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Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

NCT01917708 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2019-12-26

No results posted yet for this study

Summary

This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem cell transplant for serious non-malignant diseases as well as to assess the immunological effects of abatacept. Participants will be followed for 2 years.

Conditions

  • Hurler Syndrome
  • Fanconi Anemia
  • Glanzmann Thrombasthenia
  • Wiskott-Aldrich Syndrome
  • Chronic Granulomatous Disease
  • Severe Congenital Neutropenia
  • Leukocyte Adhesion Deficiency
  • Shwachman-Diamond Syndrome
  • Diamond-Blackfan Anemia
  • Dyskeratosis-congenita
  • Chediak-Higashi Syndrome
  • Severe Aplastic Anemia
  • Thalassemia Major
  • Hemophagocytic Lymphohistiocytosis
  • Sickle Cell Disease

Interventions

DRUG

Abatacept

All patients will receive 4 doses of abatacept in addition to standard GVHD prophylaxis with cyclosporine and mycophenolate mofetil.

Sponsors & Collaborators

  • Emory University

    lead OTHER

Principal Investigators

  • John T Horan, MD · Children's Healthcare of Atlanta/Emory University

Study Design

Allocation
NA
Purpose
SUPPORTIVE_CARE
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-01-31
Primary Completion
2019-09-19
Completion
2019-09-19
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01917708 on ClinicalTrials.gov