Participants with Sickle Cell Disease or β-Thalassemia for Beta Thalassemia

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Memorial Sloan Kettering at Basking Ridge (Consent only), Basking Ridge, NJBeta Thalassemia+2 MoreTacrolimus - Drug
Eligibility
2 - 50
All Sexes
What conditions do you have?
Select

Study Summary

This trial is studying a new way of doing an HCT: using drugs to lower the activity of the body's immune system before transplanting healthy stem cells to replace diseased or damaged cells.

Eligible Conditions
  • Beta Thalassemia
  • Thalassemia
  • Sickle Cell Disease

Treatment Effectiveness

Study Objectives

1 Primary · 0 Secondary · Reporting Duration: 1 year

1 year
Number of participants with treatment related mortality/TRM or primary graft failure

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

1 Treatment Group

Participants with Sickle Cell Disease or β-Thalassemia
1 of 1

Experimental Treatment

24 Total Participants · 1 Treatment Group

Primary Treatment: Participants with Sickle Cell Disease or β-Thalassemia · No Placebo Group · Phase 2

Participants with Sickle Cell Disease or β-ThalassemiaExperimental Group · 8 Interventions: Tacrolimus, Rituximab, Cyclophosphamide, Dexamethasone, Fludarabine, Mycophenolate Mofetil, Rabbit ATG, Bortezomib · Intervention Types: Drug, Drug, Drug, Drug, Drug, Drug, Biological, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Tacrolimus
2011
Completed Phase 4
~4510
Rituximab
1999
Completed Phase 4
~1880
Cyclophosphamide
1995
Completed Phase 3
~3920
Dexamethasone
2007
Completed Phase 4
~2420
Fludarabine
2012
Completed Phase 3
~1100
Mycophenolate Mofetil
1997
Completed Phase 4
~2330
Rabbit ATG
2001
Completed Phase 2
~40
Bortezomib
2005
Completed Phase 2
~1250

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 1 year

Who is running the clinical trial?

Memorial Sloan Kettering Cancer CenterLead Sponsor
1,846 Previous Clinical Trials
590,042 Total Patients Enrolled
Maria Cancio, MDPrincipal InvestigatorMemorial Sloan Kettering Cancer Center
2 Previous Clinical Trials
32 Total Patients Enrolled

Eligibility Criteria

Age 2 - 50 · All Participants · 6 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:

Frequently Asked Questions

Does this research project accept individuals aged 45 or older as participants?

"Per the trial's criteria, individuals between 2 and 50 years old are eligible for enrollment." - Anonymous Online Contributor

Unverified Answer

May I be eligible to join this experiment?

"Eligibility to this medical trial necessitates that patients have thalassemia and are between 2-50 years old. The goal is to recruit around 24 participants for the clinical study." - Anonymous Online Contributor

Unverified Answer

Are any enrollees being welcomed into this research initiative currently?

"Correct. Clinicaltrials.gov testifies that this trial is actively enrolling patients; it was initially posted on February 9th, 2023 and the most recent edit occurred on the 17th of February 2023. The study requires 24 individuals to be selected from 6 different medical sites." - Anonymous Online Contributor

Unverified Answer

How many centers are responsible for coordinating the progress of this trial?

"The current trial is recruiting out of 6 sites, including Montvale, Middletown and Commack. It would be beneficial to select the closest one in order to reduce travel costs incurred when joining this study." - Anonymous Online Contributor

Unverified Answer

What is the maximum capacity for participants in this experiment?

"Affirmative. Clinicaltrials.gov's records demonstrate that this clinical trial, which was initially advertised on February 9th 2023 is actively seeking participants. Around 24 people must be enrolled from 6 associated medical centres." - Anonymous Online Contributor

Unverified Answer

What health risks do those with Sickle Cell Disease or β-Thalassemia face?

"Our team at Power has assessed the safety of individuals suffering from Sickle Cell Disease or β-Thalassemia as a 2 due to its current Phase 2 status. While there is evidence affirming some measure of protection, no data exists in support of efficacy." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.