Adenosine Deaminase Severe Combined ImmunodeficiencyNCT03878069
Registry Study of Revcovi Treatment in Patients With ADA-SCID
An observational study of Adenosine Deaminase Severe Combined Immunodeficiency, run by Chiesi Farmaceutici S.p.A. at 14 sites in United States.
Trial summary
This registry study is being conducted in patients with adenosine deaminase severe combined immune deficiency (ADA-SCID) who require enzyme replacement therapy (ERT) treatment with Revcovi. Data on safety and on measures of efficacy are collected. Read more about this study
- Who is it for?
- Ages 0 months to 65 years
- Conditions the study is for
- Adenosine Deaminase Severe Combined Immunodeficiency
- What is being tested?
- elapegademase-lvlr
- Type of treatment
- Injection or infusion
- How many people
- 32 participants
- Where
- 14 sitesUnited States
- Study dates
- 30 Sep 2019to 18 Jan 2023
- How long will I take part?
- 24 Months
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 months to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with ADA-SCID who require ERT
Who may be able to join
- Male or female, aged newborn to adult
- In need of ERT treatment due to one of the following circumstances:
- Waiting to receive a stem cell transplant
- Previously declined, had been found to be ineligible for, or did not respond to a stem cell transplant
- Waiting to enrol in a clinical trial on gene therapy, or had been found to be ineligible for or had failed such a trial
- One of the following histories of ERT treatment:
Show all 9Show fewer
- Revcovi only
- Previously on Adagen but had transitioned to Revcovi
- Not yet on any ERT but about to start on Revcovi
Who cannot join
- Any condition that, in the opinion of the Investigator, makes the patient unsuitable for the study.
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
United States14 sites
UBMD Pediatrics Outpatient Center
Buffalo, New York, 14203
Duke University Hospital
Durham, North Carolina, 27705
Penn State Children's Hospital
Hershey, Pennsylvania, 17033
University of California Los Angeles
Los Angeles, California, 90095-1752
Le Bonheur Children's Hospital
Memphis, Tennessee, 38105
Children's Minnesota
Minneapolis, Minnesota, 55404
Childrens Hospital of New Orleans
New Orleans, Louisiana, 70118
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Allergy & Asthma Medical Group and Research Center, A P.C.
San Diego, California, 92123
UCSF - University of California
San Francisco, California, 94158
Seattle Children's
Seattle, Washington, 98101
St. Louis Children's Hospital - Washington University School of Medicine
St Louis, Missouri, 63110
University of South Florida Allergy Immunology Clinic
St. Petersburg, Florida, 33701
Children's National
Washington D.C., District of Columbia, 20010
About this study
Official title: Single Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement Therapy
This registry study is being conducted in patients with adenosine deaminase severe combined immune deficiency (ADA-SCID) who require enzyme replacement therapy (ERT) treatment with Revcovi. Data on safety and on measures of efficacy are collected.
Read the detailed description
Patients with ADA-SCID who require ERT will receive Revcovi on a dosage and schedule determined by the treating physician. They will be followed for safety throughout the study, and will be monitored for the efficacy markers of adenosine deaminase (ADA) activity and deoxyadenosine nucleotide (dAXP) concentration according to a suggested schedule. Some subjects will be new to ERT; some will have transitioned from Adagen, which was the ERT available before Revcovi; and some will have previously participated in an earlier Phase 3 trial of Revcovi (study STP-2279-002). Patients will be followed either until they are able to successfully undergo a stem cell transplant or stem cell gene therapy and thus no longer require ERT treatment, or until all ongoing participants have received a minimum of 24 months of Revcovi treatment.
Note: Due to the nature of this study, all analyses are descriptive and no statistical hypotheses will be tested.
- Being tested
- elapegademase-lvlr
- Keywords
- ADA-SCID, Revcovi, ERT
- Medical subject headings
- Severe combined immunodeficiency due to adenosine deaminase deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
elapegademase-lvlrBiological
Also called Revcovi
Revcovi is administered intramuscularly (i.m.). Weekly dosage is calculated in mg/kg of body weight, and can be adjusted over the course of the trial based on ADA activity and dAXP concentration as well as on clinical assessment by the treating physician.
| Study group | Role | What they receive |
|---|---|---|
| Patients with ADA-SCID in need of ERT treatment | All participants will receive Revcovi. For analysis purposes, there will be three groups, who differ with respect to ERT history:
* Adagen-naïve: Subjects starting on ERT for the first time
* Adagen-transitioning: Subjects switching from Adagen to Revcovi
* STP-2279-002 participants: Subjects who had taken part in an earlier Phase III trial of Revcovi (study STP-2279-002) in which they had been switched from Adagen to Revcovi. This last group differs from the Adagen-transitioning group in that they will have been on Revcovi the longest. Receives: Biological: elapegademase-lvlr |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Subjects Meeting the Toxicity Threshold for Deoxyadenosine Nucleotide (dAXP) Concentration at the Last Measurement
A deficiency in the ADA enzyme results in a build-up of dAXP nucleotides, which are toxic to lymphocytes and lead to impairment of immune function. A detoxified level of dAXP concentration is defined as a trough value of 0.02 millimoles per liter (mmol/L) or lower. Data are presented as the number of subjects who met the targeted threshold at the final measurement, whether that took place at Month 24 or earlier.
Measured: From enrollment to end of treatment up to Month 24
Number of Subjects Meeting the Optimal Threshold for ADA Activity at the Last Measurement
An optimal level of ADA plasma activity is defined as a trough value of 30 millimoles per hour per liter (mmol/h/L) or greater. Data are presented as the number of subjects who met the targeted threshold at the final measurement, whether that took place at Month 24 or earlier.
