini
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data. Trials, record NCT07877727. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07877727
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data
- NCT number
- NCT07877727
- Status
- Recruiting
- Enrollment
- 250 participants, estimated
- Lead sponsor
- Istituto Giannina Gaslini
- Conditions
Inherited Bone Marrow Failure Syndrome, Fanconi Anemia, Dyskeratosis Congenita, Telomere Biology Disorder, Congenital Neutropenia, Primary Immune Regulatory Disorder, Congenital Amegakaryocytic Thrombocytopenia, Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, PNH
- First posted
- 2026-10-16
- Last updated
- 2026-10-16
Source ClinicalTrials.gov
Registered outcomes1
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Early Diagnosis and Molecular Characterization of CMF and AAA | Within the 24-month project period | To improve the early diagnosis of constitutional bone marrow failure syndromes (CMF) and acquired aplastic anemia (AAA) through the integration of clinical, laboratory, immunological, and genetic data. At least 50 newly enrolled patients will be evaluated prospectively over the first 21 months, alongside approximately 200 previously referred patients analyzed retrospectively using artificial intelligence (AI). The diagnostic work-up will include the implementation of additional tests, such as telomere length assessment by flow-FISH, serum thrombopoietin measurement, and immunophenotyping by flow cytometry. Whole-genome sequencing and functional studies will be used to identify disease-causing variants, copy number variations (CNVs), and clarify variants of uncertain significance (VUS). Early and accurate diagnosis will support appropriate targeted treatment, genetic counseling, surveillance for bone marrow failure and malignancies, and identification of suitable related donors. |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
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
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT07877727
GET https://conductscience.org/api/trials/v1/NCT07877727/registered