High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 6 May 2024
• Main ID: NCT02629120
• Date of registration: 10/12/2015
• Prospective Registration: Yes
• Primary sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
• Public title: High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease
• Scientific title: High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease
• Date of first enrolment: December 17, 2015
• Target sample size: 44
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02629120
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Elizabeth M Kang, M.D.
• Affiliation: National Institute of Allergy and Infectious Diseases (NIAID)

Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

- Must have confirmed Chronic Granulomatous Disease.

- Must have sufficient complications from underlying disease to warrant undergoing
transplantation (either a history of or ongoing inflammation/CGD related autoimmunity
OR a CGD related infection while on prophylaxis) OR or have a Quartile 1 and/or 2
residual oxidase production level.

- Ages 4 years - 65 years

- HLA-matched family donor graft or an HLA matched unrelated peripheral blood stem cell
(PBSC) graft (10/10 or 9/10 mismatch) available

- Must be HIV negative

- When discharged from the hospital must be able to stay within one hour s travel of the
NIH for the first 3 months after transplantation and have a family member or other
designated companion to stay with during the post transplant period.

- Must provide a durable power of attorney for health care decisions to an appropriate
adult relative or guardian in accordance to NIH -200 NIH Durable Power of Attorney for
Health Care Decision Making .

- If of child-bearing potential, must agree to consistently use contraception from one
month prior to, and throughout, study participation, and for 3 months post-study.
Acceptable forms of contraception are:

- Contraceptive pills or patch, Norplant , Depo-Provera , or other FDA-approved
contraceptive method

- Male partner has previously undergone a vasectomy.

- Male participants will be advised to consistently use contraception throughout
study participation and for 3 months post-transplant.

EXCLUSION CRITERIA:

- Eastern Cooperative Oncology Group (ECOG) or equivalent performance status greater
than or equal to 3 (See Supportive Care guidelines, available at
http://intranet.cc.nih.gov/bmt/clinicalcare)

- Left ventricular ejection fraction < 40%

- Transaminases > 5x upper limit of normal based on the participant s clinical situation
and at the discretion of the investigator

- Psychiatric disorder or mental deficiency severe enough as to make compliance with the
HSCT treatment unlikely, and/or making regulatorily and legally effective informed
consent impossible

- Major anticipated illness or organ failure incompatible with survival from AlloPBSC
transplant

- Pregnant or lactating

- HIV positive

- Uncontrolled seizure disorder

- Individuals older than 65 are excluded. It is known from standard transplantation that
these participants have a higher risk of morbidity and mortality related to
transplantation. Given the investigational nature of this protocol, the risk benefit
ratio is not warranted to include these participants at this time.

- Any condition or circumstance which the PI feels would create difficulty in
maintaining compliance with the requirements of this protocol.

- Individuals who are not willing to submit their information as part of the Alemtuzumab
(Campath) Distribution Program application or participants whom the Distribution
Program committee has determined are not qualified to receive alemtuzumab.

--NOTE: Alemtuzumab (Campath-1H) (intravenous [IV] formulation) is no longer
distributed commercially. In order to receive product, the physician must contact the
program for the patient. If the patient is not willing to consent to submit their info
(demographics, contact information, and rationale for use) to the program such that we
can obtain the drug, then we cannot proceed with conditioning; therefore no transplant
will occur on this protocol.

- Patients with a CRP greater than 100 mg/L within 30 days of anticipated transplant.

- If the underlying inflammation is controlled for one month with repeat CRP
testing showing a level of less than 100 on at least two separate testings, the
patient will be reconsidered for transplant. If during this time period a CRP of
greater than 100 is measured, then the patient would no longer be eligible for
transplant.



Age minimum: 4 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Chronic Granulomatous Disease Transplant

Intervention(s)

Drug: Alemtuzumab

Drug: Sirolimus

Radiation: Total Body Irradiation

Drug: Cyclophosphamide

Biological: Peripheral blood stem cells

Drug: Busulfan

Primary Outcome(s)

To determine engraftment rates with the use of high cell doses, without increasing the risk of GvHD by using post-transplant cyclophosphamide and sirolimus in conjunction with a busulfan- based conditioning regimen. We will compare the incidence... [Time Frame: 5 years]

Secondary Outcome(s)

To measure the engraftment rate and the engraftment kinetics using such a regimen [Time Frame: 5 years]

To assess the level and kinetics of immune reconstitution (via chimerism) when using post- transplant cyclophosphamide [Time Frame: 5 years]

To further elucidate the factors involved in the development of GvHD and graft rejection/failure [Time Frame: 5 years]

Secondary ID(s)

16-I-0032

160032

Source(s) of Monetary Support

Please refer to primary and secondary sponsors