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Trial · Interventional

CompletedPhase 1/2Results postedRegistry data

Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy

NCTNCT01207908 (opens ClinicalTrials.gov in a new tab)Other id2010-1491
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Completed
Phase
Phase 1/2
Enrollment
44 participants (actual)
Sponsor
Children's Hospital Medical Center, Cincinnati · Other
Design
Randomized · Single masking
Countries
1 country

United States

Start
Nov 2010
Primary completion
Oct 2012
Completion
Jun 2013
First posted
23 Sept 2010 (estimated)
Last update posted
20 Jan 2021
Conditions
Duchenne Muscular Dystrophy

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20102011201220132014
  1. Ph 1/2

    Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy

    NCT01207908

    Nov 2010 to Jun 2013

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).

Interventions

  • IGF-1Drug

Arms2

  • Experimental

    IGF-1

    IGF-1 plus standard steroid treatment

  • No intervention

    Standard steroid treatment alone

    Standard daily steroid treatment for DMD

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline

    Time frame · 6 months

    Registry description

    Outcome Measure Data Table shows change of 6-Minute Walk Distance at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1).

Secondary outcomes(2)

  1. Difference in Height Velocity Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline

    Time frame · 6 months

    Registry description

    Outcome Measure Data Table shows change of height velocity at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1).

  2. Difference in North Star Ambulatory Assessment (NSAA) Score Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline

    Time frame · 6 months

    Registry description

    Outcome Measure Data Table shows change of North Star Ambulatory Assessment (NSAA) score at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1). The NSAA is a 17-item scale that grades performance of various functional skills on a scale from 0 (unable), 1 (complete independently but with modifications), and 2 (complete without compensation). The range of NSAA score is from 0 to 34. The higher score indicates better motor function.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMean· 6 months

Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline

GroupValue (meters)Spread
IGF-13.432.4
Standard Steroid Treatment Alone-5.150.2
Secondary and other outcome results(2)
Secondary outcomeMean· 6 months

Difference in Height Velocity Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline

GroupValue (cm/year)Spread
IGF-12.601.64
Standard Steroid Treatment Alone-0.061.69
Secondary outcomeMean· 6 months

Difference in North Star Ambulatory Assessment (NSAA) Score Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline

GroupValue (score on a scale)Spread
IGF-1-0.942.41
Standard Steroid Treatment Alone-0.43.0

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
IGF-1IGF-1 plus standard steroid treatment IGF-1: IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.0 / 221 / 2220 / 22
Standard Steroid Treatment AloneStandard daily steroid treatment for DMD0 / 220 / 2221 / 22

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
Male
Age
from 5 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: * DMD diagnosed with mutational testing and/or complete absence of dystrophin on muscle biopsy * Proximal pelvic girdle weakness (Gower's maneuver, difficulty with arising from floor and going up steps) * Male * Age \> 5 years of age * Bone maturation (assess by bone age x-ray): \</= 11 years of age * Daily GC (prednisone or deflazacort) therapy for \> 12 months * Ambulatory * Informed consent * Willingness and ability to comply with all protocol requirements and procedures Exclusion Criteria: * Current or prior treatment with growth hormone or IGF-1 therapy * Non-ambulatory * Pubertal (based on clinical Tanner staging examination) * Congestive cardiac failure * History of intracranial hypertension * Daytime ventilatory dependence (non-invasive or tracheostomy) * Concomitant therapy - any other medications/supplements that would be considered, in the opinion of the investigators, to affect muscle function, need to have been started 3 months prior to enrollment * Patients enrolled in other clinical drug trials * Any physical or mental conditions which may, in the investigators'opinions, render the subject unable to complete the tasks of the study appropriately * There will be no selection by ethnicity

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