Sequential and Personalized PK-guided Busulfan Administra... | NCT04451200

Studies
Sequential and Personalized PK-guided Busulfan Administration in the Frame of the Conditiong Regimen for Allo-HSCT in Patients With Malignant Hemopathies Ineligible for the Standard Myeloablative Conditioning

NCT04451200

Sponsor

Institut Paoli-Calmettes

Status

Not yet recruiting

Last Update Posted

Nov 17, 2020Actual

Enrollment

82Estimated

Phase

Phase 2

Conditions

Acute Leukemia
Mielodysplasic Syndrome
Myeloproliferative Neoplasm

Interventions

Busulfan Injection

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Status Module

Record Verification Date

Oct 2020

Overall Recruitment Status or Expanded Access Status

Not yet recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 2020Estimated

Primary Completion Date

Nov 2023Estimated

Completion Date

Dec 2028Estimated

First Submitted Date

Jun 3, 2020

First Submission Date that Met QC Criteria

Jun 29, 2020

First Posted Date

Jun 30, 2020Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Nov 16, 2020

Last Update Posted Date

Nov 17, 2020Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

FDAAA801 Violation

Not provided

Description Module

Brief Summary

Because the anti-leukemic activity of busulfan, this dug is largely used in graft conditioning but in elderly and/or cormobid patienth an excess of toxicity is observed. This study focus on the possibility of significanty reducing this toxicity by customizing the doses of busulfan to individual PK parameters.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

82Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
busulfan treatment	Experimental	Personalized BU administration	Drug: Busulfan Injection

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Busulfan Injection	Drug	injections doses will be personalized by PK at days -7 and -4	busulfan treatment

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
non-relapse mortality evaluation	non-relapse mortality evaluation	100 days post graft

Secondary Outcomes

Measure	Description	Time Frame
incidence of grade 3 or 4 toxicities	To evaluate toxicities linked to sequential bususlfan administration	1 month
graft taking after sequential busulfan conditioning	incidence of hematological reconstituation

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Adult patient up to 65 years old
Acute leukemia, myelodysplastic syndrome or myeloproliferative neoplasia eligible for an allogeneic transplant
Chemosensitive disease, in complete or partial or stable remission
Allograft from an identical HLA related donor, Haplo-identical or unrelated (HLA compatibility from 8/10 to 10/10 according to HLA-A, -B, -C, -DR, -DQ allelics)
Signed consent to participate

-. Affiliation to a social security regimen or beneficiary of this regimen
Patient not eligible for standard myeloablative conditioning due to age> = 45 years and / or the presence of an HCT-CI comorbidity score> = 3

Exclusion Criteria:

Pregnant woman, without effective contraception or breastfeeding
Person in emergency situation, patient deprived of liberty or placed under the authority of a tutor,
Impossibility of undergoing medical follow-up of the trial for geographic, social or psychological reasons
Contraindications to performing an allogeneic transplant
Previous allograft
Placental blood allograft

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

65 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Name	Role	Phone	Extension	Email
Dominique Genre, MD	Contact	+33491223778		drci.up@ipc.unicancer.fr

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

ID	Term
D001855	Bone Marrow Diseases
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases

ID	Term
D002072	Butylene Glycols
D006018	Glycols
D000438	Alcohols
D009930	Organic Chemicals
D008698	Mesylates
D000476	Alkanesulfonates
D017738	Alkanesulfonic Acids
D000473	Alkanes
D006839	Hydrocarbons, Acyclic
D006838	Hydrocarbons
D013451	Sulfonic Acids
D013456	Sulfur Acids
D013457	Sulfur Compounds