← Back to Search

Stem Cell Transplant

Stem Cell Transplant vs Best Available Therapy for Multiple Sclerosis (BEAT-MS Trial)

Phase 3

Recruiting

Research Sponsored by National Institute of Allergy and Infectious Diseases (NIAID)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

T2 abnormalities on brain Magnetic Resonance Imaging (MRI) that fulfill the 2017 McDonald MRI criteria for dissemination in space

Completion of COVID-19 vaccination series, according to the current Centers for Disease Control and Prevention (CDC) Advisory Committee on Immunization Practices (ACIP) recommendations, ≥ 14 days prior to randomization (Day 0)

Must not have

Brain MRI or cerebrospinal fluid (CSF) examination indicating a diagnosis of progressive multifocal leukoencephalopathy (PML)

History of cytopenia consistent with the diagnosis of myelodysplastic syndrome (MDS)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from visit pre-r up to 72 months (6 years)

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing whether AHSCT is better than BAT for treating people with MS who have not responded to other treatments. 156 people will be randomly assigned to either AHSCT or BAT, and followed for 72 months.

Who is the study for?

This trial is for adults aged 18-55 with treatment-resistant relapsing Multiple Sclerosis (MS), as per the McDonald Criteria, who've had at least two episodes of disease activity in the past three years despite treatment. Participants must have an EDSS score ≤6.0, be vaccinated against COVID-19 and varicella zoster, and agree to contraception use.

What is being tested?

The BEAT-MS trial compares Autologous Hematopoietic Stem Cell Transplantation (AHSCT) with Best Available Therapy (BAT) for MS that hasn't responded well to other treatments. It's a blinded study where participants are randomly assigned to one of these strategies in equal numbers.

What are the potential side effects?

Potential side effects from AHSCT may include infection risks due to immune system suppression, reactions related to stem cell infusion, and infertility. BAT side effects vary depending on the specific therapy used but can include flu-like symptoms, injection site reactions, and increased risk of infections.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My brain MRI shows changes that meet specific criteria for a diagnosis.

Select...

I completed my COVID-19 vaccination series more than 14 days ago.

Select...

I have been diagnosed with Multiple Sclerosis according to the latest criteria.

Select...

My disability level allows me to walk at least 100 meters without aid or rest.

Select...

My MS worsened despite taking approved medication for over a month.

Select...

I am between 18 and 55 years old.

Select...

My brain MRI shows specific changes that meet certain criteria for a diagnosis.

Select...

I can walk with a cane for 100 meters without help.

Select...

My condition has worsened or shown activity in the last year.

Select...

I am a candidate for high efficacy MS treatments like cladribine or natalizumab.

Select...

My MS has worsened twice or more in the last 3 years despite treatment.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have been diagnosed with PML based on brain MRI or CSF tests.

Select...

I have a history of low blood cell counts due to MDS.

Select...

I have had a solid organ transplant in the past.

Select...

I have a history of sickle cell anemia or similar blood disorders.

Select...

I have had an acute MS flare-up or steroid treatment within the last month.

Select...

I have had a stem cell transplant before.

Select...

I currently have an active stomach ulcer.

Select...

I have or had a serious autoimmune disease that needed treatment.

Select...

My diabetes is not well-managed, with an HbA1c level over 8%.

Select...

I do not have any active infections, including viral, bacterial, or fungal.

Select...

My kidney function is reduced with an eGFR below 60.

Select...

I have been diagnosed with primary progressive MS.

Select...

I have a history of neuromyelitis optica or MOG antibody disease.

Select...

I have or had liver cirrhosis.

Select...

I have or had a neurological disorder.

Select...

My liver condition meets specific health criteria.

Select...

I have had a stroke or similar brain blood flow problem.

Select...

I have or had a serious heart condition.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from visit pre-r up to 72 months (6 years)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from visit pre-r up to 72 months (6 years)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from visit pre-r up to 72 months (6 years) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Multiple Sclerosis (MS) Relapse-Free Survival

Secondary study objectives

Change in Serum Neurofilament Light Chain (NfL) Concentration

Number of Multiple Sclerosis (MS) Relapses Per Year

Proportion of Autologous Hematopoietic Stem Cell Transplantation (AHSCT) Recipients Who Experience Primary or Secondary Graft Failure

