Comparison of Two Different Doses of Rabbit ATG-Fresenius With Cyclosporin in the Treatment of Acquired Aplastic Anaemia

NCT02028416 | Unknown DMC

• 1 other identifier: NIBD2571975
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

Acquired Aplastic anemia is one of the most frequent reason of bone marrow failure in East (Pakistan).

• The first treatment option is Allogenic Bone Marrow transplantation which is an expansive treatment option and also require a full matched HLA identical donor, hence hardly 25% of our affected patients get opportunity for BMT.
• The second line treatment option caters a large chunk of patients (severe and non-severe AA) along with those who lack HLA identical donor. Previously many protocols had been used in past for ATG+CsA Treatment, this treatment protocol especially addresses the two different regimens of ATG to study its efficacy, durability and long-term effects. Following doses would be used:
• CsA+ATG @ 10mg/kg for 3 days
• CsA+ATG @ 10mg/kg for 5 days

Conditions

Aplastic Anemia

Keywords

pancytopenia; aplastic anemia; ATG; Cyclosporin

Outcome Measures

Primary Outcomes (1)

• absolute blood counts not meeting the criteria of Aplastic Anemia

  Response is defined as blood counts no longer meeting the standard ("Camitta") criteria for severe pancytopenia in SAA, equivalent to 2 of the following values obtained on 2 serial blood count measurement at least one week apart at landmark time points (3, 6, and 12 months) . -To document the number of doses required by each of the two dose schedule to produce a rise in neutrophils and platelet count to achieve a nadir absolute lymphocyte count of 200 cmm Absolute neutrophil count \> 500/ microL Platelet count \> 20,000/ microL Reticulocyte count \> 60,000/ microL

  6 months

Secondary Outcomes (4)

• transfusion dependency after ATG treatment

  2 years

• sustained improvement of blood counts post ATG

  2years

• short-term safety of ATG and clonal evolution

  26 weeks

• response rates of ATG treatment

  2 years

Study Arms (2)

ATG-Fresenius 3 Days

ACTIVE COMPARATOR

In one group Injection ATG-Fresenius will be given @ 10mg/kg will be given for 3 days along with Capsule Cyclosporin 5mg/kg for 6 months followed by very slow tapering of dose

Drug: ATG-fresenius

ATG-Fresenius 5 Days

EXPERIMENTAL

In other arm injection ATG will be given @10mg/Kg for 5 days (different dose regimen, according to the randomization) with capsule Cyclosporin@5mg/Kg (same dose) for 6 months followed by very slow tapering. There is a difference of days of treatment received i-e 5 days.

Drug: ATG-fresenius

Interventions

ATG-fresenius DRUG

we will create 2 arms as per randomization table. Both arms will receive ATG-Fresenius but with 2 different regimens. the first arm will receive ATG-F @10mg/kg for 3 days and second arm will receive ATG-F same dose but for 5 days

Also known as: Rabbit ATG, ATG-F

ATG-Fresenius 3 Days; ATG-Fresenius 5 Days

Eligibility Criteria

• Age: 2 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• i. Severe aplastic anemia characterized by: Bone marrow cellularity \<30% (excluding lymphocytes) AND
• At least two of the following:
• a. Absolute neutrophil count \< 500/ uL b. Platelet count \< 20,000/ uL c. Absolute reticulocyte count \<60,000/ uL i. Age \> 2 years old ii. Weight \> 9 kg

You may not qualify if:

• i. Diagnosis of Fanconi's anemia ii. Evidence of a clonal disorder on cytogenetics. Patients with very severe neutropenia (ANC \< 200 /uL) will be excluded iii. Failure of BMT iv. Prior immunosuppressive therapy with ATG, ALG, alemtuzumab, or high dose cyclophosphamide v. Infection not adequately responding to appropriate therapy vi. Serologic evidence of HIV infection vii. Failure to discontinue the herbal supplements or Other alternative approach of treatment within 2 weeks of enrolment viii. Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient's ability to tolerate protocol therapy, or that death within 7-10 days is likely ix. History of carcinoma that is not considered cured (except local cervical, basal cell, or squamous cell) x. Current pregnancy, or unwillingness to take oral contraceptives or refrain from pregnancy if of childbearing potential xi. Not able to understand the investigational nature of the study or give informed consent

Sponsors & Collaborators

• National Institute of Blood Disease Center, Pakistan: lead

Study Sites (1)

National Institute of Blood Diseases and Bone Marrow Transplantation

Karachi, Pakistan, 75950, Pakistan

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic; Pancytopenia

Interventions

thymoglobulin

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases; Cytopenia

Study Officials

• TAHIR S SHAMSI, MBBS,FRCPath

  NATIONAL INSTITUTE OF BLOOD DISEASES AND BONE MARROW TRANSPLANTATION

  PRINCIPAL INVESTIGATOR

• MEHWESH TAJ, MBBS, FCPS

  NATIONAL INSTITUTE OF BLOOD DISEASES AND BONE MARROW TRANSPLANTATION

  STUDY DIRECTOR

• UZMA RIZVI, MBBS

  NATIONAL INSTITUTE OF BLOOD DISEASES AND BONE MARROW TRANSPLANTATION

  STUDY CHAIR

Central Study Contacts

Mehwesh Taj, MBBS,FCPS

CONTACT

+923002581491; mehweshfaisal@gmail.com

Tahir S Shamsi, MBBS,FRCPath

CONTACT

+9233452383956; shamsi@super.net.pk

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: comparisor of two different regimens of AtG in Acquired Aplastic Anemia

Study Record Dates

• First Submitted: January 2, 2014
• First Posted: January 7, 2014
• Study Start: September 1, 2013
• Primary Completion: December 1, 2017
• Study Completion: December 1, 2017
• Last Updated: April 5, 2016

Record last verified: 2016-04

Data Sharing

• IPD Sharing: Will not share

Locations

PA (1)