Clinical trial NCT03234335: High Dose Therapy Followed by Autologous Transplantation for Myeloma Patients With Severe Renal Impairment
Official title: A Prospective, Non-interventional, Multinational Study Evaluating the Efficacy and the Safety of High Dose Therapy Followed by Autologous Hematopoietic Stem Cell Transplantation as a Frontline Therapy for Myeloma Patients With Severe Renal Impairment (IRMYG Study)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Multiple Myeloma
Renal Failure
Keywords: Multiple Myeloma
Renal Failure
Autologous Hematopoietic Stem Cell Transplantation (ASCT)
Melphalan
Lead sponsor: Centre Hospitalier Universitaire de Saint Etienne
Sponsor class: OTHER
Collaborators: name: Institut de Cancérologie de la Loire
First posted: 2017-07-31
Enrollment: 50
Sex: ALL
Minimum age (years): 66
Standard ages: OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Myeloma patients with severe renal impairment suseptible of undergoing autologous transplantation.

Brief summary: Multiple myeloma (MM) is a malignant plasma cell disorder, characterized by the presence of more than 10 % of clonal plasma cells in the bone marrow. Therapeutic intervention is recommended when at least one of the myeloma defining events occurs (CRAB features). Renal impairment (RI) is one of the most common complications of MM, accounting for 20-30 % of MM patients at diagnosis and 40-50% of patients during the course of their disease. To date, there is no defined consensus for the management of myeloma patients with renal failure. It is then of clinical importance to better considering available therapeutic options to improve responses and survival of these patients.

Detailed description: RI is associated with poor prognosis and short median survival (32 months vs 55 months for MM patients with normal renal function). Thus, RI remains a major challenge for hematologists, including decisions on optimal anti-myeloma therapy, potential dialysis, supportive care and quality of life. The combination of a proteasome inhibitor and an immunomodulator is the preferred induction treatment for newly diagnosed transplant-eligible MM patients. After induction, high-dose therapy with Autologous Stem Cell Transplant (ASCT) is the standard of care for these patients. However, concerns related to management of comorbidities and treatment side effects question about therapeutic options for patients with severe renal damage. Of interest, recent studies argued that high-dose therapy followed by ASCT could be a feasible and safe method for renal failure MM patients. Yet, these observations on small sample size patients groups need to be confirmed with standardized conditions. This study proposes to evaluate the efficacy and the safety of this therapeutic strategy in MM patients with severe renal impairment.

Eligibility criteria: Inclusion Criteria:

* Age ≤ 66 years-old
* Patients with symptomatic, measurable and newly diagnosed multiple myeloma associated:
* Severe renal failure at the time of transplantation (creatinine clearance \< 40 ml/min/1.73m², CKD-EPI: Chronic Kidney Disease Epidemiology Collaboration)
* Partial response after induction treatment
* For patients who undergo autologous transplantation, absence of known contraindication for transplantation
* Absence of amylose
* Patient affiliated to a social security regimen or beneficiary of the same
* Signed written informed consent form

Exclusion Criteria:

* Patient without at least a partial hematological response following the induction stage
* Medical history of previous malignancy
* Patient under guardianship or deprived of his liberty or any condition that may affect the patient's ability to understand and sign the informed consent (art. L.1121-6, L.112-7, L.1211-8, L.1211-9)
* Pregnant or breastfeeding woman
* Declining participation

Primary outcomes:
measure: Non Relapse Mortality post-transplantation
timeFrame: 100 days post-transplantation
Secondary outcomes:
measure: Overall survival
timeFrame: 2 years post-transplantation
measure: progression-free survival
timeFrame: 2 years post-transplantation
measure: Number of toxicities
timeFrame: 2 years post-transplantation
measure: presence of hematological response
timeFrame: 6 months
measure: Level of renal response
timeFrame: 3 months, 6 months and one year
Other outcomes:
Locations:
city: Alger
country: Algeria
facility: Centre Pierre et Marie Curie
city: Oran
country: Algeria
facility: EHU Oran
city: Liège
country: Belgium
facility: CHU Sart Tilman
city: Amiens
country: France
facility: Centre Hospitalier Universitaire d'Amiens
city: Angers
country: France
facility: Centre Hospitalier Universitaire d'Angers
city: Argenteuil
country: France
facility: Centre Hospitalier d'Argenteuil
city: Bayonne
country: France
facility: Centre Hospitalier de la Côte Basque
city: Besançon
country: France
facility: Centre Hospitalier Universitaire de Besançon
city: Boulogne
country: France
facility: Centre Hospitalier de Boulogne
city: Brest
country: France
facility: CHU de Brest
city: Caen
country: France
facility: Centre Hospitalier Universitaire de Caen
city: Cholet
country: France
facility: Centre Hospitalier de Cholet
city: Clermont-Ferrand
country: France
facility: Centre Hospitalier Universitaire de Clermont Ferrand
city: Dijon
country: France
facility: Centre Hospitalier Universitaire de Dijon
city: Grenoble
country: France
facility: Centre Hospitalier Universitaire de Grenoble
city: Limoges
country: France
facility: CHU de Limoges
city: Lyon
country: France
facility: Centre Léon Bérard
city: Montpellier
country: France
facility: Hôpital Saint-Eloi
city: Nancy
country: France
facility: Centre Hospitalier Universitaire de Nancy
city: Nice
country: France
facility: Hôpital Archet
city: Paris
country: France
facility: Institut Curie
city: Paris
country: France
facility: Groupe Hospitalier Pitié-Salpétrière
city: Paris
country: France
facility: Hôpital Saint-Antoine
city: Paris
country: France
facility: Hôpital Tenon
city: Paris
country: France
facility: Hôpital Cochin
city: Pierre-Bénite
country: France
facility: Centre Hospitalier Lyon Sud
city: Poitiers
country: France
facility: Hôpital Saint-Bernard
city: Rennes
country: France
facility: CHU de Rennes
city: Roubaix
country: France
facility: Hôpital Victor Provo (Roubaix)
city: Saint-Priest-en-Jarez
country: France
facility: CHU de Saint-Etienne
city: Saint-Quentin
country: France
facility: Centre Hospitalier de Saint Quentin
city: Strasbourg
country: France
facility: Hôpitaux Universitaires de Strasbourg
city: Beyrouth
country: Lebanon
facility: American University of Beirut
Study officials:
affiliation: CHU de Saint-Etienne
name: Jérôme Cornillon, MD
role: PRINCIPAL_INVESTIGATOR
References: