Multiple MyelomaPhase 4NCT06425276

Evaluate Safety and Efficacy of High-dose Melphalan HCL for Injection in MM Patients With Auto-HSC Transplantation

A Phase 4 study testing Melphalan Hydrochloride for Injection in people with Multiple Myeloma, run by CASI Pharmaceuticals (China) Co., Ltd. at 1 site in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The goal of this clinical trial is to learn if high-dose Melphalan HCl for Injection works to treat multiple myeloma. It will also learn about the safety of high dose Melphalan HCl for Injection. Read more about this study

Who is it for?: Ages 18 to 65 years
Conditions the study is for: Multiple Myeloma
What is being tested?: Melphalan Hydrochloride for Injection
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 67 participants
Where: 1 siteChina
Study dates: 24 Jul 2020to 18 Sep 2021
Phase: Phase 4
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 65 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Diagnosed as symptomatic multiple myeloma, according to the International Myeloma Working Group's IMWG Guidelines for the Diagnosis and Treatment of Multiple Myeloma, treatment is necessary and suitable for autologous hematopoietic stem cell transplantation;
When signing the informed consent form, males and females aged ≥ 18 years and ≤ 65 years old;
Adequate autologous hematopoietic stem cells were collected, defined as peripheral blood stem cells containing at least 2 x 106 CD34+cells/kg that have not been manipulated or refrigerated;
Important organ functions meet the following conditions:
Echocardiography indicates left ventricular ejection fraction (LVEF) ≥ 40%;
Serum total bilirubin<2 times the upper limit of normal value, alanine aminotransferase (ALT) and aspartate aminotransferase (AST)<3 times the upper limit of normal value;

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creatinine clearance rate>60 mL/min ;
Blood oxygen saturation>92% in non oxygenated state, without significant ventilation or ventilation dysfunction;
The Eastern Oncology Collaborative Group (ECOG) physical fitness status of the subjects is 0, 1, or 2;
The subject or their legal guardian voluntarily signs an informed consent form approved by the ethics committee before participating in the study, and agrees to complete the entire study treatment according to the clinical trial protocol.

Who cannot join

Multiple myeloma subjects without treatment indications;
Suffering from plasma cell leukemia;
Suffering from systemic amyloidosis;
Subjects with extramedullary plasma cell tumors did not reach PR after induction therapy;
Suffering from POEMS syndrome (multiple peripheral neuropathy, organ enlargement, endocrine disorders, M-proteinemia, skin changes);
Suffering from Fahrenheit macroglobulinemia;

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Subjects with non secretory multiple myeloma;
Subjects with active bacterial, viral, or fungal infections who require oral or intravenous antibiotic treatment according to the researcher's judgment;
The expected survival period of the subjects is less than 6 months;
Previously suffering from other malignant tumors, except for cured basal cell carcinoma or cervical carcinoma in situ. Malignant tumors that have undergone curative treatment and achieved complete remission (CR) for more than 5 years can be enrolled. If malignant tumors receive curative treatment but have achieved complete remission (CR) for ≤ 5 years, they cannot be enrolled unless approved by the sponsor;
Pregnant or lactating women;
Subjects who have fertility and are unwilling to take appropriate contraceptive measures within 3 months after signing the informed consent form until the end of treatment in this study;
Positive for human immunodeficiency virus (HIV) antibodies;
Subjects with positive hepatitis B virus DNA;
The subject receives other concurrent anti-tumor treatments (including chemotherapy, radiation therapy, hormone therapy, or immunotherapy) within 30 days prior to autologous hematopoietic stem cell transplantation, or plans to receive any such treatments before the last study visit on day 95 ± 5;
The side effects of chemotherapy drugs received before administration have not yet recovered, defined as not regressing to level 0/1 of the National Cancer Institute's Common Terminology Standard for Adverse Events (NCI-CTCAE v5.0), or at the level specified in the inclusion/exclusion criteria, except for adverse events such as hair loss that the researcher assessed and deemed not to affect the safety of the subject's participation in this study;
Allergy or intolerance to any component of the investigational drug formulation;
Participants participate in other clinical trials within one month before signing the informed consent form;
According to the researcher's judgment, subjects who are not suitable for enrollment, may affect treatment evaluation, or are at inappropriate risk.

Trial locations and contacts

1 site in 1 country.

