Complete DiGeorge AnomalyNCT05329935
Congenital Athymia Patient Registry
An observational study of Complete DiGeorge Anomaly and Complete DiGeorge Syndrome, run by Sumitomo Pharma Switzerland GmbH at 1 site in United States.
Trial summary
This Congenital Athymia Patient Registry is an observational exposure-based registry study. It uses a prospective cohort design to follow patients who have been treated with RETHYMIC. Clinical studies conducted with investigational RETHYMIC showed that treatment can result in immune reconstitution and prolong life. Read more about this study
- Who is it for?
- Ages 0 to 21 years
- Conditions the study is for
- Complete DiGeorge Anomaly, Complete DiGeorge Syndrome, Congenital Athymia
- What is being tested?
- Cultured Thymus Tissue
- How many people
- 75 participants
- Where
- 1 siteUnited States
- Study dates
- 25 May 2022to Apr 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 0 to 21 years
- All sexes
- Child, Adult
Study population: Pediatric patients diagnosed with Congenital Athymia: • Who are scheduled for implantation with RETHYMIC within the next 30 days, or who were treated with RETHYMIC within the previous 60 days.
Who may be able to join
- Pediatric patients diagnosed with Congenital Athymia:
- Who are scheduled for implantation with RETHYMIC within the next 30 days, or who were treated with RETHYMIC within the previous 60 days.
- Who have provided written informed consent directly, or written informed consent has been provided by the patient, the parent, or legal guardian.
Who cannot join
- Written informed consent cannot be obtained.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Duke University School of Medicine
Durham, North Carolina, 27710
John Sleasman, M.D., 919-684-9914, john.sleasman@duke.edu
About this study
Official title: Congenital Athymia Patient Registry of RETHYMIC
This Congenital Athymia Patient Registry is an observational exposure-based registry study. It uses a prospective cohort design to follow patients who have been treated with RETHYMIC.
Clinical studies conducted with investigational RETHYMIC showed that treatment can result in immune reconstitution and prolong life. This treatment-based registry is being conducted to learn more about the reconstitution process following treatment and the impact of treatment on longer-term survival and the occurrence of adverse events of special interest (AESI).
Read the detailed description
This Congenital Athymia Patient Registry is an observational exposure-based registry study. It uses a prospective cohort design to follow patients who have been treated with RETHYMIC.
Clinical studies conducted with investigational RETHYMIC showed that treatment can result in immune reconstitution and prolong life. This treatment-based registry is being conducted to learn more about the reconstitution process following treatment and the impact of treatment on longer-term survival and the occurrence of adverse events of special interest (AESI).
Currently, there is a single site where patients are being treated with RETHYMIC. The Investigator will be responsible for recruiting patients who have recently had (i.e., within the past 60 days) or are scheduled to have this treatment within 30 days and obtaining signed informed consent (and assent as applicable). The Investigator will collect baseline medical history and clinical data information for each enrolled subject. The start of follow-up (day 1) begins on the day following treatment surgery. Clinical data and flow cytometry test results (when conducted) will be abstracted from the medical records by Clinical Staff at baseline and during follow-up at predefined intervals.
- Being tested
- Cultured Thymus Tissue
- Keywords
- Congenital Athymia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cultured Thymus TissueBiological
Also called RETHYMIC, allogeneic cultured thymus tissue-agdc
Product will be surgically administered into the quadriceps
| Study group | Role | What they receive |
|---|---|---|
| Main cohort | All eligible patients Receives: Biological: Cultured Thymus Tissue | |
| Secondary cohort | All eligible patients who survived 1 year post treatment Receives: Biological: Cultured Thymus Tissue |
What is being measured
The primary outcome is the main question the study is designed to answer.
Vital Status
Measured: 12 months post treatment with RETHYMIC.
