UnknownPhase 2

A Novel TBI Free Conditioning Protocol for Haploidentical Transplant in Acquired Aplastic Anemia:

Pakistan30 participantsStarted 2018-07-12

Plain-language summary

Severe and very severe aplastic anemia are life threatening disorders for which allogeneic stem cell transplant is only curative treatment. However, matched sibling donor (MSD) is available in only 25-35% cases. Pakistan has a population of around 203 million but there is no donor registry available so there is no option available for matched unrelated donor (MUD) transplants . Haploidentical transplant represents only curative option for patients lacking MSD. Protocols involving post transplant cyclophosphamide require Total body irradiation (TBI) and utilize peripheral blood stem cell(PBSC) as graft source. TBI is not available in most of transplant centres across Pakistan due to lack of availability , cost and lack of expertise. The investigators have conceived a novel TBI free conditioning regimen to be used for haplo-identical Hemtopoeitic stem cell transplant in acquired aplastic anemia patients

Who can participate

Age range

2 Years – 60 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: * Age \>2 years and \< 60 years * Karnofsky performance status \>= 70% * Aplastic Anemia that meets the following criteria: i. Peripheral Blood (must fulfill 2 of 3): ii. \<500 neutrophils iii. \<20,000 platelets iv. absolute reticulocyte count \<40,000/microL * Bone Marrow (must be ): markedly hypocellular (\<25% of normal cellularity) with absence of reticulin and abnormal infiltrate Exclusion Criteria: * Presence of donor specific antibodies * Fanconi anemia * Cytogenetic abnormalities suggestive of myelodysplastic syndrome * Prior HSCT * Human immunodeficiency virus infection * Active Hepatitis B virus infection * Active /uncontrolled bacterial, viral , fungal infection or Tuberculosis * Psychiatric illness * Poor cardiac function (ejection fraction \<40%) * Poor pulmonary function (Forced vital capacity \<50% predicted) * Poor liver function (bilirubin \>= 2mg/dL) * Poor renal function (creatinine \>= 2.0mg/dL or creatinine clearance \<40)

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

Number of Participants with overall survival

Timeframe: from the date of transplant to 1 year post transplant

Trial details

NCT IDNCT03955601

SponsorNational Institute of Blood and Marrow Transplant (NIBMT), Pakistan

Sponsor typeOTHER_GOV

Study typeINTERVENTIONAL

Primary completion2020-12-30

Contact for this trial

Xanab akram

03325346564 xanab.akram@gmail.com

View on ClinicalTrials.gov More Aplastic Anemia Idiopathic trials