A Study of Subcutaneous Abatacept to Treat Diffuse Cutaneous Systemic Sclerosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02161406
• Date of registration: 03/06/2014
• Prospective Registration: Yes
• Primary sponsor: Dinesh Khanna, MD, MS
• Public title: A Study of Subcutaneous Abatacept to Treat Diffuse Cutaneous Systemic Sclerosis ASSET
• Scientific title: A Phase 2 Study to Evaluate Subcutaneous Abatacept vs. Placebo in Diffuse Cutaneous Systemic Sclerosis- a Double-blind, Placebo-controlled, Randomized Controlled Trial.
• Date of first enrolment: September 2014
• Target sample size: 88
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02161406
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Canada, United Kingdom, United States

Contacts

• Name: Dinesh Khanna, MD, MS
• Affiliation: University of Michigan

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Diagnosis of Systematic Sclerosis (SSc), as defined using the 2013 American College of
Rheumatology/ European Union League Against Rheumatism classification of SSc

2. Diffuse Systemic Sclerosis (dcSSc) as defined by LeRoy and Medsger

3. Disease duration of = 36 months (defined as time from the first non-Raynaud phenomenon
manifestation)

4. For disease duration of = 18 months: = 10 and = 35 mRSS units at the screening visit

5. For disease duration of >18-36 months: = 15 and = 45 mRSS units at the screening visit
and one of the following:

- Increase = 3 in mRSS units compared with the last visit within previous 1-6
months

- Involvement of one new body area with = 2 mRSS units compared with the last visit
within the previous 1-6 months

- Involvement of two new body areas with = 1 mRSS units compared with the last
visit within the previous 1-6 months

- Presence of 1 or more Tendon Friction Rub

6. Age = 18 years at the screening visit

7. If female of childbearing potential, the patient must have a negative pregnancy test
at screening and baseline visits

8. Oral corticosteroids (= 10 mg/day of prednisone or equivalent) and NSAIDs are
permitted if the patient is on a stable dose regimen for

- 2 weeks prior to and including the baseline visit.

9. ACE inhibitors, calcium-channel blockers, proton-pump inhibitors, and/or oral
vasodilators are permitted if the patient is on a stable dose for = 2 weeks prior to
and including the baseline visit.

Exclusion Criteria:

1. Rheumatic disease other than dcSSc; it is acceptable to include patients with
fibromyalgia and scleroderma-associated myopathy

2. Limited cutaneous systemic sclerosis or sine scleroderma at the screening visit

3. Major surgery (including joint surgery) within 8 weeks prior to screening visit

4. Infected ulcer prior to randomization

5. Treatment with any investigational agent within = 4 weeks (or 5 half-lives of the
investigational drug, whichever is longer) of the baseline visit

6. Previous treatment with cell-depleting therapies, including investigational agents,
including but not limited to, CAMPATH, anti-CD4, anti-CD5, anti-CD3, anti-CD19, and
ABA

7. Anti-CD20, and cyclophosphamide within 12 months prior to baseline visit.

8. Use of Intravenous Immunoglobulin (IVIG) within 12 weeks prior to baseline visit

9. Previous treatment with chlorambucil, bone marrow transplantation, or total lymphoid
irradiation

10. Immunization with a live/attenuated vaccine within = 4 weeks prior to the baseline
visit

11. Treatment with methotrexate, hydroxychloroquine, cyclosporine A, azathioprine,
mycophenolate mofetil rapamycin, colchicine, or D-penicillamine, within= 4 weeks prior
to the baseline visit

12. Treatment with etanercept within = 2 weeks, infliximab, certolizumab, golimumab, ABA
or adalimumab within = 8 weeks, anakinra within = 1 week prior to the baseline visit

13. Pulmonary disease with FVC = 50% of predicted, or DLCO (uncorrected for hemoglobin ) =
40% of predicted at the screening visit

14. Pulmonary arterial hypertension (PAH) as determined by right heart catheterization or
on PAH approved medications for PAH. It is acceptable to use PDFE-5 inhibitors for
Raynaud's and digital ulcers.