China1 site

Peking University People's Hospital
Beijing

Other trials in China

About this study

Official title: A Multicenter, Open-Label, Safety and Efficacy Study of High Dose Melphalan Hydrochloride for Injection for Myeloablative Conditioning in Multiple Myeloma Patients Undergoing Autologous Hematopoietic Stem Cell Transplantation

Does high-dose Melphalan HCl for Injection deplete bone marrow activity which results in a better outcome of patients'own stem cell (blood-forming cell) transplantation? What medical problems do participants have when taking high-dose Melphalan HCl for Injection? How fast is the high-dose Melphalan HCl for Injection cleared out from blood?

Participants will:

Take high-dose Melphalan HCl for Injection for 2 days
Have stem cell transplantation one day after treatment
Stay in the hospital for at least 10days and visit the clinic once every week for the first month after transplantation and every month after for checkups and tests.

Read the detailed description

Autologous stem cell transplantation (ASCT) is a standard of care in transplant-eligible MM patients which has been demonstrated with a better complete remission rate (CR) and with longer survival. High-dose melphalan (200 mg/m2) has been the most commonly used conditioning regimen for ASCT in MM. EVOMELA (melphalan HCl for Injection) stabilized with Captisol, a specially modified cyclodextrin, has been believed to have better solubility and stability upon reconstitution which result in decreased toxicity, increased convenience and flexibility of administration without comprising efficacious data. Melphalan HCl for Injection has been approved by NMPA as a standard conditioning drug for ASCT in MM patients. The current study evaluated the efficacy, safety, and PK profile for high-dose Melphalan HCl for Injection as a myeloablative conditioning regimen in Chinese symptomatic transplant-eligible MM patients.

Conditions: Multiple Myeloma
Being tested: Melphalan Hydrochloride for Injection
Medical subject headings: Multiple Myeloma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Melphalan Hydrochloride for InjectionDrug
Also called Evomela
During the Study Period, patients will receive Melphalan HCl for Injection dosed at 100 mg/m2 on Day -3 and Day -2.

Study groups in NCT06425276
Study group	Role	What they receive
High-dose Melphalan HCl for Injection treatment arm	Experimental	Patients will receive Melphalan HCl for Injection dosed at 100 mg/m2 on Day -3 and Day -2. Following 1 day of rest after the myeloablative conditioning (Day -1), patients will receive an autologous graft with a minimum cell dose of 2 × 106 CD34+ cells/kg of patient body weight (Day 0). Receives: Drug: Melphalan Hydrochloride for Injection

What is being measured

The primary outcome is the main question the study is designed to answer.

Rate of patients achieveing myeloablation
To record the percentage of patients who achieve myeloablation which is defined as absolute neutrophil count [ANC] <0.5 × 109/L, absolute lymphocyte count [ALC] <0.1 × 109/L, or platelet count <20,000/mm3 in 2 consecutive daily assessments.
Measured: 95±5 days after ASCT
Time to achieveing myeloablation
To record the time, in days, from the date of first dose of High-dose Melphalan HCL for Injection to the date of myoloablation which is defined as absolute neutrophil count [ANC] <0.5 × 109/L, absolute lymphocyte count [ALC] <0.1 × 109/L, or platelet count <20,000/mm3 in 2 consecutive daily assessments.
Measured: 95±5 days after ASCT
Time to achieveing neutrophil engraftment
To record the time, in days, from the date of Auto-HSCT to the date when absolute neutrophil count (ANC) >0.5 × 109/L in 3 consecutive daily assessments.
Measured: 95±5 days after ASCT
Time to achieving platelet engraftment
To record the time, in days, from the date of Auto-HSCT to the date when untransfused platelet measurement >20,000/mm3 in 3 consecutive daily assessments.
Measured: 95±5 days after ASCT
Incidence of Treatment-related Motality (TRM)
To characterize the safety, tolerability of High-dose Melphalan HCL for Myeloablation in MM Patients With Auto-HSC Transplantation by recording the incidence of death without relapse or progression of the disease.
Measured: 95±5 days after ASCT
Incidence and severity of AEs and SAEs, including changes in laboratory values
To characterize the safety, tolerability of High-dose Melphalan HCL for Injection for Myeloablation in MM Patients With Auto-HSC Transplantation.
Measured: 95±5 days after ASCT

Secondary outcomes (5)

