Tibulizumab for Scleroderma

Recruiting in Arlington (>99 mi)

Overseen By Chief Medical Officer

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Zura Bio Inc

Disqualifiers: Left ventricular failure, Pulmonary hypertension, Renal crisis, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

80 Participants Needed

Trial Summary

What is the purpose of this trial?

The study is a Phase 2, multi-center, randomized, double-blind, placebo-controlled study to evaluate the effects of tibulizumab over 24 weeks (Period 1) in adult participants with systemic sclerosis, followed by an open-label extension period where all active participants will receive tibulizumab and will be evaluated for an additional 28 weeks (Period 2)

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. Please consult with the trial coordinators for more details.

How is the drug Tibulizumab different from other drugs for scleroderma?

Tibulizumab may be unique in its approach to treating scleroderma by potentially targeting specific immune pathways, similar to how tocilizumab, an anti-interleukin-6 receptor antibody, is used to manage the condition. This could offer a new mechanism of action compared to traditional treatments like cyclophosphamide, which broadly suppress the immune system. ^{1 2 3 4 5}

Research Team

Study Director

Principal Investigator

Zura Bio Inc

Eligibility Criteria

This trial is for adults with systemic sclerosis, a condition where skin and connective tissues harden. Participants must meet specific health criteria to join, but the exact inclusion and exclusion details aren't provided here.

Inclusion Criteria

Fulfills classification criteria for SSc according to ACR/EULAR 2013 criteria

FVC >50% predicted

Diffusing capacity of the lungs for carbon monoxide (DLCO) ≥40% predicted (corrected for hemoglobin)

Exclusion Criteria

Current rheumatic disease other than SSc that could interfere with assessment of SSc

I have active Crohn's Disease or ulcerative colitis.

I have had serious heart, lung, kidney, stomach, or circulation problems in the last 6 months.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive tibulizumab or placebo for 24 weeks in a double-blind, placebo-controlled manner

24 weeks

Open-label extension

All participants receive tibulizumab and are evaluated for safety and efficacy over an additional 28 weeks

28 weeks

Follow-up

Participants are monitored for safety and effectiveness after the open-label extension

4 weeks

Treatment Details

Interventions

• Tibulizumab

Trial Overview The TibuSURE study tests tibulizumab against a placebo in a two-part process: first, participants are randomly assigned to receive either the drug or placebo for 24 weeks; then all get tibulizumab for another 28 weeks.

Participant Groups

Group I: Tibulizumab Experimental Treatment 1 Intervention

Subcutaneous injection

Group II: Placebo Placebo Group 2 Interventions

Subcutaneous injection

Who Is Running the Clinical Trial?

Zura Bio Inc

Lead Sponsor

Trials

1

Recruited

80+

Findings from Research

In a phase II study involving patients with systemic sclerosis (SSc), tocilizumab treatment for 48 weeks led to a significant improvement in skin scores and stabilization of lung function, as measured by the modified Rodnan Skin Score (mRSS) and forced vital capacity (%pFVC).

While tocilizumab was associated with an increased rate of serious infections compared to placebo, no new safety concerns were identified, suggesting that while the drug is effective, monitoring for infections is important during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and efficacy of subcutaneous tocilizumab in systemic sclerosis: results from the open-label period of a phase II randomised controlled trial (faSScinate). Khanna, D., Denton, CP., Lin, CJF., et al.[2019]

In a pilot study of 13 patients with recent-onset diffuse scleroderma, treatment with anti-thymocyte globulin (ATG) followed by mycophenolate mofetil (MMF) resulted in a significant decrease in skin score from 28 to 17 over 12 months, indicating improved skin condition.

While MMF was well tolerated, some patients experienced adverse effects, including serum sickness in five patients, which was manageable with corticosteroids, suggesting that while the treatment is generally safe, monitoring for side effects is necessary.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pilot study of anti-thymocyte globulin plus mycophenolate mofetil in recent-onset diffuse scleroderma. Stratton, RJ., Wilson, H., Black, CM.[2019]

Tocilizumab, administered every two weeks, showed a decrease in the modified Rodnan's score after 4 weeks in a 44-year-old female patient with systemic scleroderma, indicating its potential effectiveness in treating skin fibrosis.

The treatment was well-tolerated with no side effects reported after 6 months, suggesting that tocilizumab may be a safe option for maintaining lung function and improving symptoms in scleroderma patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tocilizumab in systemic sclerosis treatment: a case report. Kudsi, M., Khalayli, N., Tarcha, R., et al.[2023]

Cyclophosphamide is the primary treatment for progressive skin involvement and active interstitial lung disease in scleroderma, while mycophenolate mofetil shows promise as an alternative.

Newer therapies targeting T-cells (like sirolimus and alefacept) and B-cells (like rituximab) have shown encouraging results in small studies, but the safety and efficacy of antifibrotic treatments like imatinib still need further confirmation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immunotherapy of systemic sclerosis. Manno, R., Boin, F.[2022]

In a phase 3 trial involving 210 participants, tocilizumab did not significantly improve skin fibrosis compared to placebo, as indicated by the modified Rodnan skin score (mRSS) change after 48 weeks.

However, tocilizumab showed a positive effect on lung function, with a significant improvement in forced vital capacity (FVC%) compared to placebo, suggesting it may help preserve lung function in early systemic sclerosis-associated interstitial lung disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tocilizumab in systemic sclerosis: a randomised, double-blind, placebo-controlled, phase 3 trial. Khanna, D., Lin, CJF., Furst, DE., et al.[2021]

References

1. England pubmed.ncbi.nlm.nih.gov

Safety and efficacy of subcutaneous tocilizumab in systemic sclerosis: results from the open-label period of a phase II randomised controlled trial (faSScinate). [2019]

2. England pubmed.ncbi.nlm.nih.gov

Pilot study of anti-thymocyte globulin plus mycophenolate mofetil in recent-onset diffuse scleroderma. [2019]

3. England pubmed.ncbi.nlm.nih.gov

Tocilizumab in systemic sclerosis treatment: a case report. [2023]

4. England pubmed.ncbi.nlm.nih.gov

Immunotherapy of systemic sclerosis. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Tocilizumab in systemic sclerosis: a randomised, double-blind, placebo-controlled, phase 3 trial. [2021]