Measured: From enrollment to end of treatment up to Month 24
Safety of Revcovi
The number of subjects reporting adverse events (AEs). Due to the nature of ADA-SCID, only AEs deemed to be at least possibly related to Revcovi treatment were documented.
Measured: From enrollment to end of treatment up to Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2019
- Study start30 Sep 2019
- Primary completion18 Jan 2023
- Study completion18 Jan 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Adenosine Deaminase Severe Combined ImmunodeficiencyStudies of the same condition, open and under way.
- More trials run by Chiesi Farmaceutici S.p.A.The sponsor's other registered studies.
- 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
- 32 participants (actual)
- Target duration
- 24 Months
Sponsor and investigators
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03878069
- Sponsor's protocol ID
- CLI-06814AA1-01
- Status last verified
- Sep 2024
- First submitted
- 12 Mar 2019
- Last updated
- 15 Nov 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Single Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement Therapy
- ClinicalTrials.gov ID
- NCT03878069
- Acronym / sponsor ID
- CLI-06814AA1-01
- Keywords
- ADA-SCID, Revcovi, ERT
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Chiesi Farmaceutici S.p.A.Industry (pharma, biotech, devices)
- Study start
- 30 Sep 2019 (actual)
- Primary completion
- 18 Jan 2023 (actual)
- Study completion
- 18 Jan 2023 (actual)
- First posted
- 18 Mar 2019 (actual)
- Results
- Posted 15 Nov 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This registry study is being conducted in patients with adenosine deaminase severe combined immune deficiency (ADA-SCID) who require enzyme replacement therapy (ERT) treatment with Revcovi. Data on safety and on measures of efficacy are collected.
Detailed description
Patients with ADA-SCID who require ERT will receive Revcovi on a dosage and schedule determined by the treating physician. They will be followed for safety throughout the study, and will be monitored for the efficacy markers of adenosine deaminase (ADA) activity and deoxyadenosine nucleotide (dAXP) concentration according to a suggested schedule. Some subjects will be new to ERT; some will have transitioned from Adagen, which was the ERT available before Revcovi; and some will have previously participated in an earlier Phase 3 trial of Revcovi (study STP-2279-002). Patients will be followed either until they are able to successfully undergo a stem cell transplant or stem cell gene therapy and thus no longer require ERT treatment, or until all ongoing participants have received a minimum of 24 months of Revcovi treatment.
Note: Due to the nature of this study, all analyses are descriptive and no statistical hypotheses will be tested.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Target follow-up
- 24 Months
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with ADA-SCID in need of ERT treatment | – | Biological: elapegademase-lvlrAll participants will receive Revcovi. For analysis purposes, there will be three groups, who differ with respect to ERT history: * Adagen-naïve: Subjects starting on ERT for the first time * Adagen-transitioning: Subjects switching from Adagen to Revcovi * STP-2279-002 participants: Subjects who had taken part in an earlier Phase III trial of Revcovi (study STP-2279-002) in which they had been switched from Adagen to Revcovi. This last group differs from the Adagen-transitioning group in that they will have been on Revcovi the longest. |
Who may qualify
- Ages
- 0 months to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with ADA-SCID who require ERT
Key inclusion criteria (9 listed)
- Male or female, aged newborn to adult
- In need of ERT treatment due to one of the following circumstances:
- One of the following histories of ERT treatment:
Key exclusion criteria (1 listed)
- Any condition that, in the opinion of the Investigator, makes the patient unsuitable for the study.
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 |
|---|---|---|
| elapegademase-lvlrAlso: RevcoviRevcovi is administered intramuscularly (i.m.). Weekly dosage is calculated in mg/kg of body weight, and can be adjusted over the course of the trial based on ADA activity and dAXP concentration as well as on clinical assessment by the treating physician. | Biological | Patients with ADA-SCID in need of ERT treatment |
Primary outcome
Number of Subjects Meeting the Toxicity Threshold for Deoxyadenosine Nucleotide (dAXP) Concentration at the Last Measurement
Time frame: From enrollment to end of treatment up to Month 24
A deficiency in the ADA enzyme results in a build-up of dAXP nucleotides, which are toxic to lymphocytes and lead to impairment of immune function. A detoxified level of dAXP concentration is defined as a trough value of 0.02 millimoles per liter (mmol/L) or lower. Data are presented as the number of subjects who met the targeted threshold at the final measurement, whether that took place at Month 24 or earlier.
Number of Subjects Meeting the Optimal Threshold for ADA Activity at the Last Measurement
Time frame: From enrollment to end of treatment up to Month 24
An optimal level of ADA plasma activity is defined as a trough value of 30 millimoles per hour per liter (mmol/h/L) or greater. Data are presented as the number of subjects who met the targeted threshold at the final measurement, whether that took place at Month 24 or earlier.
Safety of Revcovi
Time frame: From enrollment to end of treatment up to Month 24
The number of subjects reporting adverse events (AEs). Due to the nature of ADA-SCID, only AEs deemed to be at least possibly related to Revcovi treatment were documented.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Nov 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
14 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Adenosine Deaminase Severe Combined Immunodeficiency, run by Chiesi Farmaceutici S.p.A. at 14 sites 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 9 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: finished, as of the sponsor's last update on 15 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites 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?
Yes. Results are posted in the registry (first posted 15 Nov 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Subjects Meeting the Toxicity Threshold for Deoxyadenosine Nucleotide (dAXP) Concentration at the Last Measurement (time frame: From enrollment to end of treatment up to Month 24). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Nov 2024.