NCT03444805

Brief Summary

The aim of the study is to assess the effectiveness of various post-transplant treatment management approaches on clinical and immune biological responses after Autologous Hematopoietic Stem Cell transplantation (AHSCT) for Systemic Sclerosis (SSc) as currently performed by the different treatment protocols used in routine clinical practice across Europe in various EBMT centres

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
60

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Jul 2019

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 19, 2018

Completed
4 days until next milestone

First Posted

Study publicly available on registry

February 23, 2018

Completed
1.4 years until next milestone

Study Start

First participant enrolled

July 1, 2019

Completed
2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2021

Completed
1.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 30, 2023

Completed
Last Updated

September 3, 2019

Status Verified

August 1, 2019

Enrollment Period

2.5 years

First QC Date

February 19, 2018

Last Update Submit

August 29, 2019

Conditions

Autoimmune Diseases

Keywords

Autoimmune diseasesSystemic sclerosisAutologous hematopoietic stem cell transplant (AHSCT)Post-AHSCT managementImmune reconstitution

Outcome Measures

Primary Outcomes (1)

  • Progression free survival (PFS),

    defined as survival since AHSCT without evidence of progression of SSc.

    2 years post transplant

Secondary Outcomes (10)

  • Treatment related toxicity

    100 days post-transplant

  • 100 days Treatment Related Mortality (100d TRM)

    100 days post-transplant

  • Overall Survival (OS)

    2 years post-transplant

  • Use of prednisone equivalent

    1 year and 2 years post-transplant

  • Use of immunosuppressive drugs

    2 years post-transplant

  • +5 more secondary outcomes

Study Arms (1)

NISSC-2

SSC patients treated with AHSCT

Procedure: Autologous HSCT

Interventions

Autologous HSCTPROCEDURE

1st AHSCT

NISSC-2

Eligibility Criteria

Age18 Years+
Sexall
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

All consecutive patients treated with AHSCT for progressive systemic sclerosis in participating centres

You may qualify if:

  • \- Autologous HSCT
  • \- Age above 18 years at time of transplant.
  • Established diagnosis of progressive SSc according to 2013 ACR/EULAR classification criteria

You may not qualify if:

  • Pregnancy or inadequate contraception
  • Severe concomitant disease
  • Reduced lung, cardiac or renal function
  • a. .Reduced lung function with FVC \< 50% or DLCO \< 30% (of predicted values) b; .Pulmonary arterial hypertension with baseline (resting) PASP \> 40 mmHg or mPAP \> 25 mmHg or a PASP \> 45 mmHg or mPAP \> 30 mmHg after fluid challenge or Pulmonary vascular resistance \> 3 Wood units on RHC c. Severe heart failure with Ejection Fraction \< 45% by cardiac echocardiography d. D-sign of septal bounce on cardiac MRI e. Unrevascularized severe coronary artery disease f. Untreated severe arrhythmia g. Cardiac tamponade h. Constrictive pericarditis i. Kidney insufficiency: creatinine clearance \<30ml/min Previously damaged bone marrow
  • Leukopenia \< 2.0 x 109/L (total white cell count)
  • Thrombocytopenia \< 100 x 109/L
  • Uncontrolled severe or chronic infection (Hepatitis B/C, HIV, Salmonella carrier, syphilis, tuberculosis)
  • Severe concomitant psychiatric illness (depression, psychosis)
  • Concurrent neoplasms or myelodysplasia in the past 5 years
  • Smoking (current)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Badoglio Manuela- EBMT Paris Office

Paris, 75010, France

RECRUITING

Related Links

MeSH Terms

Conditions

Autoimmune DiseasesScleroderma, Systemic

Condition Hierarchy (Ancestors)

Immune System DiseasesConnective Tissue DiseasesSkin and Connective Tissue DiseasesSkin Diseases

Study Officials

  • Dominique Farge, PhD

    EBMT ADWP

    STUDY CHAIR

Central Study Contacts

Manuela Badoglio, MS

CONTACT

+33 1 70 64 24 16manuela.badoglio@upmc.fr

Dominique Farge, PhD

CONTACT

dominique.farge-bancel@aphp.fr

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
NETWORK
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 19, 2018

First Posted

February 23, 2018

Study Start

July 1, 2019

Primary Completion

December 31, 2021

Study Completion

March 30, 2023

Last Updated

September 3, 2019

Record last verified: 2019-08

Locations

FR(1)