Red Blood Cell Precursor Formulation to Determine Increased Production (RBCPF)

An Open-Label, Pilot Study of a Red Blood Cell Precursor Formulation to Determine Increased Production in Subjects With Mild to Moderate Anemia

The objective of this study is to measure the change in hemoglobin levels after the administration of an amino acid based, RBC precursor formulation.

Study Overview

• Status: Withdrawn
• Conditions: Anemia
• Intervention / Treatment: Drug: RBCPF

Detailed Description

Medical foods are a distinct FDA regulatory category different from single molecule chemical pharmaceuticals, and from dietary supplements. The FDA has regulated amino acid preparations as drugs since the 1940s as they can elicit pharmacologic effects similar to conventional single molecule pharmaceuticals. The best known amino acid preparations are used to treat conditions such as maple syrup disease and phenylketonuria (PKU). An official definition and categorization of medical foods was made in 1988 as part of the Orphan Drug Act. Medical foods are regulated similarly to drugs except they do not require pre-approval because all ingredients are found on the FDA's generally recognized as safe (GRAS) (Sections 201(s) and 409 of the Federal Food, Drug, and Cosmetic Act) list and claims are confined to the nutritional management of a specific disease. Medical food claims must be supported by recognized scientific data as determined by medical evaluation. A GRAS substance is distinguished from a food additive on the basis of the common knowledge about the safety of the substance for its intended use. The standard for an ingredient to achieve GRAS status requires not only technical demonstration of non-toxicity and safety, but also general recognition of safety through widespread usage and agreement of that safety by experts in the field. Many ingredients have been determined by the FDA to be GRAS, and are listed as such by regulation, in Volume 21 Code of Federal Regulations (CFR) Sections 182, 184, and 186.

• Study Type: Interventional
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• United States
  • California
    • Tarzana, California, United States, 91356
      • Lawrence May, MD, Inc.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. M/F patients 18 to 75 years old, non-pregnant/lactating
2. Male patients with < Hemoglobin of 12.5
3. Female Patients with < Hemoglobin of 11
4. Diagnosis of mild to moderate anemia by study physician

Exclusion Criteria:

1. Pregnant or unwilling to use adequate birth control for the duration of the study.
2. Unwilling or unable to sign informed consent.
3. Myocardial infarction within the last 6 months.
4. Patients currently taking an erythropoietin medication and unable to discontinue for the duration of the study.
5. GI bleed in the last 6 months.
6. Inflammatory bowel disease.
7. Chronic liver disease.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: RBCPF; Treatment intervention arm	Drug: RBCPF; Other Names: Red blood cell precursor formulation

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in hemoglobin level 2 hours after administration of the first dose.	Subjects will have blood drawn at baseline and 2 hours after the administration of the treatment intervention. Then again, after 7 days.	Baseline, 2 hours and 7 days

Secondary Outcome Measures

Outcome Measure	Time Frame
CBC w/ differential	Baseline, 2 hours and 7 days
Erythropoietin level	Baseline, 2 hours and 7 days
Reticulocytes	Baseline, 2 hours and 7 days
Iron levels	Baseline, 2 hours and 7 days
Ferritin	Baseline, 2 hours and 7 days
IGG	Baseline, 2 hours and 7 days
B12	Baseline, 2 hours and 7 days

Collaborators and Investigators

• Sponsor: Targeted Medical Pharma
• Investigators: Principal Investigator: Lawrence May, MD, Lawrence May MD, Inc

Study record dates

Study Major Dates

• Study Start: October 1, 2012
• Primary Completion (Actual): May 1, 2014
• Study Completion (Actual): May 1, 2014

Study Registration Dates

• First Submitted: October 3, 2012
• First Submitted That Met QC Criteria: October 3, 2012
• First Posted (Estimate): October 5, 2012

Study Record Updates

• Last Update Posted (Estimate): May 5, 2016
• Last Update Submitted That Met QC Criteria: May 4, 2016
• Last Verified: May 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Anemia
• Other Study ID Numbers: RBC100312