Pilot Study: The Effect of Ergocalciferol on Plasma Mucin-1 Levels

NCT03747523 | Completed Early Phase 1 FDA Drug

• 1 other identifier: IRB00054784
• Study Type: interventional
• Target: 44
• Locations: 1 country
• Sites: 1

Brief Summary

To determine if a one-time oral administration of ergocalciferol results in a decline in plasma mucin-1 levels in healthy individuals and individuals with ADTKD-MUC1.

Conditions

Ergocalciferol

Keywords

Vitamin D Deficiency; Drisdol; Rickets; Plasma; Healthy Subjects; Autosomal Dominant Tubulo Interstitial Kidney Disease

Outcome Measures

Primary Outcomes (2)

• Plasma mucin-1 levels

  Plasma mucin-1 levels will be measured in both groups. using a two-tailed T-test.

  Baseline

• Plasma mucin-1 levels

  Plasma mucin-1 levels will be measured in both groups. using a two-tailed T-test.

  3 days

Secondary Outcomes (6)

• Plasma mucin-1 levels

  7 days

• Plasma mucin-1 levels

  10 days

• Urinary mucin-1 levels in healthy controls

  Baseline

• Urinary mucin-1 levels in healthy controls

  3 days

• Urinary mucin-1 levels in healthy controls

  7 days

Study Arms (2)

Healthy Group

EXPERIMENTAL

40 Healthy individuals will receive a single dose of ergocalciferol (200,000 units)

Drug: Ergocalciferol

ADTKD-MUC1 Group

EXPERIMENTAL

40 individuals with ADTKD-MUC1 (Autosomal Dominant Tubulo-Interstitial Kidney Disease- a rare disease caused by mutation in MUC1) will receive a single dose of ergocalciferol (200,000 units)

Drug: Ergocalciferol

Interventions

Ergocalciferol DRUG

Investigational drug will be administered to eligible participants in a single dose of 200,000 USP units vitamin D

Also known as: Drisdol, Calciferol, Calcidol, Vitamin D2

ADTKD-MUC1 Group; Healthy Group

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age \> 18 years and \<65 years
• Females must be non-pregnant, non-lactating and fulfilling one of the following: a. Post menopausal defined as amenorrhea for at least 12 months following cessation of all exogenous hormonal treatments. b. Status post irreversible surgical sterilization by hysterectomy, bilateral oophorectomy or bilateral salpingectomy but not tubal ligation. c.
• Use of acceptable contraceptive method: IUD with spermicide, a female condom with spermicide, contraceptive sponge with spermicide, an intravaginal system(e.g., NuvaRing®), a diaphragm with spermicide, a cervical cap with spermicide, or oral, implantable, transdermal, or injectable contraceptives, sexual abstinence, or a sterile sexual partner.
• Males must agree to avoid fathering a child (or donating sperm), and therefore be either sterile or agree to use, from the time of enrollment until 45 days after end of study, one of the following approved methods of contraception: a male condom with spermicide, a sterile sexual partner, use by female sexual partner of an IUD with spermicide, a female condom with spermicide, contraceptive sponge with spermicide, an intravaginal system (e.g.,NuvaRing®), a diaphragm with spermicide, a cervical cap with spermicide, or oral, implantable, transdermal, or injectable contraceptives.
• No serious health conditions except for estimated glomerular filtration rate \<30 ml/min/1.73 m2 (according to the modified MDRD formula) in participants with ADTKD- MUC1.
• Prior established genetic diagnosis of ADTKD-MUC1 in participants recruited with ADTKD-MUC1.
• Able to understand and comply with requirements of the entire study and to communicate with the study team.
• Living in close enough proximity to a local laboratory to obtain labwork for the study.
• Written informed consent using a document that has been approved by the Institutional Review Board.
• The participant has not taken ergocalciferol in the month prior to study initiation. If receiving cholecalciferol, the dose is \< 1000/day and has been stopped one month prior to Day 0 (On Study).
• Participants agree NOT to take ergocalciferol or cholecalciferol (except for\<500 IU that contained in a daily multi-vitamin) for six months after receiving the one-time dose of ergocalciferol.

You may not qualify if:

• Major systemic illness other than chronic kidney disease in individuals with ADTKD- MUC1.
• A history of granulomatous disorders (sarcoidosis, tuberculosis), hyperparathyroidism, or other disorders of calcium metabolism.
• A history of nephrolithiasis.
• A history of osteoporosis or osteopenia.
• A fracture or a fall that did not occur during exercise within the last six months.
• Intolerance or known allergic reaction to ergocalciferol.
• hydroxy Vitamin D level \> upper limit of normal.
• Lactating.
• Liver disease.
• Receiving glucocorticoids.

Sponsors & Collaborators

• Wake Forest University Health Sciences: lead

Study Sites (1)

Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27157, United States

Location

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MeSH Terms

Conditions

Vitamin D Deficiency; Rickets

Interventions

Ergocalciferols; Cholecalciferol

Condition Hierarchy (Ancestors)

Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Bone Diseases, Metabolic; Bone Diseases; Musculoskeletal Diseases; Metabolic Diseases; Calcium Metabolism Disorders

Intervention Hierarchy (Ancestors)

Cholestenes; Cholestanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Sterols; Vitamin D; Secosteroids; Membrane Lipids; Lipids

Study Officials

• Anthony J. Bleyer, MD, M.S

  Wake Forest University Health Sciences

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 16, 2018
• First Posted: November 20, 2018
• Study Start: March 12, 2019
• Primary Completion: December 20, 2019
• Study Completion: January 27, 2020
• Last Updated: October 29, 2020

Record last verified: 2020-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)