Home-based Training and Supplementation in DM1 Patients

NCT05848830 | Unknown Phase 3

• 1 other identifier: 15550
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

Myotonic dystrophy type 1 (DM1) is a rare genetic disease that affects about 1 in 2100 people. Patients diagnosed with DM1 present with many symptoms, however, their muscles are mainly affected. DM1 patients experience a gradual loss of muscle, followed by an increase in body fat percentage, which makes them weaker, resulting in difficulties to perform activities of daily living, such as climbing stairs, and understandably, this affects their quality of life. DM1 currently does not have a cure. Therefore, it is very important to find ways in which we can help DM1 patients to improve their symptoms, and hopefully, improve their quality of life, and possibly improve disease prognosis. Exercise is known to improve muscle quality and function. In addition, we hypothesize that a multi-ingredient supplement (MIS) for muscle health and antioxidants for fat loss, might show improved benefits on top of exercise. Therefore, we will investigate the effects of 16-week home-based concurrent training, with MIS or placebo, on body composition, and functional measures. Lastly, we will investigate muscle adaptations in DM1 and following study intervention

Conditions

Myotonic Dystrophy 1

Keywords

myotonic dystrophy, skeletal muscle, mitochondria

Outcome Measures

Primary Outcomes (2)

• Body Composition Index

  Changes in ratio of fat-free mass to fat mass index as assessed via DEXA scan

  4 months from enrolment

• VO2 Max

  Changes in cardiorespiratory fitness proxy measure as assessed via VO2 max testing

  4 months from enrolment

Secondary Outcomes (8)

• 6-minute walk test

  4 months from enrolment

• 5x sit to stand

  4 months from enrolment

• Timed up and go

  4 months from enrolment

• Leg muscle strength

  4 months from enrolment

• Grip strength

  4 months from enrolment

Study Arms (3)

DM1 + HBEXT + MIS

ACTIVE COMPARATOR

Participants will be asked to undergo 16 weeks of home-based training and asked to take one dose of a multi-ingredient supplement per day.

Dietary Supplement: Mult-ingredient supplement; Behavioral: Concurrent exercise training

DM1 + HBEXT + PLA

PLACEBO COMPARATOR

Participants will be asked to undergo 16 weeks of home-based training and asked to take one dose of a multi-ingredient supplement placebo per day.

Dietary Supplement: Placebo; Behavioral: Concurrent exercise training

CONTROL

NO INTERVENTION

Healthy control subjects who will not undergo study intervention and will be used for baseline measurements and outcomes.

Interventions

Mult-ingredient supplement DIETARY_SUPPLEMENT

A mult-ingredient supplement containing Protein, both whey and casein, Creatine, Vitamin D, and Calcium. As well as antioxidants and other dietary supplements including Vitamin E, CoQ10, Alpha Lipoic Acid, L-Arginine, Beet Root Extract, Green Coffee Bean Extract, Green Tea Extract, Black Tea Extract, Curcumin, and Forskolin

DM1 + HBEXT + MIS

Placebo DIETARY_SUPPLEMENT

The placebo sachet will contain micro-crystalline cellulose (inactive compound) and will be identical in look and taste to the active supplement.

DM1 + HBEXT + PLA

Concurrent exercise training BEHAVIORAL

All participants will undergo 16-weeks of exercise training, containing 3 days/week of resistance training and 2days/ week of aerobic training.

DM1 + HBEXT + MIS; DM1 + HBEXT + PLA

Eligibility Criteria

• Age: 19 Years - 60 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Healthy men and women
• Normal BMI (BMI 18.5 - 24.9 kg/m2)
• Male or female clinically diagnosed with DM1 (age 19 - 60 y).
• CTG repeats 100-1000.
• Normal weight (BMI 18.5 - 24.9 kg/m2) or overweight (BMI 25 - 29.9 kg/m2).
• Physically inactive (\< 1 hour of formal exercise/week).
• minute walk test score between 250 - 500 meters
• ECG with PR interval \< 225 ms and QRS duration \< 125 ms.

You may not qualify if:

• Smoking
• Obese (BMI \> 30.0 kg/m2)
• Physically active (\> 1-2 hour of formal exercise/week)
• minute walk test score \<250 meters, chronic (\> 2 weeks)
• Use of narcotic analgesic or anti-inflammatory drugs
• Type 1 or 2 diabetes (more than one anti-diabetic drug)
• Cardiovascular disease (recent myocardial infarction (\< 6 months)
• Uncontrolled hypertension requiring more than 2 medications.
• Congestive heart failure requiring more than one medication for control.
• Cardiac conduction block (as above)
• Renal disease (creatinine \> 140)
• Known liver disease
• Cognitive impairments limiting ability to provide informed consent
• Previous stroke with residual hemiparesis
• Active musculoskeletal injuries and/or severe osteoarthritis

Sponsors & Collaborators

• McMaster University: lead

Study Sites (1)

McMaster University Medical Center

Hamilton, Ontario, L8N 3Z5, Canada

Location

MeSH Terms

Conditions

Myotonic Dystrophy

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Myotonic Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Mark A Tarnopolsky, MD, PhD

CONTACT

905-525-9140; tarnopol@mcmaster.ca

Joshua P Nederveen, PhD

CONTACT

905-902-0583; nedervj@mcmaster.ca

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Both the participants and the investigators are blinded to the active and placebo groups. A third party, Gruppo Nutrition (Windsor, ON) will conduct the randomization for our supplemental interventions and manage blinding procedures. Neither the participants nor the investigators will be unblinded to the supplement randomization. Lastly, researchers will only be unblinded upon finishing the data analysis.
• Purpose: SUPPORTIVE CARE
• Intervention Model: FACTORIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator, Professor, Director of Neuromuscular and Neurometabolic Clinic

Model Details: All participants will undergo 16 weeks of home-based exercise training. Half of them are randomized in an active group with a multi-ingredient supplement, and half will be given a placebo. Both the participants and the investigators are blinded to the groups.

Study Record Dates

• First Submitted: April 28, 2023
• First Posted: May 8, 2023
• Study Start: January 1, 2024
• Primary Completion: January 1, 2025
• Study Completion: January 1, 2025
• Last Updated: October 19, 2023

Record last verified: 2023-10

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)