NCT05049668

Brief Summary

After exiting the RACE trial (NCT02099747) patients will be invited to participate in this long term follow-up study

Trial Health

75
On Track

Trial Health Score

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

Enrollment
197

participants targeted

Target at P50-P75 for all trials

Timeline
104mo left

Started Oct 2021

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
enrolling by invitation

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

Study Progress35%
Oct 2021Dec 2034

First Submitted

Initial submission to the registry

September 13, 2021

Completed
7 days until next milestone

First Posted

Study publicly available on registry

September 20, 2021

Completed
11 days until next milestone

Study Start

First participant enrolled

October 1, 2021

Completed
12.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2033

Expected
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2034

Last Updated

September 27, 2021

Status Verified

September 1, 2021

Enrollment Period

12.2 years

First QC Date

September 13, 2021

Last Update Submit

September 21, 2021

Conditions

Outcome Measures

Primary Outcomes (1)

  • Failure Free Survival

    Failure Free Survival, where treatment failure is defined as one or more of the following: death, relapse, malignant clonal evolution, need for further (e.g. transplant)

    15 years

Secondary Outcomes (12)

  • Response Rate: number of patients who reach a hematological response

    15 years

  • Overall Survival

    15 years

  • Cumulative incidence of relapse after response

    15 years

  • Cumulative incidence of clonal evolution

    15 years

  • Cumulative incidence of clinical Paroxysmal nocturnal hemoglobinuria (PNH)

    15 years

  • +7 more secondary outcomes

Study Arms (1)

RACE 1 patients

After exiting the RACE trial (NCT02099747) patient will be invited to participate in this study

Drug: ATGAM plus CsA with or without Eltrombopag

Interventions

Standard treatment with or without Eltrombopag

Also known as: hATG+CsA +/- Revolade
RACE 1 patients

Eligibility Criteria

Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients who participated in the initial RACE 1 trial

You may qualify if:

  • Subject participated in the RACE trial (NCT02099747, EudraCT number: 2014-000363-40) during which patient received ATGAM, Cyclosporine A with or without Eltrombopag.
  • Subject has provided informed consent to participate in long-term data collection

You may not qualify if:

  • None

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hospital St. Louis

Paris, France

Location

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Antilymphocyte Serumeltrombopag

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow Diseases

Intervention Hierarchy (Ancestors)

Immune SeraAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsBiological ProductsComplex Mixtures

Study Officials

  • Regis Peffault de Latour, Prof, MD

    Hospital St. Louis, Paris, France

    PRINCIPAL INVESTIGATOR
  • Antonio M Risitano, MD, PhD

    Hospital Avellino, Napels, Italy

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Target Duration
15 Years
Sponsor Type
NETWORK
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 13, 2021

First Posted

September 20, 2021

Study Start

October 1, 2021

Primary Completion (Estimated)

December 1, 2033

Study Completion (Estimated)

December 1, 2034

Last Updated

September 27, 2021

Record last verified: 2021-09

Data Sharing

IPD Sharing
Will not share

Locations