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Aplastic Anemia IdiopathicPhase 2FluCAB-PrimeNCT03955601

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

A Phase 2 study testing Haploidentical HSCT using TBI free regimen, ''ATG'' with ''Post transplant cyclophosphamide'' in people with Aplastic Anemia Idiopathic, run by National Institute of Blood and Marrow Transplant (NIBMT), Pakistan at 1 site in Pakistan.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 2 to 60 years
Conditions the study is for
Aplastic Anemia Idiopathic
What is being tested?
Haploidentical HSCT using TBI free regimen, ''ATG'' with ''Post transplant cyclophosphamide''
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
1 sitePakistan
Study dates
12 Jul 2018to 30 Jun 2021
Phase
Phase 2
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 2 to 60 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age >2 years and < 60 years
  • Karnofsky performance status >= 70%
  • Aplastic Anemia that meets the following criteria:
  • 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

Who cannot join

  • Presence of donor specific antibodies
  • Fanconi anemia
  • Cytogenetic abnormalities suggestive of myelodysplastic syndrome
  • Prior HSCT
  • Human immunodeficiency virus infection
  • Active Hepatitis B virus infection
Show all 12Show fewer
  • 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)

Trial locations and contacts

1 site in 1 country.

Xanab akram contact

03325346564

xanab.akram@gmail.com

Pakistan1 site, 1 recruiting

  • NIBMT

    Rawalpindi, Punjab Province, 46000

    Tariq Mehmood Satti, FCPS, MCPS, +92-51-9270076 ext. 201, tariqmahmood_satti@yahoo.com

    FCPS,FACP

    Recruiting

Other trials in Pakistan

About this study

Official title: A Novel TBI Free Conditioning Protocol for Haploidentical Hematopoeitic Stem Cell Transplant in Acquired Aplastic Anemia

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

Read the detailed description

Aplastic anemia is considered to be a rare and heterogenous disease with incidence of 1-2 per million in western countries. Data from Asian studies show a 3-4 fold higher incidence.Majority of newly diagnosed aplastic anemia patients are younger and in Armed forces bone marrow transplant cohort of 1324 patients 64 % patient are younger than 24 years of age and 87% patients younger than 40 years (unpublished data).There is no donor registry in Pakistan and patients lacking sibling match donor cannot proceed to stem cell transplant due lack of matched unrelated donors. Horse antithymocyte globulin is not available currently in Pakistan and response to Rabbit antithymocyte globulin is dismal as shown in number of international studies. So haploidentical stem cell transplant remains only curative option for patients lacking Matched sibling donor. Currently there are 2 major platforms used for haplo-identical stem cell transplant. Post transplant cyclophosphamide based using TBI and haplo regimen of Peking university. TBI is not available for most of our patients in Pakistan due to cost,non-availability and lack of expertise. The investigators have formulated a novel TBI free regimen incorporating Busulphan, antithymocyte globulin and using co-primed bone marrow and peripheral blood harvest to minimize graft-versus-host disease and facilitate engraftment. Post transplant cyclophosphamide, Cyclosporine and mycophenolate mofetil will be used for graft-versus-host disease prophylaxis

Being tested
Haploidentical HSCT using TBI free regimen, ''ATG'' with ''Post transplant cyclophosphamide''
Keywords
haploidentical, Aplastic anemia, TBI
Medical subject headings
Anemia, Aplastic

What participants receive

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

  • Haploidentical HSCT using TBI free regimen, ''ATG'' with ''Post transplant cyclophosphamide''Drug

    Also called ''Combined BMH'' and ''PBSC harvest''

    ''Busulphan'' will be used in place of ''TBI'' in equivalent myelotoxic dose to facilitate engraftment , ''ATG'' will be used to reduce GVHD and facilitate engraftment while ''combine PBSC'' and/OR ''Bone marrow harvest'' will be used

Study groups in NCT03955601
Study groupRoleWhat they receive
TBI free Haploidentical HSCTExperimentalRecipients will receive a reduced intensity conditioning regimen of ''Fludarabine'' 30 mg/m2 IV daily from day -7 to -3, ''Cyclophosphamide'' 14.5-30 mg/kg IV daily on day -6 and -5 , ''rabbit Antithymocyte globulin'' 5 mg /kg/day from day -6 to day-3; ''Busulphan'' IV 3.2 mg per kg/day in 02 divided doses on day -3 and day-2, ''Granulocyte Colony Stimulating factor primed Bone marrow harvest'' and/OR ''PBSC'' graft on day 0 and day +1 respectively and Graft versus host disease prophylaxis with ''post-transplant cyclophosphamide'' administered at a dose of 50mg/kg/day given daily on days +3 and +5 post-transplant and ''cyclosporine'' from day +5, ''mycophenolate mofetil'' from day+5 to day+35.
Receives: Drug: Haploidentical HSCT using TBI free regimen, ''ATG'' with ''Post transplant cyclophosphamide''

What is being measured

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

  1. Number of Participants with overall survival

    overall survival is defined as the time interval from date of transplant to death or to last follow-up, whichever occurs first.

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

Secondary outcomes (4)

  1. Number of Participants with Disease free survival

    from time of transplant to death or last follow up

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

  2. Time of Neutrophil engraftment

    first of 3 consecutive days with Absolute neutrophil count> 0.5

    Measured: from the date of transplant to 10 to day 28 post transplant

  3. Frequency of Graft versus host disease

    as per clinical and histopathological diagnosis

    Measured: from the date of transplant to acute upto 100 day post transplant, chronic >100 days post transplant

  4. Rate of Complications

    both infectious and non infectious

    Measured: from the date of transplant to 1 year from day of transplantation

Timeline

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

  1. First posted20 May 2019
  2. Study start12 Jul 2018
  3. Primary completion30 Dec 2020 (estimated)
  4. Study completion30 Jun 2021 (estimated)

Resources

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03955601
Sponsor's protocol ID
AFBMTC-HAPLO-AA
Acronym
FluCAB-Prime
Status last verified
Aug 2020
First submitted
22 Apr 2019
Last updated
11 Aug 2020
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Haploidentical HSCT using TBI free regimen, ''ATG'' with ''Post transplant cyclophosphamide'' in people with Aplastic Anemia Idiopathic, run by National Institute of Blood and Marrow Transplant (NIBMT), Pakistan at 1 site in Pakistan.

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 5 inclusion and 12 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: status not confirmed, as of the sponsor's last update on 11 Aug 2020. 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 Pakistan. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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

Are results available for this study?

Results are not available in the source record.

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

Contact Xanab akram (03325346564, xanab.akram@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants with overall survival (time frame: from the date of transplant to 1 year post transplant). 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 NCT03955601.

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 11 Aug 2020.

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