## Protocol Section
### Identification Module
**NCT ID:** NCT06430788

**Brief Title:** A Study of Emapalumab for Pediatric Aplastic Anemia

**Official Title:** Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia

#### Organization Study ID Info
**ID:** 23-278
#### Organization
**Class:** OTHER
**Full Name:** Memorial Sloan Kettering Cancer Center
### Status Module
#### Completion Date
**Date:** 2029-05-21

**Type:** ESTIMATED

#### Last Update Post Date
**Date:** 2024-05-28

**Type:** ACTUAL

**Last Update Submit Date:** 2024-05-21

**Overall Status:** RECRUITING

#### Primary Completion Date
**Date:** 2029-05-21

**Type:** ESTIMATED

#### Start Date
**Date:** 2024-05-21

**Type:** ACTUAL

**Status Verified Date:** 2024-05

#### Study First Post Date
**Date:** 2024-05-28
**Type:** ACTUAL
**Study First Submit Date:** 2024-05-21
**Study First Submit QC Date:** 2024-05-21
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** OTHER

**Name:** Memorial Sloan Kettering Cancer Center

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** True

### Description Module

**Brief Summary:** The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options.

### Conditions Module

**Conditions:**

- Aplastic Anemia

- Cytopenia

- Hypocellular Marrow

**Keywords:**

- pediatric aplastic anemia

- aplastic anemia

- cytopenia

- hypocellular marrow

- Emapalumab

- Memorial Sloan Kettering Cancer Center

- 23-278

### Design Module

#### Design Info

**Allocation:** NON_RANDOMIZED

**Intervention Model:** SEQUENTIAL

##### Masking Info

**Masking:** NONE

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 55

**Type:** ESTIMATED

**Phases:**

- PHASE2

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** Participants will first receive Emapalumab for 6 weeks. After treatment with emapalumab, participants will receive standard IST with drugs called equine anti-thymocyte globulin (hATG) and cyclosporin (CsA) in addition to a lower dose of emapalumab

**Intervention Names:**

- Biological: Emapalumab

**Label:** Emapalumab, then Standard IST

**Type:** EXPERIMENTAL

#### Arm Group 2

**Description:** Participants will first receive Emapalumab for 6 weeks. After treatment with emapalumab, participants will have a standard hematopoietic stem cell transplant (HCT).

**Intervention Names:**

- Biological: Emapalumab

**Label:** Emapalumab, then HCT

**Type:** EXPERIMENTAL

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Emapalumab, then HCT

- Emapalumab, then Standard IST

**Description:** Emapalumab is an interferon gamma (IFNγ) blocking antibody

**Name:** Emapalumab

**Other Names:**

- Gamifant

**Type:** BIOLOGICAL

### Outcomes Module

#### Primary Outcomes

**Description:** The primary objective of the study is to assess the efficacy of early upfront emapalumab on hematologic recovery within 6 weeks of starting therapy after a new diagnosis of Aplastic Anemia. Response will be determined by blood count.

**Measure:** Best Response

**Time Frame:** 6 weeks

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

* Patients undergoing workup for suspected newly diagnosed sAA:

  * Patients with severe cytopenias and a hypocellular marrow concerning for sAA
  * Patients that meet the definition for suspected sAA (Camitta Criteria) as follows:

Marrow Cellularity: \<25%, or 25-50% with \<30% residual hematopoietic cells Peripheral cytopenias (at least 2 of 3) Absolute neutrophil count (ANC): \<500 x 10\^9/L Platelets: \<20 x 10\^9/L Absolute Reticulocyte Count: \<60 x 10\^9/L

* Patients that do not have evidence of leukemia or MDS
* Patients \< 25 years of age at time of diagnosis
* Able to tolerate emapalumab and IST (with standard institutional organ function criteria)

Exclusion Criteria:

* Uncontrolled infection at presentation.
* Patients who have undergone previous treatment for sAA.
* Patients with known inherited bone marrow failure
* Patient who has completed a full workup for sAA including having results back from telomere testing, DEB and genetics (when applicable), as well as having an appropriate willing and available donor and would otherwise be admitted for HSCT within 2 weeks of enrolling on the trial
* Patients with leukemia or MDS
* Patient or parent or guardian unable to give informed consent or unable to comply with the treatment protocol including research tests.