Overall Response (ORR)
Response assessment per International Myeloma Working Group (IMWG) criteria
Measured: 95±5 days after ASCT
Tmax of Melphalan HCL for Injection derived from plasma concentrations
Tmax of Melphalan HCL for Injection derived from plasma concentrations of each administration
Measured: 24 Hours
T1/2 of Melphalan HCL for Injection derived from plasma concentrations
T1/2 of Melphalan HCL for Injection derived from plasma concentrations of each administration
Measured: 24 Hours
Cmax of Melphalan HCL for Injection derived from plasma concentrations
Cmax of Melphalan HCL for Injection derived from plasma concentrations of each administration
Measured: 24 Hours
AUC of Melphalan HCL for Injection derived from plasma concentrations
AUC of Melphalan HCL for Injection derived from plasma concentrations of each administration
Measured: 24 Hours

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

24 Jul 202018 Sep 2021

First posted22 May 2024
Study start24 Jul 2020
Primary completion8 Jul 2021
Study completion18 Sep 2021

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Multiple MyelomaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 4
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 67 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT06425276
Sponsor's protocol ID: EVOM-CL-001
Status last verified: May 2024
First submitted: 17 May 2024
Last updated: 23 May 2024
Results posted: No
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Not yet recruitingPhase 2
Study of YKST02 in Adults With Relapsed or Refractory Multiple Myeloma
Relapsed or Refractory Multiple Myeloma (RRMM)
RecruitingPhase 2
Engaging T-cells to Eliminate MRD in Newly Diagnosed Myeloma Optimizing Response With Talquetmab and Teclistamab (ROTATE)
Multiple Myeloma
Active, not recruitingPhase 3
A Phase III Study of Eque-cel in Subjects With Len-refractory RRMM (FUMANBA-03)
Multiple Myeloma
Active, not recruitingPhase 1/2
A Window of Opportunity Trial to Learn if Linvoseltamab is Safe and Well Tolerated, and How Well it Works in Adult Participants With Recently Diagnosed Multiple Myeloma Who Have Not Already Received Treatment
Multiple Myeloma
Active, not recruitingPhase 2
Testing AZD5363 as a Potential Targeted Treatment in Cancers With AKT Genetic Changes (MATCH-Subprotocol Y)
Advanced Lymphoma, Advanced Malignant Solid Neoplasm

All Multiple Myeloma trials

Study at a glance

From the registry record, last updated by the sponsor on 23 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Multicenter, Open-Label, Safety and Efficacy Study of High Dose Melphalan Hydrochloride for Injection for Myeloablative Conditioning in Multiple Myeloma Patients Undergoing Autologous Hematopoietic Stem Cell Transplantation
ClinicalTrials.gov ID: NCT06425276
Acronym / sponsor ID: EVOM-CL-001
Conditions: Multiple Myeloma
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 67 participants (actual)
Lead sponsor: CASI Pharmaceuticals (China) Co., Ltd.Industry (pharma, biotech, devices)
Study start: 24 Jul 2020 (actual)
Primary completion: 8 Jul 2021 (actual)
Study completion: 18 Sep 2021 (actual)
First posted: 22 May 2024 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Participants will:

Take high-dose Melphalan HCl for Injection for 2 days
Have stem cell transplantation one day after treatment
Stay in the hospital for at least 10days and visit the clinic once every week for the first month after transplantation and every month after for checkups and tests.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
High-dose Melphalan HCl for Injection treatment arm	Experimental	Drug: Melphalan Hydrochloride for InjectionPatients will receive Melphalan HCl for Injection dosed at 100 mg/m2 on Day -3 and Day -2. Following 1 day of rest after the myeloablative conditioning (Day -1), patients will receive an autologous graft with a minimum cell dose of 2 × 106 CD34+ cells/kg of patient body weight (Day 0).

Who may qualify

Ages: 18 to 65 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (10 listed)

Diagnosed as symptomatic multiple myeloma, according to the International Myeloma Working Group's IMWG Guidelines for the Diagnosis and Treatment of Multiple Myeloma, treatment is necessary and suitable for autologous hematopoietic stem cell transplantation;
When signing the informed consent form, males and females aged ≥ 18 years and ≤ 65 years old;
Adequate autologous hematopoietic stem cells were collected, defined as peripheral blood stem cells containing at least 2 x 106 CD34+cells/kg that have not been manipulated or refrigerated;
Important organ functions meet the following conditions:
The Eastern Oncology Collaborative Group (ECOG) physical fitness status of the subjects is 0, 1, or 2;

Key exclusion criteria (19 listed)

