X-linked Lymphoproliferative Syndrome Type 2 (XLP-2)Phase 3NCT06309823
A Single-patient Clinical Trial of MAS825 in a Patient With XIAP Deficiency
A Phase 3 study testing MAS825 in people with X-linked Lymphoproliferative Syndrome Type 2 (XLP-2), run by The Hospital for Sick Children at 1 site in Canada.
Trial summary
MAS825 is a bi-specific IgG1 monoclonal antibody that simultaneously targets IL-1 beta and IL-18, thereby neutralizing both cytokines that are thought to be integral to the pathogenesis of XLP-2. Read more about this study
- Who is it for?
- Ages 13 to 14 years, men only
- Conditions the study is for
- X-linked Lymphoproliferative Syndrome Type 2 (XLP-2)
- What is being tested?
- MAS825
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteCanada
- Study dates
- 8 Feb 2023to 17 Jan 2024
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 13 to 14 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Known patient with XIAP genetic defect under the care of the investigator
Who cannot join
- N/A
Trial locations and contacts
1 site in 1 country.
About this study
MAS825 is a bi-specific IgG1 monoclonal antibody that simultaneously targets IL-1 beta and IL-18, thereby neutralizing both cytokines that are thought to be integral to the pathogenesis of XLP-2. Clinical trials are currently examining its efficacy in other diseases associated with elevations of these cytokines, including NLRC4-associated disease and hidradenitis suppurativa. This study proposes to assess the effectiveness of MAS825 in a single patient with XLP-2, who has previously demonstrated response to blockade of IL-1 beta and IL-18. Given the lack of alternative pharmaceutical options for XLP-2, this represents the only known medication option that avoids the toxicity associated with high-dose corticosteroids and the morbidity associated with hematopoietic stem cell transplantation.
- Being tested
- MAS825
- Medical subject headings
- Lymphoproliferative Syndrome, X-Linked, 2
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MAS825Drug
MAS825 is a bi-specific IgG1 monoclonal antibody that simultaneously targets IL-1beta and IL-18, thereby neutralizing both cytokines that are thought to be integral to the pathogenesis of XLP-2.
| Study group | Role | What they receive |
|---|---|---|
| Active treatment | Experimental | Not described Receives: Drug: MAS825 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Impact of MAS825 on Physician Global Assessment Score
Likert Scale:
0 = Absent (no) disease associated clinical signs and symptoms
- = Minimal disease associated signs and symptoms
- = Mild disease associated signs and symptoms
- = Moderate disease associated signs and symptoms
- = Severe disease associated signs and symptoms
Measured: 5 years
Impact of MAS825 on Physician Severity Assessment of Disease Signs and Symptoms
The signs and symptoms assessed include: 1) Abdominal pain 2) Diarrhea 3) Fever using a Likert scale:
0 = Absent
- = Minimal
- = Mild
- = Moderate
- = Severe
Measured: 5 years
Patient/Parent Global Assessment of Disease Activity
Likert Scale:
0 = Absent (no) disease associated clinical signs and symptoms
- = Minimal disease associated signs and symptoms
- = Mild disease associated signs and symptoms
- = Moderate disease associated signs and symptoms
- = Severe disease associated signs and symptoms
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2024
- Study start8 Feb 2023
- Primary completion17 Jan 2024
- Study completion17 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for X-linked Lymphoproliferative Syndrome Type 2 (XLP-2)Studies of the same condition, open and under way.
- More trials run by The Hospital for Sick ChildrenThe 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Hospital for Sick Children
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dilan Dissanayake, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06309823
- Sponsor's protocol ID
- 1000080330
- Status last verified
- Mar 2024
- First submitted
- 23 Feb 2024
- Last updated
- 13 Mar 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-patient Clinical Trial of MAS825 in a Patient With XIAP Deficiency
- ClinicalTrials.gov ID
- NCT06309823
- Acronym / sponsor ID
- 1000080330
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1 participants (actual)
- Lead sponsor
- The Hospital for Sick ChildrenUniversity, hospital or other
- Study start
- 8 Feb 2023 (actual)
- Primary completion
- 17 Jan 2024 (actual)
- Study completion
- 17 Jan 2024 (actual)
- First posted
- 13 Mar 2024 (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.
MAS825 is a bi-specific IgG1 monoclonal antibody that simultaneously targets IL-1 beta and IL-18, thereby neutralizing both cytokines that are thought to be integral to the pathogenesis of XLP-2. Clinical trials are currently examining its efficacy in other diseases associated with elevations of these cytokines, including NLRC4-associated disease and hidradenitis suppurativa. This study proposes to assess the effectiveness of MAS825 in a single patient with XLP-2, who has previously demonstrated response to blockade of IL-1 beta and IL-18. Given the lack of alternative pharmaceutical options for XLP-2, this represents the only known medication option that avoids the toxicity associated with high-dose corticosteroids and the morbidity associated with hematopoietic stem cell transplantation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Active treatment | Experimental | Drug: MAS825 |
Who may qualify
- Ages
- 13 to 14 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Known patient with XIAP genetic defect under the care of the investigator
Key exclusion criteria (1 listed)
- N/A
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 |
|---|---|---|
| MAS825MAS825 is a bi-specific IgG1 monoclonal antibody that simultaneously targets IL-1beta and IL-18, thereby neutralizing both cytokines that are thought to be integral to the pathogenesis of XLP-2. | Drug | Active treatment |
Primary outcome
Impact of MAS825 on Physician Global Assessment Score
Time frame: 5 years
Likert Scale: 0 = Absent (no) disease associated clinical signs and symptoms 1. = Minimal disease associated signs and symptoms 2. = Mild disease associated signs and symptoms 3. = Moderate disease associated signs and symptoms 4. = Severe disease associated signs and symptoms
Impact of MAS825 on Physician Severity Assessment of Disease Signs and Symptoms
Time frame: 5 years
The signs and symptoms assessed include: 1) Abdominal pain 2) Diarrhea 3) Fever using a Likert scale: 0 = Absent 1. = Minimal 2. = Mild 3. = Moderate 4. = Severe
Patient/Parent Global Assessment of Disease Activity
Time frame: 5 years
Likert Scale: 0 = Absent (no) disease associated clinical signs and symptoms 1. = Minimal disease associated signs and symptoms 2. = Mild disease associated signs and symptoms 3. = Moderate disease associated signs and symptoms 4. = Severe disease associated signs and symptoms
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Mar 2024.
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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing MAS825 in people with X-linked Lymphoproliferative Syndrome Type 2 (XLP-2), run by The Hospital for Sick Children at 1 site in Canada.
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 1 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 13 Mar 2024. 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 Canada. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Impact of MAS825 on Physician Global Assessment Score (time frame: 5 years). 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 13 Mar 2024.