15. Subjects at risk for tuberculosis (TB). Specifically excluded from this study will be
participants with a history of active TB within the last 3 years, even if it was
treated; a history of active TB greater than 3 years ago, unless there is
documentation that the prior anti-TB treatment was appropriate in duration and type;
current clinical, radiographic, or laboratory evidence of active TB; and latent TB
that was not successfully treated (= 4 weeks).

16. Positive for hepatitis B surface antigen prior to the baseline visit

17. Positive for hepatitis C antigen, if the presence of hepatitis C virus was also shown
with polymerase chain reaction or recombinant immunoblot assay prior to baseline visit

18. Subjects at risk for tuberculosis (TB). Specifically excluded from this study will be
participants with a history of active TB within the last 3 years, even if it was
treated; a history of active TB greater than 3 years ago, unless there is
documentation that the prior anti-TB treatment was appropriate in duration and type;
current clinical, radiographic, or laboratory evidence of active TB; and latent TB
that was not successfully treated (= 4 weeks).

19. Any of the following at the screening visit: Hemoglobin <8.5 g/dL; WBC < 3,000/mm3 (<3
x 109/L); platelets < 100,000/mm3 (<3 x 109/L); serum creatinine > 2 x ULN; serum ALT
or AST > 2 x ULN

20. Severe skin thickening (mRSS 3) on the inner aspects of thighs, upper arms, or abdomen

21. Patients with a history of anaphylaxis to abatacept



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Diffuse Cutaneous Systemic Sclerosis

Intervention(s)

Drug: Placebo

Drug: Abatacept

Primary Outcome(s)

Proportion of Participants With at Least One Adverse Events (AEs) or Serious AEs (SAEs) in 1 Year [Time Frame: 52 weeks]

Change From Baseline in the Modified Rodnan Skin Score (mRSS) to Month 12 [Time Frame: Baseline and 52 weeks]

Secondary Outcome(s)

Change From Baseline to Month 12 in HAQ-DI - Eating [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in PROMIS - Fatigue [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in PROMIS-29 - Depression [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in SCTC GIT - Composite Score [Time Frame: Baseline and Week 52]

ACR CRISS at 12 Months [Time Frame: Week 52]

Change From Baseline to Month 12 in HAQ-DI - Reach [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in SHAQ-DI VAS - Breathing [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in SHAQ-DI VAS - GI Involvement [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in SHAQ-DI VAS - Burden of Digital Ulcers [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in FVC (in ml) [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in HAQ-DI - Dressing and Grooming [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in HAQ-DI - Hygiene [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in Patient Global Assessment for Overall Disease [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in PROMIS-29 - Pain Interference [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in SHAQ-DI VAS - Overall Disease [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in HAQ-DI - Grip [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in PROMIS-29 - Ability to Participate in Social Roles & Activities [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in PROMIS-29 - Anxiety [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in PROMIS-29 - Sleep Disturbance [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in Tender Joint Counts [Time Frame: Baseline and 52 weeks]

Change From Baseline to Month 12 in HAQ-DI - Common Daily Activities [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in PROMIS - Sleep Disturbance [Time Frame: Baseline and Week 52]

Change in % Predicted FVC [Time Frame: Baseline and 52 weeks]

Change From Baseline to Month 12 in PROMIS - Sleep Impairment [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in Swollen Joint Count [Time Frame: Baseline and 52 weeks]

Change From Baseline to Month 12 in HAQ-DI - Arising [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in HAQ-DI - Overall [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in Physician Global Assessment for Overall Disease [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in PROMIS 29 - Fatigue [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in HAQ-DI - Walking [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in PROMIS-29 - Pain Intensity [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in PROMIS-29 - Physical Function [Time Frame: Baseline and Week 52]

Change From Baseline to Month 12 in SHAQ-DI VAS - Raynaud's [Time Frame: Baseline and Week 52]

Secondary ID(s)

IM101-344

1UM1AI110557

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Institute of Allergy and Infectious Diseases (NIAID)

Bristol-Myers Squibb

Results

• Results available: Yes
• Date Posted: 18/06/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02161406