Multiple myeloma subjects without treatment indications;
Suffering from plasma cell leukemia;
Suffering from systemic amyloidosis;
Subjects with extramedullary plasma cell tumors did not reach PR after induction therapy;
Suffering from POEMS syndrome (multiple peripheral neuropathy, organ enlargement, endocrine disorders, M-proteinemia, skin changes);

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Melphalan Hydrochloride for InjectionAlso: EvomelaDuring the Study Period, patients will receive Melphalan HCl for Injection dosed at 100 mg/m2 on Day -3 and Day -2.	Drug	High-dose Melphalan HCl for Injection treatment arm

Primary outcome

Rate of patients achieveing myeloablation
Time frame: 95±5 days after ASCT
To record the percentage of patients who achieve myeloablation which is defined as absolute neutrophil count [ANC] <0.5 × 109/L, absolute lymphocyte count [ALC] <0.1 × 109/L, or platelet count <20,000/mm3 in 2 consecutive daily assessments.
Time to achieveing myeloablation
Time frame: 95±5 days after ASCT
To record the time, in days, from the date of first dose of High-dose Melphalan HCL for Injection to the date of myoloablation which is defined as absolute neutrophil count [ANC] <0.5 × 109/L, absolute lymphocyte count [ALC] <0.1 × 109/L, or platelet count <20,000/mm3 in 2 consecutive daily assessments.
Time to achieveing neutrophil engraftment
Time frame: 95±5 days after ASCT
To record the time, in days, from the date of Auto-HSCT to the date when absolute neutrophil count (ANC) >0.5 × 109/L in 3 consecutive daily assessments.
Time to achieving platelet engraftment
Time frame: 95±5 days after ASCT
To record the time, in days, from the date of Auto-HSCT to the date when untransfused platelet measurement >20,000/mm3 in 3 consecutive daily assessments.
Incidence of Treatment-related Motality (TRM)
Time frame: 95±5 days after ASCT
To characterize the safety, tolerability of High-dose Melphalan HCL for Myeloablation in MM Patients With Auto-HSC Transplantation by recording the incidence of death without relapse or progression of the disease.
Incidence and severity of AEs and SAEs, including changes in laboratory values
Time frame: 95±5 days after ASCT
To characterize the safety, tolerability of High-dose Melphalan HCL for Injection for Myeloablation in MM Patients With Auto-HSC Transplantation.

5 secondary outcomes

Overall Response (ORR)
Time frame: 95±5 days after ASCT
Tmax of Melphalan HCL for Injection derived from plasma concentrations
Time frame: 24 Hours
T1/2 of Melphalan HCL for Injection derived from plasma concentrations
Time frame: 24 Hours
Cmax of Melphalan HCL for Injection derived from plasma concentrations
Time frame: 24 Hours
AUC of Melphalan HCL for Injection derived from plasma concentrations
Time frame: 24 Hours

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 23 May 2024.
Individual participant dataSharing planned: No

Related trials for Multiple Myeloma

Not yet recruitingPhase 2
Study of YKST02 in Adults With Relapsed or Refractory Multiple Myeloma
Relapsed or Refractory Multiple Myeloma (RRMM)
RecruitingPhase 2
Engaging T-cells to Eliminate MRD in Newly Diagnosed Myeloma Optimizing Response With Talquetmab and Teclistamab (ROTATE)
Multiple Myeloma
Active, not recruitingPhase 3
A Phase III Study of Eque-cel in Subjects With Len-refractory RRMM (FUMANBA-03)
Multiple Myeloma
Active, not recruitingPhase 1/2
A Window of Opportunity Trial to Learn if Linvoseltamab is Safe and Well Tolerated, and How Well it Works in Adult Participants With Recently Diagnosed Multiple Myeloma Who Have Not Already Received Treatment
Multiple Myeloma
Active, not recruitingPhase 2
Testing AZD5363 as a Potential Targeted Treatment in Cancers With AKT Genetic Changes (MATCH-Subprotocol Y)
Advanced Lymphoma, Advanced Malignant Solid Neoplasm

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Kaiyan Liu principal investigator

Peking University People's Hospital

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Melphalan Hydrochloride for Injection in people with Multiple Myeloma, run by CASI Pharmaceuticals (China) Co., Ltd. at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 23 May 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Rate of patients achieveing myeloablation (time frame: 95±5 days after ASCT). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06425276.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 May 2024.