TerminatedPhase 2

Study of MGTA-145 and Plerixafor in Patients With Sickle Cell Disease

Stopped: This study was voluntarily terminated due to a business decision not to proceed, and not due to any safety issue.

United States1 participantsStarted 2022-06-24

Plain-language summary

This research study is designed to investigate a new potential medicine for mobilizing stem cells and apheresis collection in patients with Sickle Cell Disease. MGTA-145, the new potential medicine, will be given with plerixafor.

Who can participate

Age range

18 Years – 35 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Subject must be ≥18 to ≤35 years of age. * Subject must weigh ≥30 kg. * Subject must have a diagnosis of Sickle Cell Disease. Exclusion Criteria: * Subject must not have had a vaso-occlusive event (VOE) requiring a visit to a healthcare facility within 30 days of screening. * Subject must not have undergone or attempted and failed previous hematopoietic stem cell (HSC) collection. * Subject must not have had a prior autologous or allogeneic transplantation, inclusive of gene therapy. * Male subject must be willing or able to use a highly effective method of contraception for 3 months during and after treatment. * Female subject must not be pregnant or breastfeeding. If sexually active, female subject must be willing or able to use a highly effective method of contraception for 3 months during and after treatment.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Apheresis Collection Yield

Timeframe: Up to 2 days

Assess Number of Participants With Treatment Emergent Adverse Events Leading to Study Drug Discontinuation Based on the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.

Timeframe: Up to 30 days

Assess the Number of Participants With Treatment Emergent >/= Grade 3 Clinical Laboratory Abnormalities Based on the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.

Timeframe: Up to 11 days

Vital Signs - Number of Participants With Clinically Significant Changes From Baseline in Vital Signs

Timeframe: Up to 11 days

Laboratory Assessment - Number of Participants With Clinically Significant Changes From Baseline in Hematology and Clinical Chemistry Laboratory Parameters.

Timeframe: Up to 11 days

Trial details

NCT IDNCT05445128

SponsorEnsoma

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-12-08

Results submitted2025-06-19

View on ClinicalTrials.gov More Sickle Cell Disease trials

Who can participate

Age range

18 Years – 35 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Apheresis Collection Yield

Timeframe: Up to 2 days

Timeframe: Up to 30 days

Timeframe: Up to 11 days

Vital Signs - Number of Participants With Clinically Significant Changes From Baseline in Vital Signs

Timeframe: Up to 11 days

Laboratory Assessment - Number of Participants With Clinically Significant Changes From Baseline in Hematology and Clinical Chemistry Laboratory Parameters.

Timeframe: Up to 11 days