**Healthy Volunteers:** True

**Maximum Age:** 25 Years

**Minimum Age:** 0 Years

**Sex:** ALL

**Standard Ages:**

- CHILD

- ADULT

### Contacts Locations Module

#### Central Contacts

**Contact 1:**

**Email:** ovedj@mskcc.org

**Name:** Joseph Oved, MD

**Phone:** 1-833-MSK-KIDS

**Role:** CONTACT

**Contact 2:**

**Email:** boelensj@mskcc.org

**Name:** Jaap Jan Boelens, MD, PhD

**Phone:** 1-833-MSK-KIDS

**Role:** CONTACT

#### Locations

**Location 1:**

**City:** New York

**Contacts:**

***Contact 1:***

- **Name:** Joseph Oved, MD

- **Phone:** 1-833-MSK-KIDS

- **Role:** CONTACT

**Country:** United States

**Facility:** Memorial Sloan Kettering Cancer Center (All Protocol Activities)

**State:** New York

**Status:** RECRUITING

**Zip:** 10065

**Location 2:**

**City:** Cincinnati

**Contacts:**

***Contact 1:***

- **Email:** anthony.sabulski@cchmc.org

- **Name:** Anthony Sabulski, MD

- **Phone:** 513-636-3200

- **Role:** CONTACT

**Country:** United States

**Facility:** Cincinnati Children's Hospital Medical Center (Data collection only)

**State:** Ohio

**Status:** NOT_YET_RECRUITING

**Zip:** 45229

**Location 3:**

**City:** Philadelphia

**Contacts:**

***Contact 1:***

- **Name:** Tim Olson, MD, PhD

- **Phone:** 800-879-2467

- **Role:** CONTACT

**Country:** United States

**Facility:** Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)

**State:** Pennsylvania

**Status:** NOT_YET_RECRUITING

**Zip:** 19104

**Location 4:**

**City:** Richmond

**Contacts:**

***Contact 1:***

- **Name:** Joe Laver, MD, MHA

- **Phone:** 804-828-9213

- **Role:** CONTACT

**Country:** United States

**Facility:** Virginia Commonwealth Univeristy (Data Collection Only )

**State:** Virginia

**Status:** NOT_YET_RECRUITING

**Zip:** 23219

**Location 5:**

**City:** Milwaukee

**Contacts:**

***Contact 1:***

- **Name:** Larisa Broglie, MD

- **Phone:** 414-266-2420

- **Role:** CONTACT

**Country:** United States

**Facility:** Medical College of Wisconsin (Data Collection AND Data Analysis)

**State:** Wisconsin

**Status:** NOT_YET_RECRUITING

**Zip:** 53226

#### Overall Officials

**Official 1:**

**Affiliation:** Memorial Sloan Kettering Cancer Center

**Name:** Joseph Oved, MD

**Role:** PRINCIPAL_INVESTIGATOR

### IPD Sharing Statement Module

**Description:** Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.

**IPD Sharing:** YES

### References Module

#### See Also Links

**Label:** Memorial Sloan Kettering Cancer Center

**URL:** http://www.mskcc.org

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000006402

- Term: Hematologic Diseases

- ID: D000080983

- Term: Bone Marrow Failure Disorders

- ID: D000001855

- Term: Bone Marrow Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC15

- Name: Blood and Lymph Conditions

- Abbrev: All

- Name: All Conditions

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M4070

- Name: Anemia

- Relevance: HIGH

- As Found: Anemia

- ID: M4071

- Name: Anemia, Aplastic

- Relevance: HIGH

- As Found: Aplastic Anemia

- ID: M3170

- Name: Cytopenia

- Relevance: HIGH

- As Found: Cytopenia

- ID: M9490

- Name: Hematologic Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M2241

- Name: Bone Marrow Failure Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M13118

- Name: Pancytopenia

- Relevance: LOW

- As Found: Unknown

- ID: M5134

- Name: Bone Marrow Diseases

- Relevance: LOW

- As Found: Unknown

- ID: T460

- Name: Aplastic Anemia

- Relevance: HIGH

- As Found: Aplastic Anemia

### Condition Browse Module - Meshes

- ID: D000000740

- Term: Anemia

- ID: D000000741

- Term: Anemia, Aplastic

- ID: D000095542

- Term: Cytopenia

### Intervention Browse Module - Browse Branches

- Abbrev: All

- Name: All Drugs and Chemicals

- Abbrev: Infe

- Name: Anti-Infective Agents

- Abbrev: ARhu

- Name: Antirheumatic Agents

- Abbrev: Derm

- Name: Dermatologic Agents

- Abbrev: ANeo

- Name: Antineoplastic Agents

### Intervention Browse Module - Browse Leaves

- ID: M4225

- Name: Antibodies

- Relevance: LOW

- As Found: Unknown

- ID: M18961

- Name: Cyclosporine

- Relevance: LOW

- As Found: Unknown

- ID: M6730

- Name: Cyclosporins

- Relevance: LOW

- As Found: Unknown

- ID: M10184

- Name: Immunoglobulins

- Relevance: LOW

- As Found: Unknown

- ID: M10407

- Name: Interferons

- Relevance: LOW

- As Found: Unknown

- ID: M4279

- Name: Antilymphocyte Serum

- Relevance: LOW

- As Found: Unknown

- ID: M21134

- Name: Antibodies, Blocking

- Relevance: LOW

- As Found: Unknown

- ID: M10406

- Name: Interferon-gamma

- Relevance: LOW

- As Found: Unknown

### Misc Info Module

- Version Holder: 2024-05-31