+9 more

Side effects data

From 2022 Phase 3 trial • 665 Patients • NCT00567567

84%

58300-Neutrophil count decreased

70%

65800-Platelet count decreased

20%

43100-Hypokalemia

18%

44800-Infections and infestations - Other specify

18%

33300-Febrile neutropenia

17%

88500-White blood cell decreased

13%

13200-Anemia

55600-Mucositis oral

42700-Hypocalcemia

13500-Anorexia

11600-Alanine aminotransferase increased

41400-Hyperglycemia

15000-Aspartate aminotransferase increased

43300-Hyponatremia

73700-Sepsis

53700-Lymphocyte count decreased

25700-Diarrhea

57600-Nausea

65900-Pleural effusion

37500-GGT increased

41600-Hyperkalemia

10300-Abdominal pain

20500-Catheter related infection

59700-Oral pain

38900-Hearing impaired

43900-Hypoxia

17200-Blood and lymphatic system disorders - Other specify

14900-Ascites

75700-Small intestinal obstruction

87900-Vomiting

43600-Hypotension

45800-INR increased

34000-Fibrinogen decreased

23000-Confusion

42600-Hypoalbuminemia

71500-Respiratory failure

73900-Serum amylase increased

26600-Duodenal obstruction

66300-Pneumonitis

69700-Rash maculo-papular

58000-Neoplasms benign malignant and unspecified (incl cysts and polyps) - Other specify

56600-Myelitis

75600-Small intestinal mucositis

66800-Postoperative hemorrhage

43500-Hypophosphatemia

37300-Generalized muscle weakness

81200-Treatment related secondary malignancy

31200-Esophagitis

83100-Urinary tract infection

24100-Creatinine increased

11100-Acute kidney injury

62600-Pelvic pain

65300-Pharyngolaryngeal pain

31900-Eye disorders - Other specify

10900-Activated partial thromboplastin time prolonged

11800-Alkaline phosphatase increased

42500-Hyperuricemia

17400-Blood bilirubin increased

63100-Pericardial effusion

72700-Right ventricular dysfunction

37200-General disorders and administration site conditions - Other specify

40000-Hepatic failure

88200-Weight gain

41300-Hypercalcemia

54900-Metabolism and nutrition disorders - Other specify

71000-Renal and urinary disorders - Other specify

69000-Pulmonary hypertension

20100-Cardiac disorders - Other specify

22100-Colitis

44200-Ileal obstruction

81900-Typhlitis

33900-Fever

35500-Gallbladder pain

40600-Hepatobiliary disorders - Other specify

66500-Portal hypertension

12000-Allergic reaction

13100-Anaphylaxis

44700-Immune system disorders - Other specify

13400-Anorectal infection

25600-Device related infection

29500-Enterocolitis infectious

53100-Lung infection

62500-Pelvic infection

75200-Skin infection

82300-Upper respiratory infection

14500-Arterial injury

15300-Ataxia

38800-Headache

63900-Peripheral motor neuropathy

11300-Adult respiratory distress syndrome

29700-Epistaxis

78100-Stridor

68400-Pruritus

51700-Left ventricular systolic dysfunction

27800-Dyspnea

58100-Nervous system disorders - Other specify

29000-Encephalopathy

42100-Hypertension

24700-Dehydration

43200-Hypomagnesemia

31800-Extrapyramidal disorder

52600-Lipase increased

10700-Acidosis

100%

80%

60%

40%

20%

Study treatment Arm

Tandem HST (CEM), Randomly Assigned

Single HST (CEM)

Not Assigned

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: AHSCTExperimental Treatment1 Intervention

AHSCT: Myeloablative and Immunoablative therapy followed by Autologous Hematopoietic Stem Cell Transplantation Participants will undergo: 1. Mobilization and graft collection: mobilization of peripheral blood stem cells (PBSC) with cyclophosphamide, filgrastim, and dexamethasone. The autologous graft will be collected by leukapheresis and cryopreserved. 2. Conditioning: high dose myeloablative and immunoablative conditioning with a six-day BEAM chemotherapy and rabbit anti-thymocyte globulin regimen will be initiated ≥30 days after cyclophosphamide mobilization. 3. Autologous cryopreserved graft infusion: the cryopreserved peripheral blood stem cells (PBSC) graft will be thawed and infused the day following completion of the conditioning regimen. Each bag will be thawed and infused according to institutional standards consistent with the Foundation for the Accreditation of Cellular Therapy (FACT) guidelines. Participants will receive prednisone following graft infusion.

Group II: Best Available Therapy (BAT)Active Control1 Intervention

Participants randomized to BAT: Best available therapy will be selected by the Site Investigator from: Cladribine (Mavenclad®), natalizumab (Tysabri®), alemtuzumab (Campath®, Lemtrada®), ocrelizumab (Ocrevus®), ublituximab (BRIUMVI™), rituximab (Rituxan®), or ofatumumab (Arzerra®) (after approval by the FDA for relapsing MS).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Autologous Hematopoietic Stem Cell Transplantation

2017

Completed Phase 3

~2090

0 / 29Eligibility criteria met