NCT04046705: DREPA-RIC

Comparison of Allogeneic Matched Related Haematopoietic Stem Cell Transplantation After a Reduced Intensity Conditioning Regimen With Standard of Care in Adolescents and Adults With Severe Sickle Cell Disease

Status unknown Phase 3 Last updated 23 September 2019

What this trial tests

Phase 3 trial testing Allogeneic matched related haematopoietic stem cell transplantation in Sickle Cell Disease in 78 participants. Status unknown.

Timeline

15 October 2019

Primary endpoint
15 October 2024

15 October 2024

Quick facts

Lead sponsor	Assistance Publique - Hôpitaux de Paris
Phase	Phase 3
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	78
Start date	15 October 2019
Primary completion	15 October 2024
Estimated completion	15 October 2024

Drugs / interventions tested

• Allogeneic matched related haematopoietic stem cell transplantation
• Standard arm

Conditions studied

• Sickle Cell Disease — all drugs for Sickle Cell Disease →

Sponsor

Assistance Publique - Hôpitaux de Paris — full company profile →

Who can join

Adults 15 to 45, any sex, with Sickle Cell Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Although the survival of children with sickle cell disease (SCD) has dramatically improved over the last decades in the US and Europe, mortality remains high in adults. Moreover, many children and most adults develop a chronic debilitating condition due to organ damage. Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the unique curative approach; it allows the cure of more than 95% of children transplanted from a matched related donor (MRD) after a myeloablative conditioning regimen.To date, few studies have addressed the role of HSCT in SCD adults, due to the risk of graft versus host disease (GVHD) and to the toxicity expected in older patients with a higher risk of organ damage. The development of safe, non-myeloablative conditioning regimens that allow stable mixed chimerism and avoid GVHD appears as an attractive option for HSCT to cure adults with severe SCD. The investigators design a prospective multicenter trial targeting patients over 15 years with severe SCD, and compare non-myeloablative transplant (when a matched related donor (MRD) is identified) versus no HSCT (for patients lacking MRD). The main objective is to assess the benefit of HSCT on the 2-year event free survival compared to standard care. The primary endpoint is the 2-year event free survival.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure.
   Lawal RA, Walters MC, Fitzhugh CD. · · 2022 · cited 4× · PMID 36400545 · DOI 10.1016/j.hoc.2022.06.007