Trial · NCT07882680
Expanding the Spectrum of Adenosine Deaminase 2 (ADA2) Deficiency: Towards a Gene Therapy Approach
NCT07882680, Not applicable, Completed, Istituto Giannina Gaslini
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Expanding the Spectrum of Adenosine Deaminase 2 (ADA2) Deficiency: Towards a Gene Therapy Approach. Trials, record NCT07882680. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07882680
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Expanding the Spectrum of Adenosine Deaminase 2 (ADA2) Deficiency: Towards a Gene Therapy Approach
- NCT number
- NCT07882680
- Status
- Completed
- Phases
- Not applicable
- Enrollment
- 29 participants, actual
- Lead sponsor
- Istituto Giannina Gaslini
- Conditions
- Adenosine Deaminase Deficiency
- Interventions
- bone marrow aspiration and biopsy
- First posted
- 2026-10-20
- Last updated
- 2026-10-20
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Increase the number of DADA2 diagnoses | Month 36 | Increase the rate of DADA2 diagnosis through the development of a new enzymatic assay to measure ADA2 activity in dried plasma spots on filter paper and by providing access to molecular testing for patients with suspected DADA2. We will also establish an Italian cohort of patients with DADA2 through the already established Eurofever registry. |
| Primary | Functional and phenotypic characterization of the bone marrow in patients with DADA2 and preclinical studies for gene therapy. | Month 36 | The pathogenesis of hematological manifestations in DADA2 remains largely unknown. Possible mechanisms include an intrinsic defect in ADA2 in bone marrow (BM) progenitor cells or an environmental effect mediated by pro-inflammatory cytokines, as suggested by the strong interferon signature observed in patients with DADA2. The efficacy of anti-TNF treatment in these patients remains controversial, as both treatment-responsive and treatment-refractory cases have been reported, with some patients requiring hematopoietic stem cell transplantation (HSCT). It is therefore clinically relevant to investigate the potential roles of type I interferon and TNF in the development of hematological manifestations of DADA2 and to determine whether their effects occur at earlier or later stages of bone marrow damage. This could influence therapeutic decision-making by establishing whether early TNF inhibitor treatment provides a clinical benefit or slows the progression of hematological |
| Primary | Develop a Gene Therapy Approach for DADA2 | Month 36 | The development of a gene therapy approach for autologous stem cell transplantation may offer a curative treatment for DADA2. Alternative therapeutic strategies are needed to avoid potential transplant-related complications, such as graft-versus-host disease (GVHD) and veno-occlusive disease. There is a strong rationale for developing an approach based on the genetic correction of patients' hematopoietic stem and progenitor cells. Gene therapy for ADA1 deficiency, a form of severe combined immunodeficiency (SCID), has demonstrated safety and efficacy in clinical trials conducted by Unit 2 . Furthermore, studies conducted by Unit 2 and other research groups have shown that gene therapy based on the ex vivo transduction of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) using lentiviral vectors (LVs) can provide stable, long-term gene expression, even in slowly dividing cells that are difficult to transduce, including long-term repopulating hematopoietic stem ce |
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/NCT07882680
GET https://conductscience.org/api/trials/v1/NCT07882680/registered