Flow cytometry including total and naïve CD3, CD4, and CD8 counts
Measured: Immediately after the intervention/procedure/surgery
Secondary outcomes (1)
AESI classified by MedDRA coding, severity and grade
Measured: Immediately after the intervention/procedure/surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Apr 2022
- Study start25 May 2022
- Primary completionApr 2026 (estimated)
- Study completionApr 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Complete DiGeorge AnomalyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Other
- Enrolment
- 75 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sumitomo Pharma Switzerland GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05329935
- Sponsor's protocol ID
- RVT-802-4001
- Status last verified
- Aug 2025
- First submitted
- 24 Mar 2022
- Last updated
- 19 Aug 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
RETHYMIC US Prescribing Information, 2021.
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Congenital Athymia Patient Registry of RETHYMIC
- ClinicalTrials.gov ID
- NCT05329935
- Acronym / sponsor ID
- RVT-802-4001
- Keywords
- Congenital Athymia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 75 participants (estimated)
- Lead sponsor
- Sumitomo Pharma Switzerland GmbHIndustry (pharma, biotech, devices)
- Study start
- 25 May 2022 (actual)
- Primary completion
- Apr 2026 (estimated)
- Study completion
- Apr 2026 (estimated)
- First posted
- 15 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This Congenital Athymia Patient Registry is an observational exposure-based registry study. It uses a prospective cohort design to follow patients who have been treated with RETHYMIC.
Clinical studies conducted with investigational RETHYMIC showed that treatment can result in immune reconstitution and prolong life. This treatment-based registry is being conducted to learn more about the reconstitution process following treatment and the impact of treatment on longer-term survival and the occurrence of adverse events of special interest (AESI).
Detailed description
This Congenital Athymia Patient Registry is an observational exposure-based registry study. It uses a prospective cohort design to follow patients who have been treated with RETHYMIC.
Clinical studies conducted with investigational RETHYMIC showed that treatment can result in immune reconstitution and prolong life. This treatment-based registry is being conducted to learn more about the reconstitution process following treatment and the impact of treatment on longer-term survival and the occurrence of adverse events of special interest (AESI).
Currently, there is a single site where patients are being treated with RETHYMIC. The Investigator will be responsible for recruiting patients who have recently had (i.e., within the past 60 days) or are scheduled to have this treatment within 30 days and obtaining signed informed consent (and assent as applicable). The Investigator will collect baseline medical history and clinical data information for each enrolled subject. The start of follow-up (day 1) begins on the day following treatment surgery. Clinical data and flow cytometry test results (when conducted) will be abstracted from the medical records by Clinical Staff at baseline and during follow-up at predefined intervals.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Main cohort | – | Biological: Cultured Thymus TissueAll eligible patients |
| Secondary cohort | – | Biological: Cultured Thymus TissueAll eligible patients who survived 1 year post treatment |
Who may qualify
- Ages
- 0 to 21 years
- Sex
- All
- Age groups
- Child, Adult
- Study population
- Pediatric patients diagnosed with Congenital Athymia: • Who are scheduled for implantation with RETHYMIC within the next 30 days, or who were treated with RETHYMIC within the previous 60 days.
Key inclusion criteria (3 listed)
- Pediatric patients diagnosed with Congenital Athymia:
Key exclusion criteria (1 listed)
- Written informed consent cannot be obtained.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cultured Thymus TissueAlso: RETHYMIC, allogeneic cultured thymus tissue-agdcProduct will be surgically administered into the quadriceps | Biological | Main cohort, Secondary cohort |
Primary outcome
Vital Status
Time frame: 12 months post treatment with RETHYMIC.
Flow cytometry including total and naïve CD3, CD4, and CD8 counts
Time frame: Immediately after the intervention/procedure/surgery
1 secondary outcome
AESI classified by MedDRA coding, severity and grade
Time frame: Immediately after the intervention/procedure/surgery
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2025.
- BackgroundRETHYMIC US Prescribing Information, 2021.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Complete DiGeorge Anomaly and Complete DiGeorge Syndrome, run by Sumitomo Pharma Switzerland GmbH at 1 site in United States.
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 3 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 19 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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 John Sleasman, M.D. (919-684-9914, john.sleasman@duke.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Vital Status (time frame: 12 months post treatment with RETHYMIC.). The study lists 2 primary outcomes in all. 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 NCT05329935, which also lists 1 linked publication.
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 19 Aug 2025.